Ocugen Announces Positive Preliminary Safety and Efficacy Results from the Phase 1/2 Trial of Gene Therapy OCU400, for the Treatment of RP and LCA

Ocugen announced positive preliminary data among retinitis pigmentosa (RP) participants treated in the first two cohorts of the phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber Congenital Amaurosis (LCA) with mutation(s) in the CEP290 gene. These preliminary results provide support that OCU400, Ocugen’s first-in-class therapeutic approach utilizing a proprietary modifier gene therapy platform, has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration, according to a company news release. 

“It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen, said in the news release. “This is the first clinical validation of the platform where patient responses across various genetic mutations support that OCU400 has the potential to transform the lives of many patients who are struggling with debilitating blindness diseases.”

This phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 RP subjects have been enrolled in this study—10 subjects in the dose escalation and 8 subjects in the expansion phase, respectively. The age of subjects enrolled to date ranges from 18-77 years across RHO and NR2E3 gene mutations. The company further expanded the phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

In Cohort 1 and 2 of the clinical trial, 7 participants with severe vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 1010 vg/mL) or medium dose (3.33 x 1010 vg/mL) OCU400, respectively. In the preliminary data analysis, 9-month follow-up data for 3 subjects [Cohort 1], and 6-month follow-up data for 4 subjects [N=1 from Cohort 1 and N=3 from Cohort 2] were evaluated.

Results showed a favorable safety profile and visual improvements after treatment with OCU400 as measured by multi-luminance mobility testing (MLMT) and best corrected visual acuity assessment (BCVA).

Key efficacy outcomes from 7 subjects demonstrated:

• 100% of treated eyes showed a stable or improved MLMT score trend;
• 5 of 7 (71.4%) OCU400 treated eyes demonstrated a 1 or more Lux level improvement in MLMT score compared to 28.6 % of untreated eyes;
• 66.7% (2 of 3) of OCU400 treated eyes in Cohort 1 with 9-month follow-up demonstrated a 2 or more Lux level improvement in MLMT score compared to none of the untreated eyes; and
• 3 of 7 (42.9%) OCU400 treated eyes demonstrated 8-11 letters of improvement in BCVA score compared to none of the untreated eyes.

“I was not expecting such substantial improvements in visual function among the trial participants I have been working with because of the advanced stage of their retinal disease,” said David Birch, PhD, Scientific Director, Retina Foundation of the Southwest, principal investigator of the study. “I am very pleased by the outcomes I have seen in my own clinic and am hopeful that OCU400 could provide a therapeutic solution for RP patients who are not only facing loss of vision, but also challenged with the psychological burden of losing their independence.”

“The early results from patients treated in the phase 1/2 clinical trial are encouraging and support the paradigm-changing potential of modifier gene therapy technology to address unmet medical needs for patients with RP and LCA,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen. “With this favorable safety profile and positive trend in efficacy signals, we are very eager to see longer-term data, and to potentially initiate Phase 3 trials in the U.S. and EU.”

Ocugen will continue to monitor long-term safety and efficacy data from the treated patients, and advance development of OCU400 to bring a potential treatment option to RP and LCA patients.

CanSinoBIO, Ocugen’s strategic partner, provided all CMC development and clinical supplies for the phase 1/2 trial of OCU400.