The following is an excerpt from a press release from Spark Therapeutics. Read the full press release here.
Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer, announced, with a cumulative follow-up of more than 18 patient years of observation, all 15 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for severe or moderately severe hemophilia B, had discontinued routine infusions of factor IX concentrates. None of the 15 participants experienced serious adverse events, and there were no thrombotic events or factor IX inhibitors, as of the May 7, 2018 data cutoff. These data were presented by Spencer K. Sullivan, M.D., hematologist and clinical investigator, Mississippi Center for Advanced Medicine, at the World Federation of Hemophilia (WFH) World Congress in Glasgow, Scotland, during the “Free Papers: Gene Therapy” session.
“We are pleased to see all 15 participants, notably including the first four participants who have been followed for more than two years, continue to show that a single administration of SPK-9001 has resulted in dramatic reductions in bleeding and factor IX infusions, with no serious adverse events,” said Katherine A. High, M.D., president and head of research & development at Spark Therapeutics. “Our commitment to gene therapy research across our hemophilia programs remains steadfast with the goal of developing a novel therapeutic approach with a positive benefit-risk profile that aims to free patients of the need for regular infusions while eliminating spontaneous bleeding.”
Based on individual participant history for the year prior to the study, the overall ABR for all 15 participants was reduced by 98 percent (calculated based on data after week four; 97 percent based on data after infusion) to an annual rate of 0.2 bleeds per participant, compared to an annual rate of 8.9 bleeds before SPK-9001 administration. Only one participant experienced a bleeding event four or more weeks after SPK-9001 infusion.
Read the press release in its entirety here.