Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here.
Spark Therapeutics announced on Dec. 11, 2017, it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post infusion, have reduced their overall annualized bleeding rate (ABR), calculated based on data after week four, by 100 percent (calculated based on data after week four; 82 percent based on data after infusion) to a mean of 0 (1) annualized bleeds as of the data cutoff, compared to a mean of 5.5 annualized bleeds before a single administration of SPK-8011. Similarly, their overall annualized infusion rate (AIR) was reduced approximately 98 percent (calculated based on data after week four; 96 percent based on data after infusion) to a mean of 1.2 (2.5) annualized infusions as of the data cutoff, compared to a mean of 57.8 annualized infusions before SPK-8011 administration. No serious adverse events have been observed to date. These data were presented by Lindsey A. George, M.D., attending physician in the Division of Hematology at Children’s Hospital of Philadelphia and principal investigator of the trial, at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta.