Today at the 2024 meeting of the American Society of Retina Specialists in Stockholm, Michael Singer, MD, presented late-breaking data on Nanoscope Therapeutics’ multicharacteristic opsin (MCO) optogenetics gene therapy platform (Figure 1). The 100-week top-line results he presented were from the successful phase 2b/3 RESTORE randomized sham-controlled clinical trial of MCO-010 in severe vision loss from advanced retinitis pigmentosa.
The RESTORE trial compared 2 doses of intravitreal MCO-010 in 18 patients with 9 sham-controlled patients, and Dr. Singer presented first-time data at the meeting. The primary endpoint was BVCA change at week 52 and the key secondary endpoint was BCVA change from baseline at week 76. Patients received oral steroid prophylaxis.
Primary and key secondary endpoints were met, with statistical significance observed over the course of the study. Of note, at week 52 40% of treated patients achieved a vision gain that was equivalent to 3 lines of 15 ETDRS letters. At 76 weeks, that number had increased to 56%. At week 100, 30% had maintained the 3-line improvement. There were no cases of retinitis, choroiditis, vasculitis, ischemic neuropathy, hypopyon, or hypotony. There were 12 cases of mild to moderate inflammation at the time of steroid tapering, and by week 100, 1 patient was still receiving topical treatment for intraocular inflammation. There will be an additional 3-year long-term follow-up study.
BCVA was assessed using the Freiburg visual acuity test, which has been clinically validated for low-vision patients. Dr. Singer explained during panel discussion that this scale helps measure clinically meaningful impact in this patient population.
“It is very exciting that there is a potential transformative gene therapy for the treatment of retinitis pigmentosa, regardless of genotype,” Dr. Singer told Retinal Physician. “It is also very encouraging to observe durable treatment effects after a single intravitreal injection presenting significant benefit to risk profile.”
RESTORE is a randomized, controlled trial of Nanoscope’s MCO optogenetic gene therapy in patients with severe vision loss related to retinitis pigmentosa. Optogenetic therapy uses a viral vector to deliver a transgene that makes bipolar cells light sensitive, allowing them to function as de facto photoreceptors.
