By Estel Grace Masangkay

Pfizer announced two major moves that will expand its rare disease R&D activities. First, the company said it will establish a gene therapy platform through its newly inked agreement with Spark Therapeutics. Secondly, it has appointed Dr. Michael Linden, Director of the University College London Gene Therapy Consortium, as head of Pfizers gene therapy research in rare diseases area.

Late-stage gene therapy firm Spark Therapeutics will collaborate with Pfizer to develop SPK-FIX, a program incorporating a bio-engineered AAV vector as potential treatment for Hemophilia B. SPK-FIX is set to begin Phase 1/2 clinical trials in the first half of 2015. Under the terms of the agreement, Spark Therapeutics will stay at the helm of the products Phase 1/2 clinical development studies. Pfizer will take over for pivotal studies, regulatory approvals, and potential commercialization of SPK-FIX worldwide. Pfizer will pay Spark an upfront fee of $20 million with up to $260 million in milestone payments for several hemophilia B product leads that may result from the collaboration. Spark will also be eligible to receive royalties based on global sales of the resulting products.

Geno Germano, group president of Global Innovative Pharma Business at Pfizer, said, 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.

We are excited to announce our collaboration with Pfizer, as we believe it marks an important step towards bringing a potentially life-altering therapeutic to patients with hemophilia B, said Jeffrey D. Marrazzo, co-founder and CEO of Spark. The company recently announced that it has received Breakthrough Therapy Designation from the FDA for its lead drug candidate SPK-RPE65 as treatment for nyctalopia in certain patients.

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Pfizer Inks Gene Therapy Partnership With Spark Therapeutics