Vertex Branches Into Diabetes, Acquiring Semma for $950 Million

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Vertex Pharmaceuticals announced a deal to buy Semma Therapeutics. Semma Therapeutics focuses on using stem cell-derived human islets as a possible cure for type 1 diabetes.

Under the terms of the deal, Vertex will acquire all outstanding Semma shares for $950 million in cash. Semma will become a separate operating subsidiary of Vertex. Bastiano Sanna, president and chief executive officer of Semma, will join Vertex as president of Semma. Douglas Melton, who founded Semma, will continue as chair of Semma’s scientific advisory board.

Semma is working to advance Melton’s methodology for creating billions of functional, insulin-producing beta cells. These cells are grown in the laboratory from stem cells and develop into islet-like clusters. Preclinical work in animals have shown that transplanting these cells via a liver infusion, can control blood glucose levels.

The company is also working on combining the cells with a state-of-the-art cell device and immune protection strategy. The implantable device would protect the infused cells from the patient’s immune system, which theoretically would allow the beta cells to function the way they do in a person without diabetes.

On July 1, Semma announced it had shown preclinical proof-of-concept for its two lead programs in type 1 diabetes in both non-human primates and pigs. The first data showed how the SC-islets were infused into the portal vein, mimicking planned human clinical trials. The SC-islets engrafted and were functional, and they persisted over six weeks, decreasing the need for insulin by more than 60%.

In a second study in pigs, the company tested the immunoprotective device, which was designed to maintain SC-islets alive and functional without immunosuppressant drugs. The device worked, allowing the SC-islets to secrete insulin.

“The therapeutic approach pioneered by Semma has the potential to address the causal human biology of type 1 diabetes, a serious disease inadequately controlled by existing therapies,” stated David Altshuler, executive vice president, Global Research and chief scientific officer of Vertex. “Unlike insulin injections and insulin pumps, islet cell transplantation can provide physiologic regulation of blood glucose thereby potentially ameliorating or preventing both the hyperglycemic and hypoglycemic episodes associated with the current standards of care.”

Altshuler added, “Their compelling proof-of-concept data in animals demonstrates the opportunity to develop transformative and potentially curative therapies to treat people with type 1 diabetes. In addition, the acquisition of Semma continues to expand the Vertex toolbox of cutting-edge technologies and capabilities, and bolsters our team of leading scientists.”

The deal is expected to close in the fourth quarter of this year. Vertex has several drugs on the market for cystic fibrosis, including Symdeko, Orkambi and Kalydeco. Beyond cystic fibrosis, the company’s pipeline is developing therapies for sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

This deal is a move into a new treatment space.

“We see a substantial opportunity to transform the treatment paradigm for type 1 diabetes, a specialty disease cared for by endocrinologists,” said Jeffrey Leiden, Vertex’s chief executive officer.

Sanna stated, “Type 1 diabetes is a disease that afflicts millions of people worldwide and has no curative therapies available. Vertex has a proven track record of serial innovation and a deep commitment to developing transformative therapies for patients in need. Being a part of Vertex will allow the Semma team to rapidly and effectively advance our cell therapy and delivery approaches to patients who need them.”



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