Ocugen announced June 16 that the US FDA had cleared an investigational new drug (IND) application for a Phase II/III trial of its OCU410ST subretinal gene therapy candidate for ABCA4-associated retinopathies, including Stargardt disease.

The FDA previously granted rare pediatric disease and orphan drug designations for OCU410ST for Stargardt, retinitis pigmentosa 19, and cone-rod dystrophy 3.

OCU410ST also has orphan drug status in the US and EU.

The Phase I GARDian trial of OCU410ST demonstrated a favorable safety and tolerability profile, with no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis, or choroidal neovascularization.

Treated eyes showed 48 percent slower lesion growth and a nearly two-line gain in best corrected visual acuity (BCVA) at 12 months vs. untreated eyes.

The Phase II/III trial aims to enroll 51 participants with Stargardt. 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 endpoint is reduction in atrophic lesion size. Key secondary endpoints include improvements in BCVA and low luminance visual acuity compared with controls.

Patients will be followed for one year, with the filing of a biologics license application (BLA) expected in 2027.

Ocugen’s modifier gene therapy approach relies on nuclear hormone receptors that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks.

Ocugen, of Malvern, Pennsylvania, is also developing OCU400, a gene therapy candidate targeting retinitis pigmentosa, and OCU410 for geographic atrophy.

More News

Want to Read Locked Articles?

Already Have an Account?

Register A Corporate Account

Topics of Interest

Products of Interest