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Ocugen Gains FDA Alignment on Key Aspects of OCU400—Modifier Gene Therapy—Pivotal Phase 3 Study Design

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Ocugen, Inc. (NASDAQ: OCGN) Receives FDA Alignment for Phase 3 Clinical Trial Design for OCU400 Gene Therapy in Patients with Retinitis Pigmentosa
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The recent announcement by Ocugen concerning FDA alignment on Phase 3 clinical trial design for OCU400 marks a significant milestone in the development process of gene therapies targeting Retinitis Pigmentosa (RP). The Phase 3 trial is a critical stage where the safety and efficacy of a drug are rigorously tested in a larger patient population before potential approval for public use. The decision by the FDA to align on the trial design suggests a positive regulatory perspective on the preliminary data from the ongoing Phase 1/2 study.

The inclusion of a broader group of RP patients in the trial, particularly those with the common RHO gene mutation, indicates the potential for OCU400 to serve a gene-agnostic approach, which could be transformative in treating genetic conditions where multiple mutations may be involved. With orphan drug and RMAT (Regenerative Medicine Advanced Therapy) designations, OCU400 could benefit from incentives like tax credits and expedited review processes, which are crucial for a biotech firm like Ocugen in terms of financial sustainability and speed to market.

From an investment standpoint, the progression to Phase 3 trials could be a catalyst for investor confidence in Ocugen's pipeline, given the high unmet need in the RP patient community and the size of the potential market. However, investors should also consider the inherent risks in drug development, especially in advanced-stage clinical trials, which can impact timelines and financial projections.

For investors and stakeholders in the biotechnology sector, Ocugen's progress represents a potential inflection point in the company's valuation. The alignment with the FDA reduces regulatory uncertainty, often a significant risk factor in biotech investing. The company's ability to initiate a Phase 3 clinical trial for OCU400 in early 2024 could lead to an increase in capital allocation to the project, with implications for the company's cash flow and burn rate.

Given the prevalence of RP, with 110,000 patients in the United States and 1.6 million globally, the addressable market for OCU400 is substantial. Should the drug receive FDA approval, the revenue potential could be significant, factoring in the orphan drug status which often allows for premium pricing due to the lack of alternative treatments. However, the cost of conducting a Phase 3 trial is considerable and the company's financial health must be monitored closely to assess the feasibility and sustainability of the trial without overly diluting shareholder value.

Moreover, the success of OCU400 could position Ocugen as a notable player in the gene therapy space, potentially attracting partnership opportunities or making it a target for acquisition by larger pharmaceutical companies seeking to expand their portfolios in novel treatments for genetic disorders.

The biotechnology industry is highly competitive, with many companies vying to bring innovative treatments to market. Ocugen's focus on gene and cell therapies places it in a rapidly growing segment of the industry known for high investment and high potential returns. The company's strategy to develop first-in-class, gene-agnostic therapies like OCU400 could set it apart from competitors focusing on mutation-specific treatments.

Understanding the term 'gene-agnostic' is essential; it refers to therapies that are designed to be effective across a range of mutations, potentially offering a broader application and simplifying the treatment landscape for complex genetic diseases. This approach, if successful, could disrupt traditional treatment paradigms and create a new standard for care in genetic disorders.

In the long-term, if OCU400 proves to be effective and safe, it could lead to significant shifts in market dynamics, with Ocugen potentially capturing a sizeable share of the RP treatment market. This could also spur further innovation and competition in the space as other companies strive to develop similar or superior gene-agnostic therapies.

MALVERN, Pa., Dec. 21, 2023 (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 Company received alignment from FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).

“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”

During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, Ocugen received alignment on key aspects of the Phase 3 study design—including the study endpoint, patient enrollment strategy, and study duration of one year. The Phase 3 clinical trial will enroll a broader group of RP patients, including patients with the most common RHO gene mutation, based on OCU400’s potentially gene-agnostic mechanism of action.

With orphan drug and RMAT designations in place for OCU400, FDA’s alignment on key aspects of the Phase 3 study design positions Ocugen to confidently move forward in pursuing product development and licensure for OCU400.

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. Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.

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 therapeutic and clinical benefits of our product candidates, expectations for clinical trial timing and results, anticipated timing of clinical trial updates and expectations for timing and outcome of regulatory interactions, 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; that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities; that receipt of orphan drug and RMAT designations may not lead to faster development or regulatory review; and that regulatory authorities may disagree with additional aspects of our clinical trial designs or may not approve our future IND applications on the anticipated timeline or at all. 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
IR@ocugen.com


FAQ

What did Ocugen announce regarding the Phase 3 clinical trial design for OCU400?

Ocugen announced that the Company received alignment from FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).

Who is the Chairman, Chief Executive Officer, and Co-Founder of Ocugen?

Dr. Shankar Musunuri is the Chairman, Chief Executive Officer, and Co-Founder of Ocugen.

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.

What is the next step for Ocugen regarding OCU400?

The next step for Ocugen is to initiate the Phase 3 clinical trial for OCU400 in early 2024.

What designations are in place for OCU400?

Orphan drug and RMAT designations are in place for OCU400.

What is the potential impact of advancing OCU400 to Phase 3 clinical development?

Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.

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.