Vertex Pharmaceuticals is stopping development of VX-264

Hey everyone, I wanted to share an update on Vertex’s research that many of us were following with hope. Vertex recently announced they are stopping the development of VX-264, an experimental cell therapy designed to help people with type 1 diabetes. In a Phase 1/2 trial with 17 patients, VX-264—which used donor stem cells packaged in a protective implantable device to boost insulin production without needing long-term immune suppression—was found to be safe but didn’t show the expected increase in C-peptide levels (a key marker for insulin production). In simple terms, while the treatment didn’t cause harm, it wasn’t effective enough to offer the benefits we hoped for.

• Trial Discontinued:
  Vertex is halting development of VX-264, an experimental cell therapy for type 1 diabetes, after early Phase 1/2 results did not meet efficacy goals.

• Therapy Concept:
  VX-264 aimed to restore insulin production by transplanting islet cells derived from donor stem cells, using an implantable device to shield the cells from the immune system—potentially avoiding the need for chronic immunosuppression.

• Study Findings:
  In a trial with 17 patients, the therapy was found to be safe and well tolerated. However, the key measure—an increase in C-peptide levels (a marker for insulin production)—did not reach levels necessary to provide clinical benefit.

• Background:
  VX-264 emerged from Vertex’s 2019 $950 million acquisition of Semma Therapeutics, marking their foray into type 1 diabetes research.

• Next Steps for Vertex:
  While VX-264 is discontinued, Vertex is advancing another therapy, zimislecel, which involves a “naked” cell approach (without encapsulation) and requires immunosuppression. This alternative, now in Phase 3 trials, targets patients with more severe type 1 diabetes and could potentially help around 60,000 individuals.

• Additional Research:
  Vertex plans to conduct further analyses on the VX-264 data—including a closer look at the removed devices—and is exploring gene-editing techniques to further protect transplanted cells.

• Financial Note:
  The company anticipates a $400 million asset impairment charge related to VX-264, with detailed results to be shared in their upcoming financial report.

For more details and to read the full article, check it out here:
https://medcitynews.com/2025/03/vertex-cell-therapy-type-1-diabetes-t1d-immunosuppression-vrtx/

This post was researched and written by a LLM using the link above.