European Commission nods at Ocugen’s gene therapy

MALVERN, Pa. - Ocugen, Inc. (NASDAQ: NASDAQ:OCGN), a biotechnology company with a current market capitalization of $212 million, announced today that the European Commission has endorsed the European Medicines Agency’s Committee for Advanced Therapies (CAT) classification for its gene therapy product OCU400. The company’s stock, which has seen a 38% gain over the past year despite recent volatility, is currently trading at $0.73. InvestingPro analysis reveals 10+ additional exclusive insights about Ocugen’s market performance and financial health. This marks a significant step for Ocugen, as OCU400 becomes the first gene therapy for a broad retinitis pigmentosa (RP) indication to enter Phase 3 trials.

The Advanced Therapy Medicinal Product (ATMP) classification will expedite the regulatory review process for OCU400. It also enables more frequent interactions with the EMA for scientific advice as Ocugen works towards a Marketing Authorization Application (MAA) filing anticipated in 2026. This classification is particularly crucial for innovative treatments like OCU400, potentially a one-time gene therapy for life.

The Phase 3 liMeliGhT clinical trial for OCU400 is currently enrolling participants. The study includes 150 subjects divided into two arms, with one focusing on patients with RHO gene mutations and the other being gene-agnostic. According to InvestingPro data, while Ocugen maintains a healthy current ratio of 2.58 and holds more cash than debt on its balance sheet, the company is rapidly burning through its cash reserves as it advances its clinical programs. Each arm will randomize participants in a 2:1 ratio to either the treatment group or an untreated control group. The trial seeks to include patients aged eight and above with early to late-stage RP.

The positive opinion from the EMA underscores the need for gene-agnostic treatments for diseases like RP, which involve multiple mutations and affect nearly 310,000 patients in the U.S., EU, and Canada. There are currently no approved treatments that address the progression of multiple forms of RP.

OCU400 is based on the NR2E3 gene, which plays a crucial role in retinal health and function. Ocugen’s Chief Medical (TASE:PMCN) Officer, Dr. Huma Qamar, expressed optimism about the EMA’s recognition of OCU400 as the clinical trial progresses.

Ocugen’s mission is to develop and commercialize therapies and vaccines for diseases with unmet medical needs worldwide. The company’s innovative approach includes a modifier gene therapy platform that could potentially treat multiple retinal diseases with a single product.

This news article is based on a press release statement from Ocugen, Inc. It should be noted that forward-looking statements in the press release are subject to various risks and uncertainties and may differ materially from actual future results or events. InvestingPro analysis indicates the company currently maintains a ’FAIR’ overall financial health score, though analysts do not anticipate profitability this year. For comprehensive insights into Ocugen’s financial outlook and detailed analysis, investors can access the full Pro Research Report, available exclusively to InvestingPro subscribers.

In other recent news, Ocugen, Inc. has reported significant strides in its Q3 2024 earnings, particularly in its clinical programs. The company is progressing in its Phase III trial for retinitis pigmentosa, marking an important milestone in gene therapy. Additionally, Ocugen is expanding its clinical trials into Canada, broadening its market potential. Despite high operating expenses, the company reported no serious adverse events in trials. The company’s financial highlights included a cash and restricted cash total of $39 million as of September 30, 2024, and total operating expenses of $14.4 million. Ocugen also completed a $30 million debt financing, extending its runway to Q1 2026. Looking ahead, Ocugen plans to complete enrollment for its OcQ400 Phase III trial in the first half of 2025, file for regulatory approvals in 2026, and pursue commercialization by 2027. The initiation of the ORQ200 Phase 1 clinical trial is scheduled for Q4 2024.

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