Pfizer seeks to expand rare disease research

Pfizer will collaborate with Spark Therapeutics, and has also appointed Prof. Michael Linden of King's College London to lead gene therapy research in rare diseases at Pfizer

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LONDON—Rare disease are one of many areas of focus for pharma giant Pfizer Inc., which has a portfolio of 22 medicines approved globally for the treatment of rare diseases in hematology, neuroscience, inherited metabolic disorders, pulmonology and oncology. And in hopes of expanding its footprint in that area, the company has announced two undertakings to that end.
To begin with, Pfizer has established an agreement with Spark Therapeutics, a Philadelphia-based company that develops gene therapies for genetic diseases. Spark and Pfizer will develop SPK-FIX, a program that incorporates a bio-engineered AAV vector for the potential treatment of hemophilia B. Spark notes that its “proprietary, bioengineered vectors are designed to deliver a high-activity Factor IX gene to patients, enabling endogenous production of Factor IX, with the potential to be effective for a number of years.”
“Pfizer strives to provide meaningful enhancements to the lives of patients with hemophilia, and the agreement with Spark Therapeutics offers an important expansion of Pfizer’s commitment to the bleeding disorder community and builds on our leading hemophilia portfolio,” noted Geno Germano, group president, Global Innovative Pharma Business at Pfizer. “We believe the SPK-FIX program could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders.”
Under this agreement, Pfizer will pay Spark $20 million up front, and the latter will also be eligible for additional development and commercialization milestone payments of up to $260 million for multiple hemophilia B product candidates that might be developed under the collaboration. Spark is also eligible to receive double-digit royalties on global product sales. Spark will be responsible for clinical development of the program through Phase 1/2 studies, after which Pfizer will assume responsibility for pivotal studies, regulatory approvals and potential global commercialization of the product. The program is expected to begin Phase 1/2 clinical trials in the first half of 2015.
“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,” Dr. Katherine High, co-founder, president and chief scientific officer at Spark, said in a press release. “Pfizer's longtime experience in hemophilia, including strong relationships with physicians, patients and payors, as well as clinical, regulatory and commercial capabilities, will complement our team's deep knowledge of AAV-mediated gene transfer for the disease. We look forward to working with Pfizer with the goal of making gene therapy for hemophilia B a reality for patients.”
Pfizer also announced its appointment of Michael Linden, Ph.D., professor at King’s College London and director of the University College London Gene Therapy Consortium; that appointment was effective December 1. Linden will be with Pfizer for a two-year secondment to lead gene therapy research in the area of rare diseases.
“The establishment of a gene therapy group under the leadership of Prof. Linden will help Pfizer explore the potential of this important technology that could possibly benefit patients living with serious diseases,” said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “Prof. Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field.”
“The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients,” added Dr. Mikael Dolsten, president of Worldwide Research and Development at Pfizer. “By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating hematologic and neuromuscular diseases.”

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