Vertex Doses First Patient in Phase 1/2 Trial of VX-264 in Type 1 Diabetes


The phase 1/2 trial features 3 phases, with new sites expected to open in the coming months. VX-264 uses the same stem cell-derived islets used in the VX-880 program in type 1 diabetes.

Bastiano Sanna, PhD, the executive vice president and chief of Cell and Genetic Therapies at Vertex

Bastiano Sanna, PhD

Vertex Pharmaceuticals has announced amid its quarterly financial results that it has dosed the first patient in its newest cell therapy program, the phase 1/2 trial (NCT05791201) of VX-264, a fully differentiated islet cell therapy, in patients with type 1 diabetes mellitus.1

The trial is still enrolling patients, and is designed as a sequential, multipart study aimed at evaluating safety, tolerability, and efficacy of VX-264. As of the latest update on July 27, 2023, the estimated enrollment was 17 patients.2

VX-264 treatment consists of differentiated islet cells that are encapsulated in an immunoprotective device designed to shield them from the immune system and avoid immunosuppressive therapy use. It uses the same stem cell-derived, fully differentiated islets used in the VX-880 program, which recently met the primary efficacy end point in its own phase 1/2 study in this patient population,3 after being placed on a clinical hold the year prior because the FDA determined there was insufficient information to support dose escalation at the time.4

The FDA greenlit this phase 1/2 trial of VX-264 in March this year, at which time Bastiano Sanna, PhD, the executive vice president and chief of Cell and Genetic Therapies at Vertex, said in a statement, “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.”

READ MORE: Rett Syndrome Gene Therapy Trial Recommended to Dose Second Patient

The trial’s first portion, Part A, will administer patients with a partial dose of cells, staggered between patients; Part B will dose patients with a full target dose, staggered between patients; and Part C will shift to concurrent dosing. Patients are enrolled from the United States, Canada, and the Netherlands, though Vertex has noted that it aims to activate additional global sites in the coming months.

In June, when data from the VX-880 development program were announced, CGTLive™ spoke with project leader Massimo Trucco, MD, director of the Alleghany Health Network Institute of Cellular Therapeutics, who offered his perspective on the islet cell transplant procedure, discussing eligibility and applications, among other topics. He noted the use of islet cell therapies as having potential in chronic pancreatitis, which he said “can lead to cancer. So, that is a consideration for doing the auto transplant in children to avoid a problem that can develop in the future. In adults, the organs are already working suboptimally and the cell-to-liver transplant is not the best because the liver is not the best. The general body of the recipient is not working very well.”

This year has featured a number of forays into diabetes for cell therapies—most notably in June, when the FDA approved CellTrans’ donislecel allogeneic pancreatic islet cell therapy under the name Lantidra, for treating adults with type 1 diabetes who are unable to approach target glycated hemoglobin because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education.5 Additionally, in June, 5 individuals treated with Sernova’s Cell Pouch System achieved insulin independence following islet transplant to the Cell Pouch and supplemental portal vein islet infusion,6 and in February, an institutional review board gave approval to Creative Medical Technology Holdings to proceed with its phase 1/2 CREATE-1 clinical trial (NCT05626712) for the evaluation of CELZ-201 (AlloStem) in the treatment of newly diagnosed T1D.7

1. Vertex Reports Second Quarter 2023 Financial Results. News release. Vertex Pharmaceuticals. August 1, 2023. Accessed August 2, 2023.
2. A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes. NIH National Library of Medicine. Updated July 27, 2023. Accessed August 2, 2023.
3. Vertex presents positive VX-880 results from ongoing phase 1/2 study in type 1 diabetes at the American Diabetes Association 83rd Scientific Sessions. News release. Vertex Pharmaceuticals. June 23, 2023. Accessed August 2, 2023.
4. Vertex provides updates on phase 1/2 clinical trial of VX-880 for the treatment of type 1 diabetes. News release. Vertex Pharmaceuticals. May 2, 2022. Accessed August 2, 2023.
5. FDA Approves First Cellular Therapy to Treat Patients with Type 1 Diabetes. News release. FDA. June 28, 2023. Accessed August 2, 2023.
6. Sernova announces positive updated interim phase 1/2 clinical data for the Cell Pouch System at American Diabetes Association 83rd Scientific Sessions. News release. Sernova Corp. June 26, 2023. Accessed 2, 2023.
7. Creative Medical Technology Holdings announces IRB approval for FDA cleared phase 1/2 clinical trial of novel cell therapy for the treatment of type 1 diabetes. News release. Creative Medical Technology Holdings, Inc. February 8, 2023. Accessed August 2, 2023.
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