January 10, 2025 - Regulatory Roundup Updated every Friday
ViGeneron reports two key milestones for its gene therapy candidate VG901 to treat retinitis pigmentosa caused by mutations in the CNGA1 gene: The Food and Drug Administration has granted Rare Pediatric Disease Designation; and the independent Data Safety Monitoring Board has unanimously approved dose escalation in the ongoing Phase Ib clinical trial.
January 10, 2025 - Clinical Observations Updated every Friday
Oculis Holding has reported topline results from the Phase II ACUITY trial of OCS-05, a neuroprotective candidate for treatment of acute optic neuritis. The trial met its primary endpoint of safety and statistical significance on several key efficacy-based secondary endpoints, according to Oculis. The study randomized 36 patients with visual loss from unilateral acute optic neuritis. Treated patients demonstrated a 43% improvement in ganglion cell-inner plexiform layer thickness and a 28% improvement in retinal nerve fiber layer thickness, compared to placebo.
Sight Sciences reports that 36-month results of the Omni Surgical System for management of primary open-angle glaucoma (POAG) have been published in the American Journal of Ophthalmology. The real-world study evaluated 230 eyes of 196 patients with POAG through up to 36 months. The results demonstrated clinically and statistically significant reductions in intraocular pressure (IOP) through 36 months postoperatively, with mean reductions ranging from 5.6 to 7.1 mmHg. The study also reports a statistically significant decrease in medication use through 18 months. Eyes with lower baseline IOP experienced medication reduction through 36 months.
Results of the Phase Ib trial of sozinibercept combination therapy in diabetic macular edema (DME) have been published in the journal Translational Vision Science & Technology, trial sponsor Opthea reports. The trial evaluated the outcomes in patients with persistent DME previously treated with aflibercept 2 mg (Eylea, Regeneron Pharmaceuticals) only. In the trial, nine patients received sozinibercept (0.3, 1, or 2 mg) in combination with aflibercept once every four weeks for 12 weeks. Sozinibercept combination therapy was well tolerated with no dose-limiting toxicities. The trial also demonstrated a dose-response relationship of increased gains in best-corrected visual acuity (BCVA) for ascending doses of sozinibercept, with the 2 mg sozinibercept combination arm demonstrating the highest BCVA gain. All sozinibercept doses demonstrated a meaningful reduction of central subretinal thickness, a key biomarker of DME. Opthea says the data support the further investigation of sozinibercept as a treatment for DME.
South Korea-based Caregen has reported interim Phase I results from US clinical trials of CG-T5, an eye drop formulation that selectively binds to vascular endothelial growth factor receptor-2, a critical protein in abnormal blood vessel growth in retinal disease. The results demonstrated improvement in best-corrected visual acuity and central retinal thickness, along with strong safety and tolerability profiles, according to Caregen. The company says it plans to expand CG-T5’s indications in Phase II trials to include diabetic retinopathy and other conditions.
PAST ARTICLES
Data show physicians can perform office-based lens surgery safely, with adverse event rates similar to those of published literature according to a study published by iOR Partners Partners in the Journal of Cataract & Refractive Surgery. The study analyzed records of more than 18,000 consecutive patients who underwent office-based lens surgery for visually significant cataract, refractive lens exchange (RLE), or phakic IOL implantation. Rates of postoperative endophthalmitis, toxic anterior segment syndrome (TASS), and corneal edema were 0.028%, 0.022%, and 0.027%, respectively. Unplanned vitrectomy was performed in 0.177% of patients. [Read the Study]
EyeCare Partners has launched the Advanced Comprehensive Ophthalmology Fellowship (ACOF). The one-year fellowship provides comprehensive training to residency graduates on diagnostic and therapeutic techniques, surgical procedures, and clinical research, among other topics. The fellowship program starts in fall 2024 and applications are open until August 31, 2023. [Apply Here]
20/20 Onsite earned a Site Readiness Certification from Precision Vision Clinical Trial Solutions, a division of Precision Vision. 20/20 Onsite is the first mobile clinic to earn the certification, giving clinical trial sponsors a new way to expand clinical trial participation. [Learn More]
Elios Vision has completed enrollment for its pivotal trial for its Elios excimer laser procedure in combination with cataract surgery. The trial recruited over 300 patients with mild to moderate glaucoma. The procedure has already received a CE mark.
Molecure filed an Investigational New Drug application with the FDA for OATD-01, a drug developed to treat sarcoidosis. If the FDA approves the NDA, Molecure will begin a Phase II trial for the chitotriosidase (CHIT1) inhibitor. Inhibition of CHIT1 by OATD‑01 has been shown to reduce inflammation and fibrosis, the company states.
The Brazilian Health Regulatory Agency (ANVISA) has approved Tepezza for the treatment of thyroid eye disease (TED). Tepezza, from Horizon Therapeutics, is the first medicine approved in Brazil for TED the company stated. The product received FDA approval in 2020, and regulatory approval in Japan appears on the horizon (pun intended).
Visgenx announced positive topline data from a preclinical study of VGX-0111 for dry age-related macular degeneration (AMD). The study, in nonhuman primates, successfully demonstrated three parameters: VGX-0111 expresses in the target tissues, causes an increase in certain very long-chain polyunsaturated fatty acids, and is well tolerated.
Intergalactic Therapeutics also announced positive results from its nonviral gene therapy platform, IG-002, which addresses retinopathies related to the ABCA-4 gene. The platform safely achieved durable 6-month expression of human ABCA-4 in non-human primate retinas with a single subretinal administration.
Nonprofit eye bank Eversight is collaborating with Donor Network of Arizona, the state’s federally designated organ procurement organization, to provide eye banking services in the Grand Canyon state. The partnership takes effect July 1. [Learn More About Eversight]
Glaucoma surgeons take note: Cigna recently updated its Glaucoma Surgical Procedure policy to cover both ab interno canaloplasty and goniotomy (trabeculotomy) procedures for the treatment of open-angle glaucoma effective June 15, 2023. Sight Sciences announced the update because its OMNI Surgical System is FDA-cleared for canaloplasty followed by trabeculotomy to reduce intraocular pressure (IOP) in adult patients with primary open-angle glaucoma. [Review the Policy]
Horizon Therapeutics has published a new analysis from its N-MOmentum clinical trial of Uplinza. The study described an adjudication process that could improve clinical care decision-making in neuromyelitis optica spectrum disorder (NMOSD) through more accurate and consistent assessment of disease-related attacks. You can read about the approach in Multiple Sclerosis Journal.
Marinomed Biotech will launch a new Carragelose eye drop product (MAM-1001-3) in Austria in the first half of 2024, the India-based company announced. Carragelose is a virus-blocking compound; however, the company’s announcement said MAM-1001-3 is intended for dry eye relief. Learn more about Carragelose.
A study in JAMA Ophthalmology found “neighborhood-level social risk factors,” such as inequity, housing burden, and lack of transportation, may contribute to health disparities in microbial keratitis (MK) disease presentation. The findings suggest these neighborhood-level social determinants of health (SDOH) impact health outcomes much like individual-level SDOH. Read the Full Study (subscription required).
Curious about reimbursement for Harrow’s Iheezo? In this video, William Wiley, MD, explains the benefits of the FDA-approved ocular anesthetic, how he’s used it in his practice, and the way it’s reimbursed.
Last but by far not least, ophthalmologist (and OIS podcast host) Ehsan Sadri, MD, recaps a panel on the future of eye care that was part of Octane’s recent Ophthalmology Tech Forum. Companies such as Orasis Pharmaceuticals, Visus Therapeutics, and Johnson & Johnson Vision got a mention. [Watch the Video]
The FDA responded to Aldeyra Therapeutics’s New Drug Application for ADX-2191 (methotrexate for injection, USP) with a rejection. The agency found insufficient evidence of effectiveness due to “a lack of adequate and well-controlled investigations” in the literature-based NDA submission. Aldeyra stated in a company press release that it did not conduct clinical trials of the drug—developed to treat primary vitreoretinal lymphoma (PVRL)—per prior discussions with the FDA.
“While we appreciate the FDA’s position with respect to providing evidence from adequate and controlled trials, we do not currently believe that randomized clinical trials of ADX-2191 in PVRL, a rare and fatal cancer with no approved therapy, are feasible,” Aldeyra President and CEO Todd Brady, MD, PhD, responded. “Given the current shortage of methotrexate, the lack of approved therapy for PVRL, and the desire to avoid potential safety risks associated with ocular injection of compounded formulations, we look forward to discussing with the FDA the potential for making ADX-2191 available to PVRL patients under an Expanded Access Program.”
In more positive news, iStar Medical will present three-year interim data from its STAR-GLOBAL trial at the 10th World Glaucoma Congress (WGC) on June 29. The trial evaluates the MINIject, a minimally invasive glaucoma surgery implant. The device has already been implanted in over 1,000 patients across Europe and the UK.
Bausch + Lomb has launched a new eye health supplement: the PreserVision AREDS 2 Formula soft gels plus coenzyme Q10 (CoQ10). The company says it’s “the only eye vitamin that combines the exact nutrient formula recommended by the National Eye Institute (NEI) to help reduce the risk of moderate to advanced age-related macular degeneration (AMD) progression in AMD patients with CoQ10 to help support heart health.” [Check It Out]
Alcon has been busy gathering data. It now has results from the Alcon Eye On Cataract Survey, a global survey among 7,331 cataract surgery patients aged 50 and over. One finding: memory, mobility, and vision are the three most important qualities of aging among almost all respondents (83-84 %). [Get More Survey Results]
Looking for a summer read? Take a look at the Centre for Ocular Research & Education (CORE)’s latest issue of Contact Lens Update. Issue 72 focuses on corneal ectatic disorders.
The FDA granted 510 (k) clearance to the iHealthScreen iPredict Eye Screening System, the company announced recently. iPredict is an AI-enabled tool that screens for age-related macular degeneration (AMD) in patients aged 50 and older. The screening takes five minutes and provides results within 60 seconds.
The FDA also gave 510 (k) clearance to EverPatch from CorNeat Vision. EverPatch is a synthetic, non-degradable tissue-integrating matrix for use in ophthalmic surgeries. It’s designed to address the critical needs of ocular surgeons, providing a sterile and non-degradable solution for surgical patients. CorNeat will launch the product in a few ophthalmic centers in Q3.
EyeBio has launched its Phase Ib/II clinical trial to evaluate the safety and preliminary efficacy of intravitreal Restoret (EYE103) in patients with diabetic macular edema (DME) and neovascular AMD. Restoret is an investigational tri-specific antibody that acts as an agonist of the Wnt signaling pathway. It is designed to resolve residual fluid in the retina and to improve visual acuity in patients with DME and neurovascular AMD.
Sky Bioscience dosed its final patient in the final multiple ascending dose (MAD) arm of its Phase I trial evaluating the safety of SBI-100 Ophthalmic Emulsion in patients with glaucoma. The topical cannabinoid receptor type 1 (CB1R) agonist was well tolerated with only mild and moderate adverse events.
Ocular Therapeutix has topline data from its Phase I clinical trial evaluating OTX-TK1, an axitinib intravitreal hydrogel implant, for the treatment of wet AMD. Data demonstrated maintenance of controlled wet AMD subjects comparable to aflibercept injections every eight weeks with a single administration of OTX-TKI. Four subjects received rescue therapy for the first time at Month 12. No serious drug-related ocular or systemic adverse events were observed.
MediPrint Ophthalmics completed its SIGHT-2 dose-finding Phase IIb Group 1 (low-dose) study for its proprietary drug-eluting contact lens for glaucoma, LL-BMT1. The group that received LL-BMT1 achieved IOP reduction of about 5.5 mm Hg, comparable to the reference treatment. MediPrint will study the lens in two other groups for this trial.
Stealth BioTherapeutics reported a positive end-of-phase-2 meeting with the FDA about its lead drug candidate, elamipretide, developed to treat dry AMD. The FDA's meeting minutes confirmed ellipsoid zone (EZ) attenuation as an approvable endpoint in dry AMD, giving the company confidence to move forward.
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*Ocular Therapeutix and Stealth BioTherapeutics are set to showcase their latest advancements at the upcoming Retina Innovation Summit. Register here and stay updated on the cutting-edge developments shaping the field of retinal health.
Apple’s next operating system update will include new features designed to protect eye health. Apple Watch will measure time users spend in daylight so users can spend the recommended 80 to 120 minutes outdoors. The iPhone and iPad will nudge users if they keep the device too close to their face for too long, potentially reducing risk of eye strain in adults and myopia in children. Look for the new OS this fall. [See iOS 17]
GameSquare Holdings also supports healthy screen habits. It’s teamed up with Swiss vision care start-up Vivior to execute a comprehensive marketing campaign to talk about the negative effects of digital eye strain. A rewards program will help incentivize gamers to adopt healthier habits.
AOS (Advanced Ophthalmic Systems) launched a new remote visual acuity software platform. The update is designed to measure and calculate precise and reliable visual acuity test scores and offers the same level of security and compliance as previous versions. It’s available as both a stand-alone product and as part of an AOS subscription. [See the Website]
Biotrue Hydration Boost Contact Lens Rehydrating drops from Bausch + Lomb are now available at retailers throughout the United States. The preservative-free drops help combat dryness from contact lens–wearing. Relatedly, the company also launched the Infuse Multifocal silicone hydrogel (SiHy) daily disposable contact lenses.
The FDA approved Novaliq’s Vevye (cyclosporine ophthalmic solution .1%, formerly called CyclASol) for the treatment of dry eye disease. The preservative-free, oil-free drop delivered impressive results in clinical trials, with significant improvements in tCFS and dryness score at 29 days and corneal staining score improvements after two weeks. The announcement follows the approval of Bausch + Lomb/Novaliq’s Miebo last month.
Horizon Therapeutics published new data from its Phase III pivotal trial of Uplizna. The study identified important biomarker trends associated with neuromyelitis optica spectrum disorder (NMOSD, aka Devic disease) attacks. Look for the study in The Journal of Neurology, Neurosurgery and Psychiatry.
Glaukos completed enrollment and randomization in its second Phase III pivotal trial for Epioxa in less than six months. Epioxa is a next-generation corneal cross-linking therapy for the treatment of keratoconus. The first Phase III trial met its primary endpoint. Glaukos plans to submit a New Drug Application (NDA) by the end of 2024.
Eyepoint Pharmaceuticals completed enrollment in its Phase II trial evaluating EYP-1901 as a potential nine-month treatment for moderate to severe non-proliferative diabetic retinopathy (NPDR). The company enrolled 77 patients, exceeding its 60-patient target. It expects to have topline data to report in Q2 2024.
Meanwhile, Clearside Biomedical started enrollment for its Phase IIb trial for CLS-AX (axitinib injectable suspension). It plans to enroll 60 patients with wet AMD within 36 months of screening. The study will compare the efficacy of CLS-AX with aflibercept for 36 weeks.
Aviceda Therapeutics dosed its first patient in its Phase II trial for AVD-104. The intravitreal drug is a novel glycan-coated nanoparticle intended to treat patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Yuyu Pharma announced topline results from its Phase I/II trial of YP-P10 in patients with dry eye disease. Over 12 weeks, the YP-P10 exhibited a positive safety profile and was well-tolerated. Patients reported improved ocular discomfort scores by the sixth visit but no significant reduction in corneal staining scores. Mild adverse events were balanced across study groups.
Eyestem Research submitted an Investigational New Drug (IND) application to the Central Drugs Standards Control Organization, India, to begin its first-in-human trials for Eyecyte RPE. The cell therapy is being developed to treat patients with medium- and late-stage GA secondary to dry AMD.
VivaVision Biotech reports its VVN539 ophthalmic solution met its primary endpoints for the treatment of primary open-angle glaucoma or ocular hypertension in a Phase II study. The treatment was statistically superior to the vehicle at all nine time points over the 21-day study. Next step: a trial comparing VVN539 against a first-line hypotensive drug therapy in a larger patient population.
A team of researchers from Google, Verily Life Sciences, and elsewhere recently published a study that compares the performance of an automated deep learning algorithm with manual grading by ophthalmologists for identifying diabetic retinopathy in retinal fundus photographs. The specificity and sensitivity were quite good, showing promise for use in the clinical setting. Read the full study in JAMA to find out how the researchers trained and validated the model and the results.
A group of eye care companies have joined forces to raise awareness around the negatives associated with preservatives in eye drops and encourage use of preservative-free options. Called the Preservative Freedom Coalition, the organization will focus on awareness, availability, and advocacy. Founding members include Thea Pharma, The Glaucoma Foundation, The Intrepid Eye Society, National Medical Association Ophthalmology Section, and Real World Ophthalmology. Visit the organization’s webpage for details.
HOYA Vision Care has released its MiYOSMART Sun spectacle lenses in both Singapore and Canada. Available in photochromic and polarized options, the spectacles are developed to slow down myopia progression in children while protecting the eyes from the sun.
Racial and gender inequity among physicians persists, including among ophthalmologists. According to new survey results from Johnson & Johnson Vision, about a third (32%) of female ophthalmologists surveyed said their competence in medicine was questioned due to their gender. About the same number have been harassed or disrespected. The Women in Ophthalmology survey aimed to uncover barriers faced by female ophthalmologists. The survey results and their implications will be discussed at the American Academy of Ophthalmology 2023 Annual Conference on Saturday, November 4, from 3:00pm – 3:50pm PDT. [Get More Info]
President Joe Biden presented David Huang, MD, PhD, an ophthalmologist and researcher at Oregon Health & Science University; and James Fujimoto, PhD, and Eric Swanson, MS, of the Massachusetts Institute of Technology, with the National Medal of Technology and Innovation award for developing optical coherence tomography (OCT). The award is considered this country’s highest honor for technical achievement.
Bausch + Lombhas updated its eyeTELLIGENCE platform with new surgical planning software. The new software is designed deliver a “seamless, secure flow of information from the office to the OR” (operating room) ensuring surgeons can manage data and personalize their approach to each case digitally without the need for multiple documents, sources, and folders, according to a company statement. The goal is to save time and mitigate lost data.
Skye Bioscience announced Phase I results studying the safety and pharmacokinetics of SBI-100 Ophthalmic Emulsion (OE). The product targets the endocannabinoid system; specifically, the CB1 receptor, to address glaucoma. Of the 24 total patients, one patient in the single ascending dose (SAD) arm and two in the multiple ascending dose (MAD) arm experienced red eyes. Any discomfort reported was resolved in less than 15 minutes. Patients in the MAD arm with an IOP of 17mmHg or greater experienced a reduction in IOP that ranged from 14 to 31%.
Aviceda Therapeutics has completed enrollment for part one its Phase II/III SIGLEC clinical trial for AVD-104, designed to treat geographic atrophy. Part one is a multicenter, open label safety and dose escalation study with 30 patients. Part two will be a multicenter, double-masked, randomized trial to evaluate the treatment effect of AVD-104 against sham and an active comparator. The recruitment goal is 290 patients.
The FDA approved a supplemental biologics license application (BLA) for Byooviz as a biosimilar interchangeable with Lucentis (ranibizumab). Byooviz from Samsung Bioepis was initially approved in September to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), and myopic choroidal neovascularization (mCNV). The supplemental approval means Byooviz is interchangeable with Lucentis across all approved indications with safety risk.
The FDA cleared the investigational new drug application (IND) for EA-2351, a regenerative medicine developed to treat geographic atrophy (GA). The approved IND allows the developer, Endogena Therapeutics, to launch a first-in-human study in 2024.
To learn more about Endogena’s technology, listen to our podcast with CEO Matthias Steger, PhD.
Aurion Biotech dosed its first patient in its Phase I/II clinical trial for AURN001, a cell therapy developed to treat corneal edema secondary to corneal endothelial dysfunction. The primary endpoint is percentage of subjects who gain three lines of vision at six months.
The European Medicines Agency (EMA) granted Priority Medicine (PRIME) designation to ANX007 from Annexon. The drug is designed to treat GA secondary to age-related macular degeneration (AMD). The designation provides early and proactive support from the EMA.
Roche opted to stop development of vicasinabin, an oral drug it was developing to treat non-proliferative diabetic retinopathy (DR). The company dropped vicasinabin as part of a cull that also affected a cancer drug in the company’s pipeline. The news was announced during the company’s Q3 announcement. Teresa Graham, CEO of Roche Pharmaceuticals, told Fierce Biotech, “We've seen encouraging early data that would lead us to believe that further study is necessary or warranted. So I would say stay tuned, and hopefully at an upcoming medical conference, we'll have more data that we can share.”
Bausch + Lomb has launched the enVista Aspire monofocal and toric IOLs with intermediate optimized lenses (IOL) in the United States. The company reports the enVista Aspire is the only toric IOL platform available to treat less than one diopter of astigmatism at the corneal plane. [Details]
Sales of the Lucentis biosimilar ranibizumab-eqrn (CIMERLI) has exceeded 100,000 doses in its first year, according to Coherus BioSciences. The company plans to report net sales for Q3 in November. [More About Cimerli]
STAAR Surgical, which focuses its business on implantable lenses for the eye, is celebrating the 30th anniversary of its Implantable Collamer Lens (ICL). The ICL evolved into what is now the EVO ICL family of lenses. [More About EVO]
Heidelberg Engineering received FDA clearance for its all-in-one Anterion platform. The imaging platform is designed to transform anterior segment diagnostics and streamline practice workflow. It combines biometry, IOL power calculation with corneal topography and tomography, anterior chamber metrics, and high-resolution imaging.
FDA approved Qlosi (pilocarpine hydrochloride ophthalmic solution) 0.4% from Orasis Pharmaceuticals for the treatment of presbyopia in adults. The drop demonstrated efficacy 20 minutes after administration and can last up to 8 hours, as measured on day 15, according to Orasis.
On the flipside, The FDA identified “substantive review issues” in the new drug application for reproxalap, a dry eye drug from Aldeyra Therapeutics. According to US securities documents, the FDA stated Aldeyra did not have the data “to support the clinical relevance of the ocular signs to support your dry-eye indication.” As a result of the news, Aldeyra stock dropped almost 70% this week. Aldeyra is attempting to mitigate the identified issues. We’ll report updates as we get them.
Aviceda Therapeutics announced positive data from its ongoing safety studies for AVD-104, its geographic atrophy (GA) drug. No drug-related adverse events, serious adverse events, or dose-limiting toxicity have been observed.
At the recent AAO meeting, Sun Pharmaceuticals presented Phase IV data showing its dry eye drug, Cequa (cyclosporine ophthalmic solution) 0.09%, produces sustained improvement in the signs and symptoms of dry eye disease (DED). The patient population were those who were either still symptomatic and/or exhibited disease signs on Restasis therapy for at least three months, and who had a history and clinical diagnosis of dry eye disease for at least three months before screening/baseline.
The TOTAL30 Multifocal, the first and only monthly water gradient multifocal contact lens, is now available globally according to Alcon. “With the introduction of TOTAL30 Multifocal, we’re bringing the exceptional comfort of Water Gradient Technology to contact lens wearers with presbyopia who prefer a monthly replacement schedule,” said Ian Bell, Alcon’s President of Global Business and Innovation, in a statement. “The lens material approaches 100% water at the surface so all that touches your eye is a gentle cushion of moisture. This will not only transform the multifocal lens-wearing experience, but also ensure an exceptional fitting experience for Eye Care Professionals.” [See More]
AEYE Health, a company that provides AI-powered diabetic retinopathy screening, says it’s reduced the processing time for its system to under five seconds. The company reports the exam requires a single image per eye and takes less than two minutes for most patients. [See How It Works]
After four years of treatment and follow up, NVK002 (low dose atropine 0.01%), a preservative-free drop from Vyluma developed to myopia in children, shows strong safety and continued efficacy. That’s according to top-line results of the second stage of Vyluma’s Phase III study. The Prescription Drug User Fee Act (PDUFA) date for NVK002 is set for January 31, 2024.
Samsung Bioepis presented results from a post-hoc analysis to find out the biosimilarity of SB15, a proposed biosimilar to aflibercept (Eylea). In short, comparable clinical efficacy and safety was maintained in patients with neovascular age-related macular degeneration (nAMD) who switched from aflibercept to SB15.
“This post-hoc analysis provides valuable insights on the outcomes of switches from the reference biologic to SB15 and may help clinicians to make well informed decisions on potential use of SB15 if approved,” said Mourad Farouk Rezk, Vice President and Head of Global Medical and Development, Biogen Biosimilars Unit. Samsung Biologics and Biogen established Samsung Bioepis in 2012.
Genentech announced positive topline long-term results from its global Phase III BALATON and COMINO studies. The two trials evaluated extended treatment intervals with Vabysmo (faricimab-svoa) in macular edema due to branch and central retinal vein occlusion (BRVO and CRVO).
Looking at weeks 24 to 72, the results showed patients maintained vision gains achieved in the first 24 weeks of the trials. Results showed “robust and sustained drying of retinal fluid” from baseline up to week 72, as measured by reduction in central subfield thickness.
Innovent dosed its first patient in its Phase III STAR clinical trial of efdamrofusp alfa (IBI302), a recombinant fully human anti-VEGF and anti-complement bispecific fusion protein designed to treat nAMD. The company plans to enroll 600 patients. Xiaodong Sun, MD, PhD, Deputy Director of Shanghai General Hospital, Head of Ophthalmology Centre, is the Principal Investigator.
OKYO Pharma filed an investigational new drug (IND) application with the FDA to develop OK-101, a long-acting drug candidate to treat neuropathic corneal pain (NCP). Pending IND approval, study enrollment is planned to start in early 2024. Pedram Hamrah, MD, Professor and Vice Chair of Research and Academic Programs, Co-Director of the Cornea Service and Director of the Center for Translational Ocular Immunology at Tufts Medical Center, will serve as principal investigator.
BioTissue announced results from a multicenter, retrospective study conducted on patients with moderate to severe dry eye disease (DED) treated with BioTissue’s self-retained Prokera Slim Amniotic Membrane corneal bandage. Results showed a significant improvement in Dry Eye Workshop (DEWS) scores one week, one month, and three months post treatment. Baseline scores improved from 3.1 +/– 0.6 at baseline to 1.50 +/– 0.5 at one week (P = .006) and 1.2 +/- 0.4 at both one and three months (P = .004). No complications or adverse effects were reported.
Data from stage one of the Phase III clinical trial of OCS-01 showed the drug met primary and secondary endpoints, according to an Oculis statement. Results include a statistically significant improvement in best corrected visual acuity (BCVA) in patients treated with OCS-01 versus vehicle-treated patients at week six (OCS-01: 7.2 letters vs. vehicle: 3.1 letters, [P = .007]). BCVA was sustained through Week 12 (OCS-01: 7.6 letters vs. vehicle: 3.7 letters, [P = .016]).
Humonix Biosciences has developed a 3D human tissue model called the retinal vascular dysfunction model, which is a physiologically relevant 3D model of the blood-retinal barrier. The product provides a platform for testing therapies related to retinal vascular dysfunction and may speed up the drug development process.
"A reproducible and approachable model of the blood-retinal barrier needs to be more complex than a monolayer of cells; interactions between endothelial cells and pericytes are central to the development and maintenance of barrier function, a fact well recapitulated in the Humonix model," said Patricia D’Amore, PhD, MBA, Associate Chief for Basic and Translational Research, Mass Eye and Ear and Humonix Scientific Advisor. [Learn More]
Janssen, which has been part of Johnson & Johnson since 1961, is now Johnson & Johnson Innovative Medicine. The change is in name only: J&J’s pharmaceutical segment will continue to develop products for oncology, immunology, neuroscience, cardiovascular, pulmonary hypertension, and metabolism and retina.
The FDA has granted 510 (k) clearance to the IMS platform from Altris. The image and data management platform is designed to store, organize, and support in-depth analysis of Optical Coherence Tomography (OCT) scans.
Lineage Cell Therapeutics announced results from its Phase I/II study of RG6501 (OpRegen), a geographic atrophy treatment. “These data suggest that OpRegen RPE cells may provide direct support to the patients’ remaining retinal cells within atrophic areas, and that the improvements to retinal structure can be detected within the first three months following a single administration,” said Lineage CEO Brian Culley in a statement. “We look forward to additional, future clinical data updates on the OpRegen program from our partners, Roche and Genentech.”
Clearside Biomedical’s partner Arctic Vision has completed enrollment in a Phase III trial in China for suprachoroidal use of ARCATUS (ARVN001, aka XIPERE®) for the treatment of uveitic macular edema.
Kiora Pharmaceuticals received Investigational New Drug (IND) approval in Australia to enroll six additional patients in a study investigating KIO-301. The additional enrollment will enable Kiora to study the product in patients with late-stage retinitis pigmentosa and late-stage choroideremia.
Ocular Therapeutics has launched its first pivotal trial to evaluate OTX-TKI, an axitinib intravitreal implant, for the treatment of wet AMD. The company activated its first US site and plans to enroll its first patient by year’s end. The study is designed as a superiority trial that will enroll about 300 treatment-naive patients with wet AMD. Arshad M. Khanani, MD, Director of Clinical Research and Director of Fellowship at Sierra Eye Associates in Reno, Nevada, is the principal investigator for the trial.
The FDA granted Breakthrough Device designation to Toku’s CLAiR technology. The platform is intended to provide affordable, point-of-care and non-invasive evaluation for risk of cardiovascular disease (CVD) using fundus retinal images through a routine eye exam.
After a successful meeting with the FDA, Ocuphire Pharma will advance its diabetic retinopathy drug, APX3330, into Phase III clinical trials.
“Results from our phase 2 ZETA-1 results demonstrate that oral APX3330 has the potential to slow or prevent clinically meaningful progression of diabetic retinopathy, as measured by the percentage of subjects with ≥ 3-step worsening on a binocular diabetic retinopathy severity scale (DRSS), which will be the phase 3 primary endpoint,” said CEO George Magrath, MD, in a recent statement.
Ocular Therapeutics received an okay from the FDA under a Special Protocol Assessment (SPA) on the overall design of its ongoing Phase III clinical trial for Axpaxli (axitinib intravitreal implant), a potential wet AMD treatment.
Ashvattha Therapeutics has dosed its first patient in a trial to evaluate D-4517.2, a subcutaneous therapeutic developed to treat wet AMD or DME. The company plans to enroll at least 20 patients in the Phase II chronic dosing study.
ViaLase has completed enrollment in VIA-002, a pivotal trial of the ViaLase Laser, which is designed to treat adults with primary open angle glaucoma (POAG). VIA-002 is a non-inferiority efficacy study that will compare the ViaLase Laser to selective laser trabeculoplasty (SLT) according to ViaLase Chief Medical Officer Richard Lewis, MD. The study includes 152 total participants who will be randomized to either the ViaLase Laser or SLT. The primary endpoint is a reduction in mean unmedicated intraocular pressure (IOP) from baseline to six months and 12 months.
Clearside Biomedical completed recruitment for its Phase IIb clinical trial to evaluate CLS-AX (axitinib injectable suspension), a tyrosine kinase inhibitor intended to treat wet AMD. The company says it expects to have topline data in Q3 2024.
iSTAR Medical announced this week that the first patients in Ireland have been implanted with its MINIject, a minimally invasive glaucoma surgery (MIGS) device. The procedures took place at the Institute of Eye Surgery, (IOES) Mullingar.
OnPoint Vision received the green light from the FDA to begin Phase I of its pivotal clinical trial of the AccuraSee Intraocular Pseudophakic Capsular Lens (IOPCL) Magnifier (MAG) for secondary implantation in the capsular bag with a pre-existing 6mm acrylic posterior chamber IOL. The IOPCL MAG is designed to magnify near images when unilaterally implanted in low-vision pseudophakic subjects with stable AMD at least six months after cataract surgery.
SpyGlass Pharma has launched a Phase I/II study to evaluate its intraocular drug delivery platform in patients with glaucoma and visually significant cataracts. Designed to be implanted during cataract surgery, the platform is designed to deliver multiple years of bimatoprost in patients with open angle glaucoma or ocular hypertension.
Okogen has launched a Phase IIb clinical trial to evaluate OKG-0303 for acute infectious conjunctivitis (pink eye). The product is a first-in-class, fixed-dose combination product developed to treat the underlying cause and the associated signs and symptoms of both viral and bacterial forms of acute infectious conjunctivitis. Okogen will conduct the study in India.
The FDA approved Vabysmo (faricimab-svoa) for the treatment of macular edema following retinal vein occlusion (RVO) according to a press release from Genentech. The approval is based on Phase III studies that demonstrated monthly treatment with Vabysmo provided early and sustained vision improvement in people with branch and central RVO, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. This is the drug’s third indication: it’s also approved for wet age-related macular degeneration (AMD) and diabetic macular edema (DME).
Bausch + Lomb has launched SeeNa, an ophthalmic diagnostic system for refractive cataract patients, in the United States. The device is fully integrated with Bausch’s Eyetelligence surgical planning software.
“SeeNa provides a new way for busy refractive cataract practices to enhance surgical planning efficiency,” said Luc Bonnefoy, president, Global Surgical, Bausch + Lomb, in a statement. “Surgeons can gather critical measurements in a few steps, and the integrated Eyetelligence software connectivity combines these data points to help eliminate the need for multiple documents and avoid lost data and time.” [See SeeNa]
The FDA accepted Amgen’s Biologics License Application (BLA) for ABP 938, an Eyelea (aflibercept) biosimilar. The company’s Phase III study results “confirmed no clinically meaningful differences in efficacy, safety, and immunogenicity,” compared with Eylea” according to a press release.
Amydis launched a Phase II open-label, blinded assessment study for AMDX-2011P, a novel retinal tracer technology intended to help treat glaucoma. The technology is a small molecule designed to detect and quantify amyloid-beta deposits in the retina using imaging devices that are already part of a patient’s standard office visit.
Regenxbio announced more topline data from its Phase II trial of ABBV-RGX-314, a one-time gene therapy intended to treat diabetic retinopathy (DR). At two different dose levels, the therapy demonstrated stable to improved disease severity. Seven serious adverse events were reported—none of them were considered drug related.
Australian company PolyActiva reported Phase IIa results for its biodegradable ocular implant designed to provide sustained drug delivery for people with glaucoma. The company reported that patients who received the implant experienced about a 20% reduction in intraocular pressure at 12 weeks and 26 weeks. Eight patients were able to receive a second implant at 21 weeks.
Kodiak Sciences plans to reboot its clinical program for tarcocimab tedromer 5 mg in patients with moderately severe to severe DR. The company discontinued the program after two late-stage trials failed to meet their primary endpoints. The company’s Phase III GLOW study met primary endpoints. “In recent discussions with the FDA, which included the GLOW data, we believe we have a clear regulatory pathway requiring one additional positive study to support a single BLA submission for all three indications,” said CEO Victor Perlroth, MD.
Kiora Pharmaceuticals reports that results from its Phase I/II clinical trial for KIO-301, the company’s first-in-class intravitreal (IVT) molecular photoswitch, has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP) with ultra-low vision or complete blindness. The study results showed improvements in kinetic visual field, visual acuity, and light perception, among other observations.
Final data from Belite Bio’s Phase II study for Tinlarebant (LBS-008) in adolescent Stargardt disease (STGD1) shows promise. The 24-month study showed that five of the 12 patients (42%) remained free of atrophic retinal lesions after 24 months of treatment. No adverse events were reported.
Alcon has launched Clarion Vivity a non-diffractive extended depth of focus (EDOF) IOL in Canada. The lens is designed to provide vision from distance to functional near with a profile comparable to a monofocal.
Patients with diabetic macular edema (DME) continued to benefit from Eylea (aflibercept) at 96 weeks according to recently announced data from the PHOTON trial from Regeneron Pharmaceuticals. At 96 weeks, all study groups maintained improvements in Best Corrected Visual Acuity (BCVA) and decrease in central retinal thickness.
Lumenis launched its OptiPlus dual-frequency radiofrequency (RF) device in the United States. The device is used for aesthetic purposes and as a complimentary device to the Lumenis OptiLight technology, which targets dry eye disease. The device stimulates peri-orbital skin rejuvenation while also reaching into the deeper tissue to target the meibomian glands.
Bausch + Lomb has a CPT Category 1 code for Xipere, a treatment that uses suprachoroidal space to treat macular edema associated with uveitis. The CPT code will take effect January 1, 2024. The J Code is J3299.
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted marketing authorization to Yesafili, an Eylea (aflibercept) biosimilar from Biocon Biologics. The product is intended to treat AMD, visual impairment due to myopic choroidal neovascularisation, visual impairment due to diabetic macular edema, and visual impairment due to macular edema secondary to retinal vein occlusion.
Relatedly, the European Medicines Agency (EMA) recommends approval of 8 mg Eylea from Regeneron (commercialized outside the US by Bayer AG). Approval from the European Commission is pending.
Tarsier Pharma reported that its Type C meeting with the FDA was successful. Tarsier met with the FDA to discuss primary and secondary endpoints for an upcoming TRS01 trial in patients with noninfectious uveitis; specifically, uveitic glaucoma. The upcoming trial is intended to replicate Tarsier’s previous Phase III trial for the drop.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, a gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The FDA granted the designation based on efficacy data from Atsena Therapeutics.
Ocugen dosed its first patient in its Phase I/II trial for OCU410ST (AAV5-hRORA), a modifier gene therapy candidate being developed for Stargardt disease. Studies include a dose-ranging study and a dose-expansion study.
Pixium Vision SA reported data from its first-in-human trial to assess the efficacy and safety of the PRIMA photovoltaic retinal stimulation microchip. Data showed the implant was feasible and well tolerated in the study’s five participants, with no peripheral vision issues after 48 months. Patients recognized letters and sequences of letters with a clinically meaningful improvement in visual acuity of up to eight lines. The chip is designed to improve visual acuity in patients with severe central vision impairment due to dry age-related macular degeneration (AMD)
Inflammasome Therapeutics received a green light from the FDA to launch its Phase I/II trial to evaluate its inflammasome inhibitor drug, called Kamuvudines, for the treatment of geographic atrophy. “…[O]ur drugs target multiple toxic pathways—complement, amyloid beta, iron overload, retrotransposons, etc. —via their common pathway to toxicity, inflammasome activation,” Paul Ashton, PhD and CEO of Inflammasome Therapeutics, said in a statement. “We believe this approach will have a more profound effect.”
Patients with low vision now have a new AI-powered tool to help them see better. OrCam Technologies has launched OrCam Read 3, a versatile tool that patients can use as a handheld reading companion, as a next-generation magnifier, or as a stationary reader. The device is about the size of a cigar or a single Twix candy bar and does not require an Internet connection to work. When connected to the Internet, however, users can leverage AI to have the tool read text aloud and/or provide a summary of scanned text.
“Our mission was clear with the development of the OrCam Read 3,” said CEO Elad Serfaty. “We wanted to create a device that could not only act as a handheld reader but also as a magnifier and stationary reader. By combining all these features into a single device, users get enormous value that is absolutely unique in the market and unprecedented versatility in how and when they use the device … we encourage anyone who is curious to view the demos on our website or call us to arrange a demo.” [See Read 3]
Ocutrx Technologies also released a device designed to help people with see clearly, this one designed to help people with AMD. Called OcuLenz AR/XR, the headset uses augmented reality and overlays high-contrast, pixel-manipulated images onto the user's remaining viable field of view. [See OcuLenz]
ABIONYX Pharma announced results from a long-term preclinical study into CER-001, a therapy based on the recombinant apoA-I protein. The 74 animals, tested with either CER-001 alone or in combination, experienced statistically significant reductions in inflammation at six hours and 24 hours after injection.
Chengdu Origen Biotechnology and Vanotech announced dosing of the first patient in the VAN-2201 Phase I clinical trial evaluating treatment with KH631, a gene therapy for patients with wet age-related macular degeneration (wet AMD). The trial will assess safety, efficacy, and tolerability in patients with previously treated wet AMD.
A team led by researchers at the Schepens Eye Research Institute of Mass Eye and Ear have identified a potentially new strategy for glaucoma cell replacement therapy. By changing the microenvironment in the eye, the researchers could take stem cells from blood and turn them into retinal ganglion cells capable of migrating and surviving into the eye’s retina. The study was conducted in mice, but the research shows the strategy could be applied to humans. [Read the Study]
Zilia, a Quebec, Canada-based company that develops technology related to ocular biomarkers, has received 501(k) clearance from the FDA on its Zilia Ocular FC retinal camera. By assessing ocular biomarkers, the device is intended to diagnose and manage ocular disease.
Skye Biosciences has treated its first patient in a Phase II study evaluating SBI-100, a glaucoma treatment designed to target the endocannabinoid system. Skye plans to enroll 54 patients into the double-masked, randomized, placebo-controlled study.
Pune, India–based Enzene Biosciences has launched Ranibizumab, a Lucentis biosimilar, in India. The company’s pipeline includes 10 biosimilars for a range of indications.
Aldeyra Therapeutics received a Complete Response Letter from the FDA regarding Aldeyra’s NDA for reproxalap, a potential dry eye disease treatment. The letter said the NDA did not demonstrate “efficacy in treating ocular symptoms associated with dry eyes” and that “at least one additional adequate and well-controlled study to demonstrate a positive effect on the treatment of ocular symptoms of dry eye” should be conducted. A crossover trial is being proposed, subject to FDA feedback on a Special Protocol Assessment submitted in mid-November. If all goes well, Aldeyra hopes to resubmit its NDA in the first half of 2024.
OliX Pharmaceuticals, an RNAi therapy developer based in South Korea, announced Phase I study results evaluating the safety and tolerability of OLX10212, a potential AMD treatment. Out of the 15 patients treated, no adverse effects, signs of inflammation, or systemic effects were observed.
An online eye care course developed by Orbis International appears to successfully train healthcare workers to identify signs of glaucoma at a level comparable to ophthalmologists. That’s according to a new peer-reviewed study published in the British Medical Journal (Open). The study suggests that training non-ophthalmologists via a five-hour online course could help improve access to glaucoma diagnosis and treatment in low- and middle-income countries where there are few ophthalmic experts. [Read the Study]
Because AI is everywhere, the American Medical Association has released new guidance around augmented intelligence (AI aka artificial intelligence) development, deployment, and use. The principles promote development and deployment in a manner that is “ethical, equitable, responsible, and transparent,” according to an AMA statement.
“The AMA recognizes the immense potential of health care AI in enhancing diagnostic accuracy, treatment outcomes, and patient care,” said AMA President Jesse Ehrenfeld, MD, in the statement. “However, this transformative power comes with ethical considerations and potential risks that demand a proactive and principled approach to the oversight and governance of health care AI. The new AMA principles will guide the organization’s engagement with the administration, Congress, and industry stakeholders in discussions on the future of governance policies to regulate the development, deployment, and use of health care AI.” [Review the Principles Doc]
The iPredict AI System, a diagnostic that provides fully automated diabetic retinopathy (DR), AMD, and glaucoma suspect screening has received certification and registration from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) according to manufacturer iHealthScreen. The product is already in use in the United Arab Emirates, Australia, and Bangladesh.
Oculis is ramping up its Phase IIb RELIEF trial to evaluate licaminlimab (OCS-02), an anti-TNFα biologic eye drop intended to treat dry eye disease (DED). They have reached first patient first visit (FPFV) in the multicenter, randomized, double-masked, vehicle-controlled trial evaluating safety and efficacy in patients with moderate to severe DED.
EyePoint Pharmaceuticals announced favorable results from its Phase II DAVIO 2 trial of EYP-1901, an investigational sustained delivery maintenance treatment for wet AMD combining vorolanib, a selective tyrosine kinase inhibitor, with bioerodible Durasert E. According to EyePoint, EYP-1901 met its primary endpoint, with both doses demonstrating “statistical noninferiority change in best corrected visual acuity (BCVA) compared to aflibercept control and a favorable safety profile with no EYP-1901-related ocular or systemic serious adverse events (SAEs),” the company reports.
Atsena Therapeutics also announced encouraging data, theirs from an ongoing Phase I/II trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). The therapy delivered consistently meaningful vision improvements at 12 months after treatment, with no serious treatment-related adverse events.
Okyo Pharma hit a critical milestone in its Phase II trial for OK-101, a dry eye disease drug: It enrolled the last of the study’s 240 patients. Okyo expects to share topline results later this month.
Ciliatech has released a new design of its CID (cilioscleral interpositioning device) used in the cilioscleral technique to lower intraocular pressure (IOP) while preserving the eye’s anterior chamber for glaucoma treatment. The new design is intended to improve safety and performance and enhanced IOP reduction. [Learn More About CID]
HCP Cureblindness, a nonprofit dedicated to restoring sight to people worldwide, completed its first corneal transplants to four patients in Eritrea. The doctors restored vision to patients who were previously bilaterally blind.
“After cataracts, corneal blindness is the second most common reason that people are blind in Eritrea,” said Dr. Matt Oliva, who sits on the HCP Cureblindness Board and was part of the Eritrean transplant team. “When the cornea becomes opacified (typically from infection or inflammation), the only way to restore sight is a corneal transplant procedure.” [Learn More About HCP Cureblindness]
Glaukos received FDA approval for its iDose TR (travoprost intracameral implant 75 mcg), a prostaglandin analog intracameral implant designed to reduce intraocular pressure (IOP) in patients with ocular hypertension or open-angle glaucoma. The commercial launch begins in early 2024. Wholesale cost of the iDose TR is $13,950 per implant.
The FDA granted 510 (k) clearance to Belkin Vison’s Eagle glaucoma laser. The Eagle is a Q-switched, 532 nm-wavelength, frequency-doubled Nd:YAG laser intended for use in performing selective laser trabeculoplasty (SLT). It is the first and only contactless laser for glaucoma, the company reports.
Clearside Biomedical has completed randomization in its Phase IIb clinical trial to evaluate CLS-AX (axitinib injectable suspension) in patients with wet AMD. It plans to have topline data from the study by Q3 2024, according to a company press release.
Tarsus Pharmaceuticals announced topline results from its Phase IIa clinical trial, which evaluates TP-03 (lotilaner ophthalmic solution, 0.25%) or the treatment of meibomian gland disease (MGD) in patients with Demodex mites. Tarsus reports a significant and clinically meaningful increase from baseline observed in the mean Meibomian Gland Secretion Score (MGSS) of 10.5 (±1.6 standard error, SE) and 11.7 (±1.9 SE) for the twice-daily (BID) and three-times-daily arms (TID), respectively, at Day 85 (P < .001). The improvement in the mean number of meibomian glands secreting clear liquid from baseline was also statistically significant and clinically meaningful, with an increase of 4.8 (±0.8 SE) and 5.3 (±1.1 SE) glands for the BID and TID arms, respectively, at Day 85 (P<0.001). In July, the FDA approved TP-03 (Xdemvy) for Demodex blepharitis.
Exonate reports that its EXN407, a topical treatment for diabetic macular edema, met its endpoints in a Phase Ib/IIa clinical trial. The study results showed no clinically meaningful changes or trends in safety parameters, no tolerability issues, no serious or severe adverse events, and signals of biological activity.
Ocugen hit a milestone in the development of OCU410 (AAV-RORA), a modifier gene therapy candidate being evaluated for dry age-related macular degeneration (AMD). The first patient was dosed in Ocugen’s Phase I/II clinical trial in a successful surgery.
Ophtec, maker of intraocular products, has launched Precizion Go, a hybrid acrylic intraocular lens (IOL) designed to provide distance and enhanced intermediate vision. It’s available in Europe, South Africa and South Korea. Professor Youngsub Eom, MD, PhD, from the Korean University College of Medicine and Ansan Hospital, performed one of the first surgeries in the marketing study. He said, “The…Precizon Go IOL…provides excellent far and intermediate vision without causing dysphotopsia and corrects the corneal positive spherical aberration according to the pupil size." See Precizion Go.
The FDA has approved the New Drug Application (NDA) for a single administration per eye of travoprost intracameral implant (iDose TR) 75 mcg. According to a statement from drugmaker Glaukos the product is intended to lower IOP in patients with open-angle glaucoma.
The Phase III Diamond-1 trial to evaluate OCS-01 from Oculis is officially underway. The study team achieved first patient first visit: The drug will potentially treat inflammation and pain after cataract surgery.
Azura Ophthalmics announced that its Phase II study of AZR-MD-001, a drop for patients with contact lens discomfort and signs of Meibomian Gland Dysfunction (MGD), met its primary endpoints. Patients in the AZR-MD-001 treatment arm gained at least three hours of comfortable contact lens wear time every day with improvements in tear break-up time and ocular surface staining.
The FDA granted regenerative medicine advanced therapy (RMAT) designation to OCU400 from Ocugen. The gene therapy is designed to treat retinitis pigmentosa associated with RHO mutations.
Clearside Biomedical completed randomization for its Phase IIb Odyssey clinical trial of CLS-AX (axitinib injectable suspension), a product designed to treat wet age-related macular degeneration (AMD). The company expects to have topline results in Q3 2024, according to a company statement.
Outlook Therapeutics is making progress in the advancement of ONS-5010/Lytenava, the investigational ophthalmic formulation of bevacizumab: It submitted a Special Protocol Assessment (SPA) request with the FDA for the required additional adequate and well-controlled study of the wet AMD treatment.
Nicox has screened its first patient in its Phase IIIb trial to investigate the dual mechanism of action (nitric oxide and prostaglandin analog) of NCX 470 to lower IOP. The novel nitric oxide (NO)-donating bimatoprost eye drop is the company’s lead product. Nicox said it plans to enroll about 20 healthy volunteers with ocular hypertension in a double-masked, placebo-controlled study that will investigate the action of NCX 470 on aqueous humor parameters, including trabecular meshwork outflow and episcleral venous pressure. Each patient will participate in the trial for about eight days. The trial is expected to take about one year.
Formycon and its license partner Klinge Biopharma announced that the European Medicines Agency has accepted the marketing authorization application for FYB203, a biosimilar candidate to aflibercept.
Belite Bio announced the National Medical Products Administration in China has granted approval to initiate the Phase III clinical trial of Tinlarebant for geographic atrophy (GA). Known as PHOENIX, this Phase III, multicenter, double-masked, placebo-controlled, randomized, fixed-dose clinical study is designed to evaluate the efficacy and safety of Tinlarebant in patients with GA associated with dry age-related macular degeneration. PHOENIX is already underway in the United States, Taiwan, Australia, the United Kingdom, and Switzerland. Approximately 430 subjects are targeted for enrollment with a 2:1 randomization (tinlarebant:placebo).
Annexon has outlined its global registration program for ANX007, a first-in-class C1q and classical complement inhibitor, for the treatment of patients with GA, and reported it has gained alignment with the US Food and Drug Administration on a Phase III registration program that includes using, for the first-time, the prevention of ≥15-letter loss of best corrected visual acuity (BCVA) as the primary outcome measure, as well as conducting a comparison of ANX007 to an injection agent, consistent with requests for trials across ophthalmic indications. Notably, the FDA has not required Annexon to study the slowing of lesion growth as measured by fundus autofluorescence, an anatomical endpoint used for the approval of other GA programs.
Annexon’s registration program will launch with ARCHER II, a global sham-controlled trial designed to confirm the results from the Phase II ARCHER trial. Given the availability of FDA-approved treatments in the United States, Annexon said it plans to conduct its injection-controlled head-to-head study, ARROW, against pegcetacoplan. ARCHER II is expected to begin enrollment in mid-2024, followed by ARROW in late 2024.
Ocugen reports dosing the first patient in the ArMaDa Phase I/II clinical trial of OCU410 (AAV-RORA), a modifier gene therapy candidate being developed to treat dry age-related macular degeneration (AMD). This trial will assess the safety and efficacy of OCU410 for geographic atrophy secondary to dry AMD and will be conducted in two phases. Phase I is a multicenter, open-label, dose-ranging study. Phase II is a randomized expansion phase in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.
The European Commission has granted marketing authorization in the European Union for Eylea 8 mg (aflibercept 8 mg, Bayer) for the treatment of neovascular age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME), the same indications for which Regeneron Pharmaceutical’s Eylea HD has been approved in the US. Bayer holds the ex-US franchise for aflibercept.
LumiThera has filed a De Novo request with the US Food and Drug Administration to reclassify its Valeda Light Delivery System as a Class II device. Valeda uses photobiomodulation (PBM) treatment for ocular damage and disease. LumiThera says the De Novo request reflects the benefit-to-risk profile and allows for novel devices to reach the market and provides for special controls to maintain the safety and efficacy of the device. LumiThera submitted the US LIGHTSITE III clinical data as part of a technical package to the FDA under a Premarket Approval application in 2023. The company could anticipate the FDA action on the De Novo request enabling US market introduction by mid-2024.
OKYO Pharma Ltd. reports positive safety and efficacy results in the Phase II, randomized, double-masked, placebo-controlled trial evaluating the safety and efficacy of OK-101 ophthalmic solution in dry eye disease (DED). This first-in-human trial of OK-101 established a clear and informed path for further development in Phase III registration trials. The Phase II trial enrolled 240 subjects with DED dosed twice-daily (BID). Long story short: OK-101 demonstrated superiority vs. placebo at day 29 with a similar safety profile.
EyePoint Pharmaceuticals reports dosing the first patient in the Phase II VERONA clinical trial of EYP-1901 for diabetic macular edema (DME). EYP-1901 is an investigational sustained delivery therapy containing vorolanib, a selective tyrosine kinase inhibitor formulated in the bioerodible Durasert E insert. VERONA is enrolling DME patients previously treated with a standard-of-care anti-VEGF therapy. The three-arm trial is expected to enroll approximately 25 patients assigned to one of two intravitreal doses of EYP-1901 or an aflibercept control.
OcuTerra announced completion of the last patient’s final visit in the Phase II DR:EAM (Diabetic Retinopathy: Early Active Management) clinical trial of nesvategrast (OTT166), a small-molecule selective integrin inhibitor delivered via eye drop, in diabetic retinopathy.
Tarsier Pharma has received agreement from the US Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the clinical trial protocol and planned statistical analysis of the Tarsier-04 Phase III trial to evaluate TRS01 eye drops for the treatment of noninfectious uveitis, including uveitic glaucoma. A SPA agreement indicates FDA concurrence with the study protocol. Tarsier-04 will be a multicenter, randomized, double-masked, active-controlled trial. It is planned to enroll up to 300 patients in the US.
Atsena Therapeutics announced the FDA has granted Rare Pediatric Disease designation to ATSN-101, an investigational gene therapy being evaluated in an ongoing Phase I/II clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in the GUCY2D gene (LCA1). The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.
Pursuant Health has received 510(k) clearance from the FDA for its newest kiosk model equipped with retinal imaging technology. Pursuant reports the kiosk is the first to offer self-service retinal imaging, enabling individuals to capture their own images. Ophthalmologists and optometrists can remotely access the images to evaluate pathology.
LumiThera has initiated what it claims is the largest trial of the photobiomodulation (PBM) treatment platform in patients with dry age-related macular degeneration (AMD). The multicenter clinical registry study of the Valeda Light Delivery System will enroll 500 to 1,000 patients treated with PBM over the past three years. Known as EUROLIGHT, the study will initially include up to 20 European centers and could be expanded in other countries outside the Europe Union where Valeda is approved.
Aviceda Therapeutics has reported positive topline data from Part 1 of the Phase II/III SIGLEC trial of AVD-104 in patients with geographic atrophy (GA) secondary to AMD. The data demonstrated positive safety and efficacy outcomes observed at three months after a single dose of AVD-104. SIGLEC is a two-part Phase II/III US-based trial. Part 1 of the trial, a multicenter, open-label safety and dose-escalation trial of 30 patients who received a single intravitreal injection of AVD-104 and were followed for three months, has been completed.
REGENXBIO reported positive interim data from the Phase II AAVIATE trial of ABBV-RGX-314 for the treatment of wet AMD using suprachoroidal delivery. ABBV-RGX-314 is designed to be a one-time, in-office treatment that has the potential to sustain constant anti-vascular endothelial growth factor (VEGF) therapy and stabilize or improve vision long-term for wet AMD patients. The data include six-month results from two additional dose level 3 cohorts (Cohorts 5 and 6).
Ocular Therapeutix announced that the US Food and Drug Administration has agreed to a Special Protocol Assessment (SPA) agreement modification for its pivotal Phase III SOL clinical trial of Axpaxli, the axitinib intravitreal implant also known as OTX-TKI, for the treatment of wet age-related macular degeneration (AMD). The modification allows the trial to include treatment-naïve wet AMD subjects with visual acuity of approximately 20/80 or better at the initial screening. It also allows the pivotal trial to move forward. The pivotal trial would evaluate Axpaxli with a single optimized implant with a drug load of 450 µg of a more soluble form of axitinib, a configuration expected to provide a slightly increased daily drug release.
Apellis Pharmaceuticals reports that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan, known as Syfovre in the US, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Apellis says it plans to seek immediate re-examination of its application. The MAA is based on 24-month results from the Phase III OAKS and DERBY studies.
Ocular Therapeutix announces that the US Food and Drug Administration has agreed to a Special Protocol Assessment (SPA) Agreement Modification for the pivotal Phase III clinical trial of Axpaxli (axitinib intravitreal implant, also known as OTX-TKI) for the treatment of wet AMD. The SPA Agreement Modification enables the trial to include treatment-naive wet AMD subjects with visual acuity of approximately 20/80 or better at the initial screening visit. The modification also allows the pivotal trial to move forward with a single optimized implant with a drug load of 450 µg of a more soluble form of axitinib, which Ocular says is expected to provide for a slightly increased daily release of the drug.
Genentech/Roche, is reporting new 72-week data from two global Phase III studies, BALATON and COMINO, evaluating Vabysmo (faricimab-svoa) in macular edema due to branch and central retinal vein occlusion (BRVO and CRVO). Whereas available RVO treatments are typically given every one to two months, BALATON data showed nearly 60% of patients people getting Vabysmo and up to 48% in COMINO extended their treatment intervals to three or four months. Results will be presented virtually this weekend at Angiogenesis, Exudation, and Degeneration 2024, organized by Bascom Palmer Eye Institute at the University of Miami.
Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases, has launched the Phase Ib/IIa trial to investigate oral danegaptide for treatment of diabetic macular edema (DME). Danegaptide targets the core pathological events in diabetic retinopathy, including cell-cell uncoupling, apoptotic vascular cell death, and vascular leakage at earlier stages of disease progression.
SparingVision reports the Phase I/II clinical trial of SPVN06, its gene-agnostic investigational gene therapy for the treatment of retinitis pigmentosa (RP), has now progressed to the final dose cohort of the dose-escalation phase (Part 1). This positive recommendation from the Data Safety Monitoring Board represents a further step toward the commencement of the controlled part of the study (Part 2) anticipated to start in Q2 2024, in line with study development timelines.
Iridex Corp. and Imperial College Healthcare NHS Trust have enrolled the first patient in a collaborative study and registry evaluating the treatment of glaucoma with MicroPulse transscleral laser therapy (TLT) using Iridex’s Cyclo G6 laser and the MicroPulse P3 delivery device. This partnership establishes a large-scale enrollment platform to advance clinical research.
Aviceda Therapeutics reports dosing the first patient in Part 2 of the Phase II/III SIGLEC trial for AVD-104 in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). SIGLEC is evaluating safety and efficacy outcomes of AVD-104 and an active comparator (avacincaptad pegol [Syfovre, Apellis Pharmaceuticals]). Part 1 of the SIGLEC trial enrolled 30 patients who received a single intravitreal injection of AVD-104 and were followed for three months. In Part 1, all patients tolerated a single dose of AVD-104 at month three, and no drug-related ocular or systemic severe adverse reactions were observed.
Here are four clinical trial readouts from last weekend’s virtual Angiogenesis, Exudation, and Degeneration 2024, organized by Bascom Palmer Eye Institute:
- Genentech reported 72-week data from two global Phase III studies, BALATON and COMINO, evaluating Vabysmo (faricimab-svoa) in macular edema due to branch and central retinal vein occlusion (BRVO and CRVO). Available RVO treatments are typically given every one to two months, but the data showed nearly 60% of people receiving Vabysmo in BALATON and up to 48% in COMINO were able to extend their treatment intervals to three or four months apart. In addition, patients in the studies maintained vision gains achieved in the first 24 weeks of the studies for more than one year.
- 4D Molecular Therapeutics reported positive interim data from the Phase II PRISM trial evaluating intravitreal 4D-150 in wet AMD patients with severe disease activity and a high treatment burden. Twenty-four-week results from the expansion cohort showed a single intravitreal dose of 4D-150 demonstrated favorable safety results through up to 48 weeks of follow-up. The trial used two 4D-150 treatment groups: high-dose (3E10 vector genes per eye [vg/eye]); and low-dose (1E10 vg/eye). Results showed an 89% and 85% reduction in annualized anti-VEGF injection rates in the high- and low-dose arms, respectively.
- EyePoint Pharmaceuticals reported results from new subgroup analyses from the Phase II DAVIO 2 clinical trial of EYP-1901, an investigational sustained delivery maintenance treatment for wet AMD combining the selective tyrosine kinase inhibitor vorolanib with the bioerodible Durasert E intraocular insert. In patients who did not need supplemental treatment for up to six months, the EYP-1901 treatment groups had a greater change in BCVA than the aflibercept control arm. Treated patients also had an 85% average reduction in treatment burden for up to six months.
Ocuphire Pharma reported data from the Phase II ZETA-1 trial evaluating oral APX3330 in diabetic retinopathy (DR). Only 5% of APX3330-treated patients had a clinically meaningful ≥ 3-step worsening in Diabetic Retinopathy Severity Scale (DRSS) score at week 24 vs. 13% of placebo patients. APX3330 also showed favorable safety and tolerability in patients who continued taking their diabetes medications through the study duration.
Alimera Sciences reports that the UK’s National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending that phakic patients, those with a natural lens who are being treated for chronic diabetic macular edema (DME), have on-label access to Iluvien 190 µg intravitreal implant in the applicator (fluocinolone acetonide). The final guidance for Iluvien in phakic eyes will be issued after February 22, 2024, when it will be combined with a previous NICE recommendation for patients with a pseudophakic lens, and reissued as a single updated guidance from NICE. Alimera says National Health Service (NHS) England has agreed to provide funding to implement this guidance 30 days after publication, and that NHS Wales will usually fund it within two months of the final draft guidance.
The US Food and Drug Administration has cleared OK-101 as the first Investigational New Drug (IND) application for the treatment of neuropathic corneal pain (NCP), OKYIA Pharma reports. The determination greenlights a Phase II study, designed as a double-masked, randomized, 12-week placebo-controlled trial, comparing OK-101 to placebo in NCP patients. It would enroll 54 patients with confirmed NCP disease. OKYO Pharma says the trial will begin in Q2. Pedram Hamrah, MD, of Tufts Medical Center, Boston, will lead the single-center study.
Eyenovia reports that its manufacturing plant in Redwood City, CA, has successfully completed FDA inspection. The plant will primarily be used for final assembly, packaging, and labeling for Mydcombi, the tropicamide-phenylephrine combination for in-office and presurgical pupil dilation using the Optejet technology. The facility adds to existing plants in Reno, NV, as well as its contract manufacturer, Coastline International.
Opthea has completed enrollment in the COAST Phase III pivotal clinical trial of sozinibercept, also known as OPT-302, in combination with aflibercept (Eylea, Regeneron Pharmaceuticals) for the treatment of neovascular age-related macular degeneration (nAMD). Sozinibercept inhibits vascular endothelial growth factor (VEGF)-C/D, while aflibercept inhibits anti-VEGF-A. COAST is one of two Phase III trials in the sozinibercept program. The other, ShORe, is expected to complete enrollment in Q2 2024. Opthea says it intends to report topline results from these two trials by mid-2025.
Ocular Therapeutix reports the first three subjects have been screened and received their first aflibercept injection in the Phase III SOL-1 clinical trial of Axpaxli, the axitinib intravitreal implant also known as OTX-TKI, for nAMD. Axpaxli is an investigational bioresorbable, hydrogel implant incorporating axitinib, a small-molecule, multi-target, tyrosine kinase inhibitor.
The 47th Annual Meeting of the Macula Society last week in Palm Springs, CA, provided a podium for these three trial updates:
- Twelve-week data from the Phase Ib/IIa AMARONE trial of Restoret in patients with treatment-naive nAMD and diabetic macular edema (DME). Eyebiotech is developing Restoret, an investigational tri-specific Wnt agonist antibody designed to eliminate vascular leakage by activating the Wnt pathway to both restore and maintain the blood-retinal barrier. The 12-week data demonstrated that multiple monthly doses of Restoret, as both monotherapy and in combination with aflibercept, were well tolerated, with no drug-related adverse events or intraocular inflammation. Patients with DME (n=26) and nAMD (n=5) who received Restoret monotherapy showed improvements in best-corrected visual acuity and reduction in retinal thickness.
- Interim 12-month safety and efficacy results for the Phase II SKYLINE trial of AGTC-501 in X-linked retinitis pigmentosa (XLRP) demonstrated a 63% response rate in study eyes treated with high-dose AGTC-501 (6.8 E+11 vector genes per eye [vg/eye]), while low-dose AGTC-505 (7.5 E+10 vg/eye) failed to show a response rate, study sponsor Beacon Therapeutics Holdings reports. The study defines response rate as an improvement in microperimetry-measured retinal sensitivity. High-dose patients also demonstrated a robust improvement in visual function, including mean retinal sensitivity, as well as good tolerance and no associated clinically significant safety events, Beacon says.
Twenty-six-week results from the LUNA Phase II trial of the gene therapy ixoberogene soroparvovec, also known as Ixo-vec and ADVM-022, in nAMD demonstrated significant reduction in the need for anti-VEGF injections in recipients, trial sponsor Adverum Biotechnologies reports. Annualized reduction in anti-VEGF injection rates were 90% (n=19) in the 6E+10 vg/eye group and 94% (n=20) in the 2E+11 vg/eye group. Also, 68% and 85% of patients in the respective cohorts didn’t need supplemental anti-VEGF treatments. The results also demonstrated maintenance of visual acuity and central subfield thickness, an anatomical biomarker of retinal integrity.
OKYO Pharma (NASDAQ: OKYO) reports the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for OK-101 for the treatment of neuropathic corneal pain (NCP), a painful ocular surface condition for which no FDA-approved therapy exists. Based on the action, a Phase II study will evaluate OK-101 versus placebo in NCP patients. OKYO says the trial is scheduled to begin in Q2.
Palatin Technologies reports that data analysis is underway in the MELODY-1 Phase III trial of PL9643 for dry eye disease (DED). The trial is evaluating the safety and efficacy of PL9643 versus vehicle. The trial has two coprimary efficacy endpoints: one clinical sign of DED and one clinical symptom of DED. The Phase III MELODY-1 trial enrolled 575 patients in the US. The remainder of the Phase III program, which includes the Phase III safety and efficacy study MELODY-2 and the open label MELODY-3 study, are expected to begin enrolling patients in the second half of the year.
jCyte reports a “successful outcome” of its pre-Phase III Type B meeting with the US Food and Drug Administration, clearing the way for it’s the pivotal US trial of jCell, its investigative treatment for retinitis pigmentosa and other degenerative retinal disorders, in the second half of the year.
Neurophth Therapeutics says it has completed enrollment in the Phase I/II clinical trial of Opvika (esonadogene imvoparvovec) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON). The first patients were dosed in June 2023.
Ocugen reports that it has completed dosing in the first cohort of the Phase I/II GARDian clinical trial of OCU410ST (AAV5-hRORA), a modifier gene therapy candidate being developed to treat Stargardt disease, an inherited retinal disease that affects around 35,000 people in the US. Up to 10 US retinal surgery centers across are participating in the trial. In the first cohort, three patients received 200 L single subretinal administration of the low dose (3.75x1010 vg/mL) of OCU410ST. Phase I will also evaluate the safety of unilateral subretinal administration of medium dose (7.5×10E10 vg/mL), and high dose (2.25×10E11 vg/mL) OCU410ST. Phase II is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to sham.
The US Food and Drug Administration has cleared the Investigational New Drug application filed by IView Therapeutics to for the initiate a Phase I/II clinical trial of IVW-1001, a novel TRPM8 agonist—it stands for transient receptor potential melastatin subtype 8, also known as the cold and menthol receptor 1—to treat signs and symptoms of dry eye disease. iView says it anticipates launching the trials in Q2 this year.
Innovent Biologics reports results of two clinical studies of IBI311, an anti–insulin-like growth factor 1 monoclonal antibody indicated for treatment of thyroid eye disease (TED). A Phase I study in healthy volunteers showed that the drug was safe and well tolerated, with low-level treatment-emergent adverse events, most of which resolved. A Phase II trial of 33 patients randomized to IBI311 or placebo met its primary 12-week endpoint, with a response rate of 59.1% for the treatment group vs. 18.2% in the placebo arm.
Atsena Therapeutics has initiated dosing in the second cohort of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS). LIGHTHOUSE is an open-label, dose-escalation, and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients aged 6 years and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in the RS1 gene. Enrollment in the trial is ongoing.
GenSight Biologics reports initial results of new meta-analyses evaluating Lumevoq gene therapy in Leber hereditary optic neuropathy (LHON). Patients treated with Lumevoq (also known as GS010 and lenadogene nolparvovec) reportedly had a greater rate of visual recovery than did untreated patients or those treated with idebenone. The meta-analyses are the first to focus solely on patients with the m.11778G>A ND4 mutation.
Telios Pharma reports topline results from its Phase II study evaluating TL-925, a topical Bruton’s tyrosine kinase (BTK) inhibitor for individuals with moderate to severe dry eye disease (DED). This proof-of-concept, multicenter, randomized, double-masked study evaluated the safety, tolerability, and efficacy of TL-925 versus vehicle control. Some 107 patients with moderate to severe DED were dosed twice daily for 28 days. The study found TL-925 safe and well tolerated, and the intention-to-treat analysis demonstrated clinically meaningful and statistically significant efficacy relative to vehicle control across multiple signs and symptoms. Complete results will be presented at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in Seattle in May.
Valo Health has completing enrollment in the Phase II Spectra evaluating oral OPL-0401 in patients with moderately severe or severe non-proliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy (PDR). OPL-0401 is an investigational small molecule Rho kinase (ROCK) 1/2 inhibitor. The company anticipates a topline readout before year’s end.
The first patient has been randomized in the DRCR Retina Network’s Protocol AL trial evaluating Alimera Sciences’ Iluvien (fluocinolone acetonide 0.19 mg) intravitreal implant in patients with primary choroidal melanoma receiving plaque brachytherapy treatment. The trial will enroll 600 patients at risk for vision loss from radiation retinopathy, randomizing them to Iluvien or Vabysmo (faricimab 6 mg, Genentech/Roche). It will assess development of macular edema and associated long-term visual acuity effects of consistent and continuous release of corticosteroid or repeated injections of anti-VEGF initiated near the time of radiation therapy compared to observation until macular edema develops in patients at risk for radiation retinopathy.
Biotech startup Oculogenex is scheduled to take a ride to the International Space Station (ISS). Actually, its investigative gene therapy treatment for age-related macular degeneration (AMD) is. The platform was set to launch on SpaceX’s 30th Commercial Resupply Services (CRS) mission on Thursday, March 21. The goal of the research is to evaluate spaceflight as a novel biomarker of intermediate AMD, according to the ISS National Laboratory. The research aims to determine whether gene therapy can thwart spaceflight-induced retinal dysfunction and degeneration.
Ocugen reports completing dosing in the first cohort of three patients in its Phase I/II ArMaDa clinical trial for OCU410 (AAV-hRORA), a modifier gene therapy candidate being developed for geographic atrophy (GA). Up to 13 retina surgery centers across the US are participating in the clinical trial. The first cohort of patients received 200-µL single subretinal administration of the low-dose (2.5x1010 vg/mL) of OCU410.
OcuTerra Therapeutics reports not-so-good news in topline results from the Phase II DR:EAM (Diabetic Retinopathy: Early Active Management) clinical trial of nesvategrast (OTT166), a selective RGD integrin inhibitor developed as an eye drop. The trial failed to meet its primary efficacy endpoint of the percentage of patients that have a more than two-step improvement in the Diabetic Retinopathy Severity Scale (DRSS), which measures the severity of diabetic retinopathy. The trial also failed to meet key secondary efficacy endpoints: measuring the prevention of progression to vision-threatening events; amount of delayed time to intravitreal injection and/or laser treatment; and exploratory imaging endpoints. The trial did meet its primary safety endpoint. OcuTerra says it plans to evaluate its strategic alternatives and will share additional details in the future.
Innovent Biologics reports that the second Phase II clinical trial of efdamrofusp alfa (IBI302) high-dose, a recombinant human VEGFR-Fc-human CR1 fusion protein injection, met the second primary endpoint in patients in China with neovascular age-related macular degeneration (nAMD). The two trials compared IBI302 with Eylea (aflibercept 2 mg, Regeneron Pharmaceuticals). The results showed that the primary endpoint was met. At week 40, the IBI302 6.4-mg and 8-mg groups showed non-inferior best-corrected visual acuity gains compared to the aflibercept group. Average change in central subfield thickness was also comparable between the two treatment groups.
Belite Bio has submitted applications to the US Food and Drug Administration as well as the Pharmaceuticals and Medical Devices Agency in Japan to initiate the Phase II/III portion of the DRAGON II clinical trial of Tinlarebant in adolescent patients with Stargardt disease. DRAGON II is a global, multicenter, double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety and tolerability of Tinlarebant, also known as LBS-008. The trial would enroll around 60 patients, ages 12 to 20 years, including approximately 10 in Japan. They’ll be randomized 1:1 to Tinlarebant or placebo.
Outlook Therapeutics has secured a positive opinion in Europe for ONS-5010/Lytenava, its ophthalmic formulation of bevacizumab (Avastin, Genentech/Roche). The company reports that the European Medicines Agency (EMA) has issued a positive opinion on the application. The approval covers the treatment of wet age-related macular degeneration (AMD). Outlook says it’s evaluating both direct commercialization of the product and partnering in Europe on a country-by-country basis. The positive opinion supports the grant of marketing authorization by the European Commission, which is expected to make a decision within approximately 67 days following the positive opinion. The decision will apply automatically in all 27 EU member states, and, within 30 days, also to Iceland, Norway, and Liechtenstein.
Iveric Bio, an Astellas company, reports that the US Centers for Medicare and Medicaid Services has assigned a J-code for Izervay (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy secondary to age-related macular degeneration (AMD). The J-code goes into effect April 1 and will enable providers to obtain reimbursement for administering Izervay.
OKYO Pharma reports additional key findings from analyses of the clinical data set from the Phase II trial evaluating OK-101 (0.05%) ophthalmic solution in 240 patients with dry eye disease (DED). The additional findings include:
- Statistically significant improvements in ocular pain relief, tear-film break-up time, and burning/stinging, and blurred vision as early as day 15 that lasted throughout the trial.
- Improved conjunctival staining as early as day 29, again with a sustained benefit.
The observed endpoints support the proposed mechanism-of-action of OK-101 as demonstrated in preclinical animal models, OKYA says.
BlueRock Therapeutics, a clinical-stage cell therapy company and wholly owned independent subsidiary of Bayer, and the Foundation Fighting Blindness say they’re collaborating to add a new cohort to the Foundation’s Uni-Rare natural history study of people living with inherited retinal diseases (IRDs) caused by rare single-gene mutations. BlueRock plans to use study data to identify and develop outcome measures for clinical trials in IRDs. Launched in 2022, the Uni-Rare study is enrolling around 1,500 people with one of more than 300 rare genes associated with IRDs, including retinitis pigmentosa, Leber congenital amaurosis, and Usher syndrome. Study leader is José-Alain Sahel, MD, at the University of Pittsburgh School of Medicine.
The first cohort has completed dosing in the open-label, dose-escalation Phase I/II clinical trial evaluating subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene. Trial sponsor Opus Genetics says that positive safety and efficacy data from the first cohort of three adult patients enable advancing into the next highest dose, which Opus anticipates initiating in the next cohort in mid-year.
Nanoscope Therapeutics reports positive top-line results after the completion of the 2-year Phase IIb RESTORE randomized, controlled clinical trial of MCO-010, which the company describes as a mutation-agnostic gene therapy for patients with permanent and severe vision loss from advanced retinitis pigmentosa.
The trial met its primary endpoint: demonstrating a statistically significant improvement of best-corrected visual acuity (BCVA) at week 52 in both the high-dose and low-dose treatment groups vs. sham. Improvements in visual function persisted or increased after one year after a single intravitreal injection of MCO-010. BCVA improvement at week 76, a key secondary endpoint, was statistically significant in the high-dose treatment group vs. sham.
Two-year data from the Phase I/IIa trial of the gene therapy ABBV-RGX-314 (RegenxBio), a subretinal, one-time treatment for neovascular, or wet, age-related macular degeneration (nAMD), have been published in the journal Lancet. The study demonstrated that a single administration of ABBV-RGX-314 was generally well tolerated. Patients who received therapeutic doses demonstrated sustained levels of the ABBV-RGX-314 protein in the eye and stable or improved vision and retinal anatomy with few or no supplemental anti-VEGF treatments. RegenxBio also reports additional interim data from a long-term follow-up study showing that treatment was well-tolerated and demonstrated long-term treatment effect for up to four years.
LENZ Therapeutics (Nasdaq: LENZ) announces positive Phase 3 CLARITY study results for aceclidine-based eye drops LNZ100 and LNZ101, demonstrating significant near vision improvements for presbyopia patients without compromising distance vision, with LNZ100 identified as the lead candidate. With rapid onset, long-duration efficacy, and high tolerability observed across over 30,000 treatment days, LNZ100 shows promise as a therapeutic option, prompting plans for an NDA submission in mid-2024 and potential commercialization efforts by the second half of 2025 upon FDA approval. These robust study results reinforce LNZ100's potential as an effective treatment for presbyopia, with anticipated commercial efforts aiming to address the needs of millions experiencing such symptoms in the United States.
The first patient has been dosed in the Phase Ib clinical trial evaluating intravitreal injection of VG901 to treat retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene, trial sponsor ViGeneron reports. VG90 delivers a functional CNGA1 gene to retinal photoreceptor target cells. The trial is an open-label, single-arm, dose-escalation study evaluating the safety, tolerability, and preliminary efficacy of VG901.
The following trial readouts are from the annual meeting of the American Society of Cataract and Refractive Surgery in Boston:
- Results from the Phase III OPTIMIZE trial of OCS-01 for inflammation and pain after cataract surgery showed that 57.2% of treated patients were inflammation-free at day 15 (vs. 24% with vehicle), and 75.5% had no ocular pain at day 4 (vs. 52% with vehicle), trial sponsor Oculis Holding reports. Oculis says a topline readout from the second Phase III OPTIMIZE-2 trial is anticipated later this year, and that it would support a New Drug Application (NDA) submission. OCS-01 is a once-daily, topical, preservative-free corticosteroid.
Ocular Therapeutix reports positive Phase II data for Paxtrava (travoprost intracameral implant, or OTX-TIC) in patients with open-angle glaucoma or ocular hypertension. The trial showed statistically significant reductions of intraocular pressure (IOP) of ~24% to 30% with a single implant over six months. Paxtrava was also reportedly well tolerated with no detrimental impact on corneal health.
The first subject has been dosed in the LYNX-2 Phase III trial evaluating Phentolamine Ophthalmic Solution 0.75% (PS) for treatment of decreased visual acuity under low-light conditions following keratorefractive surgery, trial sponsor Ocuphire Pharma reports. The trial is being conducted under conditions of a Special Protocol Assessment with the US Food and Drug Administration.
The American Journal of Ophthalmology International has published a large-scale, comparative real-world clinical outcomes study of patients treated by three leading, minimally invasive glaucoma surgery (MIGS) technologies, trial sponsor Sight Sciences reports. Using the American Academy of Ophthalmology’s IRIS Registry, the trial evaluated two-year postsurgical outcomes among patients with primary open-angle glaucoma treated with the most commonly used US Food and Drug Administration–approved ab interno MIGS devices: Omni Surgical System, Hydrus Microstent, and iStent inject, either combined with cataract surgery or for cataract surgery alone. The study showed that patients with both high- and low-baseline intraocular pressures (IOP) — with 18 mmHg as the demarcation between the two — who had the outflow procedure using the Omni platform had the greatest reduction in IOP as well as reduction in medication use. Sight Sciences funded the study.
Ocugen reports that it has completed dosing in the second cohort of its Phase I/II ArMaDa clinical trial for OCU410 (AAV-hRORA), a modifier gene therapy candidate being developed for geographic atrophy (GA). Three subjects received 200-mL, single subretinal administration of the medium dose (5x1010 vector genes [vg]/mL) of OCU410. Up to 13 US retinal surgery centers are participating in the ArMaDa clinical trial.
Sleep technology of all things has been shown to improve visual acuity in people with wet, or neovascular, age-related macular degeneration (nAMD), according to Somavedic, the company developing the technology. The study enrolled 18 patients with newly diagnosed nAMD and active choroidal neovascularization (CNV) for whom anti–vascular endothelial growth factor (VEGF) therapy was indicated. After one month, patients using the Somavedic device had a mean improvement of +4.2 Early Treatment Diabetic Retinopathy Study (ETDRS) letters in best corrected visual acuity (BCVA), accompanied by a reduction of -7.6 μm in central retinal thickness (CRT). Patients in the control group reportedly showed minimal changes in BCVA and an increase of +75 μm in CRT.
Ocular Therapeutix reports what it says are positive topline results from the Phase I HELIOS study of Axpaxli in patients with moderately severe to severe nonproliferative diabetic retinopathy (NPDR) without diabetic macular edema (DME). The study found that Axpaxli was well tolerated overall with no inflammation observed, including no cases of iritis, vitritis, or vasculitis. Six of 13 (46.2%) patients in the Axpaxli group had a one- or two-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at 40 weeks, with two having a two-step improvement. No patients in the control group showed a one- or two-step improvement at the same time point. No patients in the Axpaxli group experienced any worsening in DRSS vs. one of eight (12.5%) in the control group at 40 weeks. Ocular reports a single injection of Axpaxli provided durable DRSS improvement up to 40 weeks, and that no patients in either arm received rescue treatments. Ocular says the results support moving directly to a Phase III study, and plans to present topline results at a future meeting.
Iyuzeh (latanoprost ophthalmic solution) 0.005% from Théa Pharma is now available in the US. The company says it’s the first and only preservative-free latanoprost for patients with primary open-angle glaucoma (POAG) and ocular hypertension (OHT).
Lumify Eye Illuminations, a new line of hypoallergenic specialty eye care products from Bausch + Lomb, is also now on the US market. The line includes eye makeup remover, a lash and brow conditioner, and an eye cream.
Orbis International announced two partnerships over the past week. With support from Alcon Foundation, OMEGA, Orbis is rolling out a surgical training program in Zambia via its Flying Eye Hospital. It also entered into a strategic partnership with Siloam Vision to expand eye care access. Orbis will use Siloam Vision’s cloud-based telemedicine platform to deliver care to people in hard-to-reach communities.
Unity Biosciences announced results from its Phase IIb study of UBX1325 (foselutoclax) for patients with wet AMD who were not responding to their current anti-VEGF therapy. Top takeaways: Patients who switched to UBX1325 maintained visual acuity through 24 weeks and had a 1.5-letter decrease at week 48.
The FDA approved Ryzumvi (phentolamine ophthalmic solution) 0.75% for the treatment of pharmacologically induced mydriasis produced by adrenergic agonists or parasympatholytic agents. Developed by Ocuphire Pharma and Viatris, Ryzumvi will be the only commercially available treatment for reversal of dilated eyes. It’s expected to be available in the US in the first half of 2024.
Dopavision has completed enrollment in MyopiaX, a proof-of-concept trial to evaluate its investigational product, MyopiaX, in pediatric participants with myopia. The one-year study is being conducted at 10 clinical sites across Europe and the UK.
Apellis has made it official: The Centers for Medicare & Medicaid Services (CMS) has assigned a permanent and product-specific J-code to the geographic atrophy drug Syfovre (pegcetacoplan injection). For those that need it, the code is J2781.
NovaBay Pharmaceuticals has launched Avenova Allograft in the United States. The prescription product is the only optic allograft manufactured using proprietary processes from BioStem Technologies. It’s intended for use as a protective covering during ocular surface repair. [Learn More About Avenova]
Visual impairment and dementia go hand in hand. Dementia affects parts of the brain that process visual information; at the same time, people with both dementia and poor vision may have an even tougher time recognizing people they know.
A UK-based study found the prevalence of visual impairment is significantly higher in people with dementia, and higher still among those in assisted living and similar communities. Over half of these issues were correctable with either glasses or cataract surgery. The findings suggest a need for a specialized eye care pathway for people with dementia and perhaps specialized optometrists. [Read the Study]
Ocugen provided an update on its Phase I/II trial for OCU400, a therapy for retinitis pigmentosa (RP) and Leber congenital amaurosis. Relaying results that include additional subjects, the data show 83% of OCU400-treated subjects demonstrated stabilization or improvements in best corrected visual acuity and 42% of the OCU400-treated patients experienced four-letter improvement, among other findings.
The FDA granted 510 (k) clearance for an AI-powered ophthalmic image management system from Altris AI. Called Altris IMS, the platform is designed to store, organize, and support in-depth analysis of Optical Coherence Tomography (OCT) scans. Altris AI Chief Medical Officer Maria Znamenska, MD, PhD, said in a statement that the platform helps physicians “detect retinal pathologies, pathological signs, and biomarkers. It means less human errors, early detection of the pathologies that can lead to blindness, and better patient outcomes as a result.”
Astellas Pharma, the new parent company of Iveric Bio, announced favorable 24-month topline results from its Phase III GATHER2 clinical trial. The trial evaluated the efficacy and safety of Izervay (avacincaptad pegol intravitreal solution), recently approved for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Results indicated slowed GA growth at 24 months and safety consistent with 12-month data.
Results from Santen’s MERCURY-3 trial support the addition of Roclanda as an alternative fixed-dose combination (FDC) treatment option for patients with primary open-angle glaucoma (POAG) or ocular hypertension. The trial compared Roclanda – an FDC of latanoprost 50 mcg/mL and netarsudil 200 mcg/mL ophthalmic solution – with FDC of bimatoprost 300 mcg and timolol maleate 500 mcg/mL. The results showed comparable intraocular pressure (IOP) lowering and sustained IOP reduction with no serious adverse events.
Tarsier Pharma announced findings from its Phase III trial on TRS01 (eye drop formulation of dazdotuftide). A post-hoc analysis that evaluated the total impact of the drug vs steroids suggests a benefit-risk advantage in noninfectious uveitis; specifically, in uveitic glaucoma. Inflammation parameters were comparable to steroids. While the trial did not meet its primary endpoint, Tarsier plans to meet with the FDA to determine the best path forward, according to a statement.
Myra Vision has launched a first-in-human study of its Calibreye System, a glaucoma drainage device with titratable outflow control designed for moderate to severe glaucoma patients.
The dry eye drug Miebo (perfluorohexyloctane ophthalmic solution) from Bausch + Lomb and Novaliq is now available in the United States. Approved by the FDA in May, it’s touted as the first and only dry eye treatment that targets tear evaporation.
LKC Technologies has released a report on diabetic retinopathy (DR) to help simplify and standardize DR assessment and management. Titled “Modern Fundamentals of Diabetic Retinopathy Management in Optometry,” the report was developed by members of a 14-member optometric task force.
“There is a dire need for an elevated protocol to guide patient care in DR,” Jeffry Gerson, OD, of Grin Eye Care in Olathe, Kansas, said in a statement. “The sad reality is that the complexity of this disease and the subjectivity of many of our tests can lead to errors and vision loss.” [Get the Report]
Heidelberg Engineering, a UK-based eye care technology company, is partnering with Orbis, an international nonprofit dedicated to preventing and treating avoidable blindness. Heidelberg will fund teaching opportunities and research; specifically, training eye care professionals around the world via Cybersight (Orbis’ telemedicine and e-learning platform). Heidelberg will also fund Orbis’ research into retinoblastoma, a rare retinal cancer that most commonly affects children under age five years.
The United States Patent and Trademark Office (USPTO) has issued a US patent to Stuart Therapeutics. The patent claims support the company’s PolyCol platform of Collagen Mimetic Peptide (CMP) therapeutic compounds. PolyCol is a tissue reparative platform used in its products in development for dry eye disease and glaucoma, among others.
Opus Genetics dosed its first patient in a Phase I/II trial of OPGx-LCA5, a gene therapy developed to treat a form of Leber congenital amaurosis (LCA). The product is an adeno-associated virus 8 (AAV8) vector designed to deliver a functional LCA5 gene to the outer retina in patients with LCA resulting from biallelic mutations in the LCA5 gene.
Final data from Alcon’s Vivity Registry Study of more than 900 cataract patients produced encouraging results: Evaluating real-world patient experiences for up to 12 months post cataract surgery with implantation of either AcrySof IQ Vivity or AcrySof IQ Vivity Toric presbyopia- correcting intraocular lens, about 75% of patients reported no difficulty with their sight for everyday life activities. Most (92%) said they were satisfied with their sight, and more than 91% reported no halos, glares, or starbursts.
CVS Health, Walgreens Boots Alliance, Boiron, and five other companies received warning letters from the FDA this week for manufacturing or marketing unapproved ophthalmic products. The products at issue are drops illegally marketed to treat conjunctivitis (pink eye), cataracts, glaucoma, among other conditions. Some of the products are labeled to contain silver, which can cause problems with long-term use. Read the announcement and review the warning letters here.
Novartis has discontinued clinical development of GT005, a one-time gene therapy for geographic atrophy. An Independent Data Monitoring Committee concluded that overall data from GT005’s Phase II study “did not support continuation of the development program.” Novartis acquired GT005 as part of its acquisition of Gyroscope Therapeutics in 2021.
A study recently published in BMJ Oncology concluded that short-term use of immunosuppressant medications to control noninfectious ocular inflammatory diseases was not associated with an increased risk of cancer.
“When we got these results, I was reassured, and I hope patients will be, too,” said lead author Jeanine Buchanich, PhD, Associate Dean for Research and Associate Professor of Biostatistics at University of Pittsburgh’s Pitt School of Public Health. “Immunosuppressants are widely used and transformative for care of patients with inflammatory diseases, but the potential concern that they carry a cancer risk has forced people to make difficult decisions without enough information. Alleviating that concern with use for inflammatory diseases will help people make the treatment decision that’s right for them.” Researchers from Mass Eye and Ear also contributed to the study. [Read the Full Study]
Neurophth Therapeutics reports that the Australian Therapeutic Goods Administration registered and approved NFS-05, a therapy developed to treat autosomal dominant optic atrophy. There are no other treatments available for this inherited neuropathy.
As part of a Phase III clinical trial, ophthalmologists at UC Davis Health in Davis, California, used an experimental gene therapy to treat a patient with wet age-related macular degeneration (AMD). The drug ABBV-RGX-314 from Regenxbio, is being tested as a one-time therapy for wet AMD and diabetic retinopathy. Review study details by using NCT05407636.
The FDA declined to approve Outlook Therapeutics’ Biologics License Application (BLA) for ONS-5010 (Lytenava), an investigational ophthalmic formulation of bevacizumab intended to treat wet AMD. The reason stemmed from “several CMC [chemistry, manufacturing, and controls] issues, open observations from preapproval manufacturing inspections, and a lack of substantial evidence,” according to a company statement. If approved, Lytenava would give physicians an alternative to off-label use of repackaged oncologic IV bevacizumab.
The first patient has been dosed in Atsena Therapeutics’ Phase I/II clinical trial for ATSN-201, a subretinal injection developed to treat X-linked retinoschisis. Review study details by using NCT05878860.
Apellis updated investors and the public on the retinal vasculitis cases reported among patients using the recently approved geographic atrophy (GA) drug, Syfovre. The company blamed the events on “internal structural variations” identified in the specific 19-gauge x 1½ inch filter needle included in certain injection kits. A causal relationship between the needles and the retinal vasculitis has not been established, however.
Apellis recommends physicians stop using the 19-gauge needles and use injection kits with 18-gauge filter needles. These kits are already in distribution. According to a statement, the company estimates events of retinal vasculitis are still “very rare at an estimated real-world rate of 0.01 percent.
Apellis shares have responded well to the news, rising from $30.76 to $42.82 in one day.
Biomarkers found via retinal imaging may indicate signs of Parkinson’s disease up to seven years before the disease manifests clinically. Researchers from Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, conducted a large study on retinal imaging and Parkinson’s disease and found individuals with Parkinson’s disease have reduced ganglion cell-inner plexiform layer and inner nuclear layer thickness. [Read the Full Study]
Are your patients asking for blue-light—filtering glasses? They may want to save their money. A recent review of 17 clinical trials found blue-light lenses found no evidence of reduced eye strain, protection against retinal damage, or improvement of sleep. [Read the Abstract]
Days after Regeneron and Bayer announced positive, 48-week data from its pivotal PULSAR trial investigating an 8 mg dose of aflibercept (Eylea HD), the FDA approved the higher-dose for wet age-related macular degeneration (AMD), diabetic macular edema (DME) and diabetic retinopathy (DR). The FDA declined to approve aflibercept 8 mg about a month ago due to “inspection findings at a third-party filler.” According to an article in Fierce Pharma, Regeneron “provided proposed corrective and preventative actions to address these observations.”
The first patient has been dosed in Neurophth’s Phase I/II clinical trial of NFS-02 in patients with Leber hereditary optic neuropathy caused by ND1 mutation. A team led by Professors Jia Qu and Rong Zhou from the Eye Hospital of Wenzhou Medical University in China is overseeing the patient’s treatment.
Amber Ophthalmics enrolled its first patient in its Phase II/III NEXPEDE-1 trial to evaluate two concentrations of Nexagon (lufepirsen ophthalmic gel) for the treatment of persistent corneal epithelial defects (PCED). Mark S. Gorovoy, MD, enrolled the inaugural patient. For study details, see NCT05966493.
The European Medicines Agency (EMA) approved ViGeneron’s clinical trial application for VG901, a gene therapy designed to treat CNGA1-associated retinitis pigmentosa (RP). The therapy uses an adeno-associated virus vector to deliver the therapy via intravitreal injection.
The EMA also accepted Iveric Bio’s marketing authorization application (MAA) for potential approval of avacincaptad pegol (Izervay) for geographic atrophy (GA) treatment. With acceptance of the MAA, the EMA will start its review for potential approval.
Nonprofit Prevent Blindness has launched a new advocacy website: Advocacy.PreventBlindness.org. Check it out for policy information, skill-building resources, representative information, and more.
Staar Surgical has teamed with Tennessee Titans quarterback Will Levis to raise awareness for Staar’s EVO Implantable Collamer Lenses (EVO ICL). The lenses are designed to correct myopia and myopia with astigmatism. “The ability to wake up with sharp, clear vision has been a game-changer,” said Levis in a press release. The Staar Surgical team talk about the company’s celebrity campaigns on a recent OIS podcast. [Listen Here]
With wildfires burning everywhere from Maui to Quebec this year, wildfire smoke has affected a large portion of the North American population. Because of this, eye health experts have raised concern over the long-term impact of this toxic smoke. Recent studies have linked wildfire smoke to ocular surface disease, though results are inconclusive. For patients who present with itchy, gritty, red, watery, irritated eyes during a period of low air quality, consider these tips from VSP Vision. Check wildfire activity using AirNow’s Fire and Smoke map.
Euclid Vision Cofounder George Glady has passed away, the company announced this week. Glady helped develop the company’s Ortho-K contact lenses, which are designed to reshape the cornea in people with myopia.
And in case you haven’t heard, Precision Lens owner Paul Ehlen died in a plane crash in Montana in late June. His plane, a vintage World War II aircraft, malfunctioned on takeoff. “Paul was an innovative and courageous man, and his entrepreneurial spirit and dedication to helping others will continue,” Precision stated. “Above all else, Paul was a family man, and our thoughts and prayers are with his wife and children whom he loved so dearly.”
A month prior, Precision Lens, which provides surgical products and intraocular lenses, was hit with a $487 million judgment in a False Claims Act and Anti-Kickback Statute case. According to an article in Mass Device, the company plans to appeal.
The FDA has accepted the new drug application (NDA) for bromfenac ophthalmic solution 0.09% for the treatment of postoperative inflammation and pain. The product is the generic equivalent of Bausch + Lomb’s Bromday and was developed by Lupin, a pharmaceutical company based in Mumbai, India. Lupin will also manufacture the drug if approved.
Sandoz is making progress on its aflibercept biosimilar. Results from its Phase III study showed therapeutic equivalence in mean change of best corrected visual acuity (BCVA) from baseline to week 8 between the aflibercept biosimilar and Eylea (aflibercept 2 mg; Regeneron). Sandoz plans to file for U.S. and EU regulatory approval in the coming months.
Rayner completed the first two implants in two pivotal market access studies in the U.S. and China for the RayOne EMV Toric IOL. The inaugural U.S. implant took place at Vance Thompson Vision in Sioux Falls, South Dakota, last week. The results of the study will support a premarket approval (PMA) submission to the FDA. The second implant took place in June at The Peking University Hospital, and the results will support approval in that country.
Regeneron and Bayer announced positive, two-year (96 weeks), topline data from its pivotal PULSAR trial investigating Eylea (aflibercept 8 mg) in patients with wet age-related macular degeneration (AMD). The study demonstrated that most patients were able to maintain or further extend dosing intervals. Specifically, 88% were on a ≥12-week dosing interval at the end of two years, and 78% maintained ≥12-week dosing intervals throughout the two-year study period, compared to 83% throughout the first year of study (48 weeks).
Topline results from Oculis’ Phase III trial for OCS-01 eye drops show that the drop met its primary endpoints: absence of inflammation at day 15 and the absence of pain at day 4. It was also well tolerated. If approved, the product will be the first once-daily, topical, preservative-free corticosteroid for treating inflammation and pain following ocular surgery. The company also announced that it has enrolled the first patient in a study to evaluate OCS-01 for the treatment of cystoid macular edema (CME).
Iveric Bio, now part of Astellas, closed out last week by announcing the FDA’s approval of what is now called Izervay (avacincaptad pegol intravitreal solution). It is the second approved treatment for geographic atrophy. Astellas/Iveric says it’s the “only approved GA treatment with a statistically significant reduction (P < .01) in the rate of GA progression at the 12-month primary endpoint across two phase 3 clinical trials.” It is expected to be available in 2-3 weeks.
Nanoscope Therapeutics announced six-month results from its Phase II clinical trial for MCO-010, for patients with Stargardt disease. Highlights include clinically meaningful improvements in best-corrected visual acuity, an approximate 3 dB gain in mean sensitivity, and no serious adverse events.
The latest from Apellis on the safety of Syfovre: To date, the company is aware of seven confirmed events of retinal vasculitis. It is also evaluating an eighth case. Apellis reported no manufacturing-related issues, and no quality issues were identified. It will continue to work with the retina community and share updates as it gets them.
The FDA cleared the investigational new drug (IND) application for a Phase I/IIa clinical trial for SKG0106 from Skyline Therapeutics. SKG0106 is a gene therapy designed to treat neovascular AMD.
Okyo Pharma, in partnership with Tufts Medical Center, in Boston, will conduct an open-label clinical trial to evaluate OK-101 in patients with neuropathic corneal pain. OK-101 is also being investigated in a Phase II trial to treat dry eye disease. The product is a lipid conjugated chemerin peptide agonist of the ChemR23 G protein-coupled receptor that is typically found on immune cells of the eye responsible for the inflammatory response.
Apellis Pharmaceuticals reports that the European Medicines Agency (EMA) has reset the review of the marketing authorization application (MAA) for intravitreal pegcetacoplan, known as Syfovre in the United States, for geographic atrophy. The EMA has stated their intent to convene a new expert group meeting. Apellis says it anticipates an opinion from the Committee for Medicinal Products for Human Use (CHMP) no later than July. This update follows a March ruling by the Court of Justice of the European Union (CJEU) on the organization of EMA’s expert groups, which has implications on EMA's policy for handling competing interests of experts. Apellis adds the EMA decision “is strictly procedural in response to the CJEU judgment and is not related to the pegcetacoplan data package.” In January, the CHMP issued a negative opinion on the pegcetacoplan MAA.
Aurion Biotech reports that it has completed dosing in the Phase I/II CLARA clinical trial of AURN001, an allogeneic cell therapy for the treatment of corneal edema secondary to corneal endothelial dysfunction. AURN001 is a combination cell therapy product comprised of neltependocel (allogeneic human corneal endothelial cells) and Y-27632 (an inhibitor of Rho-associated, coiled-coil containing protein kinase). AURN001 is intended to be administered to the eye as a one-time, intracameral injection.
The first patients in the final cohort of the Phase I/II clinical trial evaluating NPI-002, a proprietary antioxidant molecule delivered via a sustained-release implant, have been implanted, trial sponsor Nacuity Pharmaceuticals reports. The trial is evaluating NPI-002 for delayed cataract progression in patients undergoing vitrectomy. The final cohort of the trial is actively enrolling patients at three sites in Australia.
A nutritional supplement designed to ease the symptoms of dry eye has demonstrated clinically meaningful efficacy and safety in a clinical study published in Frontiers in Ophthalmology, trial sponsor Bausch + Lomb reports. The supplement is a proprietary blend of ingredients including lutein, zeaxanthin isomers, curcumin, and vitamin D3. B+L says it intends to launch the supplement later this year under the brand name Blink NutriTears. The study reportedly met both of its primary endpoints: change in tear production and participant reports of dry eye symptoms.
Theriva Biologics has reported positive topline data from the Phase I trial of its investigational oncolytic adenovirus VCN-01 in patients with intraocular retinoblastoma of the eye that has not responded to chemotherapy or radiotherapy. These patients, for whom enucleation, or removal of the eye, was the only treatment option, received two intravitreal injections of VCN-01 14 days apart. The study found that VCN-01 was well tolerated and the most frequently reported treatment-related adverse events were Grade 1 or 2. No dose-limiting toxicities and no ocular or systemic toxicities equal to or greater than Grade 3 were reported during the evaluation period. The treatment also demonstrated a signal for antitumor activity and did not appear to change the retinal function, Theriva reports. Three patients have avoided enucleation so far, one of whom has retained their eye after four years of follow-up.
Atsena Therapeutics reports positive preliminary data from the first cohort treated in the Phase I/II LIGHTHOUSE study evaluating subretinal injection of the gene therapy ATSN-201 for the treatment of X-linked retinoschisis (XLRS), an inherited, sight-threatening retinal disease. ATSN-201 utilizes AAV.SPR, a novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. LIGHTHOUSE is enrolling male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. In the first (low-dose) cohort, two of the three patients showed resolution of schisis beginning at 8 weeks after dosing, with additional and continued resolution observed through week 24. ATSN-201 was also well tolerated in all three XLRS patients in the first cohort; no serious adverse events were reported.
HanAll Biopharma has initiated a Phase III VELOS-4 study to evaluate the efficacy and safety of tanfanercept in dry eye based on the findings from the previous Phase III VELOS-3 study, in which tanfanercept demonstrated statistically significant improvement on the secondary dry eye outcome measures. The topline data from the Phase III VELOS-4 study is expected in the second half of 2025, HanAll says.
AsclepiX Therapeutics reports completing patient enrollment in the DISCOVER trial of AXT107 (gersizangitide) for the treatment of retinal vascular diseases. The trial is evaluating the safety and tolerability of three doses of AXT107 [125 µg (n=3), 250 µg (n=3), and 500 µg (n=9)] and determining the bioactivity and duration of action when injected suprachoroidally. Secondary endpoints will include efficacy as assessed by central subfield thickness and best-corrected visual acuity.
EyePoint Pharmaceuticals reports that the Phase II PAVIA trial evaluating Duravyu, the vorolanib intravitreal insert previously known as EYP-1901, did not meet its primary endpoint in patients with nonproliferative diabetic retinopathy (NPDR), although the data demonstrate that Duravyu had a biologic effect. EyePoint says it plans to provide an update on the path forward for Duravyu for NPDR following a review of the full 12-month data, which did show a favorable safety and tolerability profile with no treatment-related ocular or systemic serious adverse events reported.
Boehringer Ingelheim presented positive data from the HORNBILL Phase I/IIa study of BI 764524 for people living with diabetic macular ischemia (DMI). The study found that BI 764524 was well tolerated following intravitreal administration of single and multiple doses, meeting its primary safety endpoints and showing early signs of potential efficacy.
Inflammasome Therapeutics has dosed the first patient in the Phase I trial of the K8 intravitreal implant for geographic atrophy (GA) caused by age-related macular degeneration (AMD). Inflammasome is developing what it calls a new class of inflammasome inhibitor drugs, Kamuvudines.
From the Association for Research in Vision and Ophthalmology meeting (ARVO), Anexon presented Phase II data on ANX007 in patients with vision loss from GA. The new data are the first to show preservation of both vision and relevant anatomical structures following treatment with ANX007. Annexon says it will advance ANX007 into registrational Phase III trials in GA, the ARCHER II and the ARROW trial, a head-to-head trial with pegcetacoplan (Syfovre, Apellis Pharmaceuticals). Both are to be initiated later this year. The Phase II data showed significant protection against vision loss in the foveal and nonfoveal regions, as well as in low-light environments, along with a significant reduction in photoreceptor loss and meaningful deceleration of lesion growth.
Outlook Therapeutics has submitted a Marketing Authorization Application (MAA) to the UK Medicines and Healthcare products Regulatory Agency (MHRA) seeking authorization of ONS-5010/Lytenava, its ophthalmic formulation of bevacizumab, for the treatment of wet age-related macular degeneration (AMD).
ArcScan reports that Insight 100, a computer-controlled high-frequency ultrasound device that measures sizing of phakic intraocular lenses for treatment of myopia, has received National Medical Products Administration approval in China. The Insight 100 already has approvals in the United States and European Union.
Dosing is complete in the second cohort of the Phase I/II GARDian clinical trial for OCU410ST (AAV-hRORA), a modifier gene therapy candidate being developed as a one-time treatment for Stargardt disease, trial sponsor Ocugen reports. Six patients with Stargardt disease have been dosed in the trial to date. An additional three patients will be dosed with the high-dose cohort in the dose-escalation phase.
Oculis Holding has completed enrollment in its multicenter, randomized, double-blind, placebo-controlled Phase II ACUITY trial evaluating OCS-05 in patients with acute optic neuritis (AON). The trial is evaluating once-daily OCS-05 intravenous infusion. The study is ongoing across four sites in France. Oculis says topline results are expected in Q4.
The Food and Drug Administration has approved Biocon Biologics’ application for Yesafili (aflibercept-jbvf) as the first interchangeable Eylea biosimilar in the United States. The approval covers all the indications Eylea is approved for: neovascular age-related macular degeneration (AMD), diabetic macular edema (DME), retinal vein occlusion, and myopic choroidal neovascularization. Biocon also reported that it has secured a launch date in Canada of no later than July 1, 2025, under the terms of a settlement agreement.
The FDA also granted de novo authorization to Notal Vision for its patient self-operated Scanly home-based optical coherence tomography (OCT) device. Scanly captures spectral-domain OCT images in a 10-x-10-degree area centered on the point of fixation. The proprietary, artificial intelligence–based Notal OCT Analyzer segments and estimates the volume of disease biomarkers on OCT images. Ophthalmologists may now prescribe the Scanly Home OCT Monitoring Program, provided by the Notal Vision Monitoring Center, for remote access to high-resolution OCT images and the Notal OCT Analyzer image analysis between patients’ regularly scheduled office visits.
Clearside Biomedical reports that the journal Retina has published new guidelines for best practices for injection of ophthalmic medications into the suprachoroidal space, the area Clearside is targeting with its suprachoroidal space (SCS) injection platform. The panel that drafted the guidelines, led by Charles C. Wykoff, MD, PhD, of Retinal Consultants of Texas in Houston, established consensus on the rationale for SCS injection, including potential benefits relative to other intraocular delivery methods, and current best practices in patient preparation, pre- and peri-injection management, SCS-specific injection techniques, and postinjection management and follow-up.
Opthea reports it has completed enrollment in both the COAST and ShORe trials constituting its pivotal Phase III clinical program for sozinibercept, also known as OPT-302, for treatment of neovascular age-related macular degeneration (nAMD). COAST is evaluating sozinibercept in combination with aflibercept (Eylea, Regeneron Pharmaceuticals) 2 mg; ShORe uses ranibizumab (Lucentis, Genentech/Roche).
Sight Sciences reports the publication of two trial readouts simultaneously in the journal Clinical Ophthalmology. They are:
- One-year results of the SAHARA trial of TearCare dry eye therapy, which showed that patients previously treated with cyclosporine ophthalmic emulsion 0.05% (Restasis, AbbVie) had improvement of symptoms and key dry eye biomarkers with TearCare.
- Findings from the ROMEO study of the Omni surgical system suggested that ab interno canaloplasty coupled with trabeculotomy was as effective at lowering intraocular pressure and reducing medication usage in advanced glaucoma as it was in mild and moderate glaucoma.
The Food and Drug Administration has qualified the American Academy of Ophthalmology’s Assessment of IntraOcular Lens Implant Symptoms (AIOLIS) platform as a Medical Device Development Tool (MDDT). With the designation, medical device sponsors can use AIOLIS to clinically evaluate patients’ perception of visual disturbances following premium intraocular lens (IOL) cataract surgery. The assessment tool is the result of a multiyear collaboration among the Academy; UCLA; Ron D. Hays, PhD, an expert in patient-reported outcomes; RAND; and four IOL device manufacturers: Alcon, Bausch & Lomb, Carl Zeiss Meditec, and Johnson & Johnson. AIOLIS was field tested by 20 cataract surgeons in sites across the United States and two sites outside of the United States. This is the second MDDT the AAO has obtained; the first was for the Patient-Reported Outcomes with LASIK Symptoms and Satisfaction (PROWL-SS).
The European Medicines Agency has validated the Marketing Authorization Application for Vyluma’s lead compound, NVK002, for treatment of childhood myopia. The approval confers 10 years of data exclusivity and marketing protection. Applications for NVK002 have also been submitted in the United States and China.
Surgical robot maker AcuSurgical reports it has completed the first clinical study with the Luca surgical robot for vitrectomy procedures. The clinical study was conducted at Gent University Hospital in Belgium by Fanny Nerinckx, MD, a senior vitreoretinal surgeon. Seven patients were treated using the Luca system and all surgeries reportedly were successful.
PharmAbcine reports it will advance PMC-403, its candidate for treatment for neovascular age-related macular degeneration (nAMD), to the fourth single-dose group of 4 mg and first multiple-dose group of 3 mg in the Phase I trial. PMC-403 is a TIE2-activating antibody that aims to stabilize pathological and leaky blood vessels. TIE2 receptors, expressed on endothelial cells, are involved in vessel normalization processes such as angiogenesis and intercellular adhesion. The Phase I trial targets nAMD patients who no longer respond to anti-VEGF therapy.
Apellis Pharmaceuticals says it’s seeking a re-examination of the second negative opinion the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted on the application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA). In a press release, Apellis noted that multiple CHMP members dissented on the opinion. Apellis says it expects a final opinion in the fourth quarter of 2024. The opinion followed an Ad Hoc Expert Group meeting, in which the panel agreed that size of GA lesion is an acceptable primary outcome measure for a GA trial that microperimetry is the best available functional measure in GA.
The US Food and Drug Administration has granted approval for Vabysmo PFS (faricimab-svoa), a 6-mg single-dose prefilled syringe (PFS), Genentech/Roche reports. Indications are neovascular, age-related macular degeneration (nAMD), diabetic macular edema (DME), and macular edema following retinal vein occlusion (RVO). Vabysmo PFS delivers the same medicine as the currently available Vabysmo vials in an alternative, ready-to-use format. Vabysmo also will continue to be available in a 6-mg vial, Genentech says.
The US Food and Drug Administration approved FYB203, now known as Ahzantive (aflibercept-mrbb), a biosimilar to Eylea (aflibercept 2mg, Regeneron Pharmaceuticals), Germany-based Formycon and licensing partner Klinge Biopharma jointly announce. The FDA covers indications for age-related neovascular macular degeneration (nAMD), diabetic macular edema (DME), diabetic retinopathy (DR), and macular edema following retinal vein occlusion (RVO). Meanwhile, a decision is pending on the European Medicines Agency application for FYB203.
Another Eylea biosimilar, ALT-L9, is the subject of a Marketing Authorization Application to the European Medicines Agency, sponsor Alteogen reports. The Phase III trial for ALT-L9 enrolled more than 400 subjects across 12 countries and evaluated changes in best-corrected visual acuity (BCVA) over eight weeks as the primary endpoint. The study demonstrated therapeutic equivalence between ALT-L9 and Eylea.
Glaukos’ iDose TR anti-glaucoma implant has received a J-code from the Center for Medicare and Medicaid Services (CMS) Healthcare Common Procedure Coding System. The new J-code simplifies the reimbursement process for practitioners.
OKYO Pharma has entered into an agreement with Tufts Medical Center in Boston to evaluate OK-101 in patients with neuropathic corneal pain (NCP), with a plan to file an Investigational New Drug Application in Q4. Pedram Hamrah, MD, will be principal investigator. OK-101 is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, which is found on immune cells of the eye that cause inflammation.
South Korea–based NexThera reports that it has submitted a Phase I/IIa Investigational New Drug (IND) application to the U.S. Food and Drug Administration for NT-101, a noninvasive eye drop treatment for wet age-related macular degeneration (AMD). NT-101 is NexThera's first new drug candidate developed using its proprietary eye drop delivery platform.
OKYO Pharma has entered into an agreement with Tufts Medical Center in Boston to evaluate OK-101 in patients with neuropathic corneal pain (NCP), with a plan to file an Investigational New Drug Application in Q4. Pedram Hamrah, MD, will be principal investigator. OK-101 is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor, which is found on immune cells of the eye that cause inflammation.
South Korea–based NexThera reports that it has submitted a Phase I/IIa Investigational New Drug (IND) application to the U.S. Food and Drug Administration for NT-101, a noninvasive eye drop treatment for wet age-related macular degeneration (AMD). NT-101 is NexThera's first new drug candidate developed using its proprietary eye drop delivery platform.
Alkeus Pharmaceuticals presented interim data from the TEASE-3 study demonstrating that early-stage Stargardt disease patients treated with gildeuretinol acetate showed no disease progression and remained asymptomatic while on therapy ranging between two and six years. The study update was presented last week at the 42nd American Society of Retina Specialists (ASRS) Annual Scientific Meeting in Stockholm. TEASE-3 has enrolled a total of six patients who receive gildeuretinol as a once-a-day pill.
Eluminex Biosciences reports dosing the first patient in the LOTUS trial of EB-105 in patients with diabetic macular edema (DME). LOTUS is a first-in-human, open-label, multicenter, single injection, dose-escalation study. Preclinical studies have shown that EB-105 inhibits a number of biomarkers elevated in patients with retinal disease, including vascular endothelial growth factor A/RD, angiopoietin-2/Tie-2, and interleukin-6/-6R signaling. LOTUS is a multicenter, single intravitreal injection dose-escalation study that will evaluate three dose levels of EB-105 in patients with center-involved DME and will enroll approximately 12 patients.
The first procedure implanting the SING IMT (Smaller-Incision New-Generation Implantable Miniature Telescope) in China has been completed, Samsara Vision reports. In all, Samsara says almost 100 surgeons in 17 countries have implanted the SING IMT in more than 350 patients.
Ocugen has reported a positive outcome of the Data and Safety Monitoring Board review for its Phase I/II ArMaDa clinical trial for OCU410 (AAV5-hRORA), a modifier gene therapy candidate being developed for geographic atrophy, an advanced stage of dry age-related macular degeneration. Six subjects have been dosed in the trial so far; three with the low dose and three with the medium dose. An additional three patients will be dosed with the high dose of OCU410 in the dose-escalation phase.
Azura Ophthalmics has dosed the first patient in the Phase III ASTRO study assessing the efficacy and safety of AZR-MD-001 sterile ophthalmic ointment 0.5% in patients with clinical signs and symptoms of meibomian gland dysfunction (MGD). ASTRO (Assessment of Secretions and Treatment for Restoring Ocular surface health in patients with MGD) is a multicenter, double-masked, vehicle-controlled, randomized trial to evaluating AZR-MD-001 compared to vehicle in patients with abnormal meibomian gland function and associated symptoms of dry eye disease. It will enroll around 500 patients.
Amsterdam-based Avanzanite Bioscience reports that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for marketing authorization of Akantior (polihexanide) for the treatment of acanthamoeba keratitis in adults and children aged 12 years and older. Following the CHMP’s recommendation, the European Commission will make its final marketing authorization approval decision within approximately two months, currently expected in early August.
Skye Bioscience, the clinical-stage biopharma developing therapeutic drugs that modulate the endocannabinoid system, says it is canceling its SBI-100 ophthalmic emulsion program after the Phase IIa clinical trial in primary open-angle glaucoma (POAG) or ocular hypertension (OHT) did not meet its primary endpoint for lowering intraocular pressure (IOP). Skye says it’s shutting down its ophthalmology pipeline to focus on its metabolic program, extending its operating runway into 2027. The Phase IIa trial of SBI-100 did not achieve a statistically significant improvement in IOP over placebo.
4D Molecular Therapeutics presented supplemental data from the Phase II PRISM trial evaluating intravitreal 4D-150 in wet age-related macular degeneration (AMD), focusing on a subgroup of patients who didn’t need supplemental aflibercept injections in addition to the 4D-150 treatment. Single intravitreal 3E10 vector genes per eye (vg/eye) dose resulted in sustained reduction and stabilization of mean central subfield thickness (CST), compared to aflibercept at all timepoints. In the 3E10 vg/eye injection-free subgroup, the mean difference versus aflibercept in CST change from baseline for weeks 20 and 24 was –31 microns in favor of 4D-150.
Stealth BioTherapeutics has enrolled and dosed the first patient in the ReNEW trial in the Phase III clinical program for elamipretide in patients with dry age-related macular degeneration (AMD). ReNEW is a companion Phase III trial to ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD.
Beacon Therapeutics has dosed the first patient in its registrational VISTA clinical trial evaluating its lead asset, AGTC-501 (laruparetigene zovaparvovec), in patients with X-linked retinitis pigmentosa (XLRP). Beacon says it plans to use the data, along with previous trials, for applications in the US and Europe.
Oculis Holding has reported positive topline results from the Phase IIb RELIEF trial with licaminlimab, an anti-tumor necrosis factor–alpha biologic eye drop, in patients with dry eye disease (DED). The trial randomized 122 patients 1:1 to either licaminlimab (n=62) or vehicle (n=60). Licaminlimab demonstrated a superior treatment efficacy compared with vehicle, the company reports.
Syfovre (pegcetacoplan injection) demonstrated preserved visual function at 36 months in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), trial sponsor Apellis Pharmaceuticals reports. Data from the GALE long-term extension study were presented at the Clinical Trials at the Summit Meeting on June 8 in Park City, Utah. In a prespecified microperimetry endpoint, patients developed, on average, 2.9 fewer new scotomatous points with 36 months of continuous monthly and 2 fewer new scotomatous points with every-other-month dosing, compared to patients from the sham crossover group.
Aldeyra Therapeutics reports it has completed enrollment in a Phase III trial of topical ocular 0.25% reproxalap, an investigational treatment for dry eye disease. The trial is designed to enable potential resubmission of a New Drug Application (NDA) in the second half of 2024. The trial has enrolled 132 patients to evaluate the primary endpoint of ocular discomfort.
Beacon Therapeutics Holdings reports dosing the first patient in its registrational VISTA clinical trial with its lead asset, AGTC-501 (laruparetigene zovaparvovec). VISTA is a global randomized, controlled, masked, multicenter pivotal study evaluating the efficacy, safety, and tolerability of two dose levels of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP), a severe, inherited retinal disorder, versus an untreated control group. Beacon says it plans to use VISTA trial data, along with data from previous trials, to support a Biologics License Application (BLA) in the US and a parallel application in Europe for the treatment of XLRP.
Luminopia has reported that real-world data from the Patients Using Prescription Luminopia (PUPiL) Registry continues to demonstrate efficacy of the Food and Drug Administration-approved therapy in improving vision for children with amblyopia. The therapy uses proprietary therapeutic software with virtual-reality headsets and children's television shows. The data, presented at the European and International Strabismological Association conference in Toulouse, France, further support the topline data presented at previous scientific meetings that demonstrated the efficacy of Luminopia in the real world and, importantly, demonstrate its clinical benefit as both a first- and second-line treatment, Luminopia says.
The US Food and Drug Administration has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation for AURN001, Aurion Biotech’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease. BTD is a process designed to expedite development and review of drugs that target a serious disease for which treatment options are limited. RMAT is a part of the 21st Century Cures Act designation for regenerative therapies that target serious conditions.
Vyluma reports it had a Type A meeting with FDA Division of Ophthalmology representatives to discuss the response to FDA’s questions regarding its late-stage product candidate, atropine sulfate ophthalmic solution 0.01%, under development to treat pediatric myopia. Vyluma says it “is very encouraged by the discussion” and that it will submit answers to the FDA’s questions in Q3.
The FDA has given priority review status to Neurotech Pharmaceuticals' biologic license application (BLA) for NT-501 (revakinagene taroretcel), an investigational encapsulated cell therapy aimed at treating Macular Telangiectasia Type 2 (MacTel). This designation indicates that the agency has found the application sufficiently complete for review and intends to take action within 6 months, compared to the typical 10 months for a standard review.
Results from the first prospective clinical trial using home-based optical coherence tomography (OCT) to manage neovascular age-related macular degeneration (nAMD) have shown that users had a significant reduction in office visits and treatment frequency, while visual acuity remained stable, trial sponsor Notal Vision reports. The results were published in the journal Retina. The trial enrolled 15 patients and 21 eyes with nAMD in at least one eye from three retina specialist practices in the US over a period of six months. Users extended the intervals between office visits for treatments from every eight weeks to every 15.3 weeks. Patient adherence to home OCT use was also considered high, with an average 6.2 scans a week in each eye.
The Food and Drug Administration cleared 4D Molecular Therapeutics’ Investigational New Drug Application (IND) for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment geographic atrophy (GA). The Phase I GAZE clinical trial will assess 4D-175 in patients with GA secondary to age-related macular degeneration (AMD). 4DMT says it expects to begin enrollment in H2 2024.
Meanwhile, 4DMT also reports that it will present, at the American Society of Retina Specialists’ annual meeting, the initial interim 24-week landmark analysis from the population extension cohort of the PRISM Phase II trial evaluating 4D-150 in a broad wet AMD patient population. The meeting will be held in Stockholm from July 17 through July 20, 2024.
Alcon reports that the Unity Vitreoretinal Cataract System (VCS) and Cataract System (CS) have received Food and Drug Administration 510(k) clearance. Unity VCS and Unity CS are designed to bring significant workflow efficiencies over Alcon’s existing surgery platforms, Constellation for vitreoretinal procedures and Centurion with Active Sentry for cataract surgery. Alcon says more than 28,000 Centurion and Constellation devices in the market that will be targeted for upgrade to the Unity platform over the next decade.
Ikerian has registered four devices as Class IIa medical devices under the European Union Medical Device Regulation (MDR). The certificate applies to RetinAI Discovery, a digital data platform in ophthalmology, and the artificial intelligence-based models to identify and quantify retinal layers, retinal fluids, and retinal biomarkers. The latest MDR replaces the former European Medical Device Directive (93/42/EEC), including more rigorous requirements for clinical evaluation and postmarketing surveillance.
Chengdu Origen Biotechnology and partner Vanotech have dosed the first patient in the VAN-2201 multi-center Phase I clinical trial evaluating KH631 for wet age-related macular degeneration (AMD). This US-based, open-label, dose-escalation trial will assess the safety, tolerability, and efficacy of KH631 as a single administration gene therapy for patients with previously treated wet AMD. KH631 is a recombinant adeno-associated virus vector that encodes a human VEGF receptor fusion protein.
Arctic Vision has reported positive topline results of its Phase III clinical trial of Arcatus (ARVN001), a triamcinolone acetonide suprachoroidal injectable suspension for the treatment of uveitic macular edema (UME) in China. Marketed as Xipere in the United States, the candidate already has US Food and Drug Administration for the same indication. The new drug application has also been accepted in Australia and Singapore. The Phase III trial met its primary endpoint and secondary endpoints and demonstrated superior visual acuity improvement and edema control versus sham, Arctic reports. Originally developed by Clearside Biomedical, Xipere is licensed commercially to Bausch + Lomb in the United States and Canada.
The first patients have been enrolled in the Phase III SOL-R clinical trial evaluating repeat dosing of Axpaxli (axitinib intravitreal implant, also known as OTX-TKI) for neovascular age-related macular degeneration (nAMD), trial sponsor Ocular Therapeutix reports. SOL-R is a global, 825-patient, noninferiority study evaluating repeat dosing of Axpaxli every six months compared to 2 mg aflibercept, dosed every eight weeks. A third arm evaluating 8 mg aflibercept dosed every six months has also been incorporated into the study.
Ocugen reports it has completed dosing in the third cohort of the Phase I/II ArMaDa clinical trial of OCU410 (AAV-hRORA), a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). Three subjects received a single subretinal administration of the highest dose (200 microliters of 1.5x1011 vector genes per mL), which completed the dosing for the Phase 1 stage of the trial. The ArMaDa clinical trial for OCU410 is being performed at 14 US retinal surgery centers.
Annexon has initiated in the global pivotal Phase III ARCHER II trial evaluating ANX007 for the treatment of geographic atrophy (GA). Annexon says ARCHER II is the only GA pivotal program using visual protection as the regulatory-aligned primary endpoint. ARCHER II is a global, randomized, sham-controlled trial for which topline data are expected in the second half of 2026.
Ribomic reports that 13 pediatric patients, aged 5 to 14 years, have been enrolled in an observational Phase II study of umedaptanib pegol (anti-FGF2 aptamer) for achondroplasia. A low-dose subcutaneous study to evaluate the efficacy and safety of umedaptanib pegol in six patients who have completed the observational study is ongoing and is expected to be completed in November, Ribomic reports. The first patient has enrolled in a high-dose subcutaneous study of patients who completed the observational study.
Lupin, a global pharmaceutical company based in India, reports it has completed the global Phase III clinical study of LUBT010, its biosimilar candidate to Lucentis (ranibizumab, Genentech/Roche). The study met its primary endpoint of therapeutic equivalence in visual acuity improvement for patients with wet age-related macular degeneration, Lupin reports. Lupin has been marketing the ranibizumab biosimilar in India under the brand name RaniEyes since 2022.
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Ocular Therapeutix has received a written response from the Food and Drug Administration that its Phase III SOL-R clinical trial of Axpaxli (axitinib intravitreal implant, also known as OTX-TKI) is appropriate for use as the second adequate and well-controlled study of the drug candidate for the treatment of patients with wet age-related macular degeneration (AMD) and can be used to support a potential New Drug Application (NDA) and product label. Ocular also recently announced that the first subjects have been enrolled in the SOL-R non-inferiority study evaluating repeat dosing of Axpaxli. Enrollment in the SOL-1 superiority study evaluating a single Axpaxli implant continues.
Novaliq reports the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a marketing authorization in the European Union for Vevizye for treatment of moderate to severe dry eye disease in adult patients. Vevizye, which is also known by the development name CyclASol, is a clear ciclosporin 0.1% solution, free of oils, surfactants, and preservatives, designed to last longer on the ocular surface than existing drug. Novaliq submitted a Marketing Authorization Application (MAA) for Vevizye in July 2023.
The Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Ixo-vec for the treatment of wet age-related macular degeneration (AMD), Adverum Biotechnologies reports. The RMAT program aims to expedite the development and review processes for promising pipeline products, including genetic therapies. RMAT includes potential priority review of the Biologics License Application (BLA), and other opportunities to expedite development and review.
The Food and Drug Administration also granted Theriva Biologics Rare Pediatric Drug Designation (RPDD) for VCN-01 for the treatment of retinoblastoma. VCN-01 is a systemic, selective, stroma-degrading oncolytic adenovirus. Previously, the FDA granted orphan drug designation to VCN-01 for the same indication. VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. VCN-01 has been administered to more than 80 patients in Phase I and investigator-sponsored clinical trials of different cancers, including retinoblastoma.
Eclipse Life Sciences has enrolled and dosed the first patient in the BETTIS-1 Phase II trial for EC-104 fluocinolone acetonide (FA) extended release, intravitreal corticosteroid implant with intended 6 months durable drug release for the treatment of diabetic macular edema (DME). BETTIS-1 is a randomized, controlled, double-masked clinical trial comparing two doses (FA 0.14 mg and FA 0.092 mg) of EC-104 to Ozurdex (dexamethasone intravitreal implant 0.7 mg, AbbVie) in patients with DME with prior suboptimal clinical response to IVT antivascular endothelial growth factor (VEGF) therapy and who have been previously treated with locally administered corticosteroids without a clinically significant rise in intraocular pressure. The primary endpoint for BETTIS-1 is safety of EC-104. The secondary endpoint is efficacy based on spectral domain-optical coherence tomography (SD-OCT) images through week 24.
Sandoz reports that the Food and Drug Administration has approved the aflibercept biosimilar Enzeevu 2 mg vial kit and prefilled syringe for intravitreal treatment of neovascular age-related macular degeneration (nAMD). The launch will depend on several factors, Sandoz says, including the resolution of litigation over the platform.
Atsena Therapeutics reports the FDA has granted Rare Pediatric Disease designation (RPD) for the gene therapy ATSN-201 for the treatment of X-linked retinoschisis (XLRS), a hereditary, blinding retinal disease for which no approved treatments exist. About 30,000 males in the United States and Europe have XLRS. With the RPD, ATSN-201 will be eligible to receive a priority review voucher upon approval that could be used to advance another internal program or be sold to an outside company.
The Food and Drug Administration has granted Rare Pediatric Disease designation (RPD) for the ocular gene therapy OPGx-LCA5 to treat the inherited retinal disease Leber congenital amaurosis (LCA), sponsor Opus Genetics reports. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with inherited retinal degeneration resulting from biallelic mutations in the LCA5 gene.
Innovec Pharmaceutical Technology has received FDA clearance for its Investigative New Drug (IND) application for IVB103, an AAV-based gene therapy for neovascular age-related macular degeneration.
Eclipse Life Sciences has enrolled and dosed the first patient in the BETTIS-1 Phase II trial for EC-104 fluocinolone acetonide (FA) extended release, intravitreal corticosteroid implant with intended 6 months durable drug release for the treatment of diabetic macular edema (DME). BETTIS-1 is a randomized, controlled, double-masked clinical trial comparing two doses (FA 0.14 mg and FA 0.092 mg) of EC-104 to Ozurdex (dexamethasone intravitreal implant 0.7 mg, AbbVie) in patients with DME with prior suboptimal clinical response to IVT antivascular endothelial growth factor (VEGF) therapy and who have been previously treated with locally administered corticosteroids without a clinically significant rise in intraocular pressure. The primary endpoint for BETTIS-1 is safety of EC-104. The secondary endpoint is efficacy based on spectral domain-optical coherence tomography (SD-OCT) images through week 24.
Sandoz reports that the Food and Drug Administration has approved the aflibercept biosimilar Enzeevu 2 mg vial kit and prefilled syringe for intravitreal treatment of neovascular age-related macular degeneration (nAMD). The launch will depend on several factors, Sandoz says, including the resolution of litigation over the platform.
Atsena Therapeutics reports the FDA has granted Rare Pediatric Disease designation (RPD) for the gene therapy ATSN-201 for the treatment of X-linked retinoschisis (XLRS), a hereditary, blinding retinal disease for which no approved treatments exist. About 30,000 males in the United States and Europe have XLRS. With the RPD, ATSN-201 will be eligible to receive a priority review voucher upon approval that could be used to advance another internal program or be sold to an outside company.
NovaSight reports publication of a study in the American Journal of Ophthalmology that showed that amblyopia patients who adhered to the CureSight treatment protocol—using the company’s novel binocular eye-tracking device—achieved greater improvements in visual acuity than did those undergoing traditional occlusion therapy, or patching, in a multicenter randomized controlled trial. The study included 149 children with amblyopia who were randomized to either CureSight digitized binocular treatment or patching.
BlueRock Therapeutics, a clinical-stage cell therapy company and wholly owned, independently operated subsidiary of Bayer, reports the US Food and Drug Administration cleared the Investigational New Drug application for OpCT-001, an investigational induced pluripotent stem cell–derived therapy for the treatment of inherited primary photoreceptor diseases of the eye. BlueRock licensed OpCT-001 exclusively from FujiFilm Cellular Dynamics and Opsis Therapeutics.
Ocuphire Pharma reports that the VEGA-3 Phase III trial evaluating phentolamine ophthalmic solution 0.75% for presbyopia has dosed its first participants. The trial will enroll 545 participants with presbyopia, randomizing them 3:2 to one drop of phentolamine 0.75% or placebo daily. Participants will be followed for 48 weeks. Recruitment will occur at up to 40 sites in the US.
Clinical results of Iantrek’s uveoscleral outflow technology in 243 eyes with open-angle glaucoma were published in the Journal of Clinical Medicine. The technology is a reinforced scleral allograft implanted during surgery to facilitate uveoscleral outflow. Some 79% of eyes had phacoemulsification cataract surgery before the intervention; the remainder had stand-alone surgery. No sight-threatening or serious ocular adverse events were reported. Among treated eyes, 78.6% had intraocular pressure ≤ 18 mmHg without an increase in medications.
Outlook Therapeutics reports completing enrollment in the NORSE EIGHT clinical trial evaluating ONS-5010, an ophthalmic formulation of bevacizumab (Avastin, Genentech/Roche), in patients with neovascular age-related macular degeneration (nAMD). NORSE EIGHT is the subject of a Special Protocol Assessment agreement with the Food and Drug Administration and could be the precursor to a resubmitted Biologics License Application (BLA). NORSE EIGHT is randomizing patients 1:1 to receive 1.25 mg ONS-5010 or 0.5 mg ranibizumab intravitreal injections. Participants will receive injections at day 0 (randomization), week 4, and week 8 visits. The primary endpoint is mean change in best-corrected visual acuity from baseline to week 8. Outlook says it’s on track to report topline results by yearend, and resubmit the BLA in Q1 2025.
Viridian Therapeutics reports topline data from the THRIVE Phase III clinical trial of VRDN-001, otherwise known as veligrotug, an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, in patients with active thyroid eye disease (TED). THRIVE met the primary and all secondary endpoints at 15 weeks after five infusions of veligrotug, showing statistically significant improvement in the measured signs and symptoms of TED. A majority (53%) of treated patients achieved a proptosis response after one infusion, or three weeks after start of therapy. THRIVE enrolled 113 patients randomized to veligrotug (n=75) and placebo (n=38).
The Phase I evaluation of BI 771716, an investigational antibody fragment developed to treat geographic atrophy (GA), met its primary safety endpoint following intravitreal administration of single and multiple doses, trial sponsors Boehringer Ingelheim and CDR-Life report. The sponsors say they plan to start the Phase II trial early next year.
Merck and its EyeBio subsidiary have initiated the Phase IIb/III BRUNELLO trial evaluating Restoret (MK-3000, formerly EYE103) for the treatment of diabetic macular edema (DME). Restoret is tetravalent, tri-specific antibody. The trial is evaluating the efficacy and safety of two dose levels of intravitreal Restoret versus active control ranibizumab in patients with DME. The dual primary endpoints are safety and mean change in best-corrected visual acuity from baseline to week 52.
Alkeus Pharmaceuticals reports that the SAGA study of oral gildeuretinol acetate (ALK-001), which is being evaluated in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), demonstrated a reduction in the primary endpoint of GA lesion growth rate at 24 months: 0.25 mm²/year vs. placebo, but did not reach statistical significance (P = 0.07). Gildeuretinol did demonstrate a statistically significant reduction in the loss of low luminance visual acuity at 24 months (P = 0.03), along with a favorable safety profile consistent with other studies of gildeuretinol in Stargardt disease, Alkeus reports. Topline results will be presented next month at the American Academy of Ophthalmology annual meeting.
Iantrek reported one-year results from the prospective multicenter, real-world evidence study of open-angle glaucoma patients who underwent uveoscleral outflow enhancement surgery using Iantrek’s interventional cyclodialysis system, combined with adjunct allogeneic bio-tissue reinforcement. The bio-interventional procedure was well tolerated, with a low incidence of minor, transient adverse events. At 12 months, the mean medicated intraocular pressure (IOP) decreased 27.1% from baseline, with hypertensive eyes (baseline medicated IOP > 21 mmHg) showing a 39.7% reduction. Additionally, 81.9% of all eyes achieved a medicated IOP ≤ 18 mmHg without an increase in ocular hypotensive medications. Topline results will be presented next month at the American Academy of Ophthalmology annual meeting.
IVIEW Therapeutics reports completing patient recruitment for its Phase I/II clinical trial evaluating IVW-1001, an agonist of the transient receptor potential cation channel subfamily M member 8 (TRPM8), also known as the cold and menthol receptor 1, for the treatment of signs and symptoms of dry eye disease. The active agent is delivered via an ophthalmic eyelid wipe. IVIEW says it anticipates reporting topline data in the first quarter 2025.
Nicox SA reports that its exclusive Chinese partner, Ocumension Therapeutics, has received approval from the National Medical Products Administration of China for its New Drug Application to commercialize Zerviate (cetirizine ophthalmic solution) 0.24% in China for ocular itching associated with allergic conjunctivitis.
Opthea announced completion of its drug substance Process Performance Qualification (PPQ) campaign for sozinibercept for treatment of neovascular age-related macular degeneration. The PPQ campaign consisted of the production of three successful consecutive commercial-scale drug substance batches required for the validation of the manufacturing process. The batches have been produced following a manufacturing process development program.
Nanoscope Therapeutics reports what it describes as a “productive” End-of-Phase II meeting with the Food and Drug Administration for its clinical program evaluating MCO-010 for the treatment of severe vision loss due to Stargardt macular degeneration. The meeting supports advancing MCO-010 to a Phase III registrational trial, Nanoscope says.
The FDA has granted Orphan Drug Designation for ATSN-201 for the treatment of X-linked retinoschisis, Atsena Therapeutics reports. ATSN-201 is an adeno-associated virus vector that uses Atsena’s novel spreading capsid to achieve therapeutic levels of gene expression in photoreceptors of the central retina without the risks of foveal detachment from surgery.
The FDA issued a warning letter to Regenerative Processing Plant in Palm Harbor, FL, noting that the company’s Regener-Eyes Professional and Regener-Eyes LITE ophthalmic solutions violate the agency’s Current Good Manufacturing Practice regulations. The warning letter further notes that the products, marketed to relieve dry eye symptoms, are unapproved and include an active ingredient, namely sodium chloride, that’s not permitted for ophthalmic use. The warning letter states Regenerative distributes the products directly to optometrists, eye clinics and patients in the US.
4D Molecular Therapeutics reports data that it says show continued robust and durable clinical activity, based on longest interim follow-up data from the Phase I/II PRISM clinical trial of 4D-150 gene therapy for neovascular age-related macular degeneration. Patients in three separate groups — those with severe vision loss, the broader population, and the recently diagnosed — demonstrated reductions in annual injections ranging from 83% to 98%. In the latter group, all patients had one or fewer injections after a year. 4DMT says it’s planning for global 4FRONT Phase III development program comparing a single dose of 4D-150 to on-label aflibercept 2mg every eight weeks.
AAVantgarde Bio, an Italy-based global biotechnology company, announced that the first subject has been dosed in LUCE-1, a Phase I/II open-label clinical trial to evaluate safety and tolerability of AAVB-081, a dual-hybrid gene-therapy platform to deliver MYO7A protein in retinitis pigmentosa related to Usher Syndrome Type 1B.
Aviceda Therapeutics reports completing enrollment of the Phase IIB SIGLEC study, which compares the safety and efficacy of intravitreal AVD-104 versus Izervay (avacincaptad pegol) in patients with geographic atrophy (GA) secondary to age-related macular degeneration. In the Phase IIA study, researchers observed no drug-related ocular or systemic serious adverse reactions at three months following a single intravitreal injection of AVD-104 and concluded that the drug was well tolerated. Compared with untreated fellow eyes with GA at 3 months, as well as natural history, researchers also found that AVD-104 treatment led to greater reduction in GA lesion progression, improved imaging outcomes in the around growing GA lesions, and stabilized or improved best-corrected visual acuity.
Meanwhile, on the biosimilars front in Europe, Biogen and Samsung Bioepis report that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Opuviz 40 mg/mL, also known as SB15, a biosimilar referencing Eylea. The approval covers the same indications that Eylea has: neovascular age-related macular degeneration, retinal vein occlusion, diabetic macular edema, and myopic choroidal neovascularization. This CHMP’s positive opinion will now be referred to the European Commission which will decide whether to grant a marketing authorization for Opuviz.
However, the Committee for Medicinal Products for Human Use (CHMP) was not so judicious to Apellis Pharmaceuticals, confirming its June negative opinion on Syfovre (pegcetacoplan), the company's intravitreal pegcetacoplan for the treatment of geographic atrophy (GA). This follows on an initial negative opinion and January in which the CHMP found it could not establish a positive balance of benefits and risks for Syfovre. Apellis sought a re-examination and was allowed to submit new data in support of its application following an unrelated court ruling in Europe. The US Food and Drug Administration last year approved Syfovre for treatment of GA.
Ocuphire Pharma has reported the publication of full results from two pivotal Phase III clinical trials, MIRA-2 and MIRA-3, evaluating Ryzumvi (phentolamine 0.75%) for the treatment of pharmacologically induced mydriasis. The results appeared in the journal Ophthalmology. The results showed that fewer patients treated with Ryzumvi had residual dilation at 24 hours after they were dilated, compared to placebo-treated subjects (28% vs. 34%).
Ocular Therapeutix has announced accelerated timelines for the SOL-1 Phase III registrational clinical trial of Axpaxli in wet age-related macular degeneration. The company now expects the SOL-1 study to be fully enrolled with all patients randomized by the end of 2024, ahead of the previously reported target of the end of Q1 2024. Topline data from the SOL-1 trial are now expected during the fourth quarter of 2025.
Okyo Pharma has started screening and recruiting patients for a Phase II trial of OK-101 to treat neuropathic corneal pain. The study is a double-masked, randomized, 12-week placebo-controlled trial enrolling 48 patients. The primary endpoint is to measure pain relief using Visual Analog Scale.
The Phase IIb ODYSSEY clinical trial of CLS-AX (axitinib injectable suspension) for the treatment of neovascular age-related macular degeneration achieved both its primary and secondary outcomes, trial sponsor Clearside Biomedical reports. In participants who received CLS-AX delivered suprachoroidally, best corrected visual acuity and central subfield thickness were stable up to six months, compared to participants who received aflibercept. In addition, CLS-AX demonstrated a well-tolerated safety profile to week 36, including mandatory redosing of CLS-AX at Week 24. No patients required additional dosing up to 12 weeks, and 67% did not require any additional treatment before mandatory redosing at week 24.
Late-stage clinical biotech Nanoscope Therapeutics reports it had a “productive” Food and Drug Administration meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa. Nanoscope says that, based on the regulatory feedback, it will start the submission of a Biologics License Application (BLA) in Q1 2025. Nanoscope says the FDA acknowledged the company’s proposed next steps to facilitate a BLA submission for MCO-010. Nanoscope outlined its plans for a rolling submission based on its Fast Track Designation.
South Korea–based PharmAbcine reports that its Tie2-activating antibody candidate, PMC-403, received unanimous safety approval from the Safety Review Committee for the 4-mg single ascending dose cohort in its Phase I clinical trial for neovascular age-related macular degeneration. The company says it’s also preparing to begin enrollment for the 4-mg multiple ascending dose cohort.
The Food and Drug Administration has approved Bausch + Lomb’s enVista Envy intraocular lens (IOL). B+L says the IOL offers a continuous range of vision with “excellent” dysphotopsia tolerance on the enVista IOL platform. Envy uses ActivSync Optic intelligent energy distribution to optimize vision in a variety of lighting conditions. Envy also enables surgeons to treat a wider range of astigmatic patients with more accuracy and precision with 0.5-diopter steps (or fewer) throughout the cylinder range. The enVista Envy IOLs will be commercially available in the US on a limited basis in the coming weeks and more broadly in 2025. B+L says it’s in the process of seeking regulatory approvals for the lens in additional markets.
Sight Sciences reports publication of a study in the journal Clinical Ophthalmology that shows 52% of study participants with dry eye disease had a clinically significant improvement in reading speed of more than 10 words per minute after therapy with TearCare. Improvements on the International Reading Speed Test and the Minnesota Low Vision Reading Test reached statistical significance. The participants also demonstrated significant improvements in all dry eye disease sign metrics, including tear break-up time, meibomian gland secretion score, and corneal fluorescein staining.
LENZ Therapeutics has a couple of things going on. The company reports that the U.S. Food and Drug Administration accepted its New Drug Application for LNZ100 (1.75% aceclidine hydrochloride) for the treatment of presbyopia. The FDA has assigned a Prescription Drug User Fee Act target action date of August 8, 2025. The FDA noted that it is not planning to hold an advisory committee meeting to discuss this application.
LENZ also reported, with partner Corxel Pharmaceuticals, positive topline data from the Phase III JX07001 clinical trial of LNZ100 in patients with presbyopia in China. Among key outcomes:
- 84% and 69% of participants achieved two- and three-lines or greater improvement in visual acuity, respectively, at 30 minutes.
- 88% and 74% achieved two- and three-lines or greater improvement, respectively, at three hours posttreatment, and maintained their optimal distance visual acuity.
- 61% and 30% achieved two- and three-lines or greater improvement, respectively, at 10 hours.
No serious treatment-related adverse events were reported.
Astellas Pharma has withdrawn its marketing authorization application from the European Medicines Agency (EMA) for avacincaptad pegol intravitreal solution, distributed as Izervay in the United States, for the treatment of geographic atrophy secondary to age-related macular degeneration. The company says it decided to withdraw the application after interactions with the EMA’s Committee for Medicinal Products for Human Use.
Glaukos Corp. reports that the second Phase III confirmatory pivotal trial for Epioxa (pronounced epi-on), met its primary efficacy endpoint. Epioxa is a noninvasive drug therapy for the treatment of keratoconus, a corneal dystrophy that causes the cornea to take a cone-like shape, which can severely impact vision and the corneal structure. The trial demonstrated a clinically relevant and statistically significant improvement in maximum corneal curvature (Kmax) at 12 months from baseline between the Epioxa treated arm and the sham/placebo-controlled arm. Key topline results include:
- A Kmax treatment effect of a decrease of –1 diopter (D) at 12 months in the Epioxa-treated arm vs. the sham/placebo-controlled arm.
- 91.5% of enrolled treatment patients completed the 12-month trial vs. 90.9% of enrolled control patients, demonstrating acceptable tolerance.
- No serious ocular adverse events reported. The majority of adverse events reported were mild and transient in nature.
- No evidence of treatment-related systemic effects emerged.
- Corneal endothelial cell counts were unchanged over the 12-month evaluation period.
The trial enrolled 312 eyes. Glaukos says these results, with the already-completed first Phase III pivotal trial, are expected to support a New Drug Application for Epioxa by the end of 2024.
Alkeus Pharmaceuticals reports that topline results from the SAGA study of gildeuretinol (ALK-001) for geographic atrophy (GA) secondary to age-related macular degeneration showed a statistically significant reduction in the GA lesion growth rate of 15.3% vs. placebo from six to 24 months. And gildeuretinol-treated patients had a statistically significant visual function improvement — 4.4 fewer letters lost in low luminance visual acuity over 24 months.
EyePoint Pharmaceuticals reports the first patient has been dosed in the Phase III LUGANO clinical trial of Duravyu, formerly EYP-1901, for the treatment of wet age-related macular degeneration. Duravyu is an investigational sustained-delivery therapy using vorolanib, a selective tyrosine kinase inhibitor formulated in proprietary bioerodible Durasert E. LUGANO and LUCIA are global, randomized, double-masked, aflibercept controlled, non-inferiority Phase III trials evaluating Duravyu. Each trial is expected to enroll approximately 400 patients globally.
A separate trial, the ongoing Phase II VERONA trial evaluating Duravyu for treatment of diabetic macular edema, reported that patients receiving the 2.7-mg dose gained 8.9 letters in best-corrected visual acuity at 16 weeks and had a 68-micron reduction in central subfield thickness, a key retinal biomarker that demonstrates anatomical improvement. EyePoint says full topline data from the trial are expected in Q1 2025.
The Food & Drug Administration has authorized marketing of the Valeda Light Delivery System for treatment of patients with dry age-related macular degeneration (AMD), LumiThera reports. The de novo application is the first FDA-approved treatment for vision loss in dry AMD patients, according to LumiThera. Valeda uses photobiomodulation. The pivotal US LIGHTSITE III trial demonstrated the device improved best-corrected visual acuity over 24 months by five letters or more, equivalent to a line on the eye chart.
The FDA has also cleared HuidaGene Therapeutic’s investigational new drug application for HG202, a CRISPR/Cas13 RNA-editing therapy and the only clinical-stage RNA-targeting therapy for neovascular age-related macular degeneration. Preclinical studies showed HG202 reduced choroidal neovascularization area by 87%, outperforming both anti-VEGF antibodies and adeno-associated virus anti-VEGF gene therapy, according to HuidaGene.
The FDA also granted fast-track designation to FLQ-101, FELIQS’ lead candidate for prevention of retinopathy of prematurity. FLQ-101 can be administered daily either orally or intravenously to enhance retinal vascularization and protect against inflammation and abnormal neovascularization. FELIQS says it is preparing a Phase Ib/II study in the US and Japan in the first quarter 2025. The FDA also granted FLQ-101 orphan drug designation earlier this year. The company also says it is preparing to submit an investigational new drug application for its second asset, FLQ-104, to treat intermediate dry age-related macular degeneration in the second half of 2025.
The Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) goal date for Neurotech Pharmaceuticals’ investigative treatment for macular telangiectasia type 2 (MacTel), NT-501, by three months, to March 18, 2025. Neurotech reports the FDA imposed the extension to give the agency time to review additional data it requested from the company. NT-501 is an implant that uses Neurotech’s encapsulated cell therapy platform, a cell-based delivery system designed to provide sustained delivery of therapeutic proteins.
PulseSight Therapeutics presented new data on its lead candidate, PST-611, a DNA plasmid expressing human transferrin, in dry age-related macular degeneration/geographic atrophy at the European Association for Vision and Eye Research 27th EVER Congress 2024. PST-611 is a first-in-class nonviral vectorized therapy. The retrospective study in a large cohort of dry AMD patients confirmed higher levels of free iron and increased transferrin saturation, with the potential to preserve retinal pigment epithelium cells, in treated patients versus controls. PulseSight reports these data support continued clinical development of PST-611.
The National Eye Institute has awarded a $6.4 million grant over five years to Christopher Cioffi, PhD, of Rensselaer Polytechnic Institute in Troy, NY, and Konstantin Petrukhin, PhD, at Columbia University, to develop compounds to treat dry age-related macular degeneration and Stargardt disease. Their work has led to the discovery of an advanced preclinical candidate. The NEI funding will cover drug development work and investigational new drug–enabling toxicology studies. Their research focuses on improving delivery of retinol to the eye to suppress harmful pathological actions.
The Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) goal date for Neurotech Pharmaceuticals’ investigative treatment for macular telangiectasia type 2 (MacTel), NT-501, by three months, to March 18, 2025. Neurotech reports the FDA imposed the extension to give the agency time to review additional data it requested from the company. NT-501 is an implant that uses Neurotech’s encapsulated cell therapy platform, a cell-based delivery system designed to provide sustained delivery of therapeutic proteins.
Eyenovia reports that it is terminating the Phase III CHAPERONE trial of atropine with the Optejet dispensing platform as a treatment for pediatric progressive myopia after the trial failed to meet its primary endpoint. The trial enrolled 252 patients. The data review committee determined that the rate of myopia progression did not differ substantially between the 0.01% and 0.1% atropine metered spray treatment arms. The company says it is evaluating next steps to reduce expenses and to evaluate its strategic options.
Melt Pharmaceuticals reports positive topline results from the Phase III study evaluating the MELT‑300, a nonintravenous, nonopioid tablet for procedural sedation during cataract surgery, meeting the company’s requirements for a regulatory submission. MELT-300 combines a fixed dose of midazolam (3 mg) and ketamine (50mg) in one tablet that is administered sublingually using Catalent’s proprietary Zydis delivery platform, which dissolves in as little as 3 seconds, allowing absorption of the active ingredients across the sublingual mucosa. The Phase III trial enrolled more than 530 patients.
Adverum Biotechnologies has reported topline 52-week results from the LUNA Phase II trial of Ixo-vec for neovascular age-related macular degeneration, along with new four-year OPTIC long-term follow-up data. Topline 52-week data from LUNA showed that both doses of Ixo-vec, 6E10 and 2E11, maintained visual and anatomic endpoints for best-corrected visual acuity and central subfield thickness, and resulted in treatment burden reductions of 88% and 92%, respectively. Four-year OPTIC topline results of the 2E11 dose demonstrated an 84% reduction in treatment burden with nearly 50% of patients not needing additional injections.
Ocugen has reported positive preliminary efficacy and safety data from the Phase I dose-escalation portion of the Phase I/II ArMaDa clinical trial of OCU410 for geographic atrophy secondary to dry age-related macular degeneration. Key findings include no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and a potentially positive effect on low-luminance visual acuity (LLVA). The OCU410 Phase I trial is evaluating nine patients in three dose cohorts (low, medium, and high). The three patients in the low-dose cohort at six months demonstrated 21.4% slower lesion growth vs. untreated fellow eyes, and 100% of treated eyes showed stabilization of visual function demonstrating treatment benefit as measured by LLVA.
The European Medicines Agency Committee for Medicinal Products for Human Use reports that Astellas has withdrawn its application for an initial marketing authorization of Izervay (avancaptad pegol) for the treatment of geographic atrophy caused by age-related macular degeneration. The website PharmaPhorum also reported that the Food and Drug Administration issued a complete response letter on Astellas’ application to update the labeling for Izervay that sought to include the results of the Phase III GATHER2 trial to improve the drug's competitive profile against Syfovre (pegcetacoplan, Apellis).
Alkeus Pharmaceuticals has received Rare Pediatric Disease (RPD) and Fast Track designations from the Food and Drug Administration for the investigational oral therapy gildeuretinol (ALK-001), for the treatment of Stargardt disease. The RPD designation makes Alkeus eligible to receive a priority review voucher upon approval that could be used to advance another clinical development program. The FDA grants Fast Track designation to facilitate the development and expedite the review of drugs intended to treat a serious condition that has clinical data demonstrating the potential to address an unmet medical need. Gildeuretinol previously received Breakthrough Therapy and Orphan Drug designations from the FDA.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion for the marketing authorization of FYB203, a biosimilar candidate to Eylea (aflibercept), Formycon and licensing partner Klinge Biopharma report. Under the tradenames Ahzantive and Baiama, FYB203 has been recommended by the CHMP for approval in Europe for treating adult patients with neovascular, or wet, age-related macular degeneration, diabetic macular edema, visual impairment due to myopic choroidal neovascularization, and macular edema following retinal vein occlusion. The CHMP’s scientific assessment report forms the basis for the European Commission’s decision to grant a central marketing authorization valid in all European Economic Area countries. Formycon says it expects such approval in January 2025.
The European Commission itself has granted Sandoz marketing authorization for another aflibercept biosimilar, Afqlir, consisting of a 2-mg vial kit and prefilled syringe for intravitreal injection. Indications include neovascular age-related macular degeneration. Sandoz says launch is expected in Q4 2025.
Avirmax Biopharma has dosed the first patient in the Phase I/IIa clinical trial of ABI-110, a gene therapy drug for the treatment of wet, or neovascular, age-related macular degeneration, including polypoidal choroidal vasculopathy.
The NORSE EIGHT clinical trial of ONS-5010, an ophthalmic formulation of bevacizumab, failed to meet its primary endpoint, trial sponsor Outlook Therapeutics reports. As a result, the company says it anticipates resubmitting the biologic license application (BLA) to the Food and Drug Administration in the first quarter next year. The prespecified noninferiority endpoint at week 8 set forth in the special protocol assessment with the FDA was mean change in best corrected visual acuity from baseline to week 8, compared with ranibizumab (Lucentis, Genentech). At the same time, Outlook reported that the National Institute for Health and Care Excellence, which governs reimbursements in the UK, has recommended Lytenava, the name by which ONS-5010 is known in the UK and European Union, as an option for the treatment of wet age-related macular degeneration. Lytenava is approved in the UK and EU.
Topline results from the US Phase II clinical trial of VVN461-CS-201, a nonsteroidal dual-action janus activated kinase 1/tyrosine kinase 2 immunomodulator for the treatment of inflammation following cataract surgery, were positive, trial sponsor VivaVision Biotech reports. Among the findings: At day 14, 60% of VVN461 1% arm and 53.3% of the VVN461 0.5% arm achieved anterior chamber cell Grade 0 compared to 19.4% in the vehicle group.
EyePoint Pharmaceuticals has dosed the first patient in the Phase III LUCIA trial of Duravyu, once known as EYP-1901, for the treatment of wet age-related macular degeneration. Duravyu is an investigational sustained-delivery therapy delivering patent-protected vorolanib, a selective tyrosine kinase inhibitor formulated in proprietary bioerodible Durasert E for sustained intraocular delivery.
Ocular Therapeutix reports that more than 300 patients have been randomized in the SOL-1 Phase 3 trial of Axpaxli (axitinib intravitreal implant, also known as OTX-TKI), in wet age-related macular degeneration (AMD) and was on schedule to close randomization. The trial remains on track to report topline data in Q4 2025, Ocular states.
The Denali Phase III trial, evaluating NCX 470 in patients with open-angle glaucoma or ocular hypertension, is now fully enrolled in China, trial sponsor Nicox reports. Topline results are expected in Q3 2025.
Iantrek, a venture-backed, medical technology company, reported one-year results for the first 100 eyes enrolled in the CREST Registry, a real-world, prospective, observational study evaluating the outcomes of bio-interventional procedures in patients with open angle glaucoma. The study, published in Clinical Ophthalmology, reported on the safety and effectiveness outcomes of microinvasive cyclodialysis cleft formation with the CycloPen System, followed by scleral reinforcement with AlloFlo Bio-Tissue implantation. In patients with a baseline intraocular pressure (IOP) > 21 mmHg, 12-month mean IOP was reduced by 39.7%.
The first healthy volunteers have been dosed in the Phase I study of oral GAL-101, a small-molecule drug being investigated for the treatment of dry age-related macular degeneration and glaucoma, trial sponsor Galimedix Therapeutics announced. The study is designed to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of GAL-101. The Phase I study is planned to enroll up to 40 healthy volunteers in the single ascending dose part of the trial and 32 subjects in the multiple ascending dose part. The trial would enroll up to 120 subjects in total.
Beacon Therapeutics Holdings has reported three-month interim safety and efficacy results of the Phase II DAWN trial of AGTC-501, also known as laru-zova, a subretinal gene therapy to treat X-linked retinitis pigmentosa (XLRP). Data reported at the FLORetina-ICOOR Meeting 2024 in Florence, Italy, showed the treatment was well-tolerated with no treatment-related adverse events, including no ocular inflammatory adverse events, Beacon says, and that the benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by mutations in the RP GTPase regulator protein. Beacon also reports it is enrolling patients for the pivotal Phase II/III VISTA trial of laru-zova.
AAVantgarde Bio has been granted Orphan Drug Designation by the Food and Drug Administration for its lead program, AAVB-081 for the treatment of Usher syndrome Type 1B retinitis pigmentosa. Orphan Drug Designation provides various incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval. AAVB-081 is an intra-retinal adeno-associated virus-8–based dual hybrid product targeting MYO7A-associated Usher syndrome.
Dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis, trial sponsor Atsena Therapeutics reports. ATSN-201 uses Atsena’s proprietary gene therapy platform, adeno-associated virus SPR, to delivery therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the Food and Drug Administration.
Aurion Biotech has reported topline data from the Phase I/II clinical trial of AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction. AURN001 is a combination cell therapy product candidate comprising allogeneic human corneal endothelial cells (neltependocel) and a rho kinase inhibitor (Y-27632). AURN001 is intended to be administered to the anterior chamber of the eye as a one-time procedure.
The trial, titled CLARA, randomized 97 participants to five arms: three AURN001 doses (high, medium, low), neltependocel alone or Y-27632 alone. All three AURN001 arms demonstrated a dose-dependent response to meet the primary endpoint, which was the proportion of responders with a >15-letter improvement from baseline in best-corrected visual acuity. The high-dose arm demonstrated a statistically significant improvement, with 50% responding at six months vs. 14.3% of the Y-27632-only arm. No serious ocular adverse events were reported.
ViGeneron has received Food and Drug Administration clearance of its Investigational New Drug application for the Phase I/II study of VG801, a gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. VG801 uses ViGeneron’s proprietary dual adeno-associated virus vector technology, known as REVeRT (for REconstitution Via mRNA Trans-splicing). VG801 utilizes a novel vgAAV capsid derived from ViGeneron’s proprietary vgAAV technology and designed to enable widespread retinal transduction. The Phase I/II multicenter, open-label, dose-escalation clinical trial will evaluate the safety, tolerability, and preliminary efficacy of VG801. The study will recruit patients globally. ViGeneron says it plans to submit a Clinical Trial Application to the European Medicines Agency in the coming months.
ViGeneron also announced that the FDA has selected its proposal for the Rare Disease Endpoint Advancement pilot, a program is designed to foster innovation and advance rare disease drug development programs, one of three such programs the FDA accepts each year for fiscal years 2024 to 2027.
eyeDNA Therapeutics has been granted a Rare Pediatric Disease Designation by the Food and Drug Administration for HORA-PDE6b, its gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6b gene, specifically PDE6b retinitis pigmentosa. HORA-PDE6b is an adeno-associated virus-5–based gene therapy designed to deliver a non-mutated copy of the functional human PDE6b gene into the subretinal space.
Roche reports that the European Medicines Agency has approved Vabysmo (faricimab) 6.0 mg single-dose prefilled syringe (PFS) for use in the treatment of neovascular or wet age-related macular degeneration, diabetic macular edema, and macular edema following retinal vein occlusion.
The Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted positive opinions and recommended marketing authorizations for Celltrion’s aflibercept biosimilar Eydenzelt, also known as CT-P42. Eydenzelt 40 mg/mL solution is available in a vial and prefilled syringe. Approved indications are neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema, and myopic choroidal neovascularization. Celltrion also received approval for biosimilars to treat postmenopausal osteoporosis and arthritis and giant cell arteritis.
Recruiting through a pandemic, Nacuity Pharmaceuticals completed enrollment for its SLO-RP Phase I/II clinical trial of NPI-001 The study is designed to assess the safety, tolerability, and efficacy of NPI-001 tablets versus placebo in patients with retinitis pigmentosa (RP) associated with Usher syndrome. The company expects to have efficacy data by the end of the year.
Coave Therapeutics, a gene therapy company headquartered in France, has 12-month results from its Phase I/II trial of CTx-PDE6b, a gene therapy for RP caused by bi-allelic mutations in the PDE6B gene. Doses were well-tolerated in all groups, the company reports; however, the subgroup with less-advanced disease who received a higher dose showed positive efficacy consistently across five endpoints.
Roche Canada has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) for VABYSMO (faricimab injection), indicated for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME). The company secured a letter of intent from pCPA, which is a critical step in making VABYSMO available in many provinces throughout Canada.
Annexon announced disappointing topline results from its ARCHER Phase II trial of ANX007 in patients with geographic atrophy (GA). The primary endpoint—mean rate of change (slope) in GA lesion area compared to sham at 12 months—did not reach statistical significance. It did, however, demonstrate statistically significant, dose-dependent preservation of visual function. The company plans to engage with the FDA to determine the next step forward. “The ultimate goal for any physician is to preserve vision for our patients for as long as possible,” said investigator Jeffrey Heier, MD, Director of the Retina Service and Retina Research, Ophthalmic Consultants of Boston, in a statement. “The totality of the data on ANX007 from the ARCHER trial are promising, with the demonstrated preservation of functional vision in GA patients, regardless of their lesion location or size. I am encouraged with the overall profile of ANX007 and look forward to its continued development.”
C. Light Technologies received 510(k) clearance from the FDA for Retitrack, its retinal eye movement monitor. The device is designed to offer insight into oculomotor function by using the retina, providing medical professionals with concrete, objective metrics.
iHealthScreen submitted its 510(k) for iPredict, a fully automated artificial intelligence–based software used for early diagnosis of AMD. The tool provides retinal screening and delivers an immediate report of results.
Alimera has completed enrollment for its randomized, controlled, multicenter study designed to generate prospective data for ILUVIEN (fluocinolone acetonide intravitreal implant, 0.19mg) as a baseline therapy in patients diagnosed with DME. The study will compare ILUVIEN, a corticosteroid, with an anti-VEGF therapy. The company expects to share data in 2025.
AiViva Biopharma launched a Phase I trial for AIV007, a broad-spectrum tyrosine kinase inhibitor, targeting the convergence of fibrosis, angiogenesis, and inflammation in patients with wet AMD, and/or diabetic macular edema (DME).
Oculis reported results from stage one of its Phase III DIAMOND trial for OCS-01, an eyedrop designed to treat DME. OCS-01 showed statistically significant improvement in mean best corrected visual acuity ETDRS score from baseline to week six of 7.2 letters, compared with 3.1 letters in those who received vehicle—a results that lasted through week 12.
HanAll Biopharma Co. reported that its Phase III VELOS-3 trial didn’t demonstrate significant improvement in central corneal staining score or in Eye Dryness Score via Visual Analogue Scale at eight weeks. The study evaluated the safety and efficacy of tanfanercept, a novel, topical anti-inflammatory treatment for patients with moderate to severe dry eye disease. The company plans to continue to study tanfanercept, possibly in higher concentrations.
Last week, OIS podcast guest Natasa Jovic of RetinAI talked about the implications of AI for ophthalmologists and the eye care industry at large.
Here’s another take on AI in retina: AI expert Eric Souied, MD, PhD, head of Department of Ophthalmology at the “Hopital Intercommunal de Creteil” in France, shares his thoughts on using deep learning to screen and interpret retinal images to look for retinal lesions that may indicate diabetic retinopathy (DR). He also shares results from a retrospective analysis of 224 patients with DR using deep learning and ultra-widefield imaging. Read the Article.
Responding to the approval of MIEBO (perfluorohexyloctane ophthalmic solution) from Bausch + Lomb/Novaliq, reportedly the first FDA-approved dry eye disease treatment that targets tear evaporation, a few experts gave comments to Ophthalmology Times:
“We were a phase 3 study site and have early experience with perfluorohexyloctane,” said Laura M. Periman, MD, director of Dry Eye Services and Clinical Research, Periman Eye Institute in Seattle, Washington. “With this approval, US doctors now have access to a first-in-class alkane drop as a topical ophthalmic therapeutic for the signs and symptoms of dry eye disease.”
Ernest W. Kornmehl, MD, of Kornmehl Laser Eye Associates, Harvard and Tufts Universities said MIEBO’s advantages include “low volume, surface tension, and viscosity and are amphiphilic.” He added it will take real-world evidence from thousands of patients to know for sure whether MIEBO is a big step forward for dry eye. Thoughts? Read More About MIEBO.
The FDA approved Bausch + Lomb and Novaliq’s MIEBO (perfluorohexyloctane ophthalmic solution; fka NOV03). MIEBO is the first and only FDA-approved treatment for dry eye disease that directly targets tear evaporation.
C. Light Technologies received 510(k) clearance from the FDA for the Retitrack, a monocular, tabletop eye-movement monitor that is intended for recording, viewing, measuring, and analyzing temporal characteristics of fixation and saccadic responses when viewing a visual stimulus. The company says Retitrack is the first retinal eye-movement monitor cleared for use in the healthcare setting.
Invirsa completed enrollment of its Phase I/IIa clinical trial of a topically administered eyedrop formulation of INV-102 for dry eye disease. The company also received approval from Thailand FDA equivalent to test INV-102 in patients with acute infectious keratoconjunctivitis (AIK).
Glaucoma patients who underwent surgical procedures with the Omni Surgical System from Sight Sciences had a greater average reduction in the use of intraocular pressure-lowering medication, compared to those who had received only minimally invasive glaucoma
procedures or cataract surgery. This is what Sight Sciences found during a 12-month retrospective sub-analysis study analyzing 17,000 records from the American Academy of Ophthalmology’s (AAO) Intelligent Research in Sight (IRIS) Registry. Here's a summary of the study.
The Foundation for the National Institutes of Health (FNIH) has selected eight rare diseases the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC). The consortium’s mission is to help speed the development and delivery of customized gene therapies. Three of the rare diseases selected are in ophthalmology: Congenital Hereditary Endothelial Dystrophy, NPHP5 Retinal Degeneration, and retinitis pigmentosa. [More Info]
The FDA has approved Eyenovia’s MydCombi (tropicamide and phenylephrine hydrochloride ophthalmic spray) 1%/2.5% for inducing mydriasis for diagnostic procedures and in conditions where short-term pupil dilation is desired. According to Eyenovia, the approval represents the first approved, fixed-dose combination of tropicamide and phenylephrine in the United States. It’s also the first approved product that uses Eyenovia’s Optejet device.
On the device side, the ECHO Green Pattern Laser photocoagulator has received both FDA 510(k) clearance and a CE Mark. The ECHO from Norlase is a portable scanning laser photocoagulator that uses MEMS technology.
The FDA accepted Glaukos’s New Drug Application (NDA) for iDose TR (travoprost intraocular implant). iDose TR is designed to continuously deliver therapeutic levels of a proprietary formulation travoprost formulation from within the eye for extended periods of time. It is designed so it can be removed and replaced with a new iDose TR, making it a potential alternative to eye drop treatment. The Prescription Drug User Fee Act (PDUFA) goal date is set for December 22, 2023.
Meanwhile, the FDA accepted Genentech’s supplemental biologics license application (sBLA) for Vabysmo (faricimab-svoa) for macular edema following retinal vein occlusion (RVO) treatment. It was approved for the treatment of wet age-related macular degeneration (AMD) and diabetic macular edema (DME) in the U.S. in January 2022.
Formosa Pharmaceuticals and AimMax Therapeutics submitted their NDA for APP13007, a novel aqueous nanosuspension formulation of the corticosteroid clobetasol propionate (0.05%). The product is intended to treat inflammation and pain following ocular surgery.
University of California, Irvine, researchers may have discovered a new drug class aimed at age-related macular degeneration (AMD), diabetic retinopathy (DR), and retinitis pigmentosa (RP). Called Stress Resilience-Enhancing Drugs (SRED), the small molecule intervention enhanced resilience to acute and chronic forms of stress in the degenerating retina, thus preserving tissue structure and function. [Read the Abstract]
It's called an epidemic. Now nine pediatric ophthalmologists are calling on the medical community to recognize myopia as a disease. They advocate for myopia management by delaying onset and slowing its progression. “Myopia progression needs to be treated and controlled, not just corrected for clear vision,” the doctors write in a white paper sponsored by CooperVision. [Read the White Paper]
Enrollment is complete for Endogena Therapeutics’ Phase I/IIa trial of EA-2353, a small molecule drug for the treatment of retinitis pigmentosa (RP). The study enrolled 14 patients, with topline interim data expected in early 2024.
Smartlens has announced positive clinical results for miLens, a noninvasive, electronics-free, soft contact lens for monitoring intraocular pressure (IOP). The study reported a significant and statistically strong correlation between IOP measurements obtained with miLens and those obtained using Goldmann applanation tonometry (GAT) in twenty-five individuals.
Centricity Vision has received 510(k) clearance for the ZEPTOLink IOL Positioning System. The platform integrates the ZEPTO precision pulse capsulotomy technology with any phacoemulsification system, with the potential to streamline cataract surgery.
Nanoscope Therapeutics has announced results from its Phase IIb trial investigating MCO-010 in patients with RP. The results showed 100% of the patients treated with the gene therapy exhibited vision improvement in all tests used.
BVI has completed enrollment in its US Investigational Device Exemption (IDE) clinical study for its latest hydrophobic trifocal intraocular lens, FINEVISION HP. The study enrolled 539 patients, who will now be followed to evaluate safety and performance of the lens.
The FDA has cleared Atsena Therapeutics’ investigational new drug (IND) application for a Phase I/II clinical trial of ATSN-201. The study will examine the safety and efficacy of the gene therapy in patients with X-linked retinoschisis (XLRS).
Visus Therapeutics announced topline data from its pivotal Phase III trial of Brimochol PF, a preservative-free solution designed to treat presbyopia. The product met its primary endpoint: a proportion of subjects achieved > 15 ETDRS1 letter gain in Binocular Near Visual Acuity without a loss of > five letters at distance across all time points through six hours. [Read About the Study]
Harrow has completed the transfer of NDAs for three FDA-approved ophthalmic drugs: ILEVRO, NEVANAC, and MAXIDEX. They represent three of the five ophthalmic products Harrow purchased in January. They’re commercially available now under the Harrow name.
Théa Pharma wants to make clear: the products it acquired from Akorn Operating Company were not affected by Akorn’s recall. The company ceased operations and voluntarily recalled all its products. Théa acquired seven branded ophthalmic products from Akorn, which filed for bankruptcy in February and fired all its employees, which left the company without quality control.
Elios Vision has completed enrollment for its pivotal trial for its Elios excimer laser procedure in combination with cataract surgery. The trial recruited over 300 patients with mild to moderate glaucoma. The procedure has already received a CE mark.
Molecure filed an Investigational New Drug application with the FDA for OATD-01, a drug developed to treat sarcoidosis. If the FDA approves the NDA, Molecure will begin a Phase II trial for the chitotriosidase (CHIT1) inhibitor. Inhibition of CHIT1 by OATD‑01 has been shown to reduce inflammation and fibrosis, the company states.
The Brazilian Health Regulatory Agency (ANVISA) has approved Tepezza for the treatment of thyroid eye disease (TED). Tepezza, from Horizon Therapeutics, is the first medicine approved in Brazil for TED the company stated. The product received FDA approval in 2020, and regulatory approval in Japan appears on the horizon (pun intended).
Visgenx announced positive topline data from a preclinical study of VGX-0111 for dry age-related macular degeneration (AMD). The study, in nonhuman primates, successfully demonstrated three parameters: VGX-0111 expresses in the target tissues, causes an increase in certain very long-chain polyunsaturated fatty acids, and is well tolerated.
Intergalactic Therapeutics also announced positive results from its nonviral gene therapy platform, IG-002, which addresses retinopathies related to the ABCA-4 gene. The platform safely achieved durable 6-month expression of human ABCA-4 in non-human primate retinas with a single subretinal administration.
OcuTerra Therapeutics reports completing enrollment in the Phase II DR:EAM clinical trial evaluating topical OTT166 eye drops in adults with moderately severe to severe non-proliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy (PDR) with minimal vision loss. DR:EAM stands for Diabetic Retinopathy: Early Active Management. The trial enrolled 225 adult patients who were randomly assigned one of two doses of OTT166 or to one of two control groups receiving vehicle. OTT166 is a small-molecule integrin inhibitor. [For further details: https://www.ocuterratx.com/for-physicians]
Kodiak Sciences has scrapped its investigative program for tarcocimab tedromer, an anti-vascular endothelial growth factor (VEGF) biopolymer conjugate, after reporting disappointing topline results from two of three Phase III studies. The DAYLIGHT trial (n=557) in neovascular, or wet, age-related macular degeneration (nAMD), met its primary endpoint of noninferiority, compared to monthly aflibercept (Eylea, Regeneron Pharmaceuticals) and demonstrated tarcocimab was safe and well tolerated with a low rate of intraocular inflammation. The GLEAM and GLIMMER studies (n=460 and n=457) in diabetic macular edema (DME), comparator-controlled studies with aflibercept, failed to meet their primary efficacy endpoints of achieving noninferior visual acuity gains. Additionally, an unexpected increase in cataracts was observed over time in the tarcocimab arms of both trials.
Biocon Biologics got a favorable opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommending approval of its aflibercept biosimilar, Yesafili. The potential indications match those of the reference product, Eylea: nAMD, macular edema secondary to retinal vein occlusion, DME and myopic choroidal neovascularization. The European Commission will consider the CHMP opinion, with a decision on approval expected by the end of September.
The SAHARA trial met its primary six-month endpoint of achieving superiority of interventional eyelid procedures enabled by TearCare eye drops over Restasis (Allergan/AbbVie), trial sponsor Sight Sciences reported. The trial demonstrated an improvement of tear break-up time (TBUT) with TearCare, a key measure of aqueous retention, tear stability and the tear film’s ability to protect the ocular surface. SAHARA also showed that procedures enabled by TearCare were noninferior to Restasis eyedrops in Ocular Surface Disease Index (OSDI), the co-primary six-month endpoint.
The American Society of Retina Specialists has sent an e-mail to members informing them that it has received reports of intraocular inflammation, including six cases of vision-threatening occlusive retinal vasculitis, following injection of Syfovre (pegcetacoplan) into the eye. The notification came almost five months to the day after the Food and Drug Administration approved pegcetacoplan as the first intravitreal treatment for GA, an advanced form of dry age-related macular degeneration (AMD). The e-mail, issued as an update by the ASRS’ Research and Safety and Therapeutics (ReST) committee, noted that the reported events were observed between 7 and 13 days after injection. No specific drug lots have been implicated. “The etiology of these events is unclear, and outcomes in these patients are still evolving,” the e-mail stated. In a statement, Apellis Pharmaceuticals noted that the rate of retinal vasculitis in real-world use was about 0.01% per injection. “We are in the process of thoroughly investigating all cases with external experts, including real-world procedure technique and potential risk factors,” the statement
Clearside Biomedical initiated enrollment and dosing in its Phase IIb ODYSSEY trial of CLS-AX, an injectable suspension of the tyrosine kinase inhibitor axitinib for the treatment of neovascular, or wet, age-related macular degeneration (AMD). CLS-AX is injected into the suprachoroidal space of the eye using Clearside’s patented SCS Microinjector, which provides targeted delivery to the disease site. Topline data are expected in the Q3 2024.
Belite Bio received approval from the Taiwan Food and Drug Administration to initiate the Phase III PHOENIX clinical trial of Tinlarebant for geographic atrophy (GA) associated with dry AMD. The multicenter, double-masked, placebo-controlled, randomized, fixed-dose clinical study has been underway in the US. It will enroll approximately 430 patients with a 2:1 randomization of tinlarebant:placebo. Formerly known as LBS-008, Tinlarebant is a novel oral therapy that targets toxins in the eye that cause Stargardt disease and contribute to GA.
The FDA strongly encourages clinical trial sponsors to recruit patient populations that mimic the population of individuals who have that disease. A study published this week in Nature found that minority groups are consistently underrepresented in glaucoma and retina clinical trials. That is concerning, given primary open-angle glaucoma’s prevalence is higher among older Hispanic or Latino individuals (18%) and Black individuals (15%) than among White adults (7%) according to NIH data. Read the full study for details (subscription required).
Cognition Therapeutics dosed its first patient in its Phase II MAGNIFY study of CT1812, an experimental oral geographic atrophy (GA) treatment. The company intends to enroll 246 adult patients diagnosed with dry age-related macular degeneration (AMD) with measurable GA. Erie Retinal Surgery in Erie, PA, enrolled the first participant.
Viridian Therapeutics announced positive topline preliminary data from its Phase I/II trial of VRDN-001, a drug used to treat thyroid eye disease (TED). According to investigator Kimberly Cockerham, MD, patients in the 10 mg/kg and 3 mg/kg dose cohorts experienced reductions in proptosis as well as improvements in their clinical activity scores after receiving two infusions of VRDN-001.
Nacuity Pharmaceuticals added another site to its Phase I/II clinical trial for NPI-002, a proprietary antioxidant molecule delivered via a sustained-release intravitreal implant, developed to delay cataract progression in patients undergoing vitrectomy. The trial is already underway at the existing site: The Royal Adelaide Hospital in Adelaide, South Australia.
Upon his return to Bausch + Lomb, CEO Brent Saunders made a decidedly Brent Saunders move when he decided to acquire Xiidra and other assets from Novartis (see Friday’s newsletter for more details if you haven’t seen them already). With this deal, “B+L now owns the most complete and comprehensive portfolio of dry eye disease medications in eye care,” says Darrell White, MD, in an editorial for Healio. [Full Story]
RVL Pharmaceuticals has launched e-commerce platform called ELEVATE. ELEVATE enables providers and patients to efficiently purchase RVL products. Features included auto-refills, subscription-based pricing, home delivery, and an easy online checkout process. To see it in action visit the RVL website.
Mount Sinai’s Icahn School of Medicine recently launched the Center for Ophthalmic Artificial Intelligence and Human Health, one of the few research centers of its kind in the US. The Center aims to advance AI in ophthalmology in multiple ways. Partnering with the Windreich Department of Artificial Intelligence and Human Health, the Center will explore AI-based diagnostic care. According to a company press release, the Center also hopes to help better predict cardiovascular health, as research has shown AI-guided retinal imaging can reveal cardiovascular risk factor. [Learn More]
Will our vision get worse as the world gets hotter? According to a recent study, that could be the case. A team of researchers at University of Toronto found that adults aged 65 years and older who lived in countries with an average temperature of 60 degrees or above had a 44% higher risk of severe vision impairment than did those who lived in a country with a 50 degree or lower average temperature. While the study did not determine the cause, the researchers suspect increased ultraviolet light exposure, air pollution, infections, and folic acid degradation and potential culprits. [Read the Study]
Salvat Laboratories initiated a new study in Europe to test its corticosteroid in children up to age three years who have cataracts. The trial examines the safety and efficacy of SVT-15473 clobetasol to treat inflammation and pain in pediatric patients after cataract surgery. The study is part of an FDA approval process. It will include 60 patients. The study has enrolled four patients to date.
The FDA cleared Skyline Therapeutics’s Investigational New Drug (IND) application for Phase I/IIa clinical trials for SKG0106, a one-time intravitreally delivered recombinant AAV gene therapy for the treatment of neovascular age-related macular degeneration (nAMD). The company plans to launch its Phase I trial “soon” according to a company press release.
Formycon and Klinge Biopharma have submitted a biologics license application for FYB203, an Eyelea biosimilar, to the FDA. “With FYB203 we are going to address an important as well as growing market for the treatment of severe retinal diseases and contribute to alternative and cost-effective therapeutic options,” said Formycon CEO Stefan Glombitza, PhD, in a press release.
At the 9th Congress of the European Academy of Neurology (EAN) in Budapest this week, Horizon Therapeutics presented new data from its Phase III trial of Uplinza (inebilizumab-cdon). The data show reduced formation of subclinical (asymptomatic) transverse myelitis lesions in people with neuromyelitis optica spectrum disorder (NMOSD). Transverse myelitis lesions typically occur in people with infections or autoimmune diseases.
“This analysis offers new insights into the significance of subclinical MRI findings on the spinal cord as a potential signal of future attacks, adding to the growing list of tools available for physicians to better monitor their patients,” said Friedemann Paul, study author and Group Leader of the Clinical Neuroimmunology Department of NeuroCure Clinical Research Centre at the Charité, Berlin, Germany, in a news release.
Intergalactic Therapeutics released positive preclinical data from IG-002, its lead program for the treatment of ABCA4-related retinopathies. The gene therapy safely achieved durable six-month expression of human ABCA4 in non-human primate retinas. ABCA4 currently has no approved treatments.
Glaukos Corp. has submitted its New Drug Application (NDA) to the Food and Drug Administration for Epioxa, its next-generation corneal cross-linking therapy for the treatment of keratoconus. The NDA submission includes data from two Phase III pivotal trials of Epioxa, both of which met the primary efficacy endpoints and demonstrated favorable tolerability and safety profiles. The company anticipates Epioxa could be the first FDA-approved, noninvasive corneal cross-linking therapy that does not require removal of the corneal epithelium.
Ocugen reports that its Data and Safety Monitoring Board (DSMB) has cleared continuation of the Phase I/II ArMaDa clinical trial of OCU410, a gene therapy designed as a one-time treatment for geographic atrophy caused by dry age-related macular degeneration. The DSMB reviewed data from 15 participants in the ongoing Phase II study and found that OCU410 demonstrated an acceptable safety and tolerability profile, Ocugen says. The trial, conducted in 13 US retinal centers, is evaluating the the safety and efficacy of OCU410 in 45 patients randomized into two dosing groups or a control group.
Regeneron Pharmaceuticals reports the Phase III QUASAR trial of Eylea HD (aflibercept 8 mg) for macular edema following retinal vein occlusion has met its primary endpoint, which consisted of equivalent improvement in visual acuity, compared to Eylea 2 mg at 36 weeks. Regeneron says the data will be submitted to regulatory authorities around the world, with a submission to the Food and Drug Administration planned for the first quarter of 2025.
Galimedix Therapeutics has launched the Phase II study of GAL-101 eye drops for treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration. The study is planned to enroll up to 110 patients, who will be randomized 1:1 to receive the study drug or placebo. The primary endpoint of the trial is reduction in rate of change of GA lesion size. The trial will also evaluate the reduction in rate of change in the area of photoreceptor degeneration and various functional outcome measures, including retinal sensitivity on microperimetry with patient-tailored grid design.
ViGeneron reports two key milestones for its gene therapy candidate VG901 to treat retinitis pigmentosa caused by mutations in the CNGA1 gene: The Food and Drug Administration has granted Rare Pediatric Disease Designation; and the independent Data Safety Monitoring Board has unanimously approved dose escalation in the ongoing Phase Ib clinical trial.
Oculis Holding has reported topline results from the Phase II ACUITY trial of OCS-05, a neuroprotective candidate for treatment of acute optic neuritis. The trial met its primary endpoint of safety and statistical significance on several key efficacy-based secondary endpoints, according to Oculis. The study randomized 36 patients with visual loss from unilateral acute optic neuritis. Treated patients demonstrated a 43% improvement in ganglion cell-inner plexiform layer thickness and a 28% improvement in retinal nerve fiber layer thickness, compared to placebo.
Sight Sciences reports that 36-month results of the Omni Surgical System for management of primary open-angle glaucoma (POAG) have been published in the American Journal of Ophthalmology. The real-world study evaluated 230 eyes of 196 patients with POAG through up to 36 months. The results demonstrated clinically and statistically significant reductions in intraocular pressure (IOP) through 36 months postoperatively, with mean reductions ranging from 5.6 to 7.1 mmHg. The study also reports a statistically significant decrease in medication use through 18 months. Eyes with lower baseline IOP experienced medication reduction through 36 months.
Results of the Phase Ib trial of sozinibercept combination therapy in diabetic macular edema (DME) have been published in the journal Translational Vision Science & Technology, trial sponsor Opthea reports. The trial evaluated the outcomes in patients with persistent DME previously treated with aflibercept 2 mg (Eylea, Regeneron Pharmaceuticals) only. In the trial, nine patients received sozinibercept (0.3, 1, or 2 mg) in combination with aflibercept once every four weeks for 12 weeks. Sozinibercept combination therapy was well tolerated with no dose-limiting toxicities. The trial also demonstrated a dose-response relationship of increased gains in best-corrected visual acuity (BCVA) for ascending doses of sozinibercept, with the 2 mg sozinibercept combination arm demonstrating the highest BCVA gain. All sozinibercept doses demonstrated a meaningful reduction of central subretinal thickness, a key biomarker of DME. Opthea says the data support the further investigation of sozinibercept as a treatment for DME.
South Korea-based Caregen has reported interim Phase I results from US clinical trials of CG-T5, an eye drop formulation that selectively binds to vascular endothelial growth factor receptor-2, a critical protein in abnormal blood vessel growth in retinal disease. The results demonstrated improvement in best-corrected visual acuity and central retinal thickness, along with strong safety and tolerability profiles, according to Caregen. The company says it plans to expand CG-T5’s indications in Phase II trials to include diabetic retinopathy and other conditions.
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Rich Kirkner
Contributing Editor
rkirkner@ois.net