December 8, 2014
By Jessica Wilson, BioSpace.com Breaking News Staff
Spark Therapeutics, a Philadelphia-based gene therapy company, announced this weekend that it has entered into a partnership with Pfizer to develop and commercialize SPK-FIX, a potential treatment for hemophilia B.
Under the terms of the deal, Pfizer will pay Spark an upfront fee of $20 million. Additional milestone payments to Spark could total $260 million for the development of multiple hemophilia B products.
Spark will fund the Phase 1/2 studies of SPK-FIX, while Pfizer will pay for pivotal studies, regulatory approvals and global commercialization of the products. In addition, Spark is entitled to receive double-digit royalties based on global product sales.
“We are excited to announce our collaboration with Pfizer, as we believe it marks an important step towards bringing a potentially life-altering therapeutic to patients with hemophilia B,” said Jeffrey Marrazzo, co-founder and chief executive officer of Spark, in a statement.
A rare inherited hematologic disorder, hemophilia B, is characterized by a mutation in the Factor IX, or FIX, gene, which leads to deficient blood coagulation and an increased risk of hemorrhaging.
Working at the Children’s Hospital of Philadelphia, Spark and its founding scientific team have spent two decades researching and developing the components on which the company’s integrated gene therapy platform is built. Spark’s gene therapy platform, SPK-FIX, uses bio-engineered adeno-associated virus (AAV) vectors to deliver a high-activity Factor IX gene to the liver. The safety and proof-of-concept in expressing Factor IX in the liver have been demonstrated in prior clinical trials conducted by Spark’s scientific team.
Katherine High, co-founder, president and chief scientific officer of Spark, pioneered the development of AAV-mediated gene therapy for hemophilia.
“We believe Pfizer is the ideal partner for our hemophilia B program. Pfizer is a leader in hemophilia, developing the first recombinant Factor IX product,” High said in a statement. She also noted Pfizer’s “longtime experience in hemophilia,” its “strong relationships with physicians,” and its “clinical, regulatory and commercial capabilities” as factors in Spark’s decision to form the partnership.
Pfizer also emphasized the synergy between the two companies. “With their experience in the field of gene therapy…we believe that Spark’s team of scientists and clinicians will complement Pfizer‘s expertise in helping to bring a new therapy to patients,” said Kevin Lee, senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “Spark‘s hemophilia B program has the potential to build on our leading hemophilia portfolio and could offer patients with this bleeding disorder a potential new treatment option,” he concluded.
