Article source: EyeWorld.org
Nanoscope Therapeutics announced publication of positive results from its Phase 2 trial online in the journal eClinical Medicine ahead of print in The Lancet. The trial, according to the company, is the “first and only clinical trial to demonstrate vision improvements in patients with Stargardt disease.” The open-label study assessed safety and efficacy of MCO-010 (sonpiretigene isteparvovec) in 6 participants with severe vision loss due to the disease. In all patients there was a mean improvement of +5.5 and +9.0 ETDRS letters without and with a wearable low-vision aid, respectively, the company reported. For patients who had atrophy confined to the macula, there was a mean BCVA improvement at week 48 of +12.0 and +32.0 ETDRS letters, without and with a wearable low-vision aid, respectively. There were no serious adverse events during the 48-week study period. The company plans to begin a Phase 3 trial by the end of this year with topline results expected in 2027. MCO-010 is described as “a one-time, in-office intravitreal gene-agnostic therapy designed to restore vision in patients with advanced retinal degeneration.”
