Spark Therapeutics: Follow-up Luxturna data shows no significant change
Spark Therapeutics announced new three-year follow-up data from the continuing Phase 3 trial of LUXTURNA, an investigational, potential one-time gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease. These data were presented at the American Academy of Ophthalmology Retina Subspecialty Day by Albert M. Maguire, M.D., a principal investigator of the Phase 3 clinical trial, professor of ophthalmology at the Scheie Eye Institute at the University of Pennsylvania's Perelman School of Medicine and attending physician in the Division of Pediatric Ophthalmology at Children's Hospital of Philadelphia. "These data provide additional support information on the efficacy, durability and safety of investigational LUXTURNA, showing no statistically significant change in the primary endpoint from the initial gain in functional vision at the three-year time point for the original intervention group and at the two-year time point for the original control group after crossover," said Katherine High, M.D., president and head of Research and Development at Spark Therapeutics. "We believe the data from these participants further support the positive benefit-risk profile of investigational LUXTURNA for patients with vision loss due to confirmed biallelic RPE65-mediated IRD."