Spark Therapeutics Announces $15 Million Milestone Payment from Pfizer for Progress in Hemophilia B Gene Therapy Program

Second milestone achieved under 2014 global license agreement with Pfizer

PHILADELPHIA, Jan. 04, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that it has earned a $15 million payment from Pfizer Inc. (NYSE:PFE) for achieving a pre-specified safety and efficacy profile development milestone in the ongoing hemophilia B Phase 1/2 trial of investigational SPK-9001. SPK-9001 has received breakthrough therapy and orphan product designations from the U.S. Food and Drug Administration.

"We continue to make strong, tangible progress with our hemophilia pipeline, and achievement of this second milestone marks further advancement in the development of our investigational gene therapy for hemophilia B," said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. "We look forward to reporting additional data as we continue to document the clinical experience with SPK-9001."
Under the terms of the license agreement with Pfizer, Spark Therapeutics received a $20 million upfront payment upon entering into the agreement in 2014, and a $15 million milestone payment in December 2015 for progress with the development program. The company is eligible to receive up to an additional $230 million in aggregate for achieving future development and commercial milestones, as well as royalties calculated as a low-teen percentage of net sales on any potential SPK-FIX products. Spark Therapeutics maintains responsibility for the clinical development of SPK-FIX product candidates through the completion of Phase 1/2 trials. Thereafter, Pfizer has responsibility for further clinical development, achieving regulatory approvals and potential global commercialization.
The SPK-FIX program leverages a more than two-decade long history of hemophilia gene therapy research and clinical development conducted by Spark Therapeutics and its founding scientific team. SPK-9001 is a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant enabling endogenous production of factor IX.