September 11, 2015
By Alex Keown, BioSpace.com Breaking News Staff
PHILADELPHIA – Gene therapy company Spark Therapeutics is looking to calm investors in anticipation of results for its Phase III clinical trial of its lead program, SPK-RPE65, for the treatment of RPE65-mediated inherited retinal dystrophies after some late-stage changes were announced, the Street reported this morning.
During a conference call on Sept. 9, Spark’s executives sought to quell any investor concerns, saying that although some changes were made, the primary endpoints of the trial remain the same. On Sept. 8, Spark Therapeutics announced a database lock for the trial and also announced the modifications, including designation of pupillary light reflex as an exploratory endpoint and the analysis of the mobility test change score for an assigned first eye as a secondary endpoint. That change resulted in three secondary endpoints – full-field light sensitivity threshold testing, the first eye mobility test change score, and visual acuity – which will be tested hierarchically in that order.
Since the announcement of the additions to the study, investors got nervous and began selling off the . Since Sept. 8’s high of $47 per share, the stock had seen a steady decline. Spark’s stock was moving in both directions this morning, hitting a high of $44.21 per share, before dropping to $43.46 per share, falling past its opening price of $44.03 per share.
Following the stock’s decline, executives reinforced in the conference call that the additional data being sought in the late-stage trial would provide a “stronger package of clinical data to eventually present to regulatory agencies,” the Street said in its report, which is something the company said when it announced the changes.
Jeffrey D. Marrazzo, president and co-founder of Spark Therapeutics, said the increased information being sought would only benefit the trial when it went before regulatory bodies, including the U.S. Food and Drug Administration (FDA).
Spark said it anticipates final results of the SPK-RPE65 trial in October and plan to submit a Biologics Licensing Application to the FDA in 2016. SPK-RPE65 has orphan designation in the United States (U.S.) and the European Union, and breakthrough therapy designation in the U.S.
“We are leveraging SPK-RPE65 to address a broad spectrum of IRDs (inherited retinal dystrophies),” the company said on its website.
SPK-RPE65 has the potential to address IRDs caused by mutations in the RPE65 gene, for which there currently are no pharmacologic treatments, Spark said on its website. Mutations in the RPE65 gene are linked to subtypes of Leber’s congenital amaurosis (LCA type 2) and retinitis pigmentosa (RP type 20). The company said it estimates there are approximately 3,500 individuals in the United States and five major European markets with these forms of IRDs.
On Aug. 5, the company reported its earnings ratings at .60 per share, missing the mark set by analysts. Still, analysts at J.P. Morgan Chase & Co. reiterated their rating of “buy” for Spark’s stock.
