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Ocugen, Inc. Announces U.S. FDA Clearance of IND Amendment to Initiate OCU400 Phase 3 Clinical Trial — First Gene Therapy to Enter Phase 3 with a Broad Retinitis Pigmentosa Indication

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Ocugen, Inc. announces FDA clearance for Phase 3 clinical trial of OCU400 gene therapy for retinitis pigmentosa, a significant milestone in the treatment of RP patients. The trial includes 150 participants with a gene-agnostic approach and aims to address unmet medical needs in RP treatment.
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The FDA's clearance of Ocugen's IND amendment to start a Phase 3 clinical trial for OCU400 marks a significant progress in the field of gene therapy for retinitis pigmentosa (RP). With RP being a group of rare genetic disorders that cause retinal degeneration and subsequent vision loss, the development of a broad-spectrum gene therapy is noteworthy. The gene-agnostic approach of the trial is particularly interesting, as it suggests a potential to treat a wide array of RP mutations beyond the limited scope of existing therapies.

From a biotechnology perspective, the use of a novel endpoint like the Luminance Dependent Navigation Assessment (LDNA) in the trial indicates a tailored approach to measuring treatment efficacy. This could set a precedent for future clinical trials in ophthalmology and gene therapy. The market potential for OCU400, given the 110,000 RP patients in the US alone, is substantial. However, the success hinges on the trial's outcomes and the therapy's safety and efficacy profile. Investors should monitor the progress of this trial closely as it could have a significant impact on Ocugen's valuation and the broader gene therapy market.

Ocugen's advancement into Phase 3 trials for OCU400 presents a potential inflection point for the company's stock. Given the orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designations, Ocugen could benefit from various incentives, including tax credits and a potentially expedited FDA review process. The market for RP treatments is underserved, with only one marketed product addressing a single gene mutation out of the many associated with RP. Hence, a successful broad-spectrum treatment could disrupt the market.

The strategic decision to collaborate with leading retinal surgeons and trial sites could enhance the trial's execution and support post-trial commercialization efforts. The biotechnology sector often experiences significant stock volatility around clinical trial results and a positive outcome for Ocugen's Phase 3 trial could lead to substantial appreciation in stock value. Conversely, any setbacks could lead to a sharp decline. Long-term investors might see this as a growth opportunity, while risk-averse stakeholders should consider the inherent uncertainties of clinical trials.

The economic implications of Ocugen's Phase 3 clinical trial for OCU400 are multifaceted. On one hand, the development of a treatment for a rare condition like RP aligns with public health interests in addressing unmet medical needs. On the other hand, the cost-effectiveness of such therapies is often a concern, especially in the context of healthcare budgets and insurance coverage.

Given the orphan drug status of OCU400, Ocugen could set a high price point for the therapy, which might lead to discussions about affordability and access. The broad RP indication for OCU400 suggests a larger target patient population, which could justify the investment in development from a cost-recovery standpoint. However, payers will likely scrutinize the value proposition of the therapy in terms of quality-adjusted life years (QALYs) and other health economics outcomes. The success of OCU400 could stimulate further investment in gene therapies, but it is essential to balance innovation with considerations of economic sustainability in the healthcare system.

MALVERN, Pa., April 08, 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 that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

“The initiation of the OCU400 Phase 3 clinical trial is a significant milestone for patients with RP and a pivotal event for Ocugen as a company,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven’t had a treatment option.”

The Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

In the Phase 1/2 OCU400 clinical trial, a Multi-Luminance Mobility Testing (MLMT) scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used—Luminance Dependent Navigation Assessment (LDNA)— that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.

“A sensitive mobility course, LDNA, was developed by Ocugen in collaboration with FDA for the Phase 3 clinical trial to allow enrollment of patients with early to advanced stages of disease,” said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are extremely encouraged that with this Phase 3 study design more than 50% of intent to treat RHO patients would meet the responder criteria, which is demonstrating 2 or greater Lux level of improvement after one year of treatment based on Phase 1/2 study results.”

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation.

“We believe that the gene-agnostic clinical trial design provides an appropriate therapeutic option to include patients who have greater potential of benefiting from treatment,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “We are looking forward to working with our selected trial sites and leading retinal surgeons to deliver this novel modifier gene therapy to potentially address unmet medical needs.”

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, 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

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 significance of the FDA clearance for Ocugen?

The FDA clearance allows Ocugen to initiate a Phase 3 clinical trial for their gene therapy product candidate OCU400, a major milestone in the treatment of retinitis pigmentosa.

How many participants are included in the Phase 3 clinical trial?

The Phase 3 trial will include 150 participants, with one arm having the RHO gene mutation and the other arm being gene agnostic.

What is the primary functional endpoint for the Phase 3 trial?

The primary functional endpoint for the Phase 3 trial is the Luminance Dependent Navigation Assessment (LDNA), which includes a wider range of light intensity and Lux levels.

How many patients in the United States and globally are affected by retinitis pigmentosa?

Approximately 110,000 patients in the United States and 1.6 million patients globally are affected by retinitis pigmentosa, with more than 10% having the RHO genetic mutation.

What designations has OCU400 received from the FDA?

OCU400 has received orphan drug and RMAT designations from the FDA.

Ocugen, Inc.

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About OCGN

ocugen, inc., is a rapidly growing ophthalmology company developing a rich clinical pipeline of innovative therapies that address rare and underserved ocular disorders. the company’s lead programs in ocular graft versus host disease (ocu300) and chronic dry eye disease (ocu310) are expected to enter pivotal clinical trials in 2018. ocu300 received the first and only orphan drug designation for ocular graft versus host disease, providing certain regulatory and economic benefits. ocugen is also developing novel biologic therapies for retinitis pigmentosa (ocu100) and wet amd (ocu200), as well as a groundbreaking modifier gene therapy platform with potential to address a broad spectrum of inherited retinal disorders (ocu400). for more information, please visit www.ocugen.com.