Spark Therapeutics, Pfizer to develop new treatment paradigm for hemophilia B

Both the firms will jointly work on a worldwide basis with the aim of bringing an important investigational therapy to patients.

Pfizer senior vice president and chief scientific officer Rare Disease Research Unit Kevin Lee said: "With their experience in the field of gene therapy, as well as in the research and development of potential novel treatments for hemophilia, we believe that Spark’s team of scientists and clinicians will complement Pfizer’s expertise in helping to bring a new therapy to patients.

"This agreement reinforces Pfizer’s longstanding commitment to the hemophilia community. 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."

As part of the deal, Spark will receive an upfront payment of $20m and will be eligible for additional development and commercialization milestone payments of about $260m for multiple hemophilia B product candidates that may be developed under the collaboration.

In addition, Spark will be responsible for conducting all Phase I/II trials while Pfizer will assume responsibility for pivotal studies, any regulatory approvals and potential global commercialization of the product.

Based on global product sales, Spark is also entitled to receive double-digit royalties.

Spark’s bioengineered vectors are designed to deliver a high-activity Factor IX gene to patients, allowing endogenous production of Factor IX, with the potential to be effective for a number of years.