DALLAS, Oct. 31, 2024 / — Nanoscope Therapeutics, Inc., a BLA filing-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that positive 2-year results from the Phase 2b RESTORE randomized, controlled clinical trial (RCT) of its lead program, MCO-010, a mutation-agnostic gene therapy for patients with permanent and severe vision loss from advanced retinitis pigmentosa (RP) were presented by Allen C. Ho, MD, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope, on October 20, 2024 at the recently concluded AAO 2024 Annual Meeting.

During his presentation titled “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),” Dr. Ho highlighted the measured benefit of ~0.3 LogMAR (3-line, 15-letter equivalent) mean best-corrected visual acuity (BCVA) improvement that was maintained at later time points over the course of the sham-controlled trial. Additional findings included:

• High-dose MCO-010 group had mean improvements of 0.337 ± 0.0829 and 0.539 ± 0.1032 LogMAR (vs. sham-control, p = 0.0209 and 0.0014) at weeks 52 (primary) and 76 (key secondary), respectively, and low-dose MCO-010 group had mean improvements of 0.382 ± 0.1244 and 0.374 ± 0.1332 LogMAR (vs. sham-control, p = 0.0290 and 0.0652) at the same timepoints (previously announced).
• At week 52, 7 of 18 (39%) MCO-010 dosed individuals had BCVA improvement ≥0.3 LogMAR; 10 of 18 (56%) reached this threshold at week 76.
• Longitudinal analysis shows mean BCVA improvement with statistical significance compared to sham-control at weeks 36-88 visits.
• BCVA area under the curve profiles up to weeks 52, 76, and 100 were 5 times greater and statistically significant for both MCO-010 dose groups in comparison to sham-control.
• MCO-010 was well tolerated with no serious adverse events reported in MCO-010 groups. Anterior chamber cell (44% MCO-010, 22% sham-control) and ocular hypertension (39% MCO-010, 11% sham-control) were the most commonly reported adverse events. These adverse events resolved or were controlled with topical medication.

“The durable statistically significant vision improvement achieved at multiple time points during the 2-year study is highly noteworthy. This degree of improvement has never been observed in a randomized, controlled trial of a highly heterogeneous severe vision-loss patient population,” said Dr. Ho. “These findings deliver hope to patients and ophthalmologists that a solution is close to being found. We are finally on the brink of an impactful in-office, mutation-agnostic gene therapy for people with severe vision loss.”

RESTORE is the first-ever RCT to show statistically significant visual acuity improvement in the RP patient population and a key part of the upcoming BLA submission planned for Nanoscope’s RP program. The RESTORE study design has been previously announced.

“Nanoscope is proud to be a leader in the optogenetic space, advancing a mutation and disease agnostic therapy designed for optimal performance with rapid kinetics and sensitivity across the visible light spectrum and at ambient light levels,” said Sulagna Bhattacharya, co-founder, and Chief Executive Officer of Nanoscope. “We recognize the high unmet need, and we are working diligently to bring MCO-010 to these patients with permanent and progressive vision loss from retinal degeneration. They are our North Star.”

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

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