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聽SPK-8011聽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聽SPK-8011.聽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聽SPK-8011聽administration. No serious adverse events have been observed to date. These data were presented by聽Lindsey A. George, M.D., attending physician in the聽Division of Hematology聽at Children鈥檚 Hospital of聽Philadelphia聽and principal investigator of the trial, at the 59th American Society聽of Hematology聽(ASH) Annual Meeting and Exposition in聽Atlanta.