“Substantial improvements in visual function” that were “sustained and sometimes transformative” have been observed in patients with advanced retinitis pigmentosa (RP) treated with MCO-010 – an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy.
The observations were published alongside topline results from Nanoscope Therapeutics’ Phase 2b multi-centre, randomised, double-masked, sham-controlled RESTORE clinical trial of MCO-010.
Nanoscope Therapeutics, a clinical-stage biopharmaceutical company, has received both orphan drug and fast track designations from the United States Food and Drug Administration (FDA) for MCO-010.
In the RESTORE trial, 18 patients with severe vision impairment due to RP received a single intravitreal injection of MCO-010 while nine received a sham procedure. Results showed vision function improvements after treatment with MCO-010 consistent with previous studies, and a favourable safety profile.
The primary outcome measure was mean change in multi-luminance y-mobility test (MLYMT, vision-guided mobility) score vs. placebo. Other key efficacy assessments included the multi-luminance shape discrimination test (MLSDT, near object recognition), and best-corrected visual acuity (BCVA).
For the MLYMT and MLSDT, a two or more luminance level change is considered clinically meaningful. For BCVA, a 0.3 LogMAR change is considered clinically meaningful, with negative change indicating improved visual acuity.
Of MCO-010 treated patients, 88.9% demonstrated a two or more luminance level improvement in MLYMT or MLSDT at 12 months compared to 44.4% receiving placebo (p <0.05); seven of 18 MCO-010 treated patients improved by -0.3 LogMAR or more in BCVA compared to one of nine receiving placebo.
