OIS RETINA PRESENTING COMPANIES
Anders Haegerstrand, MD, PhD, CEO - Annexin Pharmaceuticals
Annexin Pharmaceuticals is a publicly listed (NASDAQ/First North, Sweden) clinical biotech advancing ANXV (a human recombinant protein Annexin A5 ) first-in-class biologic with a broad therapeutic potential as well as a protein- drug conjugate ANXV-chemo. IT is based on research from Karolinska Institutet .The Company focuses on ophthalmology ( clinical stage) and immuno-oncology.
Retinal Vein Occlusion is the primary indication in focus. ANXV has a potential for first in line first in class biologic therapy. The mode of action is non-overlapping to the standard of care (anti-VEGF).
ANXV has completed a Phase 1 trial and demonstrated favorable safety and tolerability profile.
The Phase 2a clinical trial in Retinal Vein Occlusion is approved by the FDA and is currently enrolling in the USA.
The CMC process is optimized.
The IP portfolio is strong with the key patents recently issued in major territories.
Annexin management are looking at strategic options that would facilitate long term development of ANXV/ANXV-conjugate.
Jamie Dananberg, MD, Chief Medical Officer - Annexon BioSciences
Annexon is pioneering a class of new complement medicines for patients with classical complement-mediated disorders of the body, brain and eye. We are conducting ongoing clinical trials in multiple serious autoimmune, neurodegenerative and ophthalmic diseases.
Beth Marsh, VP, North America Sales and Marketing - Apellis
Apellis is a global biopharmaceutical company that leverages courageous science, creativity, and compassion to lead the development of therapies that target the complement system. Apellis is committed to addressing the unmet medical needs of patients and eye care professionals worldwide, with the goal of slowing progression of Geographic Atrophy.
Sharon Bakalash, MD, PhD, Executive Director - Astellas Pharma
Astellas Pharma US, Inc., is a U.S. affiliate of Tokyo-based Astellas Pharma Inc., with its headquarters located in Northbrook, Illinois. More than 3,000 Astellas employees are based in the United States. Astellas is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products.
Charles P. Semba, MD, Chief Medical Officer - Eluminex Biosciences
Clinical stage, privately-held (Series B+), bi-coastal biotech (Suzhou BioBAY/Mountain View, CA) focused on the development of advanced protein therapeutics for ophthalmics, regenerative medicine, and dermal/facial aesthetics. EB-105 is lead retina asset and is the world's first true tri-specific fusion antibody targeting VEGF-A/PlGF, Ang-2, and IL-6R for diabetic macular edema. Entering clinic mid-year 2024 (first-in-human) in the United States.
Matthias Steger, PhD, MBA, Co-Founder & CEO - Endogena Therapeutics
Endogena Therapeutics is a clinical-stage biotech company that discovers and develops first-in-class endogenous regenerative medicines to repair and regenerate tissues and organs. Our approach has the potential of a paradigm shift to treat degenerative conditions related to ageing and genetic disorders.
Endogena is focusing its drug discovery and development efforts on indications with high unmet medical needs. Recent discoveries in stem cell biology together with technological advances for phenotypic, functional screening, have unlocked our ability to harness the potential of adult stem- and progenitor cells. Our novel drug discovery approach is based on the concept of selective regulation of these endogenous cells for controlled tissue repair by small molecules.
Endogena’s lead program for retinitis pigmentosa is in clinical phase. We are working with leading vitreoretinal specialists across the US and Europe to bring a novel treatment paradigm to retinitis pigmentosa patients who are losing their vision with no broadly applicable treatment available.
Scott Jones, Chief Commercial Officer - EyePoint Pharmaceuticals
EyePoint Pharmaceuticals (Nasdaq: EYPT) is a clinical-stage biopharmaceutical company committed to developing and commercializing therapeutics to help improve the lives of patients with serious retinal diseases. The Company's pipeline leverages its proprietary bioerodible Durasert E ™ technology for sustained intraocular drug delivery. The Company’s lead product candidate, EYP-1901, is an investigational sustained delivery.
Kenichiro Kuninobu, PhD, CEO - FELIQS Corporation
FELIQS is an early stage(IND-ready) lifescience company with focus in retinal diseases.
Our programs are as follows:
- Retinopathy of prematurity (ROP) – FLQ-101, a lipid peroxidation inhibitor repurposed to prevent ROP. FLQ-101 will be in Phase I/II dosing planned in the US in 2024.
- Age-related macular degeneration (AMD) – FLQ-104, a proprietary lipid peroxidation and ferroptosis inhibitor for earlier intervention to treat Intermediate Dry AMD. We are targeting completion of all IND-enabling studies by the end of 2025.
We are currently raising a 25M Series A and we are actively looking for investors to participate in this round.
Bob Katz, CEO - ForwardVue Pharma
ForwardVue Pharma is a physician-founded ophthalmic start-up developing a unique small molecule compound to significantly advance the treatment of chronic retinal diseases, including AMD, DME & DR. Our unique bioresorbable implant is the only entrant in retina space (clinical or commercial) with a highly differentiated, multi-target mechanism of action, an established human safety data profile and pre-clinical in vivo results. In multiple pre-clinical validated models of retina disease, we have demonstrated superior potency to the gold-standard treatments, i.e., Eylea.
Our results indicate that both better visual outcomes and reduced treatment burden (injections) are achievable in a simple monotherapy. We have formulation, compelling pre-clinical results and successful pre-IND engagement with FDA. We’re developing a simple, potent and safe treatment for a rapidly expanding market.
Max Cotler, Chief Operating Officer - GelMEDIX
GelMEDIX is a seed-stage, Harvard and UCLA founded, cell therapy company, with an initial focus on restoring vision via retinal cell therapy while overcoming the fundamental challenges of current technologies. The company’s initial target indication, Geographic Atrophy (GA), is the leading cause of blindness in American adults, affecting over one million individuals, with forty-two percent legally blind. The first and recently approved treatments have marginal clinical benefit (20% slowing GA lesion growth). Only a regenerative cell replacement therapy has the promise to restore vision for GA patients and bring back their quality of life. Current retinal cell therapies in early clinical development, specifically using suspensions or sheet implants of retinal pigment epithelial (RPE) cell approaches, have shown encouraging, early vision benefits in GA patients one year after transplantation and, importantly, with no evidence of rejection. However, both these approaches suffer from fundamental procedural limitations that lead to intolerable adverse event rates (i.e., retinal membrane formation, retinal detachment/hemorrhage), potentially limiting their utility.
Brian Levy, OD, MSc, CEO - InflammX Therapeutics
Startup developing a new drug to treat the autoimmune signs and symptoms resulting from the dysregulated innate immune system called the NLRP3 inflammasome. This innate system was only discovered in 2002, and once activated by certain disease states, leads to perpetuated autoimmunity. The drug we are developing is orally administered QD and it's MOA is selective inhibition of the dysregulated NLRP3. The drug achieves this goal by working upstream at the cellular lever where the activation primarily occurs. The drug target is the pathological connexin43 (cx43) hemichannel. Physiologically the Cx43 hemichannel forms and resides in the cell membrane of most of the organs in the human body. In the physiological state they remain closed until they dock with another hemichannel in an adjoining cell. At this time both hemichannels open to create the gap junction between cells. This activity facilitates cell-cell communication. However, as a result of certain disease states, the Cx43 hemichannel becomes overexpressed crowding the cell membrane, The result is premature opening and the release of ATP into the extracellular space. The ATP signals the NLRP3 to assemble and activate multiple proinflammatory cytokines. If allowed to continue to perpetuate, the immune system becomes autoinflammatory resulting in chronic disseminated pathology. The InflammX drug Xiflam acts on a cytoplasmic loop of the pathologically open hemichannels to close them. This in turn shuts down the extracellular release of ATP and the perpetuated signaling of the NLRP3.
Our primary clinical indications are the intermediate form of dry macular degeneration (iAMD), diabetic retinopathy (DR) and diabetic nephropathy (DN).
Patientsare treated bilaterally with an orally administered drug QD. The DR/DN study is currently underway and we are currently seeking to close a round of financing for the iAMD clinical trial.
Pierre Dromel, PhD, Founder & CEO - InGel Therapeutics
InGel is a preclinical biotech company with the mission to bring back light to patients whose world has gone dark. Having spun out of Professor Michael Young’s lab at Harvard in 2022, InGel combines tissue engineering and regenerative medicine to target retinal degeneration. Its lead program, IGT001, is a mutation-agnostic approach to Retinitis Pigmentosa. Leveraging the key protein factors secreted by its proprietary cell line, IGT001has generated encouraging data in restore visual functions across multiple preclinical models. The company is currently raising its seed round with the goal to take IGT001 to the clinic, by the end of 2025.
Marion R. Munk, MD, PhD, CMO - Isarna Therapeutics
Isarna Therapeutics is a Munich, Germany based clinical stage company, developing innovative therapies to address high unmet need in retinal pathologies. Antisense-based therapies, with ISTH0036 as lead compound are currently tested in Phase 2 studies in AMD and DME, which block the production of Transforming growth factor beta, (TGF-b), one of the key factors in the development of retinal edema and fibrosis. Current treatments do not suppress the development of fibrosis, so a high unmet need is addressed to prevent this important aspect of ocular pathologies.
John Pollack, MD, Chief Medical Officer - jCyte
jCyte is in late-stage development of a first-in-class allogeneic cell therapy for retinitis pigmentosa. The treatment is a minimally-invasive intravitreal injection than can be performed with topical anesthetic.
Brian Strem, CEO - Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan retinal diseases. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of posterior non-infectious uveitis. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase. In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on Twitter and LinkedIn as well as to visit our website and/or subscribe to email alerts.
Samarendra Mohanty, PhD, Co-Founder & President - Nanoscope Therapeutics
Nanoscope Therapeutics is a clinical-stage biopharmaceutical company developing gene therapies to cure genetic diseases that cause vision impairment and blindness.
These diseases include Retinitis Pigmentosa, Dry Age-related Macular Degeneration (AMD), and Stargardt Disease. We are developing a proprietary platform technology, employing ambient light-sensitive Multi-Characteristic Opsins (MCO) that has potential to treat a broad range of genetic illnesses.
Richard Small, CEO - Neurotech Pharmaceuticals
Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies to slow the progression of Macular Telangiectasia (MacTel) Type 2 and other chronic retinal diseases through its core platform technology, Encapsulated Cell Therapy (ECT). To learn more, visit https://www.neurotechpharmaceuticals.com/.
Sanjay Nayak, MBBS, PhD, Chief Strategy Officer - Ocular Therapeutix
Ocular Therapeutix, Inc. is a biopharmaceutical company committed to enhancing people’s vision and quality of life through the development and commercialization of innovative therapies for retinal diseases and other conditions of the eye. Ocular’s program for retinal disease is AXPAXLI™ (axitinib intravitreal implant, also known as OTX-TKI), based on its ELUTYX™ proprietary bioresorbable hydrogel-based formulation technology. AXPAXLI is currently in the first of two planned pivotal Phase 3 trials for wet age-related macular degeneration (AMD): the “SOL-1 trial,” and a Phase 1 clinical trial for the treatment of diabetic retinopathy (DR). The clinical portfolio also includes PAXTRAVA™ (travoprost intracameral implant, also known as OTX-TIC), currently in a Phase 2 clinical trial for the treatment of primary open-angle glaucoma, or ocular hypertension.
Ocular’s expertise in the formulation, development, and commercialization of innovative therapies and the ELUTYX™ platform, supported the FDA approval and launch of its first commercial drug product, DEXTENZA®, an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery and ocular itching associated with allergic conjunctivitis. ELUTYX™ is also the foundation for two other clinical-stage assets, OTX-CSI (cyclosporine intracanalicular insert) for the chronic treatment of dry eye disease, and OTX-DED (dexamethasone intracanalicular insert) for the short-term treatment of the signs and symptoms of dry eye disease, and several preclinical programs.
Riad Sherif, MD, CEO - Oculis
Oculis is a global biopharmaceutical company purposefully driven to save sight and improve eye care. Oculis’ highly differentiated clinical-stage pipeline includes: OCS-01, a topical candidate in Phase 3 for diabetic macular edema and inflammation and pain following ocular surgery; OCS-02, a topical biologic candidate in Phase 2b for dry eye disease and uveitis; and OCS-05, a disease modifying candidate for acute optic neuritis and other neuro-ophthalmic disorders. Headquartered in Switzerland and with operations in the US, Oculis’ goal is to deliver life-changing eye treatments to patients worldwide. The company is led by an experienced management team and supported by leading international healthcare investors.
David Esposito, CEO - ONL Therapeutics
ONL Therapeutics (ONL) is a clinical-stage biopharmaceutical company committed to developing first-in-class therapeutics to protect and improve the vision of patients with retinal disease. By advancing a breakthrough technology designed to protect key retinal cells from Fas-mediated cell death, ONL is pioneering a new approach to preserving vision. ONL is developing a platform of products for use in a wide range of blinding diseases, including retinal detachment, glaucoma, AMD and IRD.
Frederic Guerard, PharmD, CEO - Opthea, Limited
Opthea (ASX/NASDAQ:OPT) is a biopharmaceutical company developing novel therapies to address the unmet need in the treatment of highly prevalent and progressive retinal diseases, including wet age-related macular degeneration
(wet AMD) and diabetic macular edema (DME).
Opthea’s lead product candidate, sozinibercept, is being evaluated in two pivotal Phase 3 clinical trials (COAST, NCT04757636, and ShORe, NCT04757610) for use in combination with standard-of-care anti-VEGF-A monotherapies to improve overall efficacy and deliver superior vision gains compared to standard-of-care anti-VEGF-A agents.
Houman Hemmati, MD, PhD, Co-Founder & CMO - Optigo Biotherapeutics
Optigo Biotherapeutics is an innovative pre-clinical stage biotherapeutics company working to develop cutting-edge treatments to address vision loss from chronic ophthalmic diseases, many of which are currently treated by monthly or bimonthly injections of drugs into the eye(s) by a retina specialist.
We believe that by reducing the frequency at which each dose of drug is delivered, we will reduce the inconvenience, discomfort, risk and expense of existing treatments. In doing so, we hope to reduce the number of patients dropping out of their treatment cycles or having to skip doses due to inconvenient timing.
Optigo Bio's pipeline therapy for retinal neovascular diseases and platform technology to enhance durability of approved and future drugs to treat Geographic Atrophy aim to prevent vision loss from these blinding retinal diseases with far fewer injections into the eye.
Russell Trenary III, President & CEO - Outlook Therapeutics
Outlook Therapeutics is a biopharmaceutical company working to develop and launch ONS-5010/ LYTENAVA™ (bevacizumab-vikg) as the first FDA-approved ophthalmic formulation of bevacizumab for use in retinal indications, including wet AMD, DME and BRVO. If ONS-5010 ophthalmic bevacizumab is approved, Outlook Therapeutics expects to commercialize it as the first and only FDA-approved ophthalmic formulation of bevacizumab for use in treating retinal diseases in the United States, United Kingdom, Europe, Japan and other markets. Outlook Therapeutics expects to submit ONS-5010 ophthalmic bevacizumab to the U.S. FDA as a BLA under the PHSA 351(a) regulatory pathway.
Patrick Ruane, PhD, Chief Operating Officer - Re-Vana Therapeutics
Re-Vana Therapeutics is a venture backed, pioneering ocular drug delivery and therapeutic company focused on developing the world’s first sustained-release large molecule biologics to treat serious eye diseases. Our innovative platform technologies, EyeLief®, EyeLief SD™, and OcuLief®, enable the efficient delivery of biologics and small molecules, expanding global treatment opportunities. With a multi-billion-dollar market segment, our non-surgical approach offers a significant advantage over traditional treatments.
Ramanath Bhandari, MD, Founder, Interim CEO & Chairman - RevOpsis
At RevOpsis, we are focused on enabling the development of our proprietary Rev-Mod Platform across a broad range of therapeutic areas in collaboration with capable leaders in the industry. Partnerships and licensing are a key part of our commitment to address unmet needs for patients worldwide.
The Rev-Mod Platform uses a fully human modular system to generate proprietary multispecific biologic solutions. We are actively seeking partners to increase the impact of our technology in the numerous races against challenging multifactorial diseases.
The Rev-Mod Platform is exclusive to RevOpsis Therapeutics with granted patents that provide strong global IP support to readily generate proprietary therapeutics in any of our Multi-Mod biologic formats.
Peter Francis, MD, Chief Medical & Scientific Officer - Ray Therapeutics
Ray Therapeutics Inc (RayTx) is a late-stage preclinical Bay Area biotechnology company pioneering the first-to-market precision optogenetic therapy for vision restoration that is highly active in ambient lighting (and will not require light-enhancing goggles). Our studies have shown for the first time that our proprietary ChRown optogenetic protein can restore high levels of visual acuity, contrast sensitivity and visual field.
RayTx lead asset, RTx-015, is expected to address the huge unmet medical in people suffering from many forms of retinal blindness (independent of the underlying genetic cause), including those with advanced age-related macular degeneration. RayTx has partnered with a world-leading AAV gene therapy manufacturer is utilizing their proprietary commercial manufacturing process and analytics to maximally de-risk clinical development. RayTx anticipates clinical trials will begin by the end of 2023 in our initial indication, retinitis pigmentosa.
Ram Palanki, PharmD, Executive Vice President, Commercial Strategy & Operations - REGENXBIO
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.
Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes.
We are currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. Our product candidates all utilize AAV viral vectors from our proprietary gene delivery platform, which we call our NAV® Technology Platform. In addition to our internal product candidate programs, we also selectively license our NAV® Vectors to other leading biotechnology companies.
Carlos Ciller, CEO - RetinAI
RetinAI is a healthcare technology company developing solutions to accelerate decision workflows for pharma and clinics. With improved platforms for harmonizing and aggregating medical data at-scale and advanced analytics of AI, the company improves understanding of patients' disease and outcomes through enhanced knowledge of biomarker-based insights and clinical data.
Tom Reeves, Co-Founder, President & CEO - Ripple Therapeutics
Ripple Therapeutics is a clinical stage company focused on improving ophthalmic therapeutics with controllable, sustained drug delivery. Our patented technology platform is based on a discovery that drugs can be engineered into controlled release pharmaceuticals without polymers or excipients. These proprietary prodrugs undergo surface erosion to give zero order release kinetics and are highly engineerable to tailor drug dose and duration to the specific indication.
While our drug delivery technology can be applied to multiple therapeutic areas, we have chosen to focus on ophthalmology because of what we see as major opportunities in both retinal diseases and glaucoma. We believe we can address the gaps in the current standards of care in these billion $+ markets whether they be duration of effect and consequent treatment burden, safety, cost and/or compliance.
Ripple’s first product – IBE-814 IVT – is an intravitreal Dexamethasone prodrug implant targeting diabetic macular edema (DME) and retinal vein occlusion (RVO) with a 6-9 month duration and an improved safety profile vs. the current products on the market. A Phase 2 trial (RIPPLE-1) has been completed in Australia, New Zealand, Hong Kong, and Canada with 60 patients treated and encouraging results for both safety and efficacy as well as reduction in treatment burden.
We are also actively working on developing products with our technology platform for intermediate dry AMD and geographic atrophy, where the need for a sustained release solution is evident. We are focused on multiple drug targets including complement inhibitors, mitochondrial protection, neuroprotection and lipid deposition.
Our glaucoma pipeline includes both intracameral implants for IOP lowering and intravitreal implants for IOP lowering and neuro protection. We have two primary products we are developing: RTC-620 is an intracameral Bimatoprost implant with a 6 month duration and RTC-1119 is an intracameral Latanoprost implant with an 18 month duration. Both of these prodrug implants have no polymers or excipients, and the implant degradation supports safe retreatment. We have compelling preclinical efficacy (IOP lowering and pupillometry) and safety (endothelial cell loss and histology) for both products
Reenie McCarthy, CEO - Stealth BioTherapeutics
We are an innovative biopharmaceutical company developing therapies to treat the mitochondrial dysfunction associated with genetic mitochondrial diseases and many common age-related diseases.
Our mission is to lead the development of mitochondrial medicine to improve the lives of patients with diseases involving mitochondrial dysfunction, an area of high unmet clinical need.
Eric Schlumpf, President & CEO - Stuart Therapeutics
Stuart Therapeutics (Stuart) is a clinical stage pharmaceutical company focused on the development of a unique synthetic protein designed to target a previously undruggable target in the human body: helical collagen. The company’s platform technology, called PolyColTM (PolyCol), is a series of synthesized amino acid sequences designed to specifically bind to and repair disease-damaged helical collagen in the extracellular matrix and in membranes. These helical collagen structures play an important role in healthy tissues, including the regulation of inflammation, cell growth and proliferation, among many other processes.
Stuart was founded in 2018 to champion a new approach to the treatment of chronic diseases. Each of our founders has been affected directly or indirectly by chronic eye disease, and we have a passion for the pursuit of new and effective solutions for these indications.
Stuart’s areas of interest are Dry Eye Disease, Glaucoma, Dry Age-Related Macular Degeneration and Myopia.
Daphne Haim-Langford, PhD, CEO - Tarsier Pharma
Tarsier® Pharma is a late clinical-stage company dedicated to the development and commercialization of a breakthrough therapeutic approach for better and safer treatment of autoimmune and inflammatory ocular diseases. The TRS Platform Technology is a bio-inspired immunomodulator designed to treat blinding inflammatory ocular diseases, and is currently being developed as both eye drops and intravitreal injections.
Ocular inflammations are a group of diseases affecting the front and back of the eye. Non-infectious anterior and posterior uveitis are the most known ocular inflammatory diseases. However, ocular inflammation is a critical underlying mechanism in the pathology of other inflammatory eye diseases, such as non-evaporative dry eye, diabetic macular edema (DME), and dry age-related macular degeneration (AMD). Hundreds of millions of people in all age groups are suffering from ocular inflammatory diseases worldwide, conditions that pose severe risks of vision loss and blindness.
With a leadership comprised of a global team of experienced, motivated, and dedicated professionals, and backed by investors who are focused on strategic investments in ophthalmology, we plan to leverage our new immunomodulatory approach to disrupt how ocular inflammatory diseases are being treated.