Phase III trial of SPK RPE65 (gene therapy product) in retinal dystrophies meets primary endpoint- Spark Therapeutics

Spark Therapeutics has announced positive top-line results from a Phase III pivotal trial of SPK RPE65 (gene therapy product) for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs). The Phase III trial met its primary endpoint, with patients receiving SPK RPE65 demonstrating improvement of functional vision in the intervention group compared to the control group, as measured by the change in bi-lateral mobility testing between baseline and one year. These results represent the first successful randomized, controlled Phase III trial completed in gene therapy for a genetic disease. Spark Therapeutics also announced today that it intends to file a Biologics License Application with the FDA in 2016.

IRDs are a group of rare blinding diseases that are caused by mutations in one of more than 250 genes, leading to visual impairment. These diseases include retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), both of which can cause progressive and total blindness.