Retinitis Pigmentosa Gene Therapy

Ocugen has dosed the first patient with retinitis pigmentosa (RP) in its phase 3liMeliGhT trial (NCT06388200) of OCU400 gene therapy.1

“Patients with RP associated with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit to RP patients,” Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair, Ocugen, said in a statement.1 “OCU400 is a novel modifier gene therapy approach that could initiate a paradigm shift in the treatment of RP and to field of ophthalmology.”

The phase 3 trial will last 1 year and have a sample size of 150 participants, at least 8 years of age, half with RHO gene mutations and half that have gene agnostic RP. Each arm is randomized 2:1 to receive either 2.5 x 1010 vg/eye of OCU400 or control. The study’s primary endpoint is achieving an improvement of at least 2 Lux levels from baseline in the study eyes on Luminance Dependent Navigation Assessment (LDNA). Ocugen announced that it had received investigational new drug application clearance for a phase 3 trial of OCU400 in April 2024.2

“The current OCU400 Phase 3 study is very exciting and gives hope for thousands of individuals with RP,” Benjamin Bakall, MD, PhD, Director, Clinical Research, Associated Retina Consultants (ARC) and Clinical Assistant Professor, University of Arizona, College of Medicine – Phoenix, added.2 “I am encouraged that we may have a potential treatment option to preserve or improve the vision in RP patients regardless of gene mutation, and very pleased that the first patient dosing in the Phase 3 liMeliGhT clinical trial was performed at ARC.”

READ MORE: Multicharacteristic Opsin Gene Therapy Improves BCVA, MLSDT in Retinitis Pigmentosa

OCU400 has previously been evaluated in a phase 1/2 trial, data from which suggests positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. In this study, most patients (89%; n = 16/18) demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline, and 80% (n = 8/10) of patients with an RHO mutation experienced either preservation or improvement in MLMT scores from baseline. Most (78%; n = 18) also had preservation or improvement in treated eyes in MLMT scores from baseline.

“We are grateful for our continued collaboration with Dr. Bakall and the team at ARC,” said Huma Qamar, MD, MPH, Chief Medical Officer, Ocugen, added.1 "We are excited to expand our enrollment to include more centers and patients representing a diverse array of RP gene mutations, which will be a validation of this novel gene therapy platform. We will provide updates as our progress continues."

“Each clinical milestone achieved by OCU400 brings us closer to providing a one-time treatment for life to patients living with RP,” Shankar Musunuri, PhD, MBA, Chairman, CEO and cofounder, Ocugen, added.1 “Dosing the first patient is especially significant and makes our dedication to serving RP patients—300,000 in the U.S. and Europe and 1.6 million worldwide—more tangible.”
REFERENCES
1. Ocugen, Inc. Announces First Patient Dosed in Phase 3 liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication. June 20, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-3-liMeliGhT-Clinical-Trial-for-OCU400-First-Gene-Therapy-in-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html
2. 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. News release. Ocugen. April 8, 2024. https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-us-fda-clearance-ind-amendment-initiate/