ORLANDO — Vertex Pharmaceuticals said a third patient in a Phase 1/2 trial assessing its islet cell therapy as a treatment for type 1 diabetes has been fully insulin-independent for at least a year.
The company previously said that two patients enrolled in the study were no longer using insulin. A third went off insulin but later had to restart treatment.
On Friday, Vertex shared new data at the American Diabetes Association’s Scientific Sessions for the therapy, VX-880, for 12 patients who received the full dose, up from the six patients the company has previously reported data on. All 12 received a one-time single infusion of VX-880, with each one hitting recommended HbA1c levels. Out of the 12 patients, 11 have either reduced or stopped using insulin.
Based on these data, the company said it now plans to expand the study to 37 patients.
Felicia Pagliuca“The kind of results that we’ve seen with the cell therapy sets up the entire field for future therapeutic development based on this kind of technology,” Felicia Pagliuca, Vertex’s disease area executive for type 1 diabetes, told Endpoints News.
The primary endpoints of the trial are eliminating severe hypoglycemic events (SHEs), which occur when a patient needs assistance to recover from a severe low blood sugar event that can be life-threatening, and hitting recommended HbA1c levels. All 12 patients had no SHEs during the evaluation period, which started 90 days after the infusion. The secondary endpoint is independence from insulin.
Only three patients so far have at least 12 months of follow-up, which is needed to include them in the endpoint data. All of those patients met both endpoints.
Ten patients completed their 180-day check in. Of those 10, seven are no longer using insulin, and two are using 70% less insulin than before they enrolled in the study.
“What we’re seeing is that patients not only no longer require insulin, but that their ability to control blood sugar is significantly improved simultaneously,” Pagliuca said. “For any person with type 1 diabetes, treatment with insulin is essential, so no one could actually stop taking insulin unless there was some other source, and clearly, in this study, the source of that insulin is coming from these infused cells.”
There were no serious adverse events related to VX-880, and the majority of adverse events were mild or moderate in nature. Earlier this year, there were two patient deaths in the study, and Vertex temporarily paused it. The company has since said the deaths were not treatment-related.
Pagliuca said the company is “eager to engage with regulators to figure out exactly what data we need to get to registration for VX-880.”
Vertex is already preparing to manufacture its type 1 diabetes candidates. In April, it announced that it had paid $25 million upfront to TreeFrog Therapeutics for its cell manufacturing technology.
Despite its efficacy, there are still challenges to treating patients with this kind of therapy. Patients have to be on chronic immunosuppressive therapy to stop their own immune systems from destroying the islets implanted by the infusion.
Vertex is in the early stages of developing a fix for that: VX-264, which is currently in Phase 1/2 development, is designed to not need immunosuppressants. The trial is set to wrap up in May of 2026, according to ClinicalTrials.gov.