Ocugen Doses First Patient in Phase 3 Clinical Trial for OCU400 Gene Therapy for RP

Ocugen announced that the first patient has been dosed in its phase 3 liMeliGhT clinical trial for OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

“Each clinical milestone achieved by OCU400 brings us closer to providing a one-time treatment for life to patients living with RP,” Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen, said in a company news release. “Dosing the first patient is especially significant and makes our dedication to serving RP patients—300,000 in the US and Europe and 1.6 million worldwide—more tangible.”

The phase 3 liMeliGhT clinical trial was informed by positive phase 1/2 OCU400 data that suggests positive trends in best-corrected visual acuity (BCVA) and multi-luminance mobility testing (MLMT), and low-luminance visual acuity (LLVA) among treated eyes. In that trial, 89% (16/18) of RP subjects demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline; 80% (8/10) of RHO mutation subjects experienced either preservation or improvement in MLMT scores from baseline; and 78% (14/18) of subjects demonstrated preservation or improvement in treated eyes in MLMT scores from baseline.

The phase 3 study—with the duration of 1 year—will have a sample size of 150 participants: one arm of 75 participants with RHO gene mutations 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. Patients 8 years of age and older, with early through late-stage advancement of RP, are being recruited to participate in the liMeliGhT study.

Luminance Dependent Navigation Assessment (LDNA)—a more sensitive and specific measurement of function than MLMT used in previous phase 3 clinical trials—is the primary endpoint for the study. The phase 3 liMeliGhT study will focus on the proportion of responders, in treated and untreated groups, achieving an improvement of at least 2 Lux levels from baseline in the study eyes.

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA, and that the EMA provided acceptability of the US-based trial for submission of a Marketing Authorization Application (MAA). With the first dosing of the phase 3 clinical trial, OCU400 remains on track for the 2026 BLA and MAA approval targets.