Nanoscope Planning Phase 3 Clinical Trial of its Optogenetic Therapy for People with Advanced Stargardt Disease

Nanoscope Therapeutics has announced plans to initiate a Phase 3 clinical trial of its MCO-010 optogenetic therapy for people with advanced Stargardt disease. The decision comes after an End-of-Phase 2 meeting with the US Food & Drug Administration (FDA) for its six-patient Phase 2 Stargardt disease trial.

Nanoscope plans to begin site selection for the Stargardt disease Phase 3 trial in Q4 2024 and patient dosing in Q1 2025. The FDA confirmed that change in best corrected visual acuity (BCVA) is an appropriate primary outcome measure for the Phase 3 trial. The company plans to enroll legally blind patients as young as 12 who have BCVA worse than 20/200 and mutations in ABCA4 or other genes causing Stargardt-disease-like conditions. Patients will be randomized to receive either MCO-010 or placebo.

Nanoscope previously reported plans to seek FDA approval for MCO-010 for treating people with advanced retinitis pigmentosa. The company also has a Phase 2 clinical trial underway of MCO-010 for people with geographic atrophy (advanced dry macular degeneration).

MCO-010 is designed for people who have lost most or all of their photoreceptors, the cells that make vision possible. The treatment, a small drop of liquid delivered by an intravitreal injection, uses a human-engineered virus to deliver copies of a Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease. The company says that MCO-010 is enabling the recipient’s bipolar cells to sense ambient light, thereby working as a backup system for lost photoreceptors. The approach doesn’t use goggles or eyewear to enhance the visual information coming into the retinas. It’s also designed to work regardless of the mutated gene causing the retinal disease.