Health Canada approves Ocugen's Phase 3 clinical trial for OCU400

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Ocugen Inc announced that Health Canada provided a “No Objection Letter” to initiate the OCU400 Phase 3 liMeliGhT clinical trial in Canada.

According to a news release, OCU400 is a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP). The candidate is based on a nuclear hormone receptor gene called NR2E3, which regulates diverse physiological functions within the retina, such as photoreceptor maintenance and development, metabolism, inflammation, phototransduction, and cell survival. The company noted that retinal cells in RP patients have a dysfunctional gene network, and OCU400 resets this network to reestablish a healthy cellular homeostasis—which has the potential to improve vision in patients with RP.¹

Shankar Musunuri, PhD, MBA, chairman, CEO, and co-founder of Ocugen, pointed out in a news release that 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.

“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,” Musunuri said.

The company noted in its news release that there currently are about 10,000 patients in Canada with RP and 1.6 million patients globally. The Phase 3 study in Canada will enroll up to 50 subjects across a maximum of 5 sites for the liMeliGhT clinical trial.

More than 200 mutations in more than 100 genes have been connected to RP. The Phase 3 study, spanning 1 year, will enroll 150 participants divided into 2 study arms. One included 75 participants with RHO gene mutations and another with 75 participants who are gene agnostic. In each arm, participants will be randomized in a 2:1 ratio to receive either treatment (2.5 x 1010 vg/eye of OCU400) or remain in an untreated control group, respectively. The liMeliGhT study is recruiting patients aged 8 and older, covering the full spectrum from early to late stages of RP progression.¹

An enhanced sensitive and specific measurement of functional vision test—Luminance Dependent Navigation Assessment (LDNA)—is the primary endpoint for the study. Specifically, the primary endpoint is a measurement of the change in functional vision from baseline to week 52 as measured by the ability of a study participant to navigate through a maze (the LDNA). Those who demonstrate an improved ability to navigate the maze in dimmer light (i.e., by ≥2 Lux levels) compared to baseline will be classified as "responders" to the therapy. The liMeliGhT study will focus on the proportion of responders in both the treated and untreated eyes.¹

“Establishing clinical sites in Canada may expedite recruitment and open doors for broader commercialization with the U.S. and Europe,” Huma Qamar, MD, MPH, chief medical officer at Ocugen, said n the news release. “With only one currently approved treatment targeting a single mutation associated with RP, there remains a significant unmet medical need, and patients worldwide are eager for new therapeutic options. It is highly rewarding to extend our efforts into a new region and offer hope to Canadian patients with RP.”

According to the news release, Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. OCU400 remains on track for the 2026 BLA and MAA approval targets.

Reference:

1. Ocugen. Ocugen, Inc. Announces Health Canada Approval to Initiate Phase 3 Clinical Trial for OCU400 – Modifier Gene Therapy for Broad Retinitis Pigmentosa Indication. GlobeNewswire News Room. Published August 26, 2024. Accessed August 26, 2024. https://www.globenewswire.com/news-release/2024/08/26/2935386/0/en/Ocugen-Inc-Announces-Health-Canada-Approval-to-Initiate-Phase-3-Clinical-Trial-for-OCU400-Modifier-Gene-Therapy-for-Broad-Retinitis-Pigmentosa-Indication.html