Gene Therapy Partnership Dissolves, Sending Shockwaves Through Biotech Industry
Pfizer’s Sudden Exit Leaves Sangamo Reeling
In a stunning move, Pfizer Inc. has terminated its partnership with Sangamo Therapeutics Inc. to develop a groundbreaking gene therapy for hemophilia A, sending Sangamo’s shares into a tailspin. The biotech company’s stock plummeted as much as 56% on Tuesday, its largest decline in 16 years.
A Promising Treatment in Jeopardy
The experimental medicine, giroctocogene fitelparvovec, had shown remarkable promise in a pivotal late-stage trial, meeting its primary goal. Sangamo had been expecting Pfizer to file for US and European approval in early 2025. However, Pfizer’s sudden change of heart has left Sangamo scrambling to find a new partner or alternative solutions to continue developing the treatment.
Sangamo CEO Expresses Disappointment and Surprise
“We were extremely surprised and very disappointed” by Pfizer’s decision, said Sangamo CEO Sandy Macrae in a phone interview. The company was informed of the decision on December 22, and Macrae emphasized that Sangamo is now considering “all options” to move forward with the drug.
Liquidity Concerns and Milestone Payments
Sangamo has struggled with liquidity issues, with only $39.2 million in cash and cash equivalents as of September 30. The company had been eligible for up to $220 million in milestone payments under the agreement with Pfizer. With a market value of just below $500 million before the news broke, Sangamo’s financial situation has become even more precarious.
Hemophilia A: A Complex and Challenging Condition
Hemophilia A is a debilitating genetic disorder that makes patients more susceptible to bleeding and typically requires lifelong therapy. Despite the availability of at least a dozen approved treatments, there remains a significant need for innovative solutions. BioMarin Pharmaceutical Inc.’s gene therapy for hemophilia A, for example, has been a commercial disappointment.
Pfizer’s Shift in Focus
Pfizer cited limited interest in an additional gene therapy option for hemophilia A as the reason for its exit. The pharmaceutical giant has been seeking new opportunities as sales of its Covid-19 vaccine and treatment have declined. Shares of Pfizer rose as much as 0.9% following the announcement.
Uncertainty Looms for Sangamo’s Future
As Sangamo navigates this unexpected setback, the company’s future hangs in the balance. Will it find a new partner to help bring its promising gene therapy to market, or will it be forced to explore alternative paths? One thing is certain: the biotech industry is watching with bated breath as Sangamo charts its next move.
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