Pfizer Amends Gene-Therapy PactBy
Pfizer and Spark Therapeutics, a Philadelphia, Pennsylvania-headquartered biopharmaceutical company, have amended their license agreement for SPK-9001, an investigational gene therapy for hemophilia B.
In December 2014, Pfizer signed an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered adeno-associated virus (AAV) vector for the potential treatment of hemophilia B. In 2016, the companies were granted breakthrough therapy designation by the US Food and Drug Administration for SPK-9001, the companies’ lead investigational candidate for hemophilia B.
Spark Therapeutics will enroll up to five additional participants in the current Phase I/II clinical trial who will receive SPK-9001 manufactured using an enhanced process to test its comparability to the SPK-9001 received by the first 10 participants enrolled in the ongoing trial.
Spark Therapeutics is transferring the enhanced SPK-9001 manufacturing process to Pfizer, which intends to use material generated with this process in the Phase III clinical trial of SPK-9001. The activities outlined in the license agreement as amended will occur in parallel to Pfizer’s ongoing preparation to assume responsibility for SPK-9001 after the transfer of the investigational new drug application to Pfizer.
Subject to the amendment, Spark Therapeutics will receive from Pfizer an initial $10-million cash payment and up to an additional $15 million in potential milestone payments upon completion of certain transition activities.