MALVERN, PA Ocugen, Inc. (NASDAQ: OCGN) announced a positive outcome from the Data and Safety Monitoring Board (DSMB) review of its Phase 1/2 ArMaDa clinical trial for OCU410, a gene therapy candidate aimed at treating geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. and Europe.

To date, six patients with GA have participated in the trial. Three received a low dose of the therapy, while three others received a medium dose. The next phase will involve dosing an additional three patients with a high dose of OCU410.

The DSMB has recommended to proceed with dosing subsequent GA subjects with the high dose of OCU410 in the dose-expansion phase of the study and concurrently initiate Phase 2 dosing, said Dr. Peter Chang, Chair of the DSMB for the OCU410 clinical trial. No serious adverse events related to OCU410 have been reported to date in both low- and medium-dose cohorts. I believe that this marks a critical next step towards determining the maximum tolerated dose for OCU410 and is an important milestone for its clinical development.

Huma Qamar, Chief Medical Officer of Ocugen, expressed optimism about the therapys potential. We are delighted to report a second positive DSMB recommendation for the treatment of GA, which significantly builds on the favorable safety and tolerability profile exhibited by OCU410, she said. We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection.

The ArMaDa trial aims to assess the safety and efficacy of OCU410 administered subretinally. This trial is structured in two phases:

Phase 1: This is a multicenter, open-label, dose-ranging study. It includes three dose levels: low (2.510 vg/mL), medium (510 vg/mL), and high (1.510 vg/mL).

Phase 2: This phase will be a randomized, outcome assessor-blinded, dose-expansion study. Participants will be randomly assigned to one of two OCU410 treatment groups or an untreated control group in a 1:1:1 ratio.

The DSMBs positive review is significant for several reasons.

Safety and Tolerability: Initial results indicate that OCU410 is safe and well-tolerated at both low and medium doses. This builds confidence in the therapys potential for broader application.

Innovative Treatment Approach: Current treatments for GA target only the complement pathway and require multiple annual injections. OCU410 offers a different approach by addressing several pathways, including complement, lipid metabolism, inflammation, and oxidative stress. This could provide a more comprehensive and long-term benefit to patients.

Reduced Treatment Burden: If successful, OCU410 could reduce the number of required treatments to a single sub-retinal injection. This would significantly ease the treatment burden on patients compared to the frequent injections needed with existing therapies.

Gene therapy represents a frontier in medical research, offering hope for conditions that currently have limited treatment options. The success of OCU410 could pave the way for similar therapies targeting other complex diseases. As the population ages, the incidence of age-related conditions like GA is expected to rise, making innovative treatments even more crucial.

Ocugens progress with OCU410 thus far signals a promising development in the fight against dAMD. If the therapy continues to show positive results, it could transform the standard of care for millions of patients affected by this debilitating condition.

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Ocugen's Gene Therapy Shows Promise in Early Trial for Age-Related Macular Degeneration - MyChesCo

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