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FDA Clears IND Amendment to Initiate Phase 2/3 Trial of Ocugen's OCU410ST for Stargardt Disease

The phase 2/3 clinical trial for OCU410ST, a modifier gene therapy candidate, will enroll 51 participants diagnosed with Stargardt disease. The primary objective of the trial is to evaluate the reduction in atrophic lesion size.

Ophthalmology Management June 17, 2025

Ocugen, Inc. announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies). The FDA previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3, the company said in a press release. 

Positive data from the phase 1 GARDian trial for OCU410ST demonstrated:

  • A favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest
  • Considerably slower lesion growth—48% at 12-month follow-up in evaluable treated eyes when compared to untreated eyes
  • Statistically significant (p=0.031) improvement with clinically meaningful, nearly 2-line gain in visual function (BCVA) at 12-month follow-up in evaluable treated eyes when compared to untreated eyes

According to Ocugen, the phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5×10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial, the company said, is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in best-corrected visual acuity (BCVA) and low-luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company’s Biologics License Application (BLA).

The OCU410ST phase 2/3 pivotal confirmatory trial represents a major advancement as Ocugen’s second late-stage clinical program, the company said in the press release. It plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing 3 BLAs over the next 3 years.