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Ocugen Inc announces FDA clearance of IND amendment to Initiate OCU400 Phase 3 clinical trial


The Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic.

(Image Credit: AdobeStock/maurice norbert)

(Image Credit: AdobeStock/maurice norbert)

Ocugen Inc announced the FDA has cleared its Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen, pointed out in a news release the initiation of the OCU400 Phase 3 clinical trial is a significant milestone for patients with RP and a pivotal event for Ocugen as a company.

“OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication,” Musunuri said in the news release. “Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven’t had a treatment option.”

According to the company, the Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

Moreover, in the Phase 1/2 OCU400 clinical trial, a Multi-Luminance Mobility Testing (MLMT) scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used—Luminance Dependent Navigation Assessment (LDNA)— that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.1

“A sensitive mobility course, LDNA, was developed by Ocugen in collaboration with FDA for the Phase 3 clinical trial to allow enrollment of patients with early to advanced stages of disease,” Arun Upadhyay, PhD, chief scientific officer at Ocugen, said in the news release.

Upadhyay said the company was encouraged that with this Phase 3 study design more than 50% of intent to treat RHO patients would meet the responder criteria, which is demonstrating 2 or greater Lux level of improvement after one year of treatment based on Phase 1/2 study results.

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation.

“We believe that the gene-agnostic clinical trial design provides an appropriate therapeutic option to include patients who have greater potential of benefiting from treatment,” sHuma Qamar, MD, chief medical officer at Ocugen, said in the news release. “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.”

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.1

According to the company,OCU400 is its gene-agnostic modifier gene therapy product based on NHR gene, NR2E3NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.1

About RP

RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP.

  1. Ocugen. Ocugen, Inc. Announces U.S. FDA Clearance of IND Amendment to Initiate OCU400 Phase 3 Clinical Trial — First Gene Therapy to Enter Phase 3 with a Broad Retinitis Pigmentosa Indication. GlobeNewswire News Room. Published April 8, 2024. Accessed April 8, 2024. https://www.globenewswire.com/news-release/2024/04/08/2859013/0/en/Ocugen-Inc-Announces-U-S-FDA-Clearance-of-IND-Amendment-to-Initiate-OCU400-Phase-3-Clinical-Trial-First-Gene-Therapy-to-Enter-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html
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