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MALVERN, Pa. - Ocugen, Inc. (NASDAQ: OCGN), a $330 million market cap biotechnology company whose stock has surged over 33% in the past six months, announced Monday that the U.S. Food and Drug Administration has cleared an Investigational New Drug amendment to begin a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy for Stargardt disease. According to InvestingPro data, the company maintains a healthy liquidity position with a current ratio of 2.6, though it faces significant R&D expenses.
The therapy has previously received Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for treating ABCA4-associated retinopathies, including Stargardt disease.
The planned Phase 2/3 trial will enroll 51 participants with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST in the eye with poorer visual acuity, while 17 will serve as untreated controls. The primary endpoint will measure reduction in atrophic lesion size, with secondary endpoints including improvements in visual acuity.
According to the company’s press release, Phase 1 trial data showed a favorable safety profile with no serious adverse events related to the treatment. The data also indicated 48% slower lesion growth at 12 months in treated eyes compared to untreated eyes, and a statistically significant improvement in visual function.
"The initiation of this pivotal Phase 2/3 study represents a significant milestone in our commitment to bringing transformative genetic therapies to individuals affected by Stargardt disease," said Dr. Huma Qamar, Chief Medical Officer at Ocugen.
Stargardt disease is the most common form of inherited macular degeneration, affecting approximately 100,000 patients in the U.S. and Europe combined. The condition typically develops during childhood or adolescence, causing progressive vision loss due to photoreceptor cell degeneration in the retina.
Ocugen plans to use one-year follow-up data to support a Biologics License Application, which the company aims to submit in 2027. With a beta of 4.24, indicating high volatility compared to the market, investors seeking detailed analysis can access comprehensive financial health metrics and 14 additional ProTips through InvestingPro’s exclusive research reports, which provide in-depth analysis of over 1,400 US stocks.
In other recent news, Ocugen, Inc. reported its first-quarter 2025 financial results, revealing a net loss of $15.3 million, or $0.05 per share, which was slightly better than the forecasted loss of $0.06 per share. The company also announced a significant licensing agreement with a leading Korean pharmaceutical company for its gene therapy product OCU400, aimed at treating retinitis pigmentosa. This deal includes upfront fees and development milestones totaling up to $11 million, along with sales milestones and royalties on net sales in Korea. Additionally, Ocugen’s gene therapy product OCU410ST received Rare Pediatric Disease Designation from the FDA, targeting ABCA4-associated retinopathies.
H.C. Wainwright adjusted its price target for Ocugen to $7.00, down from $8.00, while maintaining a Buy rating on the stock. The firm highlighted the potential of Ocugen’s OCU400 due to its gene-agnostic approach, which could offer broader applications for retinitis pigmentosa treatment compared to single-gene therapies. Ocugen is advancing its OCU400 through Phase 3 clinical development, with plans to file a Biologics License Application by 2026. The company is also progressing with its Phase 3 Limelight study, focusing on OCU400.
These developments reflect Ocugen’s strategic focus on expanding its gene therapy pipeline and addressing unmet medical needs. Investors and analysts are closely monitoring Ocugen’s progress, given the company’s recent achievements and future plans in the biotechnology sector.
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