Vertex: FDA halts Phase 1/2 diabetes study

 -Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today provided updates on its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemia. Two patients in Part A received VX-880 at half the target dose. The first patient dosed in Part A of the study achieved insulin independence at Day 270, with a HbA1c of 5.2%. The second patient dosed in Part A has shown robust increases in fasting and stimulated C-peptide, and reductions in exogenous insulin requirements through Day 150. Taken together, the data from the first two patients in Part A established proof-of-concept for VX-880. Per the study protocol, the Independent Data Monitoring Committee reviewed the totality of the safety and efficacy data from the first two patients dosed in Part A of the study and recommended advancement to Part B, where patients receive the full target dose of VX-880. The first patient to receive the full target dose has achieved the Day 29 follow-up milestone.

Across the program, VX-880 has been generally well tolerated to date. There have been no serious adverse events (SAEs) considered related to VX-880. The majority of adverse events (AEs) were mild or moderate in all patients treated to date. The safety profile was generally consistent with the immunosuppressive regimen used in the study and the perioperative period.

The company also announced the VX-880 Phase 1/2 study has been placed on clinical hold in the U.S. by the Food and Drug Administration (FDA) due to a determination that there is insufficient information to support dose escalation with the product.

"We are surprised by the clinical hold placed on the study. The results from the first two patients treated with half the target dose establish proof-of-concept by demonstrating that VX-880 can restore glucose-regulated insulin production and improve glycemic control. Indeed, achievement of insulin independence by the first patient is a landmark milestone. Further, the totality of the safety and efficacy data for all three patients dosed to date gives us high confidence in our benefit-risk assessment of VX-880 and its potentially transformative profile," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "We are committed to working with the FDA to understand and address their questions, so that the trial can resume at U.S. sites as soon as possible."

https://finance.yahoo.com/news/vertex-provides-updates-phase-1-123000988.html