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.

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 21^st 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.

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.

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.