Vertex eyes type 1 diabetes cure with biotech acquisition
Will acquire Semma Therapeutics for $950m
American pharma company Vertex will acquire Semma Therapeutics, a biotech focused on finding a cure for type 1 diabetes.
Vertex will pay $950m in cash for the Cambridge, MA-based biotech, and will acquire all outstanding shares of the company. Semma will become a subsidiary of Vertex, while the biotech’s CEO Bastiano Sanna is set to join Vertex to continue as the president of Semma.
Semma was founded to develop therapies for type 1 diabetes patients who depend on insulin injections. According to Vertex, the biotech is pioneering the use of stem cells as a potential cure for type 1 diabetes. The biotech has advanced the ability to produce large quantities of functional human pancreatic beta cells that restore insulin production and ameliorate hypoglycaemia in animal tests.
In addition to this breakthrough, the biotech has created a device that captures and protects these cells from the immune system, which in turn allows durable implantation without the need for ongoing immunosuppressive therapy.
Jeff Leiden, president and CEO of Vertex
“This acquisition aligns perfectly with our strategy of investing in scientific innovation to create transformative medicines for people with serious diseases in specialty markets,” said Jeff Leiden (pictured above).
“We are excited to work with the talented scientists at Semma to build on their significant progress toward providing effective and potentially curative cell therapy options for people living with type 1 diabetes,” he added.
This acquisition follows a string of deals from Vertex, which is seeking to diversify its portfolio beyond its cystic fibrosis franchise. The company has been a pioneer in the CF therapy area, and recently filed its landmark triple combination therapy with the FDA. If approved, the therapy could transform the treatment of the disease, and could be used to treatup to 90% of all cystic fibrosis patients worldwide.
Earlier this year, Vertex also eyed the market for Duchenne muscular dystrophy, when it made a deal to acquire US biotech Exonics Therapeutics. Exonics is developing gene-editing therapies for DMD and other genetic neuromuscular disease. Vertex paid $245m upfront for the CRISPR/Cas-9-focused company, with another $750m in the future in milestone payments.
The pharma company then paid $175m upfront to expand its collaboration with the Swiss company CRISPR Therapeutics, with milestones that could drive the value of the deal above the $1bn mark. That deal also focuses on gene-editing technology directed at DMD and DM1.
Following the acquisition of Semma, Vertex is likely to find a rival in Novo Nordisk, which last year said it has reached a milestone in the same area of stem cell therapy in type 1 diabetes. The first clinical trials for both companies could begin within the next few years, setting up a potential race to first-in-class.
美国制药公司 Vertex 将收购 Semma Therapeutics,这是一家专注于寻找 1 型糖尿病治愈方法的生物技术公司。Vertex 将以 9.5 亿美元现金收购这家总部位于马萨诸塞州坎布里奇市的生物技术公司,并将收购该公司所有已发行股份。Semma 将成为 Vertex 的子公司,而生物技术公司的 CEO Bastiano Sanna 将加入 Vertex 继续担任 Semma 的总裁。Semma 公司成立的目的是为依赖注射胰岛素的 1 型糖尿病患者开发疗法。据 Vertex 介绍,该生物技术正在率先将干细胞作为一种潜在的治愈 1 型糖尿病的方法。该生物技术在动物试验中已经提高了产生大量功能性人胰腺 β 细胞的能力,这些细胞能够恢复胰岛素的产生并改善低血糖。除了这项突破,该生物技术还创造了一种设备,能够捕获并保护这些细胞免受免疫系统的攻击,进而实现持久植入,而不需要持续的免疫抑制治疗。Vertex 总裁兼首席执行官杰夫莱顿 (Jeff Leiden) 表示:“此次收购与我们投资科学创新的战略完美契合,从而在专业市场上为患有严重疾病的人创造变革性的药物。”“我们很高兴与 Semma 的天才科学家们合作,在他们为 1 型糖尿病患者提供有效和潜在治愈性细胞治疗选择方面取得重大进展,”他补充说。此次收购是在 Vertex 公司一系列交易之后进行的,Vertex 公司正在寻求将其投资组合多元化,以超越其囊性纤维化特许经营权。该公司一直是 CF 疗法领域的先驱,最近向 FDA 提交了其里程碑式的三联组合疗法。如果获得批准,该疗法可以改变该病的治疗方法,并可用于治疗全世界高达 90% 的囊性纤维化患者。今年年初,Vertex 公司也瞄准了杜氏肌营养不良症的市场,当时该公司达成了收购美国生物技术公司 Exonics Therapeutics 的交易。Exonics 正在开发针对 DMD 和其他遗传性神经肌肉疾病的基因编辑疗法。Vertex 先付 2.45 亿美元收购了这家专注于 CRISPR/Cas-9 的公司,未来还有 7.5 亿美元里程碑付款。随后,这家制药公司支付了 1.75 亿美元的先期费用,以扩大与瑞士 CRISPR Therapeutics 公司的合作,里程碑事件可能将这笔交易的价值推至 10 亿美元以上。该交易还集中在针对 DMD 和 DM1 的基因编辑技术。继收购 Semma 之后,Vertex 很可能在诺和诺德找到竞争对手,后者去年曾表示,在 1 型糖尿病干细胞治疗的同一领域达到了里程碑。两家公司的首批临床试验可能会在未来几年内开始,建立起一场潜在的 first-in-class 竞赛。

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