An announcement today by Spark Therapeutics brings the world one step closer toward a commercially available gene therapy treatment. This morning Spark released positive results from the Phase 3 pivotal trial of its lead gene therapy product candidate, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies, more commonly known as LCA.
“We saw substantial restoration of vision in patients who were progressing toward complete blindness,” said Albert M. Maguire, MD, principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania. “The majority of the subjects given SPKRPE65 derived the maximum possible benefit that we could measure on the primary visual function test, and this impressive effect was confirmed by a parallel improvement in retinal sensitivity. If approved, SPK-RPE65 should have a positive, meaningful impact on the lives of patients with this debilitating condition.”
The phase 3 study evaluated 31 patients, 21 of whom were treated and 10 who represented controls. Patients treated with SPK-RPE65 were able to navigate through a mobility course more effectively at varying levels of light after treatment was provided. They also were found to have improved full-field light sensitivity threshold testing, which is a measure of physiologic function of the retina. In addition, there were no serious adverse events or concerning immune responses observed in the clinical trial. These results show evidence of success of the gene therapy treatment and
“These results are the culmination of more than a decade of work of many dedicated individuals to correct the underlying cause of RPE65-mediated blindness through the one-time administration of a gene therapy,” said Jean Bennett, MD, PhD, professor of ophthalmology and director of the Center for Advanced Retinal and Ocular Therapeutics at the Perelman School of Medicine of the University of Pennsylvania. “We are excited about the potential impact that the results will have on the treatment of this and other blinding conditions.”
Currently, there are no commercially available gene therapy treatments in the United States and only one in the Western world. These positive results bring Spark one step closer to approval for SPK-RPE65, which would also be the first gene therapy treatment for a retinal degenerative disease. Successful progress and approval of SPK-RPE65 bodes well for Choroideremia, currently in Phase 1/2 clinical trials by Spark. With the success and experience from the RPE65 program now established, the Choroideremia community hopes that their disease will follow a similar, if not simpler, path through the clinical trial and regulatory process.
For more information on Spark Therapeutics, you can navigate to their website at www.sparktx.com. In addition, patients can contact Spark directly at 800-SPARKTX or email@example.com for more information.