Nanoscope Plans Phase 3 Trial of MCO-010 to Treat Stargardt Macular Degeneration

Nanoscope Therapeutics announced the completion of an End-of-phase 2 (EOP2) meeting with the FDA for its clinical program evaluating MCO-010 for the treatment of severe vision loss caused by Stargardt macular degeneration (SMD), supporting advancement of MCO-010 to a phase 3 registrational trial.

As outlined by the company, key outcomes of the EOP2 meeting with the FDA included the following:

• 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 age 12 years, 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 a randomized, controlled gene therapy trial for Stargardt disease using Nanoscope’s mutation-agnostic therapeutic approach, stated the company.