NEW YORK – Ocugen on Monday said it was cleared to begin a Phase III trial in Canada of its gene therapy OCU400 for the treatment of retinitis pigmentosa.
The Phase III liMeliGhT trial will evaluate OCU400 in 150 patients who are 8 years and older with retinitis pigmentosa. The study will comprise two arms: 75 participants with RHO gene mutations and 75 participants who are gene agnostic. Within each arm, patients will be randomized 2-to-1 to receive either OCU400 or no treatment.
The researchers hope to evaluate if OCU400 can be a gene-agnostic treatment for retinitis pigmentosa, which has been associated with mutations in more than 100 genes. The trial is currently ongoing in the US.
"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 retinitis pigmentosa," Ocugen CEO Shankar Musunuri said in a statement. "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 liMeliGhT study's primary endpoint is participants' change in functional vision from baseline to week 52 as measured by the ability of a study participant to navigate through a maze using a functional vision test called the Luminance Dependent Navigation Assessment (LDNA). Researchers will classify responders as those who are able to navigate the LDNA in dimmer light than the baseline test.
Earlier this month, Ocugen made OCU400 available to more retinitis pigmentosa patients in the US through an expanded access program. OCU400 has received orphan drug and regenerative medicine advanced therapy designations from the US Food and Drug Administration, and Ocugen said it remains on track to submit regulatory applications for the therapy in the US and Europe for potential approval in 2026.