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Shares of Sangamo Therapeutics have plummeted more than 56% over the past few days after Pfizer announced it had terminated its partnership to develop an investigational gene therapy product to treat hemophilia A. 

The drug, giroctocogene fitelparvovec, is specifically designed to treat adults with moderate to severe hemophilia A. 

As a result of Pfizer’s decision, Sangamo will regain full development and commercialization rights for its therapy. 

This announcement marks a sudden change of direction for Pfizer, which previously expressed commitment to investing in experimental treatments for diseases like hemophilia. 

In July, Pfizer announced positive results from a Phase 3 trial evaluating Sangamo’s gene therapy, noting that the drug demonstrated superiority compared to prophylaxis. The pharma giant also presented results at the 66th annual ASH Meeting and Exposition in December. 

Sangamo CEO Sandy Macrae expressed disappointment about Pfizer’s decision to step aside, especially after noting that the therapy had demonstrated significant potential for hemophilia A patients. 

“We are surprised and extremely disappointed by Pfizer’s decision to end our collaboration so close to the anticipated BLA and MAA submissions,” Macrae said in a statement. 

Sangamo stated that Pfizer indicated that it was discussing data from the gene therapy with regulatory authorities. 

It also mentioned that the pharma giant indicated to Sangamo that the drug’s Biologics License Application (BLA) and Marketing Authorization Application submissions (MAA) to the Food and Drug Administration were anticipated to occur in the spring. 

However, Pfizer will now effectively terminate its license agreement with Sangamo in April. Sangamo said that all trial patients under Pfizer will continue to be monitored as previously planned. 

The gene therapy maker also said it is committed to continuing the program and will potentially seek a new collaborator. 

“We are committed to exploring the optimal path forward for this important treatment, including seeking the right partner with the focus and understanding of the genomic medicine commercial environment to bring this medicine to patients,” said Macrae. 

While gene therapy trials have shown positive results for symptom management, commercial troubles around therapies on the market suggest that patients may be more interested in using existing drugs, even if they need to be administered more frequently. 

Analysts have noted that these products aren’t necessarily a top priority for pharma giants as many manage symptoms rather than curing diseases. 

For example, BioMarin won FDA approval for a gene therapy in 2023, but it hasn’t found many patients who are willing to try the product. The biotech announced sales of $16 million over the first nine months of 2024 and mentioned that it’s focused on cutting costs and growing revenues in the future. 

Despite the current market reactions toward gene therapies, Sangamo is still committed to the development of more therapies. 

The company also recently announced partnerships with Astellas Pharma and Genentech, which it believes will help carve a path forward for neurology genomic medicine. Sangamo added that it expects the enrollment of patients in a Phase 1/2 study of a therapy for idiopathic small fiber neuropathy this year. 

“We remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025,” Macrae stated.