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Vertex Pharmaceuticals Ends VX-264 Study in Diabetes After Failing to Meet Efficacy Endpoint
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Vertex Pharmaceuticals Ends VX-264 Study in Diabetes After Failing to Meet Efficacy Endpoint
Mar 31, 2025 4:42 AM

07:23 AM EDT, 03/31/2025 (MT Newswires) -- Vertex Pharmaceuticals ( VRTX ) said late Friday it will discontinue its VX-264 cells plus device program for type 1 diabetes as a phase 1/2 study did not meet the efficacy endpoint.

The company said there was no significant increase in C-peptide, a marker of insulin production, although VX-264 was found to be generally safe and well tolerated.

Vertex said it would conduct further analyses, including of explanted devices, to understand the findings better.

Additionally, Vertex said it is on track to complete enrollment and dosing this summer for a phase 3 study of investigational islet cell therapy zimislecel for type 1 diabetes patients with severe hypoglycemic events and impaired awareness of hypoglycemia.

Global regulatory submissions are planned for 2026, Vertex said.

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