May 11, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Pfizer announced inked a global collaboration and license deal with Richmond, Calif.-based Sangamo Therapeutics . The companies will work together to develop gene therapy programs for Hemophilia A.
Under the deal, Pfizer is paying Sangamo $70 million upfront. Sangamo will conduct the Phase I/II clinical trials of SB-525 as well as taking on some manufacturing operations. Pfizer will handle subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products.
Sangamo will be eligible for possible milestones up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for any additional Hemophilia A gene therapy product candidates that get developed. Sangamo will also be eligible for tiered double-digit royalties on net sales.
Sangamo will also collaborate with Pfizer on manufacturing and technical operations using viral delivery vectors.
“Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A,” said Mikael Dolsten, president of Worldwide Research and Development for Pfizer, in a statement. “Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing. We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.”
Pfizer has an ongoing development relationship with Philadelphia-based Spark Therapeutics for SPK-9001 for Hemophilia B. In March, the companies announced SPK-9001 had received support through the European Medicines Agency (EMA)’s PRIority Medicines (PRIME) program. SPK-9001 is being evaluated in an ongoing Phase I/II. It received orphan product designation from the U.S. Food and Drug Administration (FDA) in September 2015, and in July 2016, received breakthrough therapy designation.
Sangamo popped at the news of the deal, jumping from $4.40 on May 8 to a current price of $6.42.
“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Sandy Macrae, Sangamo’s chief executive officer, in a statement. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our groundbreaking research into new genomic therapies to treat serious, genetically tractable diseases.”
Sangamo hopes to move SB-525 into the clinic later this quarter. SB-525 is a recombinant adeno-associated virus (rAAV) vector. A gene therapy product, the virus carries a functional copy of the factor VKKK gene into the patient’s liver cells. If it works as designed, the functional genes will increase blood levels of the deficient clotting factor.
John Carroll, writing for Endpoints News, said, “The deal puts Pfizer up against BioMarin , which has been beaming recently about the data that it’s been seeing in Hemophilia A. But Pfizer starts well behind BioMarin. Last summer the biotech said that it nailed down hard evidence that its gene therapy for Hemophilia A demonstrated a high chance of restoring patients’ natural clotting abilities, reducing or eliminating bleeding episodes and pointing them down the road to a normal life. And company investigators pushed BMN 270 into a Phase IIb study aimed at gaining an accelerated approval for a potential blockbuster.”
MedCityNews notes, “There will no doubt be many complex problems to solve along the way. And even after commercialization, success is not guaranteed. The world’s first gene therapy, UniQure’s Glybera, was officially withdrawn from the European market last month due to a lack of demand. That’s not surprising given the therapy’s $1 million price tag.”