Pfizer, Sangamo enter deal to develop haemophilia A gene therapy

Pfizer entered a deal with Sangamo Therapeutics to develop and commercialise gene therapy programmes for haemophilia A, including the latter's SB-525, which is expected to enter the clinic this quarter, the companies reported. Under the agreed terms, Pfizer will make an upfront payment of $70 million, with Sangamo eligible to receive potential milestone payments of up to $475 million, including up to $300 million linked to SB-525.

"Pfizer has made significant investments in gene therapy over the last few years," remarked Mikael Dolsten, president of global R&D at Pfizer, adding "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." SB-525 is comprised of a recombinant adeno-associated virus (AAV) vector carrying a Factor VIII gene construct driven by a synthetic, liver-specific promoter.

Sangamo indicated that it is on track to start a Phase I/II study to evaluate the safety of SB-525 and to measure blood levels of Factor VIII protein and other efficacy endpoints. Under the deal, Sangamo will be responsible for conducting the Phase I/II trial, as well as certain manufacturing activities. Meanwhile, Pfizer will be responsible for subsequent research, development, manufacturing and commercialisation activities for SB-525 and additional products.

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The companies noted that Sangamo is eligible to receive potential milestone payments of up to $175 million for additional haemophilia A gene therapy product candidates, along with tiered double-digit royalties on net sales. Shares in Sangamo jumped as much as 41 percent on the news.

Michael Goettler, global president of rare diseases at Pfizer, noted that the deal with Sangamo bolsters its pipeline in both gene therapy and haemophilia. In 2014, Pfizer entered an agreement with Spark Therapeutics to develop SPK-FIX, a programme incorporating a bio-engineered AAV vector for the potential treatment of haemophilia B. "We have an intention to become a leader in gene therapy and this is another step," Goettler added.

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