Pfizer Makes Investments in Gene Therapy to Expand Rare Disease Research

Christina Loguidice

On December 8, 2014, Pfizer announced two strategic decisions to expand the company’s rare disease research and development activities. First, it partnered with Spark Therapeutics for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bioengineered adeno-associated virus (AAV) vectors as a potential treatment of hemophilia B. The goal of the program is to bring an important investigational therapy to patients, and the SPK-FIX program is expected to enter phase 1/2 clinical trials in the first half of 2015. Second, Pfizer has appointed Michael Linden, PhD, professor at King’s College London and director of the University College London Gene Therapy Consortium, to lead gene therapy research in the rare disease area. Professor Linden will be with the company for a 2-year secondment.

The Spark Therapeutics Partnership

In the United States, hemophilia B affects approximately 4,000 male patients. Currently, treatment consists of recurrent intravenous infusions of plasma-derived or recombinant factor IX to prevent and control bleeding episodes. Spark’s proprietary, bioengineered AAV vectors were developed to deliver a high-activity factor IX gene to patients, enabling endogenous production of factor IX, with the potential to be effective for several years. 
“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,” said Mikael Dolsten, MD, PhD, Pfizer’s president of Worldwide Research and Development, in a Pfizer press release. “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,” he said.
Under the terms of the agreement with Spark, Pfizer will provide an upfront payment of $20 million to Spark and make additional development and commercialization milestone payments of up to $260 million for multiple hemophilia B product candidates that may be developed under the collaboration. In addition, Spark could receive double-digit royalties based on global product sales. Spark will conduct all phase 1/2 studies and Pfizer will be responsible for pivotal studies, any regulatory approvals, and potential global commercialization of the product. 
“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,” said Katherine High, MD, a hematologist and Spark’s co-founder, president, and chief scientific officer, in a Spark 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,” she added.

Adding Expertise to Pfizer’s Gene Therapy Research Team

Effective December 1, 2014, Professor Michael Linden has been part of Pfizer’s team to advance gene therapy research within the company’s rare disease research area. Professor Linden has made numerous contribitions to the field of gene therapy, including determining the biochemical and structural features of the AAV Rep proteins, which orchestrate all aspects of the AAV lifecycle, and proposing a mechanism of site-specific integration. He has also been actively engaged in translational projects aimed at developing treatments for Parkinson’s disease, spinal muscular atrophy, lysosomal diseases, and a variety of eye diseases using AAV vectors.
“The establishment of a gene therapy group under the leadership of Professor Linden will help Pfizer explore the potential of this important technology that could possibly benefit patients living with serious diseases,” said Kevin Lee, PhD, senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “Professor Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field,” he noted.
Image courtesy wikimedia commons.
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