Date: 2017-01-04
Type of information: Milestone
Compound: SPK-FIX
Company: Pfizer (USA - NY) Spark Therapeutics (USA - PA)
Therapeutic area: Hematological diseases - Genetic diseases - Rare diseases
Type agreement: development
Action mechanism: gene therapy. Hemophilia B is a serious and rare inherited hematologic disorder, characterized by a mutation in the Factor IX, or FIX, gene, which leads to deficient blood coagulation and an increased risk of bleeding or hemorrhaging. Spark’s development program for hemophilia B, SPK-FIX, is advancing proprietary, bioengineered adeno-associated virus (AAV) vectors that deliver a high-activity Factor IX gene to the liver. SPK-FIX leverages a long track record of hemophilia B gene therapy research conducted by Spark and its founding scientific team which, in prior clinical trials, has demonstrated safety and proof-of-concept in expressing Factor IX in the liver.
Disease: hemophilia B
Details: * On December 8, 2014, Pfizer announced two strategic decisions to expand the company’s rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of Hemophilia B expected to enter Phase 1/2 clinical trials in the first half of 2015. Spark Therapeutics and Pfizer will collaborate to progress the clinical program for SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B. Under the terms of the agreement, Spark will maintain responsibility for clinical development through Phase 1/2 studies. Pfizer will assume responsibility for pivotal studies, any regulatory approvals and potential global commercialization of the product.
Financial terms: Under the terms of the collaboration agreement, Spark received a $20 million upfront payment and is eligible to receive up to $245 million in aggregate additional development and commercial milestones, as well as royalties calculated as a low-teen percentage of net product sales. Spark 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, any regulatory approvals and potential commercialization.
Latest news: * On January 4, 2017, Spark Therapeutics announced that it has earned a $15 million payment from Pfizer 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 FDA. Under the terms of the license agreement with Pfizer, Spark Therapeutics 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. * On December 9, 2015, Spark Therapeutics announced that it has earned a $15 million milestone payment from Pfizer under the companies’ global collaboration for the potential development and commercialization of SPK-FIX product candidates for the treatment of hemophilia B. This is the first milestone achieved under the agreement that was entered into in December 2014.
