Vertex's diabetes cell therapy spurs insulin production in a half-dozen patients

Vertex could be “leading the path to insulin independence," according to analysts, after another batch of data showed that the off-the-shelf diabetes cell therapy stimulated insulin production in all evaluable patients, and that two were now insulin independent. 

The pharmaceutical company reported that all six evaluable patients dosed in a phase 1/2 trial testing VX-880 “demonstrated endogenous insulin secretion,” according to a release issued late Friday evening. All six patients also were found to have improved blood sugar levels and reduction or elimination of external insulin control. All patients with at least 90 days of follow-up had no reported severe hypoglycemic events. 

Two patients that had at least one year of follow-up since their last infusion are insulin-independent and had 95% time-in-range when under continuous glucose monitoring. They also both had eliminated severe hypoglycemic events between 90 days and one-year post-treatment and a reduction in blood sugar levels, hitting the study’s primary endpoint. Vertex reported that VX-880 has so far been well-tolerated with no serious side effects arising from treatment.

Vertex’s Type 1 diabetes disease area head, Felicia Pagliuca, Ph.D., said in a release that the data were “a foundation advance.” Trevor Reichman, M.D, a surgeon at the University of Toronto, called the data “truly remarkable.” 

Analysts at Evercore said VX-880, “continues to deliver impressive efficacy in more patients with longer follow-up.”

The latest results further validate the potential displayed in Vertex’s previous data releases. Back in October 2021, Vertex surprised with a small batch of data that showed one patient was producing insulin after treatment with VX-880.

Vertex is already off to races with newer generations of the drug, including VX-264, a similar therapy that’s delivered via a channel array device to avoid immunosuppressants. The FDA approved the treatment’s first phase 1 trial earlier this year and recruitment began at the end of May, according to the clinical trial record

And Vertex recently locked arms with CRISPR Therapeutics in a new partnership to combine the latter’s gene editing tech to a new crop of diabetes treatments. CRISPR is already working with Vertex-acquired ViaCyte on a couple of clinical-stage diabetes treatments, including VCTX210A, which just completed an open-label phase 1 study, according to an update to the trial record posted Monday. The company said in a statement to Fierce Biotech that the trial was "largely a safety study, and we saw no major safety concerns." The asset doesn't appear to be a part of Vertex's future plans, however. 

"We’re focused on advancing the next-generation program, VCTX-211, in addition to our own VX-880 and VX-264 programs," according to the statement. 

Editor's note: This story was updated with a statement from Vertex regarding the clinical development of VCTX210A.