Sangamo secures FDA’s Fast Track Designation for haemophilia candidate
pharmafile | May 17, 2017 | News story | Research and Development, Sales and Marketing | FDA, Pfizer, Sangamo Therapeutics
Sangamo Therapeutics has announced that the FDA has granted its cDNA gene therapy candidate for haemophilia A Fast Track Designation. The candidate, SB-525, is set to enter clinical trials, at Phase 1/2 level, this year with the results expected by late 2017, early 2018.
SB-525 uses a recombinant adeno-associated virus to deliver a human Factor VIII cDNA construct and proprietary, synthetic liver-specific promoter to the nucleus of liver cells with a single infusion. The therapy is designed as a single treatment strategy intended to provide continuous, therapeutic expression of Factor VIII protein.
Early studies, in non-human primates, have investors excited that this particular treatment could become best-in-class for haemophilia A.
The news comes only a week after Sangamo and Pfizer had entered into a potential $545 million deal. Pfizer was attracted to the licensing partnership as a result of Sangamo’s four lead products, one of which includes SB-525. The deal involved $70 million in cash up-front, with a further $475 million possible in milestone payments.
The deal also meant that should SB-525 prove successful in clinical trials, Pfizer will take over the work of developing the candidate beyond Phase 1/2, as well as being responsible for manufacturing and commercialisation activities.
“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer, on the announcement of the partnership. “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 ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”
Ben Hargreaves
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