Ocugen, Inc., a biotechnology firm specializing in the discovery, development, and commercialization of innovative gene and cell therapies, along with vaccines, has announced the approval of its Investigational New Drug (IND) amendment by the U.S. Food and Drug Administration (FDA) to commence a Phase 3 clinical trial of OCU400. This therapy is designed to address retinitis pigmentosa (RP).

Dr. Shankar Musunuri, Ocugen's Chairman, CEO, and Co-Founder, highlighted the significance of this milestone, stating, “The initiation of the OCU400 Phase 3 clinical trial marks a crucial moment for RP patients and represents a pivotal event for Ocugen. OCU400 is breaking new ground as the first gene therapy program to advance to Phase 3 with a comprehensive RP indication. This offers real hope for all RP patients who have been lacking treatment options thus far.”

The Phase 3 trial will involve 150 participants, divided into two arms: one consisting of 75 participants with the RHO gene mutation and the other arm comprising 75 participants who are gene agnostic. Within each arm, participants will be randomized 2:1 to either the treatment group (receiving 2.5 x 1010 vg/eye of OCU400) or the untreated control group.

In the Phase 1/2 trial of OCU400, the primary functional endpoint was assessed using the Multi-Luminance Mobility Testing (MLMT) scale. For the Phase 3 trial, an updated mobility course called Luminance Dependent Navigation Assessment (LDNA) will be utilized. LDNA incorporates a broader range of light intensity (0.04-500 Lux) and Lux Levels (0-9), ensuring a consistent correlation between Lux level and Lux intensity.

Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen, highlighted the development of LDNA in collaboration with the FDA, emphasizing its sensitivity and its ability to accommodate patients with varying stages of disease severity. He stated, “We are optimistic that with this Phase 3 study design, more than 50% of intent-to-treat RHO patients would meet the responder criteria, showing a Lux level improvement of 2 or greater after one year of treatment, as observed in the Phase 1/2 study.”

Currently, there are approximately 110,000 RP patients in the United States and 1.6 million globally, with over 10% of them having the RHO genetic mutation.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, emphasized the gene-agnostic approach of the clinical trial design, offering a suitable therapeutic option for patients who have a higher potential to benefit from treatment. She expressed anticipation for collaborating with selected trial sites and leading retinal surgeons to deliver this innovative modifier gene therapy to 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 targeted BLA approval in 2026.

OCU400 is Ocugen’s gene-agnostic modifier gene therapy product based on the NHR gene, NR2E3. NR2E3 regulates various physiological functions within the retina, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks. OCU400 aims to reset altered cellular gene-networks and establish homeostasis, potentially improving retinal health and function in patients with inherited retinal diseases.

RP comprises a group of rare, genetic disorders characterized by the breakdown and loss of cells in the retina, leading to vision loss and blindness. Presently, there are no approved treatments that effectively slow or halt the progression of multiple forms of RP. Proposed treatments include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. However, current gene-replacement therapies are limited to treating a single mutation. Therefore, the development of gene-agnostic therapies offers greater hope for RP patients, especially when multiple and unknown genes are involved.

 

Source: globenewswire.com

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