GenSight Biologics, a biopharma company that discovers and develops innovative gene therapies for neurodegenerative retinal diseases and diseases of the central nervous system, announced that enrollment in REVERSE, a phase III clinical trial of GS010 in the treatment of Leber’s Hereditary Optic Neuropathy (LHON), has been successfully completed.
REVERSE is the first of two parallel randomized, double-masked, sham-controlled, multi-center pivotal phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene. REVERSE enrolled 36 patients with an onset of vision loss of 7-12 months, while RESCUE, the second trial, is currently enrolling 36 patients with an onset of vision loss of less than six months. Both studies are being conducted in seven centers in Europe and in the U.S.
“This is an exciting time for patients and physicians, who are waiting for a safe and efficient treatment for LHON. We are encouraged by the safety and pharmacodynamics results seen in the phase I/II study, and are now only a year away from a potential transformative treatment for LHON, should the results be positive,” commented Pr. Nancy J. Newman, M.D., investigator of the study and director of the Section of Neuro-Ophthalmology, Emory University School of Medicine, Atlanta, Georgia (USA).
Topline results of REVERSE at 48 weeks are expected at the end of Q1 2018.
RESCUE is expected to complete enrollment by the end of H1 2017.
GS010 has been granted Orphan Drug Designation both in the U.S. and in Europe.