Promising new data from a trial of Vertex’s early phase islet cell therapy VX-880 for type 1 diabetes (T1D) trial showed three patients, with 12 months of follow-up, reached insulin independence. All of the dozen patients in the trial showed improvement over all—they received the full dose of VX-880 and demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90.
VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.
The incidence of type 1 diabetes is estimated at 15 per 100,000 people worldwide.Vertex says VX880 can repair the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production. VX-880 has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations by the U.S. FDA, and PRIME designation by the European Medicines Agency.
“These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for T1D,” said Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex. “As we plan toward pivotal development, we are pleased to have secured regulatory approval to expand study enrollment and look forward to advancing this program for patients who have long awaited a transformative therapy.”
In the trial all 12 patients demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90. At the latest visit, all patients had improved glycemic control and achieved ADA-recommended targets for both HbA1c below 7.0% and time-in-range above 70% on continuous glucose monitoring. Nearly all participants (11 of 12) had a reduction or elimination of exogenous insulin use at their last visit. All patients had elimination of SHEs)from Day 90 onward.
“The data we’ve seen to-date in this study are extremely exciting. Stem cell-derived islets regulate blood glucose control as well as natural human islets,” said Piotr Witkowski, MD, PhD, professor of surgery and director, Pancreatic, and Islet Transplant Program, University of Chicago Medicine, one of the investigators on the study, and a member of Vertex’s VX-880 Steering Committee. “The marked improvements seen across several key glycemic measures, the elimination of severe hypoglycemic episodes, and the reduction or total elimination of reliance on exogenous insulin have the potential to fundamentally change the treatment landscape for T1D and alleviate the significant burden this disease carries for patients.”
These data were presented during the American Diabetes Association 84th Scientific Sessions Conference on June 21, 2024, in Orlando, Florida at an invited talk, “Update on Clinical Trials Using Stem Cell Replacement,” as part of the joint ADA/IPITA symposium from 3:45–5:15 p.m. ET.
The clinical trial is a Phase I/II, multi-center, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. Based on the positive data shown to date, the study has been expanded to approximately 37 patients.
