Vertex Pharmaceuticals has revealed ‘remarkable’ data from an early-stage study evaluating its stem-cell derived therapy VX-880 in type 1 diabetes (T1D).
In this study, a T1D patient received a single infusion of the therapy at half the target dose in combination with immunosuppressive therapy.
According to Vertex, this patient demonstrated successful engraftment as well as immediate and robust improvements across several key markers – such as increases in fasting and stimulated C-peptide as well as improvements in glycemic control and a reduction in the need for medical insulin.
In the year prior to VX-880 treatment, this patient had five severe and potentially life-threatening cases of low blood sugar.
“These results from the first patient treated with VX-880 are unprecedented,” said Bastiano Sanna, executive vice president and chief of Cell and Genetic Therapies at Vertex.
“What makes these results truly remarkable is that they were achieved with treatment at half the target dose. While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” he added.
Based on the early data, Vertex is now planning to continue progressing the Phase I/II programme for the therapy, with multiple active sites in the US. A clinical trial application has also been approved in Canada.