Vertex Pharma reports positive results from phase 1/2 study of VX-880 to treat type 1 diabetes

Vertex Pharmaceuticals presented new data from its phase 1/2 trial of VX-880, a stem cell-derived islet cell therapy, for type 1 diabetes (T1D) patients with severe hypoglycemia. The trial, involving 12 patients who received a full dose, showed consistent positive results, indicating VX-880’s transformative potential.

At baseline, patients had undetectable fasting C-peptide, recurrent hypoglycemic events, and required an average of 39.3 units of insulin daily. Post-infusion, all patients exhibited islet cell engraftment and glucose-responsive insulin production by Day 90. They achieved improved glycemic control,  with nearly all reducing or eliminating insulin use. All patients eliminated severe hypoglycemic events during the evaluation period.

VX-880 was generally well tolerated, with mostly mild or moderate adverse events. The therapy aims to restore glucose regulation by reintroducing pancreatic islet cell function. VX-880 has RMAT and Fast Track designations by the FDA and PRIME designation by the EMA. The study is expanding to approximately 37 patients.