Spark Therapeutics Announces Results from Trial of SPK-RPE65 for Genetic Blinding Conditions

October 10, 2015

Spark Therapeutics today announced positive results from their Phase 3 pivotal trial for a gene therapy product looking to be used to treat RPE65-Mediated Inherited Retinal Dystrophies (IRDS).

The pivotal trial met its conclusion by demonstrating improvement of functional vision in the intervention group compared to the control group, as measured by the change in bilateral mobility testing between baseline and one year.

There were no serious adverse events related to SPK-RPE65 or deleterious immune responses observed in the trial. Overall, adverse events related to the administration procedure were consistent with observations in earlier studies of SPK-RPE65.

In addition, subjects who received SPK-RPE65 outperformed control subjects across the first two secondary endpoints: full-field light sensitivity threshold testing and the mobility test change score for the first injected eye. The third secondary endpoint, visual acuity, did not show statistically significant evidence of benefit. All reported p-values reflect results from the intent- to-treat (ITT) population, the most stringent efficacy analysis population described in the statistical analysis plan (SAP).

“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 SPK- RPE65 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.”

Based on these results, Spark intends to file a Biologics License Application with the U.S. Food and Drug Administration in 2016 as the first step in executing its global regulatory and commercialization strategy.



Read more on this story here.

About Spark Therapeutics

Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark’s initial focus is on treating rare diseases where no, or only palliative, therapies exist. Spark’s most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, recently reported positive top- line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark’s validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Spark builds on two decades of research, development and manufacturing at The Children’s Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more, please visit

Stay Connected

Sign up for updates straight to your inbox.