Recent studies show promise in islet cell therapy for type 1 diabetes
Vertex announce positive results from ongoing trial of VX-880 for the treatment of type 1 diabetes presented at the American Diabetes Association (ADA) 84th Scientific Sessions.
Vertex Pharmaceuticals Incorporated recently presented new data from its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes.
The treatment is designed for people living with type 1 diabetes reporting impaired hypoglycaemic awareness and experiencing severe hypoglycaemic events.
Researchers presented latest study reports at an invited talk, entitled Update on clinical trials using stem cell replacement, during the American Diabetes Association 84th Scientific Sessions Conference on 21st June 21, in Orlando, Florida, as part of the joint ADA/IPITA symposium.
The presentation included updated figures on 12 patients who received the full dose as a single infusion in Parts B and C of the trial are consistent with previously reported positive results in the VX-880 trial and reinforce the transformative potential of this therapy.
All patients in the study had undetectable fasting C-peptide (a marker of endogenous insulin secretion), a history of recurrent severe hypoglycaemic events in the year prior to screening, and required an average of 39.3 (min, max; 19.8, 52.0) units of insulin per day.
Following a single infusion of VX-880 at the full dose, all 12 patients demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90.
At the latest visit, all patients had improved glycaemic 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.
In addition, during the evaluation period, all patients reported no further severe hypoglycaemic events.
Finally, all three patients who had at least one year of follow-up, and are therefore evaluable for the primary endpoint, met the primary endpoint of elimination of severe hypoglycaemic events (from Day 90 after infusion) with HbA1c less than 7.0% and the secondary endpoint of insulin independence.
Studies found that VX-880 has been generally well tolerated.
Researchers said majority of adverse events were mild or moderate, and there were no serious adverse events related to VX-880 treatment.
In previous tests two patient deaths were reported, however, both were unrelated to treatment with VX-880.
Researchers added: “The safety profile of the treatment is generally consistent with the immunosuppressive regimen used in the study, the infusion procedure, and complications from long-standing diabetes.”
Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, said: “These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for type 1 diabetes.
“As we plan toward pivotal development, we are pleased to have secured regulatory approval to expand study enrolment and look forward to advancing this program for patients who have long awaited a transformative therapy.”
Piotr Witkowski, M.D., Ph.D., 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, said: “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.
“The marked improvements seen across several key glycaemic measures, the elimination of severe hypoglycaemic episodes, and the reduction or total elimination of reliance on exogenous insulin have the potential to fundamentally change the treatment landscape for type 1 diabetes and alleviate the significant burden this condition carries for patients.”
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