Vertex halts VX-264 but advances zimislecel trial for T1D

BOSTON - Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), a prominent player in the biotechnology industry with a market capitalization of $126 billion and strong year-to-date returns of nearly 25%, has announced the completion of enrollment and dosing for parts A and B of its Phase 1/2 study for VX-264, a type 1 diabetes (T1D) treatment. According to InvestingPro analysis, the company maintains a healthy financial position with a current ratio of 2.69, indicating strong liquidity. Despite the therapy being well tolerated, efficacy data indicated it fell short of expectations, leading to the decision not to progress VX-264 to further clinical trials.

The study’s Part B aimed to evaluate the safety and change in peak C-peptide during a mixed-meal tolerance test at Day 90. While the safety endpoint was met, the efficacy results did not demonstrate the necessary benefit, as increases in C-peptide were not at the levels required. Vertex plans to conduct additional analyses of the explanted devices to gain a deeper understanding of the results.

In contrast, Vertex’s other T1D therapy, zimislecel (formerly VX-880), is progressing positively. The Phase 3 portion of the Phase 1/2/3 study for zimislecel is on track to complete enrollment and dosing by the first half of 2025, with plans for global regulatory submissions in 2026. Zimislecel has received multiple designations from health authorities, including Regenerative Medicine Advanced Therapy and Fast Track from the FDA, PRIME from the EMA, and an Innovation Passport from the UK MHRA. Vertex is preparing for potential market launch by expanding its manufacturing and commercial capabilities. With annual revenue exceeding $11 billion and revenue growth of 11.7% in the last twelve months, the company appears well-positioned to support this expansion. If approved, zimislecel could serve an estimated 60,000 people with severe T1D in the U.S. and Europe.

Vertex also continues to explore additional research-stage immunoprotective approaches for T1D, aiming to reduce or eliminate the need for standard immunosuppressive regimens. These include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel encapsulation devices for islet cells.

Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, expressed gratitude to all participants of the VX-264 study and emphasized the company’s commitment to advancing the ’cells plus device’ program. She also highlighted the promising progress of the zimislecel program and the potential impact it could have on patients with T1D.

This update is based on a press release statement from Vertex Pharmaceuticals.

In other recent news, Vertex, Inc. reported its fourth-quarter financial results, which met analyst expectations with adjusted earnings per share of $0.15, slightly surpassing the anticipated $0.14. The company recorded revenue of $178.5 million, marginally exceeding the consensus of $176.9 million and reflecting a 15.2% year-over-year increase. However, Vertex’s guidance for the first quarter of 2025 fell short of analyst projections, estimating revenue between $175 million and $178 million, compared to the expected $180.3 million. For the full year 2025, the company forecasts revenue in line with the consensus estimate of $764 million.

In a separate development, DA Davidson reaffirmed its Buy rating on Veritex Holdings, maintaining a price target of $34. The firm expressed confidence in Veritex’s potential for improved profitability and highlighted its strategic positioning in the Texas banking market. Meanwhile, Vertex Pharmaceuticals’ newly approved non-opioid pain medication, Journavx, has been included in UnitedHealth’s coverage but is categorized in a higher-cost tier. This tier 3 designation suggests patients may face higher costs compared to other drugs on lower tiers. These recent developments provide insight into the strategic and financial maneuvers of these companies.

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