Ocugen, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP). The Phase 3 study will have a sample size of 150 participants?one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

In the Phase 1/2 OCU400 clinical trial, a Multi-Luminance Mobility Testing (MLMT) scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used?Luminance Dependent Navigation Assessment (LDNA)? that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation. Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA.

With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.