Health Canada Approves Ocugen's Phase 3 Trial for OCU400 Gene Therapy

Ocugen, Inc., a biotechnology company dedicated to the innovation, development, and commercialization of breakthrough gene and cell therapies and vaccines, recently announced a significant advance in their OCU400 Phase 3 clinical trial for retinitis pigmentosa (RP) in Canada. On August 26, 2024, Health Canada issued a “No Objection Letter,” allowing Ocugen to initiate the liMeliGhT clinical trial in the country. This trial is a crucial step for OCU400, a gene therapy product designed to treat RP, a genetic disorder that leads to vision loss and blindness.

The expansion into Canada is a strategic move aimed at reaching a more extensive patient population and addressing the diverse gene mutations responsible for RP. Dr. Shankar Musunuri, Ocugen’s Chairman, CEO, and Co-Founder, emphasized the importance of this milestone, noting that the concurrent trials in Canada and the United States could accelerate the availability of a gene-agnostic treatment for approximately 110,000 RP patients in these regions.

Retinitis pigmentosa affects around 10,000 individuals in Canada and 1.6 million globally. The Canadian Phase 3 trial plans to enroll up to 50 participants across five sites. RP is characterized by mutations in over 100 genes, and the study will involve 150 participants over the course of a year. These participants will be divided into two groups: one with RHO gene mutations and the other gene-agnostic. Within each group, participants will be randomly assigned in a 2:1 ratio to either receive the treatment (2.5 x 10^10 vg/eye of OCU400) or be part of the untreated control group.

The primary endpoint of the trial is the Luminance Dependent Navigation Assessment (LDNA), which measures changes in functional vision from baseline to week 52. Participants’ ability to navigate a maze in varying light conditions will be assessed, with those showing improved navigation in dimmer light classified as “responders.” The trial aims to determine the proportion of responders among treated and untreated eyes.

Dr. Huma Qamar, Ocugen’s Chief Medical Officer, highlighted the potential benefits of establishing clinical sites in Canada, including faster recruitment and expanded commercialization opportunities in the U.S. and Europe. Given the limited treatment options currently available for RP, the introduction of a new therapeutic option is eagerly anticipated by patients worldwide. Ocugen’s efforts to extend their research into Canada offer renewed hope for RP patients.

Previously, Ocugen announced that OCU400 had received orphan drug and RMAT designations from the U.S. FDA. The company is on track to meet its 2026 targets for Biologics License Application (BLA) and Marketing Authorization Application (MAA) approvals.

OCU400 leverages a nuclear hormone receptor gene, NR2E3, that regulates various physiological functions within the retina, such as photoreceptor development, metabolism, phototransduction, inflammation, and cell survival. In patients with RP, retinal cells suffer from a dysfunctional gene network, and OCU400 aims to reset this network to restore healthy cellular homeostasis, potentially improving vision.

RP encompasses a group of rare genetic disorders that deteriorate retinal cells, causing vision impairment and eventual blindness. While there are currently no treatments to halt the progression of multiple RP forms, various proposed treatments include gene replacement therapy, retinal implants, transplantation, stem cells, vitamin therapy, and pharmacological treatments. However, current gene therapies are limited to single mutations and do not address the overall genetic defect. Consequently, broad-spectrum, gene-agnostic therapies like OCU400 offer greater hope for RP patients.

Ocugen, Inc. remains committed to discovering, developing, and commercializing innovative gene and cell therapies, biologics, and vaccines to improve global health. Through courageous innovation and leveraging unique intellectual and human resources, the company aims to make a significant impact on patients’ lives. Their modifier gene therapy platform holds potential for treating multiple retinal diseases, and their research extends to infectious and orthopedic diseases to address various unmet medical needs.