Ocugen (NASDAQ:OCGN) is positioning its ophthalmology gene therapy portfolio around treatments for inherited retinal diseases and dry age-related macular degeneration, with pivotal-stage programs in retinitis pigmentosa, Stargardt disease and geographic atrophy, Chairman, CEO and Co-Founder Shankar Musunuri said during a Canaccord discussion.
Musunuri said the company is targeting biologics license applications, or BLAs, next year for its retinitis pigmentosa and Stargardt programs, while its geographic atrophy program is expected to follow on a longer timeline. He said the company’s objective is to pursue approvals in major markets, including the U.S., Europe and Japan, and to improve patient access in parallel with regulatory work.
Modifier Gene Therapy Platform
For retinitis pigmentosa, Ocugen is developing OCU400, which uses the Nr2e3 modifier gene. Musunuri said Nr2e3 affects phototransduction pathways, which are particularly relevant in retinitis pigmentosa as patients can lose peripheral and night vision before central vision.
For Stargardt disease and geographic atrophy, Ocugen is developing treatments using the RORA modifier gene. Musunuri said RORA is intended to regulate multiple disease-related pathways, including oxidative stress, lipid metabolism, inflammation and the complement system. He said the company believes the therapy may help restore cellular homeostasis and create a healthier environment for retinal cells.
Musunuri said Ocugen’s intellectual property extends beyond ophthalmology into neurological applications, though he characterized that area as a future opportunity.
Stargardt and Geographic Atrophy Programs
OCU410ST, the company’s Stargardt disease candidate, is in a Phase II/III study enrolling patients ages 3 and older, from early through advanced stages of disease. Musunuri said the study was endorsed by the European Medicines Agency and that the FDA allowed Ocugen to convert its Phase II study into a combined Phase II/III trial following a small Phase I study.
The company expects top-line data from the Stargardt study in the second quarter of next year, Musunuri said. Ocugen then plans to file a BLA and pursue market authorization in parallel within several months of the results. If development proceeds according to plan, he said approval and launch could occur in 2028.
Musunuri contrasted the program with oral therapies that may target individual disease pathways and require ongoing dosing. He said Ocugen’s subretinal gene therapy is designed as a one-time administration, while emphasizing that efficacy and safety data will determine its potential differentiation.
OCU410, Ocugen’s geographic atrophy candidate, uses the same RORA construct as OCU410ST but at a different dose, Musunuri said. The geographic atrophy program has received the FDA’s Regenerative Medicine Advanced Therapy, or RMAT, designation. He said the company has received FDA clearance to begin a single Phase III trial and is working with the EMA to align the study as a global trial.
Retinitis Pigmentosa Readout and Filing Plans
Ocugen expects first-quarter top-line data next year from its Phase III trial of OCU400 in retinitis pigmentosa. The study includes 140 patients, uses a 2:1 treatment-to-control ratio and covers more than 30 mutations, according to Musunuri.
The primary assessment uses a mobility test designed to measure patients’ ability to navigate under low-light conditions. Musunuri said the company refined the test with FDA input and also plans to track low-luminance visual acuity as a secondary and longer-term measure. He said the company observed approximately two lines of low-luminance visual acuity improvement in treated eyes among Phase I/II patients over three years.
Musunuri said Ocugen has treated more than 325 patients across its clinical trials and expanded-access program, including more than 200 patients with retinitis pigmentosa. He said the company has not observed serious adverse events related to its gene therapy programs in those populations.
On manufacturing, Musunuri said Ocugen has completed process performance qualification validation runs needed for an OCU400 BLA submission and has commercial-scale material that could be used for supply. The company expects to complete the BLA within months after the Phase III data. Under RMAT, it may be eligible for a rolling submission, although Musunuri said the final clinical module would start the FDA review clock.
Capital Runway and Commercialization
Musunuri said a recently announced $130 million convertible note financing is expected to fund Ocugen into 2028. He said overall spending is not expected to increase substantially next year because recruitment for two Phase III programs was completed this year, while the geographic atrophy study begins.
The company is also evaluating regional partnerships and non-dilutive funding opportunities, particularly outside the U.S., Musunuri said. He said Ocugen intends to remain opportunistic regarding U.S. commercialization and may consider additional equity financing if needed to support a U.S. launch.
About Ocugen (NASDAQ:OCGN)
Ocugen Inc is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing gene therapies to treat rare inherited retinal diseases, as well as vaccines designed to address unmet needs in infectious diseases. Headquartered in Malvern, Pennsylvania, the company applies its proprietary gene therapy platform to create novel treatments aimed at preserving and restoring vision, while leveraging strategic partnerships to broaden its vaccine pipeline.
In its gene therapy portfolio, Ocugen is advancing multiple programs targeting retinal disorders.
