PARIS–(BUSINESS WIRE)–Regulatory News:
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that enrollment in REFLECT, a Phase III clinical trial of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON), was successfully completed ahead of schedule.
REFLECT is a multi-center, randomized, double-masked, placebo-controlled study to evaluate the efficacy and safety of bilateral injections of GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4) mutation. Enrolling the target number of 90 subjects was originally anticipated to be completed in September 2019; instead the 98th subject enrolled in the trial was treated on July 2.
“The conclusion of enrollment for the REFLECT study is a milestone in the story of gene therapy for Leber Hereditary Optic Neuropathy,” commented Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology, Emory University School of Medicine, Atlanta, USA, and Principal Investigator in REFLECT. “The recruitment of nearly 100 patients in less than two years is a tribute to the support of the LHON community in this partnership among researchers, clinicians and patients trying to bring therapy and hope to this blinding disease.”
The trial was designed and agreed under a Special Protocol Assessment (SPA) with the Food and Drug Administration (FDA) in the United States but is not required for the Marketing Authorization Application (MAA) in the European Union, RESCUE and REVERSE being considered by the EMA as pivotal for filing in the EU. The trial enrolled subjects with vision loss up to 1 year in duration and is underway across multiple centers in the United States, Europe, and Taiwan. In the active arm, GS010 was administered as a single intravitreal injection to both eyes of each subject. In the placebo arm, GS010 was administered as a single intravitreal injection to the first affected eye, while the fellow eye received a placebo injection.
The primary endpoint for the REFLECT trial is the best corrected visual acuity (BCVA) change from baseline reported in LogMAR at 52 weeks post-treatment in the second affected/not yet affected eye. Secondary efficacy endpoints include: BCVA reported in LogMAR at 2-years post-treatment in the second affected/not yet affected eye compared to both placebo and the first affected eye receiving GS010, OCT, contrast sensitivity and quality of life.
The first subject was treated in March 2018; topline Week 52 results are expected to be available in the third quarter of 2020.
GS010 has Orphan Drug Designation both in the United States and in Europe.