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Spark Therapeutics Announces Presentation of Additional Phase 3 and Durability Data on SPK-RPE65 at The Retina Society 48th Annual Scientific Meeting

Rapid and Sustained Difference Observed Between Intervention and Control Subjects Across Multiple Parameters Throughout Entire Phase 3 Study Period

Durable Effect Seen Over Three Years, With Observation Ongoing, in Functional Vision and Light Sensitivity in Phase 1 Trial

PARIS, Oct. 10, 2015 (GLOBE NEWSWIRE) -- Spark Therapeutics, Inc. (ONCE) announced today the presentation of additional data from the Phase 3 pivotal trial as well as continued durability data from an earlier Phase 1 study of SPK-RPE65, its lead product candidate for the treatment of RPE65-mediated inherited retinal dystrophies (IRDs).

Principal Investigator Stephen R. Russell, MD, of the Stephen A. Wynn Institute for Vision Research at the University of Iowa, presented Phase 3 data highlighting the rate, breadth and magnitude of changes seen across the primary endpoint and first secondary endpoint following administration of SPK-RPE65. In addition, Dr. Russell presented data on the three-year durability of improvements in the same measures of functional vision and light sensitivity in a cohort of subjects from an earlier Phase 1 trial.

Today’s presentation builds on top-line results of a randomized controlled multi-center Phase 3 trial announced by Spark on October 5, which demonstrated a highly statistically significant improvement in the intervention group compared to the control group in the primary endpoint and two of three secondary endpoints. There were no serious adverse events related to SPK-RPE65 in the Phase 3 trial.

Significant Improvements and Strong Parallels in Mobility and Light Sensitivity Testing

Data presented today highlighted a mean improvement in the functional vision of intervention subjects (n=20) of 1.9 specified lux levels, compared with an improvement of 0.2 specified lux levels in control subjects (n=9) as measured by the change in bilateral mobility testing (MT) between baseline and one year in the modified intent-to-treat (mITT) population. The mITT population (n=29) includes all subjects that received SPK-RPR65, and only those who continued beyond the baseline study visit. Two subjects in the intent-to-treat (ITT) population (n=31) that were randomized but never received SPK-RPE65 are excluded from this efficacy analysis population. Thirteen of the 20 subjects receiving SPK-RPE65 were able to pass the MT at one lux at year one, demonstrating maximum improvement measurable on the MT score. None of the nine control subjects followed was able to pass MT at one lux at year one.

In a corresponding finding in the first secondary efficacy endpoint, full-field light sensitivity threshold testing (FST) for white light, intervention subjects demonstrated a highly statistically significant mean improvement of -2.06 log10 (candela second/m2) compared with decline of 0.04 log10 (candela second/m2) among control subjects (all analyses in mITT population).

“It’s exciting to see a consistency of improvement between the functional vision and visual function. The parallel effect seen in the prompt response in both the primary and first secondary endpoints highlights a critically important finding from the trial: that functional improvements measured through the mobility test...