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Data and Safety Monitoring Board Approves Simultaneous Enrollment in Cohort 3 and Phase 2 Initiation in OCU410 ArMaDa study for Geographic Atrophy

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Ocugen announced a positive DSMB review for its Phase 1/2 ArMaDa clinical trial of OCU410, a gene therapy for geographic atrophy in dry age-related macular degeneration. The trial showed safety and tolerability in low and medium doses, with no serious adverse events. The DSMB approved simultaneous enrollment in the high-dose cohort and Phase 2 initiation. OCU410 aims to provide a long-term benefit with a single subretinal injection, addressing multiple pathways of dAMD. The trial involves a dose-ranging Phase 1 and a randomized, blinded Phase 2 study.

Positive
  • Positive DSMB review for Phase 1/2 trial.
  • Established safety and tolerability for low and medium doses.
  • No serious adverse events reported.
  • DSMB supports high-dose cohort and Phase 2 initiation.
  • Potential for OCU410 as a one-time treatment for GA.
  • OCU410 addresses multiple pathways of dAMD.
  • Phase 2 to include randomized, blinded study.
Negative
  • Trial still in early phases, no efficacy data yet.
  • High-dose cohort just beginning, long-term safety unknown.
  • Potential risks and side effects not fully understood.

The commencement of the Phase 2 dosing for Ocugen's OCU410 following the DSMB's positive safety review is a significant milestone in the drug's development. With no serious adverse events reported in low- and medium-dose cohorts, the data suggest that the therapy is well-tolerated at these levels, which is important in moving forward to higher doses. This approval not only validates the safety profile of OCU410 but also facilitates accelerated clinical progression. For investors, the lack of adverse events increases confidence in the drug's safety, potentially impacting Ocugen's stock positively as the trial progresses.

Ocugen's focus on geographic atrophy (GA), a severe form of dry age-related macular degeneration affecting millions, addresses a critical unmet need in ophthalmology. The current treatments for GA require multiple annual injections and OCU410’s potential as a one-time treatment with a single sub-retinal injection is highly compelling. If successful, this would be a game-changer, significantly improving patient adherence and quality of life. This differentiates OCU410 in a competitive market, potentially leading to a significant market share if it reaches commercialization.

The positive DSMB recommendation for high-dose and Phase 2 initiation in Ocugen's OCU410 ArMaDa study is an encouraging development from a financial perspective. Ocugen's progress in the clinical trial stages indicates potential future revenue streams, particularly if OCU410 proves to be an effective treatment for GA. The estimated patient population in the U.S. and Europe provides a sizable market opportunity. If OCU410 advances successfully through the trial phases and gains FDA approval, Ocugen could see substantial financial growth.

Investors should note the strategic advantage of OCU410 in targeting multiple pathways in GA treatment. This could position Ocugen favorably against competitors that focus solely on the complement pathway. However, it is important to consider the long-term financial implications, including the costs associated with extensive clinical trials and the potential need for additional funding. Ensuring a strong cash position to support these trials will be important for Ocugen moving forward.

From a market research perspective, OCU410’s advancement into Phase 2 signifies a pivotal moment for Ocugen. The ability to move forward without significant safety concerns in earlier trial phases provides a competitive edge. The market for GA treatments is poised for innovation and Ocugen’s approach of a one-time gene therapy for GA could disrupt the existing treatment paradigm. This innovation aligns with broader trends in biotechnology, where gene and cell therapies are increasingly viewed as solutions for chronic conditions.

The approval process and subsequent market introduction will be critical. Investors should keep an eye on the competitive landscape, regulatory milestones and how Ocugen positions OCU410 in the market. Success in Phase 2 will likely boost Ocugen’s market valuation and investor sentiment, but failure or delays could have the opposite effect. Understanding these dynamics is important for making informed investment decisions.

  • Established Medium Dose as Safe and Tolerable Dose in Current OCU410 Clinical Trial
  • DSMB Recommends Continuing with High-Dose Cohort Dosing with Concurrent Phase 2 Dosing

MALVERN, Pa., May 31, 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 and vaccines, today announced a positive outcome of the Data and Safety Monitoring Board (DSMB) Review for its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV5-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. and Europe combined.

Six subjects with GA were dosed in the Phase 1/2 clinical trial to date—three subjects were dosed with the low dose and three subjects were dosed with the medium dose. An additional three patients will be dosed with the high dose of OCU410 in the dose-escalation phase.

“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, MD, FACS, DSMB Chair for the OCU410 clinical trial. “No serious adverse events (SAEs) 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.”

"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," said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. "We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection. The currently approved treatments for GA target only the complement pathway and require approximately 6-12 intravitreal injections annually. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress, providing long-term benefit to patients."

The ArMaDa clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.

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 (ROR 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 as well as inhibiting the complement system in in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA 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, 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 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 is the latest update on OCU410 for geographic atrophy?

Ocugen's OCU410 has received a positive DSMB review, allowing for high-dose cohort dosing and Phase 2 initiation.

How many subjects were involved in the OCU410 clinical trial?

Six subjects with geographic atrophy have been dosed in the Phase 1/2 trial so far.

Are there any serious adverse events reported for OCU410?

No serious adverse events related to OCU410 have been reported in the low and medium dose cohorts.

What is the significance of the DSMB's recommendation for OCU410?

The DSMB's recommendation to proceed with high-dose and Phase 2 dosing marks an important milestone for OCU410's clinical development.

What makes OCU410 different from other treatments for geographic atrophy?

OCU410 is a potential one-time treatment targeting multiple pathways of dry AMD, unlike current treatments that require multiple annual injections.

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