Vertex: Positive Phase 2 Data in Third Proof-of-Concept with VX-150

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced positive results of a Phase 2 study of the investigational NaV1.8 inhibitor VX-150 in patients with pain caused by small fiber neuropathy. The study met its primary endpoint and showed that treatment with VX-150 demonstrated statistically significant and clinically meaningful pain reduction, as measured by the within-group change from baseline in the weekly average of daily pain intensity on the 11-point numeric rating scale (NRS) at Week 6. VX-150 was generally well tolerated in this study (see also Vertex Pharmaceuticals Incorporated).

This Phase 2 study in patients with small fiber neuropathy is the third positive proof-of-concept study for VX-150 and provides further validation of the potential role of NaV1.8 inhibition in the treatment of multiple pain conditions. A Phase 2b dose-ranging study of VX-150 following bunionectomy surgery is currently ongoing to support potential pivotal development. Additionally, the company is advancing multiple pain molecules through late-stage preclinical development and anticipates initiating clinical development with the first of these molecules in 2019.

“We are excited to now have three positive proof-of-concept studies that validate the potential role for NaV1.8 inhibitors to treat a variety of pain conditions,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “These results show a potential path for the treatment of pain at a time when there is great clinical and societal need for new medicines. We continue to progress VX-150 toward pivotal development and also plan to advance additional NaV1.8 pain medicines into clinical development beginning in 2019.” About the Phase 2 Study in Patients with Small Fiber Neuropathy The data announced were from a Phase 2, randomized, double-blind, placebo-controlled, 6-week study that evaluated treatment with VX-150 (n = 46) or placebo (n = 43) in patients with chronic pain caused by small fiber neuropathy. In this study, 1250mg of VX-150 was dosed orally every 24 hours. Efficacy Results The study met its primary endpoint, showing a statistically significant mean within-group change from baseline of -2.02 points for those treated with VX-150 in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6 (p<0.0001). The mean within-group change from baseline for those treated with placebo was -0.93.

A second pre-specified analysis compared those patients randomized to VX-150 or placebo. This analysis demonstrated a treatment difference in the mean change from baseline of -1.09 (95% CI: -1.88 to -0.29) in the weekly average of daily pain intensity on the 11-point NRS, as reported in the daily diary, at Week 6. This treatment difference was observed as early as Week 1, and was sustained through the six-week treatment period.

https://www.marketscreener.com/VERTEX-PHARMACEUTICALS-11321/news/Vertex-Pharmaceuticals-Announces-Positive-Phase-2-Data-in-Third-Proof-of-Concept-Study-with-the-Na-27802726/