Stargardt Macular Degeneration: First randomized, controlled gene therapy trial planned
Nanoscope Therapeutics announced a productive End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for its clinical program evaluating MCO-010 for the treatment of severe vision loss due to Stargardt Macular Degeneration (SMD), supporting advancement of MCO-010 to a Phase 3 registrational trial. The MCO-010 gene therapy utilizes an intraocular injection for delivery of a gene that encodes for the ambient light-sensitive MCO protein into retinal cells.
Key outcomes of the End-of-Phase 2 meeting discussion included:
- The FDA provided clear input on the proposed Phase 3 registrational trial in patients with SMD intended to support safety and effectiveness of MCO-010. The proposed study design will consist of a single MCO-010 intravitreal dose group, randomized 1:1 to a control cohort receiving a sham injection.
- The FDA confirmed that a change in best corrected visual acuity (BCVA) that employs testing on the ETDRS Chart is appropriate for use in a primary efficacy endpoint assessing change from baseline to Week 52 in the proposed Phase 3 trial.
- The FDA provided valuable input on statistical methods for the proposed primary and secondary endpoints that will inform further engagement with FDA to align on statistical analyses.
- The FDA offered continued discussion and assistance to plan further evaluation of the multi-luminance shape discrimination test to support use as a key secondary endpoint.
- Alignment was achieved on the enrollment of legally blind patients with vision worse than 20/200, and as young as 12 years old, for the upcoming study, extending the population evaluated from that studied in Phase 2.
- The FDA agreed on the sufficiency of the current nonclinical package to support a future BLA submission.
The planned Phase 3 trial will be the first randomized, controlled gene therapy trial for Stargardt disease.
Nanoscope Therapeutics is developing optogenetic gene therapies using Multi-Characteristic Opsin (MCO) to re-sensitize the retina to detect low light levels. These therapies have the potential to restore vision in millions of visually impaired individuals suffering from retinal degenerative diseases, including Retinitis Pigmentosa, Stargardt Disease and Dry Age-related Macular Degeneration (AMD).
The MCO-010 gene therapy utilizes a convenient and well-established intraocular injection for delivery of a gene that encodes for the ambient light-sensitive MCO protein into retinal cells. It is in clinical development for retinitis pigmentosa (RP) and Stargardt disease. MCO-010 has been granted Orphan Drug and Fast Track designation by the US Food and Drug Administration for both diseases.
MCO-010 recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for Retinitis Pigmentosa (RP) (NCT04945772). The company has also completed the STARLIGHT Phase 2 trial of MCO-010 in Stargardt disease patients (NCT05417126).
Source: Nanoscope Therapeutics