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Ocugen, Inc. Announces Positive Scientific Advice from the European Medicines Agency Related to the Approval Pathway for OCU400—Modifier Gene Therapy for Broad Retinitis Pigmentosa Indication

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Ocugen, Inc. announces positive EMA opinion on OCU400 Phase 3 clinical trial for retinitis pigmentosa, aligning with FDA clearance. EMA's review supports the trial's design and statistical analysis, paving the way for Marketing Authorization Application submission. OCU400, a gene therapy, targets RP patients with RHO gene mutations and gene agnostic mutations. The positive opinion from EMA streamlines the approval process, potentially reducing time and costs for EU marketing authorization, with OCU400 on track for BLA and MAA approvals by 2026.
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The endorsement from the EMA for Ocugen's Phase 3 liMeliGhT clinical trial is a pivotal moment for the company. The trial's design and the EMA's receptiveness to the U.S.-based study for a future MAA submission suggest a streamlined pathway towards approval in the European market. The biotechnology sector often faces regulatory hurdles and this development could significantly reduce both time and financial resources required for Ocugen to bring OCU400 to market.

OCU400's progression to Phase 3 as a gene therapy for RP, a condition with limited treatment options, positions it as a potential first-mover in the market. The Orphan Drug Designation previously granted by the EU could provide market exclusivity benefits, enhancing the commercial potential of OCU400. Investors should monitor the enrollment and execution of the Phase 3 study, as any deviations from the planned sample size or treatment protocols could impact the trial's integrity and the projected 2026 BLA and MAA approval targets.

From an economic perspective, the EMA's positive opinion on OCU400 could foster a competitive advantage for Ocugen within the EU's healthcare system. As a gene-agnostic therapy for a rare condition like RP, OCU400 may command premium pricing, especially if it demonstrates efficacy across multiple gene mutations. The healthcare economics of gene therapies are complex, with factors such as long-term effectiveness and cost-savings from reduced disease burden playing critical roles in pricing and reimbursement discussions.

Investors should consider the broader implications of OCU400's potential approval. The therapy's success could lead to increased investor confidence in Ocugen's pipeline and could also influence the valuation of other companies developing gene therapies for rare diseases. The cost implications for healthcare providers and payers will be substantial and Ocugen's ability to navigate pricing and reimbursement negotiations will be important for the therapy's commercial success.

Analyzing the market dynamics, Ocugen's announcement could signal a shift in investor sentiment towards companies with innovative treatments for genetic disorders. The RP market is currently underserved and OCU400's gene-agnostic approach could cater to a broad patient population. Market research indicates a growing demand for personalized medicine and OCU400's potential to treat multiple gene mutations could set a new standard in the field.

It is essential for investors to track the competitive landscape, as other biotech firms may accelerate their own gene therapy programs in response to Ocugen's advancements. Additionally, successful commercialization will depend on Ocugen's marketing strategy and its ability to establish partnerships for distribution within the EU. The market's response to the EMA's opinion and subsequent trial outcomes will offer further insights into the commercial viability of OCU400 and its impact on Ocugen's stock performance.

MALVERN, Pa., April 10, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).

EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP). 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.

The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication. Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU.

“We are very grateful to EMA for their collaborative discussions and support in providing a gene-agnostic therapeutic option to RP patients with severe unmet medical need,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This positive opinion is a critical step in providing our game-changing modifier gene therapies to patients globally.”

The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.

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 did the EMA review for OCU400 Phase 3 clinical trial?

The EMA reviewed the study design, endpoints, and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa.

What is the sample size for the Phase 3 liMeliGhT study of OCU400?

The study will have a sample size of 150 participants, with one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic.

What is the treatment group in the Phase 3 trial of OCU400?

Participants will be randomized 2:1 to the treatment group receiving 2.5 x 10^10 vg/eye of OCU400 and the untreated control group.

What is the significance of the positive EMA opinion for Ocugen?

The positive opinion from EMA will potentially reduce the time and cost to gain marketing authorization in the EU for OCU400, aligning with FDA clearance and supporting global availability of the gene therapy.

What are the approval targets for OCU400?

OCU400 remains on track for BLA and MAA approvals by 2026, following the positive EMA opinion.

Who is the Chairman, CEO, and Co-founder of Ocugen?

Dr. Shankar Musunuri is the Chairman, CEO, and Co-founder of Ocugen.

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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.