Vertex Pharmaceuticals The company announced positive data from an ongoing Phase I/II clinical trial of VX-880 for the treatment of type 1 diabetes.
VX-880 is an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy.
The study enrolled patients with impaired hypoglycemic awareness and severe hypoglycemic events (SHE).
This multicenter, open-label, single-arm study is designed to evaluate the safety and efficacy of VX-880.
In the initial study, 17 patients have now completed enrollment, with 14 participants having been vaccinated in parts A, B, and C, and the remaining participants expected to be vaccinated shortly.
At the start of the study, all 14 treated patients had undetectable fasting C-peptide levels, a history of recurrent SHE, and a mean daily insulin requirement of 39.3 units.
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Following VX-880 infusion, all patients demonstrated islet cell engraftment and glucose-responsive insulin production by day 90.
As positive results were observed, the study was expanded to include approximately 37 patients.
In this study, all 12 patients who received the full dose of Part B and Part C showed improvement in glycemic control and achieved American Diabetes Association-recommended goals for HbA1c and time in range.
Remarkably, nearly all participants (11 of 12) had reduced or no need for exogenous insulin at the time of their last visit, and furthermore, all patients were confirmed to be free of SHE after day 90.
The three patients who completed at least 1 year of follow-up achieved the primary endpoint of HbA1c <7.0% and elimination of SHE, as well as the secondary endpoint of insulin independence.
The treatment was found to be well tolerated, with most adverse events being mild or moderate.
No serious adverse events related to VX-880 were reported, and the two patient deaths that occurred were determined to be unrelated to treatment.
“These remarkable data further support the evidence that VX-880 is a potential treatment for type 1 diabetes,” said Carmen Bozic, executive vice president and chief medical officer, Vertex Global Medicines Development and Medical Affairs.
“As we move forward with our plans for this important development, we are pleased to have received regulatory approval to expand study enrollment and look forward to advancing this program for patients who have been waiting for a breakthrough treatment for so long.”
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