- The FDA has instituted a clinical hold on Vertex Pharmaceuticals Incorporated's (NASDAQ:VRTX) VX-880 Phase 1/2 study due to a determination that there is insufficient information to support dose escalation with the product.
- VX-880 is an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for type 1 diabetes (T1D).
- Three patients have been dosed in the Phase 1/2 study with VX-880. Two patients received half the target dose of cells in Part A of the study.
- A third patient has received the full target dose in Part B of the study.
- The first patient achieved insulin independence at Day 270, with an HbA1c of 5.2%. The second patient has shown robust increases in fasting and stimulated C-peptide and reductions in exogenous insulin requirements through Day 150
- The third patient received the full target dose of VX-880 and has reached the Day 29 follow-up visit.
- Early data show increasing fasting C-peptide and improving glycemic control through Day 29.
- Across the program, VX-880 has generally been well-tolerated to date. There have been no serious adverse events (SAEs) related to VX-880. Most adverse events (AEs) were mild or moderate in all patients treated to date.
- Patient 3 had one SAE of neutropenia, which was not considered related to VX-880.
- Price Action: VRTX shares are down 5.74% at $257.55 during the market session on the last check Monday.
