Ocugen reports preliminary safety, efficacy results Phase 1/2 trial of OCU400

Ocugen announced positive preliminary data among retinitis pigmentosa 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 mutations and Leber Congenital Amaurosis with mutation 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. 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. 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 or medium dose OCU400, respectively. In the preliminary data analysis, 9-month follow-up data for 3 subjects, and 6-month follow-up data for 4 subjects were evaluated. Results showed a favorable safety profile and visual improvements after treatment with OCU400 as measured by multi-luminance mobility testing and best corrected visual acuity assessment. Key efficacy outcomes from 7 subjects demonstrated: 100% of treated eyes showed a stable or improved MLMT score trend; 5 of 7 OCU400 treated eyes demonstrated a 1 or more Lux level improvement in MLMT score compared to 28.6 % of untreated eyes; 66.7% 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 OCU400 treated eyes demonstrated 8-11 letters of improvement in BCVA score compared to none of the untreated eyes.