Vertex Pharmaceuticals has presented positive data from its ongoing Phase I/II clinical trial of VX-880 for the treatment of T1D.
VX-880 is an investigational stem cell-derived, fully differentiated islet cell therapy.
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The study enrolled patients with impaired hypoglycaemic awareness and severe hypoglycaemic events (SHEs).
This multicentre, open-label, single-arm study is designed to evaluate the safety and efficacy of VX-880.
The original study, now fully enrolled with 17 patients, has dosed 14 participants across Parts A, B and C, with the remaining set to receive their doses shortly.
At the study’s outset, all 14 dosed patients had undetectable fasting C-peptide levels, a history of recurrent SHEs, and an average daily insulin requirement of 39.3 units.
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By GlobalDataPost-infusion of VX-880, all patients showed islet cell engraftment and glucose-responsive insulin production by day 90.
The positive outcomes observed have led to an expansion of the study to include approximately 37 patients.
In the study, 12 patients who received the full dose in Parts B and C have all demonstrated improved glycaemic control, achieving the American Diabetes Association’s recommended targets for HbA1c and time-in-range.
Remarkably, nearly all participants (11 out of 12) had reduced or eliminated their need for exogenous insulin at their last visit. Additionally, all patients have seen an elimination of SHEs from day 90 onwards.
Three patients who have completed at least one year of follow-up have met the primary endpoint of SHE elimination with HbA1c below 7.0% and the secondary endpoint of insulin independence.
The treatment was found to be well tolerated with most adverse events being mild or moderate in nature.
No serious adverse events linked to VX-880 were reported, and the two patient deaths that occurred were deemed unrelated to the treatment.
Vertex Global Medicines Development and Medical Affairs executive vice-president and chief medical officer Carmen Bozic said: “These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for T1D.
“As we plan toward pivotal development, we are pleased to have secured regulatory approval to expand study enrolment and look forward to advancing this programme for patients who have long awaited a transformative therapy.”
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