- An experimental cell therapy for Type 1 diabetes has hit a significant milestone, as its developer, Vertex, announced Wednesday the start of initial testing in humans.
- Vertex got its hands on the therapy, known as VX-880, through the $1 billion acquisition of Semma Therapeutics. Semma was co-founded by Douglas Melton, whose lab at Harvard University gave rise to VX-880.
- An early-stage study of the therapy was posted March 8, and expects to enroll about 17 patients who have Type 1 diabetes along with severely low blood sugar and impaired ability to sense it. The study will look primarily at how safe and tolerable the treatment is, as well as the proportion of participants who aren't having severe hypoglycemic events afterwards. It's estimated that data collection for the primary endpoint will wrap up in early 2024.
Vertex raised eyebrows when it announced the Semma deal in 2019. Though Semma's focus on stem cells and regenerative biology did fit into Vertex's strategy of collecting cutting-edge drug technologies, Type 1 diabetes seemed like a far cry from the other diseases that the biotech had set its sights on.
However, Vertex has argued Type 1 diabetes, which affects more than 1 million people in the U.S., is a logical target for a company trying to establish a bigger presence in specialty care drugs.
Vertex also believes Semma's work could meaningfully advance Type 1 diabetes care. At the time of its acquisition, Vertex noted how animal testing had shown Semma's therapy was able to produce large amounts of working human pancreatic beta cells that restore insulin secretion and improve low blood sugar.
What's more, Semma had developed a novel device to protect those cells from the immune system and allow patients to receive treatment without ongoing immunosuppressive therapy.
"Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets," Bastiano Sanna, Vertex's head of cell and genetic therapies, said in a March 10 statement.
Vertex also announced that the Food and Drug Administration has given VX-880 Fast Track Designation, which could speed its development and review.
Given that human testing only just begun, however, it will be a few years before Vertex gets a clear view of how VX-880 affects patients. And it's behind another developer, San Diego-based Viacyte, which has a similar, rival program already in clinical trials.
While the company remains confident, drug development is rarely a smooth process, meaning VX-880 may well run into challenges along the way. Vertex knows the challenges well, having recently hit a notable setback with another one of its experimental drugs. The drug was in mid-stage testing for an inherited disorder called AAT deficiency, which leaves patients with too little of a lung-protecting protein, but safety signals led Vertex to stop the program in October.
In providing that update, Vertex reminded investors it had a second drug for AAT deficiency also in mid-stage testing. Even so, investors have become increasingly worried about Vertex finding success beyond its work in cystic fibrosis, so the apparent failure of one of the company's most advanced programs led to a major stock drop.
Shares of Vertex now trade at $213 apiece, roughly the same price as in March 2020. Though the company saw a 50% growth in revenue last year, and expects another 10% this year, analysts think the stock could stay weighed down until the company shows that its experimental drugs have a shot at coming to market.