Data and Safety Monitoring Board Reviews Interim Safety Data of Phase 2 Subjects of OCU410 ArMaDa Clinical Trial for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

Rhea-AI Summary

Ocugen (NASDAQ: OCGN) announced positive safety data from the Data and Safety Monitoring Board (DSMB) review of their OCU410 ArMaDa Phase 1/2 clinical trial for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The DSMB assessed data from 15 Phase 2 subjects, finding OCU410 to be safe and well-tolerated with no serious adverse events reported.

The Phase 2 study involves 45 subjects randomized across two treatment groups and one control group. Unlike current GA treatments requiring 6-12 annual injections, OCU410 aims to be a one-time treatment. The trial showed promising Phase 1 results, including reduced lesion growth, preserved retinal tissue, and improved low luminance visual acuity. The study is being conducted at 13 retinal surgery centers across the U.S., with dosing expected to complete in early 2025.

Positive

• Phase 2 DSMB review confirms favorable safety and tolerability profile
• No serious adverse events reported in the clinical trial
• Positive Phase 1 results showing reduced lesion growth and preserved retinal tissue
• Potential one-time treatment versus current treatments requiring 6-12 annual injections
• OCU410 addresses all four cellular pathways of dAMD versus current therapies targeting only one

Negative

• Phase 2 trial still ongoing with final results pending
• Dosing completion not expected until early 2025

Insights

Medical Research Analyst

<p>The interim safety data from OCU410's Phase 2 trial represents a important milestone in the development of a potentially transformative treatment for Geographic Atrophy (GA). The absence of serious adverse events in the first 15 subjects and the DSMB's approval to continue are particularly noteworthy given the complexity of subretinal gene therapy administration. </p><p>The trial's design is robust, with <b>45 subjects randomized across two treatment doses (5×10^10 vg/mL and 1.5×10^11 vg/mL) and a control group</b>. What sets OCU410 apart is its comprehensive approach targeting all four pathological pathways of dry AMD, unlike current treatments that focus solely on complement inhibition. The potential for a <b>one-time treatment</b> versus the current standard of <b>6-12 annual injections</b> could revolutionize patient care.</p><p>The preliminary Phase 1 data showing reduced lesion growth and preserved retinal tissue, coupled with improvements in low luminance visual acuity, suggests potential efficacy beyond just safety. With trial completion expected in early 2025, the next 12 months will be critical for validating these early positive signals.</p>

Market Research Analyst

<p>The positive safety profile and continued trial progression significantly de-risk Ocugen's OCU410 program. With a current market cap of <money>218.6 million</money>, the company appears undervalued considering the potential market opportunity in GA, which affects millions globally. The one-time treatment approach could command premium pricing if approved, especially given the current treatment burden of frequent injections.</p><p>The expanding trial network to <b>13 leading retinal surgery centers</b> demonstrates strong investigator interest and positions the company well for potential commercialization. The comprehensive mechanism of action addressing all four disease pathways could provide a competitive advantage over existing treatments, potentially capturing significant market share in the growing GA treatment landscape.</p>

• OCU410 has a very favorable safety and tolerability profile
• No serious adverse events related to the study drug have been reported, such as exudation, infectious endophthalmitis, intraocular Inflammation, anterior ischemic optic neuropathy, or vasculitis

MALVERN, Pa., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).

“The DSMB assessed data on 15 subjects from Phase 2. Initial data indicates that OCU410 appears to be safe and well-tolerated,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events (SAEs) related to OCU410 have been reported to date.”

The ArMaDa clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410 in subjects with GA. Phase 2 is an ongoing, randomized, outcome assessor-blinded, dose-expansion study in which 45 subjects are randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups (5×10¹⁰ vg/mL or 1.5 ×10¹¹ vg/mL) or an untreated control group.

“Currently approved treatments for GA require 6-12 intravitreal injections annually and frequent injections are a burden on patients and caregivers,” said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. “We are very enthusiastic about the potential of OCU410 to serve as a game-changing, one-time treatment for life for patients with GA.”

Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the ArMaDa clinical trial demonstrated: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA).

dAMD is a multifactorial disease involving genetic and environmental factors that is one of the world’s leading causes of blindness in people aged 50 years and older. Four cellular pathways drive the pathology of dry AMD: lipid metabolism, inflammation, oxidative stress, and complement. Currently approved therapies target only the latter, while OCU410 addresses all four and thereby helps reestablish retinal homeostasis.

The ArMaDa clinical trial is currently being performed at 13 leading retinal surgery centers across the U.S. Dosing in the OCU410 ArMaDa clinical trial will be completed in early 2025 and the Company will continue to provide 9- and 12-month efficacy updates from Phase 1.  

About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV5-hRORA as a one-time gene therapy for the treatment of GA.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com 

FAQ

What are the key findings from OCU410's Phase 2 DSMB review for OCGN?

The DSMB review of 15 Phase 2 subjects showed OCU410 has a favorable safety profile with no serious adverse events reported, allowing the trial to continue.

How many patients are enrolled in the OCU410 Phase 2 ArMaDa trial for OCGN?

The Phase 2 trial includes 45 subjects randomized in a 1:1:1 ratio across two OCU410 treatment groups and one untreated control group.

What advantages does OCGN's OCU410 offer over current GA treatments?

OCU410 is designed as a one-time treatment, compared to current treatments requiring 6-12 annual injections, and targets all four cellular pathways of dAMD versus just one.

When will OCGN complete dosing in the OCU410 ArMaDa clinical trial?

Dosing in the OCU410 ArMaDa clinical trial is expected to be completed in early 2025.

What were the Phase 1 results of OCGN's OCU410 ArMaDa trial?

Phase 1 showed positive preliminary results including reduced lesion growth, preserved retinal tissue, and improved low luminance visual acuity, with no drug-related serious adverse events.