Christine Kay, MD, on the 152-week results of the RESTORE study for retinitis pigmentosa

Christine Kay, MD, a vitreoretinal surgeon and IRD specialist from Gainesville, Florida, presented the results of the RESTORE trial for MCO-010 optogenetic therapy for retinitis pigmentosa. The trial was a 152-week analysis of a novel gene therapy targeting bipolar cells.

The study involved 27 patients with advanced retinitis pigmentosa and severe vision loss, randomized into low dose, high dose, and sham arms. Patients received a single intravitreal injection with an oral corticosteroid taper. The therapy used an AAV2 delivery method with an mGluR6 promoter. The trial successfully met its primary endpoint, with approximately 40% of active treatment arms gaining 0.3 logMAR (equivalent to three lines of visual acuity). Both high and low dose groups showed statistically significant improvements compared to the sham arm. The key secondary endpoint demonstrated that visual acuity improvements remained stable for up to two and a half years. Safety was a crucial aspect of the study. The most common adverse events were anterior chamber cell and ocular hypertension, primarily occurring during injection or steroid tapering. Most patients were able to be tapered off steroids within one year, with no serious adverse events reported. The therapy proved to be gene-agnostic, targeting patients with severe vision loss (20/200 or below). Some patients experienced remarkable improvements, with super responders achieving visual acuity up to 20/50. The therapy was particularly meaningful for patients with hand motion or lower visual acuity, offering hope for those with limited treatment options. Kay emphasized the clinical significance of the results, noting that patients with this level of vision typically do not experience spontaneous improvement. The 0.3 logMAR improvement was considered clinically meaningful, exceeding natural history variability and meeting FDA criteria for vision improvement. Based on these promising results, Nanoscope initiated a rolling BLA submission with the FDA. The study highlighted the potential of optogenetic therapy as a groundbreaking treatment for patients with retinitis pigmentosa, offering a new avenue of hope for those with severe vision loss.