Spark Therapeutics has earned a $15 million milestone payment from Pfizer for achieving a pre-specified safety and efficacy profile in the ongoing hemophilia B Phase 1/2 trial of SPK-9001. SPK-9001 has received breakthrough therapy and orphan product designations from the U.S. FDA.
 
Spark Therapeutics received $20 million upfront under the agreement initiated in 2014, and a $15 million milestone in December 2015 for development progress. The company is eligible to receive as much as an additional $230 million for future development and commercial milestones, as well as royalties on sales. Spark is responsible for clinical development of SPK-FIX product candidates through Phase 1/2 trials and Pfizer has responsibility for further clinical development, achieving regulatory approvals and potential global commercialization.
 
“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.”