Ocugen has recieved clearance from the FDA for its IND amendment to the Phase III trial of OCU400. Image credit: Shutterstock / Wirestock Creators

Ocugen will soon be initiating a Phase III trial of its gene therapy candidate OCU400 for retinitis pigmentosa (RP).

The company announced an investigational new drug (IND) amendment for the Phase III trial was cleared by the US Food and Drug Administration (FDA).

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The Phase III study will enrol 150 participants in two arms; one arm of patients with the RHO gene mutation and the other arm of patients who are gene agnostic. In each arm, patients will be randomised to the treatment group and untreated control group.

Chief medical officer, Dr. Huma Qamar, said: “We believe that the gene-agnostic clinical trial design provides an appropriate therapeutic option to include patients who have a greater potential of benefiting from treatment. We are looking forward to working with our selected trial sites and leading retinal surgeons to deliver this novel modifier gene therapy to potentially address unmet medical needs.”

For the Phase III OCU400 clinical trial, the Luminance Dependent Navigation Assessment (LDNA) will be used to measure the primary endpoint. LDNA was developed by Ocugen in collaboration with the FDA for the Phase III trial to allow enrolment of patients with early to advanced stages of disease. The assessment covers a broader spectrum of light intensity and Lux Levels.

In December 2022, OCU400 received orphan drug designation from the FDA with regenerative medicine advanced therapy designation (RMAT) being granted in December 2023. The company hopes to submit a Biologics License Application (BLA) for OCU400 by 2026.

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OCU400 is an adeno-associated virus serotype 5 capsid containing the gene for human nuclear hormone receptor NR2E3. It acts by targeting centrosomal protein of 290 kDa (CEP290) and rhodopsin.

GlobalData predicts a sales forecast for OCU400 of $274m in 2029.

GlobalData is the parent company of the Clinical Trials Arena.

RP landscape

RP comprises a cluster of uncommon eye conditions that cause degeneration of the retina, the light-sensitive layer of tissue at the back of the eye, finally leading to a progressive loss of vision.

A GlobalData report estimates that across the 16 major pharmaceutical markets (16MM: Australia, Brazil, Canada, China, France, Germany, India, Italy, Japan, Mexico, Russia, South Africa, South Korea, Spain, UK, and US), there will be 1,043,381 diagnosed prevalent cases of RP in 2024, which is expected to increase to 1,088,269 diagnosed prevalent cases in 2029.

Currently, Roche is the only company dominating the RP market with its gene therapy-based drug Luxturna, which is approved in multiple geographies.