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Spark Lights Up On Cure For Blindness

Spark Lights Up On Cure For Blindness
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  • Shares of Spark Therapeutics Inc (NASDAQ: ONCE) opened higher by more than 40 percent on Monday.
  • Spark's product candidate, SPK-RPE65, targets a group of rare blinding conditions known as inherited retinal dystrophies.
  • Spark announced on Monday its SPK-RPE65 met the goals of a late-stage study.

Shares of Spark Therapeutics, a company that engaged in developing products in the field of gene therapy, surged higher by more than 40 percent Monday morning after the company announced that its product candidate (SPK-RPE65) met the goals of a late-stage study.

According to a company press release, "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."

The press release continued that there were "no serious adverse events" related to SPK-RPE65 or "deleterious immune responses" that were observed in the trial, Overall, adverse events were "consistent with observations in early studies."

"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."

Following the study, Spark intends to file a Biologics License Application with the US Food and Drug Administration in 2016 as the first step in executing its global regulatory and commercialization strategy.

CNBC commented on the data, noting that the therapy's results represents a "historic milestone."

A conference call was held Monday morning at 8:30 to further discuss the results. A replay will be available for one week and can be accessed through the firm's investor relations website.

Posted-In: Albert M. Maguire Blindness Spark Therapeutics SPK-RPE65Biotech Movers General Best of Benzinga


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