Nanoscope Shares Positive 2-Year Trial Results of MCO-010 for Retinitis Pigmentosa

DALLAS, Oct. 31, 2024 – Nanoscope Therapeutics, Inc., a biotechnology company at the stage of filing a Biologics License Application (BLA), is developing gene therapies for retinal degenerative diseases. The company recently shared promising 2-year outcomes from its Phase 2b RESTORE clinical trial of MCO-010, a gene therapy designed for patients with severe vision loss due to advanced retinitis pigmentosa (RP). These findings were presented by Dr. Allen C. Ho, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor of Nanoscope, during the AAO 2024 Annual Meeting on October 20, 2024.

In 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 showcased significant improvements in visual acuity. Specifically, the patients demonstrated an average improvement of ~0.3 LogMAR (equivalent to a 3-line, 15-letter gain) in best-corrected visual acuity (BCVA) that was sustained throughout the study period.

Key findings include:
- The high-dose MCO-010 group showed mean visual acuity improvements of 0.337 ± 0.0829 and 0.539 ± 0.1032 LogMAR at weeks 52 and 76 respectively, compared to the sham-control group (p = 0.0209 and 0.0014).
- The low-dose MCO-010 group experienced mean improvements of 0.382 ± 0.1244 and 0.374 ± 0.1332 LogMAR at the same time points (p = 0.0290 and 0.0652).
- At the 52-week mark, 39% of MCO-010 treated patients had a BCVA improvement of ≥0.3 LogMAR, increasing to 56% by week 76.
- Longitudinal analysis indicated that BCVA improvements were statistically significant compared to the sham-control from weeks 36 to 88.
- The BCVA area under the curve (AUC) profiles at weeks 52, 76, and 100 were five times greater and statistically significant for both dosage groups compared to sham-control.

The therapy was well tolerated, with no serious adverse events reported among the MCO-010 groups. The most common adverse events, such as anterior chamber cell presence and ocular hypertension, occurred more frequently in the MCO-010 groups compared to the sham-control group but were manageable with topical medication.

Dr. Ho emphasized the significance of these results, noting that the sustained, statistically significant vision improvement observed during the 2-year study period is unprecedented in trials involving a diverse and severely vision-impaired patient population. He expressed optimism that this therapy could soon offer a viable solution for patients suffering from severe vision loss.

The RESTORE trial represents the first randomized controlled trial to show statistically significant visual acuity improvement in RP patients, marking a critical milestone for Nanoscope's RP program and its forthcoming BLA submission. The trial's design had been previously detailed.

Sulagna Bhattacharya, co-founder and CEO of Nanoscope, highlighted the company's leadership in developing optogenetic therapies that are mutation and disease agnostic, with optimal performance and sensitivity across the visible light spectrum. She reiterated the company's commitment to addressing the unmet needs of patients with retinal degeneration and their dedication to bringing MCO-010 to those with permanent and progressive vision loss.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics Inc. is focused on developing gene-agnostic optogenetic therapies to restore sight in patients blinded by retinal degenerative diseases, for which there is currently no cure. Their lead asset, MCO-010, has shown positive results in the RESTORE Phase 2b clinical trial for retinitis pigmentosa. The company has also completed the Phase 2 STARLIGHT trial for Stargardt patients and is planning a Phase 3 registrational trial in early 2025. MCO-010 has been granted FDA Fast Track and orphan drug designations for both RP and Stargardt. Nanoscope is also working on preclinical assets, including a non-viral laser-delivered gene therapy for geographic atrophy.