VRTX Provides Mixed Updates for Type 1 Diabetes Pipeline Candidates

Vertex Pharmaceuticals Incorporated VRTX announced updates on its two novel investigational candidates, zimislecel (formerly VX-880) and VX-264, which are being developed in separate studies for the treatment of type 1 diabetes (T1D).

Zimislecel is designed for the transplantation of islet cells alone, using immunosuppression to protect the implanted cells. The candidate is in pivotal late-stage development for treating patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia.

Meanwhile, VX-264 is being developed following a unique approach that involves the implantation of the islet cells inside an immunoprotective device.

Year to date, shares of Vertex have rallied 20.4% compared with the industry’s rise of 3.5%.

Update on VRTX's Zimislecel Program

The company is currently developing zimislecel in the phase III portion of the ongoing phase I/II/III study for treating patients with T1D with SHEs and impaired awareness of hypoglycemia.

Enrollment and dosing in this study are expected to be completed later in the first half of 2025. Vertex plans to submit regulatory filings seeking approval for zimislecel in T1D in 2026.

Per management, the initial approval for zimislecel could serve around 60,000 people with severe T1D across the globe.

Zimislecel could benefit patients with recurrent SHEs despite the availability of other quality options in the United States and Europe.

VRTX to Stop Further Development of VX-264 in T1D

Vertex is evaluating VX-264 (cells + device) in a phase I/II study for treating T1D.

Enrollment and dosing in parts A and B of this study have been completed. Part B of this study consists of two primary endpoints – safety and change in peak C-peptide during a mixed-meal tolerance test (MMTT) from baseline at day 90.

The study failed to meet the efficacy endpoint as it did not show meaningful increases in C-peptide, a marker of insulin production, at levels required to deliver benefit.

Vertex has decided to stop further clinical development of VX-264 for treating T1D.

VRTX's Zacks Rank & Stocks to Consider

Vertex currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Jazz Pharmaceuticals, Inc JAZZ, Dynavax Technologies Corporation DVAX and Krystal Biotech, Inc. KRYS, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Jazz Pharmaceuticals’ earnings per share have increased from $22.06 to $23.33 for 2025. During the same time, earnings per share estimates for 2026 have increased from $23.13 to $23.35. Year to date, shares of JAZZ have risen 0.8%.

JAZZ’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.20%.

In the past 60 days, estimates for Dynavax’s earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share estimates have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have increased 1.5%.

DVAX’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%.

In the past 60 days, estimates for Krystal Biotech’s earnings per share have increased from $5.50 to $7.06 for 2025. During the same time, earnings per share estimates for 2026 have increased from $9.15 to $11. Year to date, shares of KRYS have rallied 15.1%.

KRYS’ earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.29%.

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