Clinical Trials

RESTORE was a multicentre, randomised, double-masked, sham-controlled phase 2b trial that studied the efficacy and safety of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for the treatment of RP, irrespective of gene mutation.

Within the trial, 18 patients with severe vision impairment due to RP were given a single intravitreal injection of MCO-010, while nine received a placebo. Results after a 12-month period showed vision function improvements after treatment with MCO-010, the data for which came from the Multi-Luminance Y-Mobility Test (MLYMT, vision-guided mobility). Other efficacy assessments included the Multi-Luminance Shape Discrimination test (MLSDT, near object recognition) and Best-Corrected Visual Acuity (BCVA).

In addition to the efficacy results, MCO-010 was well tolerated with no serious ocular or systemic adverse events.

Victor H Gonzalez MD, founder of Valley Retina Institute, McAllen, Texas and RESTORE investigator, stated: “I have had the privilege of observing substantial improvements in visual function in several patients who have enrolled in MCO-010 clinical trials over the past year. The fact that we see sustained and sometimes transformative gains in vision function, allowing them to walk in the clinic with more certainty, after a single treatment is remarkable and unprecedented. These participants who were living with severe vision impairment due to RP now have an improved quality of life, very different from before receiving MCO- 010. In addition, MCO-010’s favourable safety profile further strengthens my confidence in this groundbreaking treatment that I expect to become an important treatment option for people with advanced RP.”