The diabetes 'cure' Vertex acquired in its 2019 Semma buyout? It's headed to the clinic
The stem cell-derived therapy at the center of Vertex’s $950 million buyout of Semma Therapeutics in 2019, one that features prominently in the company’s overall cell and gene therapy push, is one step closer to reaching the clinic.
Vertex is expected to launch a Phase I/II trial in the first half of this year after the type 1 diabetes treatment received IND clearance from the FDA on Thursday, the company announced. The program, dubbed VX-880, will be evaluated in type 1 diabetes patients with severe hypoglycemia and those who struggle to perceive the onset of hypoglycemia, like young children.
Researchers will try to determine the safety and proper dosage in the study, which will be single-arm and open-label. Vertex plans to enroll about 17 patients. VX-880 itself uses stem-cell derived pancreatic islet cells to try to restore the body’s ability to produce glucose in combination with immunosuppressive therapy to protect the cells.
The program has been described as a potential “cure” for type 1 diabetes and comes from research done by Harvard’s Doug Melton. In 2014, Melton published a study showing the potential for using stem cells to create insulin-producing pancreatic beta cells, inserted in bulk into mice, that were successfully protected from an immune response.
That led him to launch Semma the next year with a $44 million Series A round, followed by a $114 million Series B in 2017 after his team figured out how to upscale the technology for humans. It involved creating an implantable, credit card-sized device containing the beta cells that function like a healthy pancreas.
Melton said at the time that it proved challenging to develop such a membrane with pores large enough for molecules to pass through, but small enough to be protected from immune cells. He compared the membrane to a tea bag that couldn’t be overloaded, given that they would need some 150 million cells in order to provide the natural insulin.
All that work led up to two studies that presented promising results in June 2019. In the first study, Semma’s stem cell-derived islets performed as hoped for, producing insulin, in a study involving non-human primates whose immune systems had been flattened to prevent a rejection. Then in a study in two pigs, a package of engineered islets contained in one of the implantable membranes successfully generated insulin without needing an immunosuppressant.
About two months later, Vertex swooped in with their buyout offer to gamble on the curative potential on the treatment. It came as part of a string of flashy deals, one that saw the big cap biotech snap up Exonics’ gene editing approach to Duchenne and Ribometrix’s RNA drugging capabilities in 2019 collaborations.
Stacked on top of a longtime partnership with CRISPR Therapeutics, Vertex’s dealmaking continued into 2020, leading to a pact with Skyhawk Therapeutics last month. The former had demonstrated landmark data in its sickle cell disease program, while the latter is another RNA drugging outfit.
