Nanoscope Therapeutics Inc. announced a productive end-of-phase 2 (EOP2) meeting with the 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.
Key outcomes of the EOP2 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.
“Patients suffering from Stargardt macular degeneration face a substantial disease burden with no restorative treatments presently available,” said Sulagna Bhattacharya, co-founder and CEO of Nanoscope. “Following the positive results from the phase 2 STARLIGHT clinical trial, the outcome of this meeting strengthens our conviction to advance clinical development of MCO-010 in this indication. We appreciate the FDA’s guidance and look forward to finalizing preparation for the phase 3 trial.”
“The current paradigm for managing Stargardt disease is one of diagnosis, followed by vision rehabilitation and a lifetime sentence of declining vision to severe blindness. The advancement of Nanoscope’s groundbreaking therapy for Stargardt to a phase 3 registrational trial moves us much closer to having a viable restorative option for patients whose vision has been lost due to this terrible disease,” said Samuel Barone, MD, CMO
The planned phase 3 trial will be the first randomized, controlled gene therapy trial for Stargardt disease.
Nanoscope’s mutation-agnostic therapeutic approach has the potential to improve vision for many others suffering from severe vision loss, including macular degeneration patients.