Vertex Pharmaceuticals has discontinued the development of VX-264, a type 1 diabetes (T1D) cell therapy delivered via an implantable device, after clinical trial data showed the treatment did not produce sufficient levels of insulin.
The decision follows a Phase I/II study (NCT05791201) in which the therapy, designed to work without the need for immunosuppression medications, failed to generate high enough levels of C-peptide, a biomarker of insulin production.
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The therapy involved implanting insulin-producing cells into patients using a device intended to protect the cells from both the autoimmune attack that causes T1D and the immune response to new cells entering the body. While the treatment was found to be generally safe, Vertex reported that the device appeared to hinder its effectiveness.
A 90-day analysis showed C-peptide increases were not observed at levels necessary to deliver a clinical benefit. As a result, Vertex said it would not advance VX-264 further, but “plans to conduct further analyses, including of explanted devices, to better understand these findings”, as per the 28 March announcement.
Despite the setback, Vertex continues to develop another T1D therapy, zimislecel, which is currently being investigated in a Phase III trial (NCT04786262). Zimislecel is administered alongside immunosuppressants to aid cell grafting. The trial is expected to complete enrolment and dosing in H1 2025, with regulatory submissions planned for next year. The therapy has received fast track designation from the US Food and Drug Administration (FDA), and priority medicines (PRIME) designation from the European Medicines Agency (EMA). If approved, zimislecel is projected to generate $554m in 2030, according to GlobalData’s Pharma Intelligence Center.
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Vertex Pharmaceuticals’ chief medical officer Carmen Bozic said: “Today’s data show that more work needs to be done to advance the ‘cells plus device’ programme, and we are committed to doing so. Equally, we are very pleased with the rapid progress of our zimislecel programme.”
The broader T1D treatment landscape continues to see new developments. In January 2025, Sana Biotechnology reported early data from its allogeneic cell therapy, UP421. The donor-derived cell therapy showed signs of insulin production in a patient four weeks after transplantation, with detectable C-peptide levels and an increase in response to a mixed meal tolerance test.
According to a report on GlobalData’s Pharma Intelligence Center, the global T1D market is projected to reach $17.5bn by 2033. However, the report also highlights high clinical trial failure rates and regulatory barriers as potential barriers to future growth in the space.
