Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones.
The Big Pharma paid $70 million to license the candidate in 2017 and handed over a further $55 million in milestones as it took the program through to a phase 3 readout last year. The pivotal trial met its primary endpoint, but, with BioMarin’s Roctavian showing clinical wins are no guarantee of commercial success in the space, Pfizer has decided to cut its losses rather than seek approval as previously planned.
Pfizer’s decision sent Sangamo’s share price plummeting 56% to $1.02 on Dec. 31. Investors had flagged the progression of the partnered gene therapy, giroctocogene fitelparvovec, as a potential source of cash for Sangamo.
The biotech ended September with around $39 million to its name. After factoring in a subsequent $10 million payment from Genentech, Sangamo said it only had the cash to fund operations into the first quarter of 2025. A deal with Astellas gave Sangamo a further $20 million last month, but the biotech is still racing toward the end of its cash runway.
Milestones from Pfizer could have helped Sangamo navigate the cash crisis. Sangamo CEO Sandy Macrae, Ph.D., told investors on an earnings call in November that Pfizer could pay up to $220 million in regulatory and commercial milestones over the next two years.
With Pfizer returning the rights to the gene therapy, Sangamo said it “intends to explore all options to advance the program, including seeking a potential new collaboration partner.” A new deal could secure Sangamo an upfront cash injection and an ongoing infusion of financial milestones, but Pfizer’s decision to pass on the program raises doubts about the attractiveness of the gene therapy to other drugmakers.
The travails of BioMarin’s Roctavian have potentially weakened Sangamo’s hand. BioMarin broke new ground by winning FDA approval for the gene therapy in 2023 but has found few takers for the product. The biotech posted Roctavian sales of $16 million over the first nine months of 2024 and is now focused on cutting costs and growing revenues to the extent that the product is profitable.
Roctavian’s commercial troubles suggest patients are content to stick with existing drugs such as Roche’s once-weekly treatment Hemlibra rather than join the vanguard of people trying gene therapies. Better gene therapies or commercialization strategies could tip the balance, but Pfizer has concluded that its dollars and attention are better spent on assets other than giroctocogene fitelparvovec.