Nanoscope Therapeutics Inc. announced positive 3-year follow-up data from the REMAIN study, the long-term extension of the phase 2b/3 RESTORE trial evaluating MCO-010, the company's proprietary optogenetic therapy for patients with retinitis pigmentosa (RP).

The REMAIN results demonstrated durable and clinically meaningful vision improvements following a single intravitreal injection of MCO-010, with a favorable safety and tolerability profile maintained through 3 years, the company said in a press release. 

In the 152-Week REMAIN long-term follow-up study, patients maintained an average best-corrected visual acuity (BCVA) gain from baseline of approximately 0.3 LogMAR (equivalent to 3 lines or 15 letters on a standard ETDRS vision chart), through Week 152. BCVA-Area Under the Curve profiles across both RESTORE and REMAIN show fivefold greater vision gains compared to sham treatment. In addition, no serious ocular adverse events were reported among treated patients. Only 1 mild case of inflammation required topical steroids; 14 of 15 treated patients required no ongoing inflammation management at Week 152, the company reported.

MCO is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including RP. Stargardt disease, and geographic atrophy. By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO treatment does not require genetic testing, invasive surgery, or repeat dosing, the company said.

MCO-010 has been granted Fast Track and Orphan Drug designation by the US Food and Drug Administration, and a rolling Biologics License Application for RP is currently underway, The European Medicines Agency has granted MCO-010 5 Orphan designations across a wide spectrum of retinal degenerations involving photoreceptor loss.