Vertex Pharmaceuticals announced Tuesday that the U.S. Food and Drug Administration has withdrawn its clinical hold on the company’s Phase I/II clinical trial of VX-880, a pancreatic islet cell replacement therapy for people with Type I Diabetes (TID).
The lift on the partial clinical hold will allow the trial to continue moving forward. The Phase I/II trial is a multi-center, single-arm, open-label study that will register approximately 17 T1D patients with impaired hypoglycemic awareness and severe hypoglycemia. It is designed to assess the safety and efficacy of VX-880 in three parts, with each section evaluating a different dose in patients. According to the most recent announcement, Vertex has dosed three patients in the study to date.
One patient who received half the target dose achieved insulin independence at day 270 of the study with an HbA1c of 5.2%. The second patient, who was given half the target dose, showed robust increases in fasting and stimulated C-peptide, an assessment of insulin production, and reductions in exogenous insulin requirements through day 150 of the study, Vertex said. Both patients moved on to the second part of the study, where they would receive the full target dose of VX-880.
Additionally, no serious adverse events were reported in those who had taken the drug and most adverse events reported were mild or moderate. VX-880 was well tolerated and had a safety profile consistent with the immunosuppressive regimen used in the study.
Read more here