The poster child for gene therapy and beating pole for short sellers, Spark Therapeutics, just kicked all of its haters in the nuts.
Spark Therapeutics announces positive top-line results from the Phase 3 pivotal trial of SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies; primary and first two secondary endpoints met (43.93) The pivotal trial met its primary endpoint (p = 0.001), 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 (p < 0.001) and the mobility test change score for the first injected eye (p = 0.001). The third secondary endpoint, visual acuity, did not show statistically significant evidence of benefit (p = 0.17). 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). 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.
Let there be light. Shares of ONCE are now up 67% in pre-market trading.
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I have this one in my “should have” portfolio.
Yikes.