Vertex Discontinues Development Of Type 1 Diabetes Candidate After Disappointing Data

Vertex Pharmaceuticals Inc VRTX announced on Friday updates about its type 1 diabetes (T1D) portfolio.

The company said it would not be advancing further in VX-264 clinical trials. Vertex plans to conduct further analyses, including of explanted devices.

• Vertex completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) study and the planned analysis at Day 90 for Part B.
• In Part B of the study, participants received the full dose of the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device.
• In Part B, there were two primary endpoints: safety and change in peak C-peptide during a mixed-meal tolerance test (MMTT) from baseline at Day 90.
• VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint.
• Increases in C-peptide, a marker of insulin production, were not observed at levels necessary to deliver benefit.

Zimislecel (formerly VX-880), Vertex’s investigational fully differentiated islet cell therapy with standard immunosuppression, is in the Phase 3 portion of the Phase 1/2/3 study in patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia.

• The is on track to complete enrollment and dosing in the first half of 2025, setting up global regulatory submissions in 2026.

Vertex is investing in expanding its manufacturing and commercial capabilities to ensure launch readiness. If approved, eligible patients across the U.S. and Europe with recurrent SHEs despite best available care could benefit from zimislecel. Vertex anticipates that initial approval could serve approximately 60,000 people with severe T1D.

Vertex is also pursuing research-stage T1D programs. These approaches include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices to encapsulate islet cells.

William Blair wrote,”We view the discontinuation of VX-264 as disappointing given that this would have expanded Vertex's type 1 diabetes market significantly from that being addressed from zimislecel; however, we did not ascribe any value for the early-stage program in our model. Vertex's prioritization on the clinical advancement and potential launch of zimislecel for 60,000 severe T1D patients is prudent given the positive data to date, and approval in the T1D space would serve to further the company's diversity of commercial assets beyond cystic fibrosis and acute pain.”

Analyst Myles Minter reiterated an Outperform rating.

VRTX Price Action: Vertex stock was down 2.09% at $482.36 at publication Monday.

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