Spark Therapeutics Inc (ONCE) Tanks as Hemophilia A Drug Disappoints in Trial; J.P. Morgan Shares Two Cents


Spark Therapeutics Inc (NASDAQ:ONCE) tanked over 35% today after data from a Phase 1/2 study showed that patients suffering from hemophilia A may not benefit from the company’s gene therapy.

Spark presented additional Hemophilia A data on four patients treated with SPK-8011 at doses of 5E11vg/kg and 1E12vg/kg. Unfortunately, there was no difference in Factor VIII levels between patients given the low 5E11 dose, and the mid 1E12 dose. Moreover, there was a good amount of inter- and intra-patient variability in Factor expression.

J.P. Morgan Cory Kasimov commented, “Though admittedly early, we were quite underwhelmed by the results from ONCE’s update for SPK-8011 in Hemophilia A presented this morning at ASH (significant variability and lack of consistency in factor expression levels). We’ve argued in the past that gene therapy for hemophilia should not be considered a zero sum game, but this update clearly places BMRN in an even stronger leadership position. More patients, higher doses, and longer duration of follow-up will obviously be necessary to more clearly elucidate SPK-8011’s potential. With that said, following these mixed results, we suspect it will take much more substantial and compelling subsequent updates for investors to assign meaningful credit to this program. Given the severity of this morning’s selloff, combined with the co’s still novel gene therapy platform and progress on multiple fronts, our OW rating is unchanged.”

Katherine A. High, president and head of research and development at Spark, said, “While still early in the dose-escalation clinical trial of SPK-8011, we are encouraged these data show clinically significant reductions in ABR and AIR with no serious adverse events reported to date […] We look forward to continuing to enroll additional participants to further increase our understanding of how to optimize the potential efficacy of this investigational gene therapy.”

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