Ocugen Receives Health Canada Approval to Initiate Phase 3 Clinical Trial for Retinitis Pigmentosa

Ocugen announced that Health Canada has provided a “No Objection Letter” to initiate the phase 3 Limelight clinical trial for OCU400 in Canada. OCU400 is a modifier gene therapy product candidate designed for the treatment of retinitis pigmentosa (RP).

"Expanding the clinical trial to Canada is significant as it will provide an opportunity to reach a broader patient population encompassing many gene mutations associated with RP," Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen, said in a company news release. "The Health Canada trial will run in parallel with the US FDA trial, expediting the ability to potentially provide a gene-agnostic treatment option to approximately 110,000 patients in the United States and Canada."

The phase 3 study in Canada will enroll up to 50 participants across a maximum of five sites as part of the Limelight clinical trial. The trial aims to offer a broad treatment option, addressing over 200 mutations in more than 100 genes linked to RP. The study, which spans 1 year, will include 150 participants, split into two groups: 75 with RHO gene mutations and 75 who are gene agnostic. Participants will be randomized in a 2:1 ratio to receive either OCU400 treatment (2.5 x 10^10 vg/eye) or remain in an untreated control group. The study targets patients aged 8 and older, covering the entire spectrum of RP progression, from early to late stages.

The primary endpoint for the study is the Luminance Dependent Navigation Assessment (LDNA), a sensitive and specific measurement of functional vision. The endpoint will assess the change in functional vision from baseline to week 52 by evaluating participants' ability to navigate a maze under varying light conditions. Participants who show improved navigation in dimmer light (by ≥2 Lux levels) compared to baseline will be classified as "responders" to the therapy. The study will focus on the proportion of responders in both treated and untreated eyes.

OCU400 has previously received orphan drug and RMAT (Regenerative Medicine Advanced Therapy) designations from the FDA, positioning the therapy on track for Biologics License Application (BLA) and Marketing Authorization Application (MAA) approvals by 2026.