Sangamo Therapeutics Faces Major Setback as Pfizer Halts Gene Therapy Development for Hemophilia A
8 months ago

Sangamo Therapeutics' shares faced a significant decline on Tuesday following an announcement that its partner, Pfizer, has opted not to move forward with the development of a new gene therapy designed to treat hemophilia A, a serious bleeding disorder. This partnership initially aimed to deliver innovative treatments to patients suffering from this condition. The collaboration resulted in the development of giroctocogene fitelparvovec, a new experimental therapy, as both companies worked on global development and commercialization strategies for gene therapies dedicated to hemophilia A.

Sangamo had previously transferred both the manufacturing technologies and the investigational new drug application to Pfizer in late 2019, highlighting the depth of their cooperation. However, the latest news indicates that Pfizer will cease any further action regarding the biologics license application and marketing authorization application for this therapy, including any exploration of its potential commercialization.

As a result, Sangamo announced that the collaboration and licensing agreement will be terminated effective April 21. On the trading floor, Sangamo's shares plummeted by 54% that day, a clear reflection of the disappointing news, while Pfizer experienced a modest increase of 0.5% in its share prices, demonstrating the market's segmented reactions to the developments. Sangamo's Chief Executive, Sandy Macrae, expressed surprise and extreme disappointment over Pfizer's decision, emphasizing the company's commitment to finding the optimal path forward for this critical treatment.

He mentioned efforts to seek out the right partner to continue development and deliver the therapy to patients who need it. Pfizer, in its statement sent to MT Newswires, elaborated on its rationale behind the decision, citing an extensive analysis of clinical trial data, feedback from key opinion leaders, and a sluggish interest in hemophilia A gene therapy from patients with moderate to severe conditions.

The pharmaceutical giant concluded that the current landscape reflected limited interest in pursuing additional gene therapy options for this patient demographic at this time. Despite the setback, Sangamo remains hopeful. Earlier this July, Pfizer had reported that a phase 3 trial of giroctocogene fitelparvovec successfully met its primary and key secondary objectives, demonstrating superiority compared to existing prophylaxis treatments.

Previous communications from Pfizer had led Sangamo to anticipate the submission of the biologics license and marketing authorization applications early in the following year. Looking ahead, Sangamo has plans to initiate patient enrollment for a phase 1/2 study of ST-503 targeting idiopathic small fiber neuropathy in mid-2025, alongside filing for clinical trial authorization for its prion disease program expected in the fourth quarter.

Both initiatives remain contingent on securing further funding and support..

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