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.
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