Vertex Pharmaceuticals has announced that VX-548 met the primary endpoint in a Phase II dose-ranging trial in people with painful diabetic peripheral neuropathy (DPN).

The company has said that all dose groups treated with VX-548 had a statistically significant and clinically meaningful reduction in the primary endpoint of change from baseline on the Numeric Pain Rating Scale (NPRS) at week 12 in the trial (NCT05660538).

VX-548, an investigational oral selective NaV1.8 inhibitor, was generally well tolerated at all doses tested in the study with most adverse events (AEs) recorded as mild to moderate. There were no serious adverse events (SAEs) related to VX-548.

Patients were randomised to four treatment arms: VX-548 once daily at 69mg (high dose), 46mg (mid dose), or 23mg (low dose), and a comparator arm of pregabalin 100mg three times a day.

Vertex has plans to advance VX-548 into pivotal development following discussions with regulators. Vertex has also initiated a second Phase II study of VX-548 in peripheral neuropathic pain. Three Phase III studies of VX-548 in acute pain are on track to read out in Q1 2024.

According to GlobalData’s Pharmaceutical Intelligence Centre, Vertex have conducted 15 clinical trials with VX-548, one is planned, three are ongoing and 11 are completed. If approved, GlobalData predicts a global sales forecast of $582m in 2029.

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Statistical and meaningful improvement across all arms

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 arm mean change was -2.09.

All VX-548 dose groups had sustained a reduction in pain from week one, with pain continuing to decrease until week five. This was then maintained for the remainder of the study.

In the responder analysis, more than 30% of patients treated with VX-548 achieved ≥50% reduction in all dose groups, and more than 20% of patients in the mid- and high-dose groups achieved ≥70% reduction in the weekly average of NPRS at week 12.

In the pregabalin reference arm, 22% of patients achieved ≥50% reduction and 10% achieved ≥70% reduction in the weekly average of NPRS at week 12.

VX-548 was generally well tolerated with the majority of the AEs being mild or moderate in severity. The most common AEs were creatinine clearance decrease, dizziness, peripheral oedema and weight increase.

There were no SAEs related to VX-548 or pregabalin in the study. There was one death in the mid-dose VX-548 group due to atherosclerotic cardiovascular disease, which was not related to the study drug.