Vertex Pharmaceuticals reports results from Phase 2 study of VX-548

Pharmaceuticals announced positive results from its Phase 2 dose-ranging study of the selective NaV1.8 inhibitor VX-548 in people with painful diabetic peripheral neuropathy, or DPN. Treatment with all doses of VX-548 resulted in a statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline in the weekly average of daily pain intensity on a Numeric Pain Rating Scale, or NPRS, at Week 12. The study also included an active reference arm of pregabalin to support the evaluation of the VX-548 treatment effect. VX-548 was generally well tolerated at all doses tested in the study. Most adverse events, or AEs, were mild to moderate and there were no serious adverse events, or SAEs, related to VX-548. The study’s primary endpoint was change from baseline in the weekly average of daily pain intensity at Week 12 in patients with painful DPN dosed with VX-548 using the standard pain assessment Numeric Pain Rating Scale, or NPRS. This 11-point scale ranges from 0 to 10. Patients were randomized to four treatment arms: VX-548 once daily at 69 mg, 46 mg, or 23 mg, or the reference arm of pregabalin 100 mg three times per day, or TID, for 12 weeks. All VX-548 treatment groups showed statistically significant and clinically meaningful reductions from baseline in pain with mean change in NPRS at Week 12 of -2.26, -2.11 and -2.18 at the high, mid and low doses, respectively. The pregabalin reference arm mean change from baseline in NPRS at Week 12 was -2.09 and is provided for context. All VX-548 dose groups had sustained mean reductions in pain from baseline starting at Week 1, with pain continuing to decrease until Week 5, which was then maintained throughout the treatment period. Secondary and other endpoints were supportive of the study’s primary endpoint.