The stem cell-derived therapy at the center of Vertex’s $ 950 million buyout of Semma Therapeutics in 2019, which plays a prominent role in the company’s overall cell and gene therapy push, is the clinic’s achievement Step closer.
Vertex is expected to start a Phase I / II trial in the first half of this year after the type 1 diabetes treatment received IND approval from the FDA on Thursday, the company said. The program, named VX-880, is being evaluated in patients with type 1 diabetes with severe hypoglycemia and in those who have difficulty noticing the onset of hypoglycemia, such as in young children.
The researchers will try to determine the safety and proper dosage in the study, which will be one-armed and open-ended. Vertex plans to enroll around 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 by Doug Melton of Harvard. In 2014, Melton published a study showing the potential for using stem cells to make pancreatic insulin-producing beta cells that were introduced into mice in large quantities and successfully protected from an immune response.
That led him to start Semma with a Series A round worth $ 44 million the next year, followed by a Series B round worth $ 114 million in 2017 after his team figured out how to improve technology for people. The goal was to create a credit card-sized implantable device that contained the beta cells that function like a healthy pancreas.
Melton said at the time that it was proving difficult 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 because it would take around 150 million cells to provide the natural insulin.
All of this work led to two studies that produced promising results in June 2019. In the first study, Semma’s stem cell-derived islets performed as hoped and produced insulin in a study of non-human primates whose immune systems were flattened to prevent rejection. In a study in two pigs, a pack of engineered islets contained in one of the implantable membranes successfully produced insulin without the need for an immunosuppressant.
About two months later, Vertex pounced on the cure potential of the treatment with its takeover bid. It was part of a series of eye-catching deals where big-cap biotechnology put Exonics’ gene-editing approach to Duchenne and Ribometrix’s RNA drugging capabilities to the test in 2019.
Due to a longstanding partnership with CRISPR Therapeutics, Vertex continued its business through 2020, which resulted in a pact with Skyhawk Therapeutics last month. The former had shown landmark data in its sickle cell disease program, while the latter is another RNA drug outfit.