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Saturday / July 13.
HomeminewsPositive Results for RP Optogenetic Trial

Positive Results for RP Optogenetic Trial

“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.