Nanoscope Therapeutics Inc. is poised to initiate a Phase 3 clinical trial for MCO-010, a gene therapy aimed at treating severe vision loss caused by Stargardt Macular Degeneration (SMD). This decision follows a successful End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), providing a pathway for potential restorative treatment for this inherited disease affecting children and young adults.
FDA Guidance on Phase 3 Trial Design
The FDA provided key guidance on the design of the Phase 3 registrational trial. The study will involve a single intravitreal dose of MCO-010, with participants randomized 1:1 to either the treatment group or a control group receiving a sham injection. A crucial aspect of the trial will be the assessment of changes in best-corrected visual acuity (BCVA) using the ETDRS chart, serving as the primary efficacy endpoint, measuring the change from baseline to Week 52.
Statistical Methods and Secondary Endpoints
Valuable input was also provided by the FDA regarding statistical methods for both primary and secondary endpoints. Nanoscope intends to further engage with the FDA to align on statistical analyses. The multi-luminance shape discrimination test is under consideration for further evaluation as a key secondary endpoint.
Expanding Patient Enrollment
A significant outcome of the EOP2 meeting was the alignment on enrolling legally blind patients with vision worse than 20/200, and as young as 12 years old. This expands the patient population beyond that evaluated in the Phase 2 STARLIGHT clinical trial (NCT05417126). The FDA also confirmed that the existing nonclinical data package is adequate to support a future Biologics License Application (BLA) submission.
Addressing Unmet Needs in Stargardt Disease
Stargardt disease, often referred to as juvenile macular dystrophy, is a leading cause of vision loss in children and young adults. It is characterized by the degradation of photoreceptor cells in the macula, leading to a loss of central vision. Currently, there are no approved treatments for Stargardt disease, and management primarily involves diagnosis and vision rehabilitation.
MCO-010: A Mutation-Agnostic Approach
MCO-010 is a gene-agnostic optogenetic therapy being developed for various retinal degenerative diseases. It has already received FDA fast track designation and orphan drug designation for both Retinitis Pigmentosa (RP) and Stargardt disease. Positive results from the Phase 2 STARLIGHT trial (NCT05417126) have demonstrated the potential of MCO-010 to improve vision in patients with Stargardt disease.
Future Directions
Nanoscope's planned Phase 3 trial will be the first randomized, controlled gene therapy trial for Stargardt disease, marking a significant step forward in addressing this unmet medical need. The company's mutation-agnostic therapeutic approach holds promise for improving vision in individuals with severe vision loss, including those with macular degeneration.
