Allen C. Ho, MD: Efficacy, Safety of MCO-010 for Retinitis Pigmentosa at 2 Years

MCO-010 optogenetic therapy achieved meaningful improvement in best-corrected visual acuity (BCVA) endpoints with statistical significance, according to Phase 2b/3 clinical trial data in patients with severe vision loss from retinitis pigmentosa (RP).¹

In an interview with HCPLive, presenter Allen C. Ho, MD, director of retina research and co-director of the retina service at Wills Eye Hospital, and chief medical advisor of Nanoscope Therapeutics, described the RESTORE program meeting its primary safety and efficacy endpoints for patients with RP, with durable effect at both the 52-week and 76-week endpoints.

“RESTORE is an optogenetic program that met its safety and efficacy endpoints for patients with severe vision loss due to retinitis pigmentosa, I look forward to describing to patients what I have done for 30 years by not trying to create false hope, but hopefully giving them some real hope with this strategy,” Ho told HCPLive.

The Phase 2b/3 clinical trial evaluated the efficacy and safety of a single intravitreal injection of MCO-010 in subjects with retinitis pigmentosa. Participants received high-dose (1.2E11 gc/eye; n = 9) or low-dose (0.9E11 gc/eye; n = 9) MCO-010 or intravitreal sham injection (n = 9) at Day 0.

Primary and key secondary endpoints included the mean best-corrected visual acuity (BCVA) change from baseline as measured by Freiburg visual acuity testing at Weeks 52 and 75, respectively. Overall, Ho and colleagues found MCO-010 had remained well-tolerated and led to no reports or serious adverse events (SAEs).

Compared with sham, high-dose MCO-010 demonstrated improvements of 0.337 ± 0.0829 (P = .0209) and 0.539 ± 0.1032 (P = .0014) logMAR. Low-dose MCO-010 showed improvements of 0.382 ± 0.1244 (P = .0290) and 0.374 ± 0.1332 (P = .0652) logMAR at Weeks 52 and 76, respectively, versus sham.

Overall, Ho indicated meaningful BCVA improvements were observed over the 2 years of study, suggesting its potential for US Food and Drug Administration (FDA) approval. Based on regulatory feedback provided by the FDA, Nanoscope Therapeutics announced plans to initiate Biologics License Application submission in Q1 2025.²

“I'm excited about the possibilities,” Ho told HCPLive.¹ “We had a meeting with the FDA, and the next steps include, for this fast-tracked orphan drug designation, a biologic license application in the first quarter of 2025.”

Disclosures: Relevant disclosures for Ho include 4DMT, Apellis, Genentech, Iveric Bio, Nanoscope, Regeneron, and others.

References

1. _{Ho AC. Efficacy and Safety of MCO-010 Optogenetic Therapy for Vision Restoration in Patients With Severe Vision Loss Due to RP: Two-Year TOPLINE Results From a Phase 2b/3 Randomized, Sham-Controlled Clinical Trial (RESTORE). Paper presented at the American Academy of Ophthalmology (AAO) 2024 Meeting. Chicago, Illinois. October 18-21, 2024.}
2. _{Iapoce C. Nanoscope signals BLA submission for MCO-010 for retinitis pigmentosa. HCP Live. October 10, 2024. Accessed October 24, 2024. https://www.hcplive.com/view/nanoscope-signals-bla-submission-for-mco-010-for-retinitis-pigmentosa.}