Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes

Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes (T1D). The VX-264 program does not require the use of immunosuppression, which may broaden the population of people with T1D that this investigational therapy could reach.

Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2023 to study the safety, tolerability and efficacy of VX-264 in patients with T1D. The company previously received approval from Health Canada on the Clinical Trial Application (CTA) for VX-264, and the Phase 1/2 trial is ongoing in Canada.

“VX-264 uses the same stem cell-derived pancreatic islet cells as our VX-880 program where we’ve already demonstrated proof-of-concept, with the addition of a proprietary immunoprotective device that allows us to eliminate the need for immunosuppression,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “We are excited to see our second program in T1D advancing into the clinic and look forward to bringing transformative, if not curative, therapies to T1D patients who are waiting.”

Vertex is pursuing multiple investigational approaches using stem cell-derived islets with the aim of replacing the insulin-producing islet cells that are destroyed in people with T1D. Vertex’s first clinical investigational program in T1D, VX-880, is a stem cell-derived, fully differentiated, insulin-producing islet cell replacement therapy used in combination with immunosuppression. Vertex has demonstrated clinical proof-of-concept in the VX-880 program, and the Phase 1/2 clinical study is ongoing in the U.S. and Canada.