Multi-Characteristic Opsin Gene Therapy for Retinitis Pigmentosa Improves Multiple Measures of Vision


Updated data from the RESTORE study on MCO-010, Nanoscope Therapeutics' investigational gene therapy, were presented at the 2023 ASGCT meeting.

Updated data from the phase 2 RESTORE study (NCT04945772) have shown that MCO-010 (Nanoscope Therapeutics) multicharacteristic opsin gene therapy was well-tolerated and yielded clinically meaningful improvements on multiple measures of vision in patients with advanced retinitis pigmentosa (RP).

These data were presented at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place May 16-20 in Los Angeles, California, by David Liao, MD, ophthalmologist, Retina Vitreous Associates Medical Group.

“[A] substantial body of clinical evidence now exists demonstrating MCO-010 improves functional vision with a favorable safety profile in patients with severe vision loss due to RP, a condition for which there are new approved treatment,” Liao and colleagues wrote in the poster.

Liao and colleagues found that in 18 treated patients, 88.9% (n = 16) had a clinically meaningful 2 or more luminance level improvement in vision guided mobility or object recognition and patients had a mean improvement in best corrected visual acuity (BCVA) of –0.34 logMAR. All treated patients improved on at least 1 efficacy measure, including multi-luminance mobility testing (MLYT), multi-luminance shape discrimination (MLSDT), or BCVA. Specifically, for MLYT, 67% (n = 12) of treated patients improved by at least 2 levels compared to 33% (n = 3) of control patients. On MLSDT, 56% (n = 10) of treated patients improved by at least 2 levels compared to 22% (n = 2) control patients; and on BCVA, 39% (n = 7) of treated patients improved by at least 0.3 logMAR compared to 1 control patient.

MCO-010 was well-tolerated, with no serious ocular or systemic adverse events (AEs). AEs were similar across study arms, with similar demographics between cohorts. Cellular inflammation was self-limiting or treated with steroids and idiopathic orbital inflammation did not require treatment in 16 patients. There were no cases of retinitis, choroiditis, vasculitis, ischemic neuropathy, hypopyon, or hypotony in treated patients.

“MCO-010 has the potential to meet a high unmet need and may yield substantial benefit to patients with severe vision loss due to advanced RP that have no available treatments today. The therapy showed consistency of response, with visual function improvements and safety profile consistent with previous studies. The next steps will be to engage with FDA and other regulatory agencies on the future of MCO-010, with the goal of expeditiously getting this novel therapy to patients,” Liao said.

Liao D, Gonzalez V, Emmanuelli A, et al. Optogenetic therapy with MCO-010 for vision restoration in patients with severe sight loss due to retinitis pigmentosa: The Phase 2b RESTORE study. Presented at: 2023 ASGCT Annual Meeting; May 16-20; Los Angeles, California. Poster #808 
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