Nanoscope Therapeutics has announced the expected advancement of MCO-010 for the treatment of Stargardt Macular Degeneration (SMD) to a Phase 3 registrational trial following a productive end-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA).
Nanoscope Therapeutics has said that 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, randomised 1:1 to a control cohort receiving a sham injection.
“Patients suffering from Stargardt Macular Degeneration face a substantial disease burden with no restorative treatments presently available,” said Sulagna Bhattacharya, Co-founder and Chief Executive Officer 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 finalising 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,” added Samuel Barone, M.D., Chief Medical Officer.
“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.”
The planned Phase 3 trial will be the first randomised, 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.
