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Why Spark Therapeutics Is Up Over 50%

Spark Therapeutics Inc. (NASDAQ: ONCE) made history Monday morning when it announced successful Phase 3 results in gene therapy for a genetic disease. Basically, the company announced positive top-line 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.

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

As a side note to the positive results, 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.

Albert M. Maguire, M.D., principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania, said:

We saw substantial restoration of vision in patients who were progressing toward complete blindness. 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.

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Kathy High, M.D., co-founder, president and chief scientific officer of Spark, also commented:

This is an important moment for the field of gene therapy, and demonstrates Spark’s ability to carefully and precisely integrate technologies and approaches across a range of disciplines to move the concept of gene therapy toward a therapeutic reality for patients. We wish to thank the trial participants and families as well as the investigators and all who contributed to this groundbreaking trial and successful result.

Shares of Spark closed Friday up 3.7%, at $43.93 in its 52-week trading range of $36.96 to $79.50. In early trading indications on Monday, shares were up 59% at $70.00. The stock has a consensus analyst price target of $74.33.

By Chris Lange


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