Pfizer’s research partnership with Sangamo Therapeutics produced a hemophilia A gene therapy that reached FDA discussions about a regulatory submission. That’s as far as the alliance will go. Pfizer is terminating the seven-year-old pact, a move that comes before the pharmaceutical giant must pay costly milestone payments for a product with uncertain commercial prospects..

辉瑞与Sangamo Therapeutics的研究合作产生了一种血友病a基因疗法，该疗法引起了FDA关于监管提交的讨论。这就是联盟的目标。辉瑞（Pfizer）正在终止这项为期七年的协议，此前这家制药巨头必须为商业前景不确定的产品支付昂贵的里程碑付款。。

According to Sangamo, Pfizer said the termination reflects its decision to not go forward with regulatory submissions for the hemophilia A gene therapy, giroctocogene fitelparvovec. The termination was announced after Monday’s market close. When the termination takes effect in April, Sangamo will regain all rights to the gene therapy.

据Sangamo称，辉瑞公司表示，终止协议反映了其决定不继续提交血友病A基因治疗giroctocogene fitelparvovec的监管文件。周一收盘后宣布终止。当终止协议于四月生效时，桑加莫将重新获得基因治疗的所有权利。

The Brisbane, California-based biotech said it still aims to advance the program and will explore all options, including seeking a new collaboration partner to take the therapy through regulatory review and commercialization..

总部位于加利福尼亚州布里斯班的生物技术公司表示，它仍将致力于推进该计划，并将探索所有选择，包括寻求新的合作伙伴，通过监管审查和商业化来实施该疗法。。

Giroctocogene fitelparvovec is a functioning version of the gene that codes for factor VIII, the clotting protein that’s deficient in hemophilia A patients. The one-time treatment is intended to enable patients to produce factor VIII, bringing that protein closer to normal levels. Under the collaboration agreement signed in 2017, Sangamo was responsible for Phase 1/2 development of the gene therapy.

Giroctocogene fitelparvovec是编码因子VIII的基因的功能版本，因子VIII是血友病a患者缺乏的凝血蛋白。一次性治疗旨在使患者产生因子VIII，使该蛋白质接近正常水平。根据2017年签署的合作协议，Sangamo负责基因治疗的1/2期开发。

Pfizer’s responsibility spanned late-stage development, regulatory submissions, and commercialization..

辉瑞的责任跨越了后期开发、监管提交和商业化。。

Sponsored Post

赞助帖子

The Funding Model for Cancer Innovation is Broken — We Can Fix It

癌症创新的资助模式被打破了-我们可以修复它

Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.

缩小癌症健康公平差距需要通过新的资助方法实现医学突破。

By Eve McDavid - CEO of Mission-Driven Tech

作者Eve McDavid，任务驱动技术公司首席执行官

This past summer, Pfizer reported preliminary Phase 3 results showing the gene therapy led to statistically significant reductions in annualized bleeding rates through 15 months. The pharma giant said it planned to meet with regulators. According to Sangamo, Pfizer had said it expected U.S. and European regulatory submissions would happen in early 2025.

去年夏天，辉瑞公司报告了初步的第三阶段结果，显示基因治疗导致15个月的年出血率在统计学上显着降低。这家制药巨头表示，计划与监管机构会面。据Sangamo称，辉瑞曾表示，预计美国和欧洲的监管报告将于2025年初提交。

As recently as last month, Pfizer indicated it was discussing the data with regulators..

就在上个月，辉瑞表示正在与监管机构讨论数据。。

Hemophilia gene therapies have made it through regulatory review. Pfizer did it earlier this year, winning FDA approval for Beqvez, a hemophilia B gene therapy that was licensed from Spark Therapeutics. But commercializing pricey hemophilia gene therapies has proven to be difficult. For patients who can manage hemophilia with infusions of clotting proteins or chronic dosing of certain drugs, one-time treatment from gene therapy has been a tough sell.

血友病基因疗法已通过监管审查。。但事实证明，将昂贵的血友病基因疗法商业化是困难的。对于那些可以通过输注凝血蛋白或长期服用某些药物来治疗血友病的患者来说，基因治疗的一次性治疗一直是一个艰难的选择。

Newer hemophilia drugs are entering the market, giving patients even more choices. Pfizer has one of them with Hympavzi, a once-weekly injectable drug approved by the FDA in October for both hemophilia A and B..

较新的血友病药物正在进入市场，给患者更多的选择。辉瑞公司（Pfizer）有一种含有Hympavzi的药物，Hympavzi是一种每周一次的可注射药物，于10月份被FDA批准用于治疗a型和B型血友病。。

The commercialization challenges facing hemophilia gene therapies are forcing companies to make hard choices. Lackluster sales of Roctavian, a BioMarin Pharmaceutical gene therapy for hemophilia A approved last year, have led that company to explore options including divestiture of the product. Now Pfizer has decided not to proceed with Sangamo’s hemophilia A gene therapy..

血友病基因疗法面临的商业化挑战迫使公司做出艰难的选择。去年批准的用于治疗血友病a的生物素药物基因疗法Roctavian销售低迷，导致该公司探索包括剥离该产品在内的各种选择。现在辉瑞决定不进行桑加莫血友病A基因治疗。。

Under the gene therapy alliance, Sangamo received $70 million up front. According to the biotech’s financial reports, it had received $55 million in milestone payments so far. Up to $220 million in additional milestone payments remained outstanding. Sangamo was counting on the Pfizer payments for its survival..

在基因治疗联盟的领导下，桑加莫获得了7000万美元的预付款。根据生物技术公司的财务报告，到目前为止，它已经收到了5500万美元的里程碑付款。高达2.2亿美元的额外里程碑付款仍未支付。桑加莫依靠辉瑞公司的付款来生存。。

presented by

提交人

Sponsored Post

赞助帖子

3D Medical Animation Services: A Revolution in Healthcare Education and Marketing

3D医学动画服务：医疗保健教育和营销的革命

3D medical animation services have emerged as a groundbreaking tool in the healthcare industry, revolutionizing the way medical concepts are taught, understood, and marketed.

3D医学动画服务已成为医疗行业的一种突破性工具，彻底改变了医学概念的教学、理解和营销方式。

By Start Grid

Collaborations with Novartis and Biogen ended last year, leading Sangamo to implement a corporate restructuring and layoffs. Sangamo has since inked deals with Genentech and Astellas Pharma, but these agreements come with small upfront payments and milestones that may be years away. In its financial reports, Sangamo said it has explored the possibility of filing for bankruptcy protection.

与诺华和Biogen的合作于去年结束，导致Sangamo实施了公司重组和裁员。桑加莫后来与基因泰克和阿斯特拉斯制药签署了协议，但这些协议带来了小额预付款和里程碑，可能还需要数年的时间。Sangamo在其财务报告中表示，已探讨申请破产保护的可能性。

The company’s cash position as of Sept. 30 was $39.2 million, according to its report for the third quarter of 2024. Sangamo said it expected to have enough cash to last only into the first quarter of 2025..

根据2024年第三季度的报告，截至9月30日，该公司的现金状况为3920万美元。Sangamo表示，预计其现金充足，只能维持到2025年第一季度。。

Sangamo needs cash to support its pipeline of neurology genomic medicines, including a gene therapy for Fabry disease. In October, the FDA confirmed to the company that Phase 1/2 data would be sufficient to support a regulatory submission under the accelerated approval pathway. The company planned a submission for the second half of 2025..

桑加莫需要现金来支持其神经病学基因组药物的管道，包括法布里病的基因治疗。10月，FDA向该公司确认，第1/2阶段的数据足以支持加速批准途径下的监管提交。该公司计划在2025年下半年提交。。

In the announcement of the Pfizer termination, Sangamo said it believes it can chart a path forward for its programs, but the company acknowledged that additional funding is necessary for advancing each of them, including the hemophilia A gene therapy. In a prepared statement, Sangamo CEO Sandy Macrae said the company was surprised and disappointed by Pfizer’s decision to end the collaboration so close to the expected regulatory submissions..

在宣布辉瑞终止协议时，桑加莫表示，它相信自己可以为其计划制定一条前进的道路，但该公司承认，推进每一项计划都需要额外的资金，包括血友病a基因治疗。Sangamo首席执行官桑迪·麦克雷（SandyMacrae）在一份准备好的声明中表示，辉瑞决定终止合作，如此接近预期的监管提交，公司对此感到惊讶和失望。。

“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,” he said.

他说：“我们致力于探索这一重要治疗的最佳途径，包括寻找关注和了解基因组医学商业环境的正确合作伙伴，将这种药物带给患者。”。

Illustration: Kuzma, Getty Images

插图：库兹马，盖蒂图片

Topics

主题

Clinical Trials

临床试验

gene therapy

基因治疗

hemophilia A

A型血友病

Pfizer

辉瑞公司

Sangamo Therapeutics

Sangamo Therapeutics公司

MedCity News Daily Newsletter

MedCity新闻每日通讯

Sign up and get the latest news in your inbox.

注册并在收件箱中获取最新消息。

Enter your email address

输入您的电子邮件地址

Subscribe Now

立即订阅

We will never sell or share your information without your consent. See our privacy policy.

未经您同意，我们绝不出售或共享您的信息。请参阅我们的隐私政策。

More from MedCity News

更多来自MedCity新闻

Healthcare IT in 2025: Triage for the Age of AI

2025年的医疗保健IT：AI年龄的分类

The Evolving Landscape of Medicaid and CHIP: Implications for Children’s Oral Health

医疗补助和CHIP的发展前景：对儿童口腔健康的影响

Healthcare Moves: A Monthly Summary of Hires, Exits and Layoffs

医疗保健举措：每月招聘、离职和裁员摘要

Axsome Aims for FDA Filing in Alzheimer’s Agitation Despite Mixed Bag of Phase 3 Data

Axsome旨在FDA提交阿尔茨海默病激动症的申请，尽管第三阶段数据混杂