Vertex begins human testing for diabetes cell remedy

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Diving letter:

  • An experimental cell therapy for type 1 diabetes reached a significant milestone when the developer Vertex announced the start of the first tests in humans on Wednesday.
  • Vertex got its hands on the therapy known as the VX-880 through its acquisition of Semma Therapeutics, valued at $ 1 billion. Semma was co-founded by Douglas Melton, whose laboratory at Harvard University produced VX-880.
  • An early-stage study of the therapy was published March 8th and expects to include approximately 17 patients with type 1 diabetes, severely low blood sugar, and impaired consciousness. The study primarily examines how safe and tolerable the treatment is and how high the proportion of participants is who no longer have severe hypoglycemic events afterwards. It is estimated that data collection for the primary endpoint will be completed in early 2024.

Dive Insight:

Vertex raised its eyebrows when it announced the Semma deal in 2019. Though Semma’s focus on stem cells and regenerative biology fitted into Vertex’s strategy of collecting cutting-edge drug technologies, type 1 diabetes seemed a long way from the other diseases targeted by biotechnology.

However, Vertex has argued that type 1 diabetes, which affects more than 1 million people in the US, is a logical target for a company trying to build a larger presence in specialty medicines.

Vertex also believes Semma’s work could make meaningful progress in type 1 diabetes care. At the time of the acquisition, Vertex found that animal studies had shown that therapy with Semma was able to produce large quantities of functioning human pancreatic beta cells that restore insulin secretion and improve blood sugar levels.

In addition, Semma had developed a novel device to protect these cells from the immune system and to enable patients to receive treatment without ongoing immunosuppressive therapy.

“Our approach is the only one that produces fully differentiated and fully functional insulin-secreting pancreatic islets,” says Bastiano Sanna, Vertex’s head of cell and gene therapies, said in a March 10 statement.

Vertex also announced that the Food and Drug Administration has awarded the VX-880 Fast Track designation, which could expedite development and review.

Given that human testing has only just begun, it will be a few years before Vertex has a clear idea of ​​how the VX-880 affects patients. And it’s behind another developer, San Diego-based Viacyte, who has a similar, competing program already in clinical trials.

While the company remains confident, drug development is rarely a smooth process, which means the VX-880 may encounter challenges along the way. Vertex knows the challenges well after recently suffering a notable setback with another of its experimental drugs. The drug was in the mid-stage of testing for a hereditary condition called AAT deficiency, in which patients do not have enough lung-protective protein. However, the safety signals led Vertex to abandon the program in October.

With this update, Vertex reminded investors that there was a second drug for AAT deficiency even in mid-stage testing. Even so, investors have become increasingly concerned that Vertex is succeeding beyond its work on cystic fibrosis. The apparent failure of one of the company’s most advanced programs resulted in a significant decline in shares.

Vertex’s stock now trades at $ 213 each, roughly the same as it was in March 2020. Though the company posted 50% sales growth last year and expects another 10% this year, analysts believe the stock could stay weighed until the company shows its experimental drugs have a chance of getting to market .