Nanoscope Therapeutics Reports 3-Year Vision Improvements from REMAIN Study of MCO-010 in RP

Nanoscope Therapeutics has announced three-year follow-up data from the REMAIN study, the long-term extension of its pivotal Phase 2b/3 RESTORE trial investigating MCO-010, a proprietary optogenetic therapy developed to treat retinitis pigmentosa (RP). The new data highlight the durability of vision improvements and reinforce the long-term safety of the therapy.

Long-Term Vision Gains Sustained Through Week 152

The REMAIN study showed that a single intravitreal injection of MCO-010 resulted in clinically meaningful and sustained vision improvements in RP patients for up to 152 weeks. Participants maintained an average Best Corrected Visual Acuity (BCVA) gain of approximately 0.3 LogMAR, equivalent to an improvement of three lines (15 letters) on a standard ETDRS chart.

Compared with sham-treated patients, the BCVA Area Under the Curve (AUC) data from the RESTORE and REMAIN trials demonstrated a fivefold greater vision gain in the MCO-010 group, underscoring the therapy’s durable efficacy.

“The sustained visual gains demonstrated over 3 years are truly remarkable for this population,” said Allen C. Ho, MD, Director of Retina Research at Wills Eye Hospital and Chief Medical Advisor to Nanoscope. “These patients typically lose about 1.5 lines of vision every five years and often hit legal blindness as early as age 20. The ability to not just slow loss but restore visual function for several years represents a significant therapeutic advance.”

Strong Safety and Tolerability Profile Maintained

Throughout the 152-week follow-up, MCO-010 continued to show a favorable safety and tolerability profile. No serious ocular adverse events were reported. Only one case of mild inflammation required short-term treatment with topical steroids, and 14 of 15 treated patients needed no ongoing inflammation management at the study’s final time point.

Clinical and Regulatory Momentum

“These long-term results underscore the lasting impact of MCO-010 in patients who currently have no treatment options,” stated Sulagna Bhattacharya, CEO of Nanoscope Therapeutics. “As we advance our rolling Biologics License Application (BLA) with the FDA, we are preparing for a potential commercial launch of what could become the first approved optogenetic therapy for retinal disease.”

She also noted that the durability observed in REMAIN highlights the broader promise of the MCO platform as a one-time treatment for a wide range of retinal degenerative diseases.

MCO Platform: Intravitreal Therapy for Retinal Degenerative Conditions

Nanoscope’s Multi-Characteristic Opsin (MCO) platform is a disease-agnostic, optogenetic therapy designed to restore vision in patients with advanced retinal degenerative diseases, including retinitis pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA).

Delivered via a one-time intravitreal injection, MCO-010 activates bipolar retinal cells to become light-sensitive, bypassing degenerated photoreceptors and restoring visual function. The platform is designed to function without the need for genetic testing, avoids invasive surgery, does not require repeat dosing, and is compatible with standard retina clinic workflows, making it a practical option for broad clinical adoption.

Regulatory Designations Support MCO-010’s Advancement

MCO-010 has received both Fast Track and Orphan Drug Designations from the FDA. In Europe, the EMA has granted five Orphan designations for MCO-010 across multiple retinal degenerative indications, supporting its potential as a first-in-class treatment.