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MALVERN, Pa. – Ocugen, Inc. (NASDAQ: NASDAQ:OCGN), a biotechnology firm specializing in gene therapies for blindness trading at $0.65 per share, has received a positive opinion from the European Medicines Agency’s Committee for Advanced Therapies (CAT) for two of its products. The company’s stock has declined over 46% in the past six months, though according to InvestingPro analysis, the stock appears undervalued based on its Fair Value assessment. The European Commission has granted Advanced Therapy Medicinal Product (ATMP) classification to OCU410 and OCU410ST, which are being developed for geographic atrophy (GA) and Stargardt disease, respectively.
GA, a stage of dry age-related macular degeneration, impacts approximately 2-3 million individuals across the United States and Europe. While two therapies exist in the U.S., neither has received approval in Europe. Stargardt disease, which affects an estimated 100,000 people in the U.S. and Europe, currently has no approved treatments.
Dr. Shankar Musunuri, CEO of Ocugen, expressed that the ATMP classification is a significant step toward addressing these unmet medical needs. The company has completed dosing for Phase 2 of the OCU410 ArMaDa clinical trial and anticipates initiating Phase 3 next year. InvestingPro data shows the company maintains a healthy current ratio of 2.58, with more cash than debt on its balance sheet, though it’s currently burning through cash rapidly as it advances its clinical programs. The goal is to file for Marketing Authorization Application (MAA) and Biologics License Application (BLA) in 2028. Similarly, the FDA endorsed a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST last week, aiming for BLA and potential MAA submissions in 2027.
OCU410 and OCU410ST target the four pathways associated with dAMD and Stargardt disease, delivered via a single subretinal injection. Preliminary trial results for OCU410 show a 44% reduction in lesion growth and a 10-letter improvement in visual function at nine months. For OCU410ST, six-month Phase 1 data revealed a 52% lesion growth reduction and a statistically significant 10-letter improvement in visual function.
The ATMP classification accelerates regulatory review and allows for more frequent interaction with the EMA for scientific advice. Ocugen plans to begin the Phase 2/3 pivotal confirmatory clinical trial for OCU410ST by mid-2025.
Both gene therapy candidates use an adeno-associated virus (AAV) platform to deliver the RORA gene, which is involved in lipid metabolism and has anti-inflammatory properties. Dr. Huma Qamar, Chief Medical (TASE:BLWV) Officer at Ocugen, highlighted the promising structural and functional outcomes and the safety profile of these treatments.
Ocugen’s mission is to discover, develop, and commercialize novel therapies for a range of diseases. The company’s breakthrough gene therapy platform has the potential to treat multiple retinal diseases with a single product. With a market capitalization of $190 million and an EBITDA of -$51 million in the last twelve months, investors should note that analysts don’t expect profitability this year. For deeper insights into Ocugen’s financial health and growth prospects, including 8 additional ProTips and comprehensive valuation metrics, check out the full research report available on InvestingPro. This news is based on a press release statement.
In other recent news, Ocugen, Inc. has reported its Q3 2024 earnings, highlighting significant progress in its clinical programs. The company is advancing its Phase 3 trial for retinitis pigmentosa, marking a key milestone in gene therapy. Ocugen’s financial report revealed operating expenses of $14.4 million, with research and development costs accounting for $8.1 million. The company also completed a $30 million debt financing, extending its financial runway to Q1 2026.
In addition, the European Commission has endorsed Ocugen’s gene therapy product OCU400, classifying it as an Advanced Therapy Medicinal Product, which will expedite its regulatory review process. This classification supports the ongoing Phase 3 liMeliGhT clinical trial for OCU400, currently enrolling participants. Meanwhile, H.C. Wainwright has raised its price target for Ocugen to $8.00, maintaining a Buy rating, following promising clinical data from the Phase 1 portion of the ArMaDa Study for OCU410.
The study indicated that OCU410 showed promising efficacy signals in treating geographic atrophy secondary to dry age-related macular degeneration. Ocugen’s Chief Medical Officer expressed optimism about the EMA’s recognition of OCU400, as the company progresses through its clinical trials. These recent developments reflect Ocugen’s ongoing efforts to address unmet needs in ophthalmology through its gene therapy initiatives.
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