A Vertex Prescription drugs drug considered as a successor to a non-opioid capsule that gained a landmark FDA approval earlier this yr has failed a mid-stage medical trial, dealing a setback to the corporate’s technique of constructing a ache drug franchise to diversify its income streams.
Primarily based on the medical trial outcomes, Vertex won’t advance the drug, VX-993, to Section 3 testing as a monotherapy for treating acute ache, the corporate introduced after Monday’s market shut.
The ache method of Boston-based Vertex focuses on blocking sodium channels, pathways within the peripheral nervous system that transmit ache alerts. By blocking these alerts within the periphery, this method is meant to keep away from the dependancy dangers posed by opioid medication that hit targets within the central nervous system.
VX-993 is designed to dam a sodium channel referred to as NaV1.8, the identical goal as Journavx, Vertex’s twice-daily ache capsule accredited in January. With VX-993, Vertex was creating each oral and intravenously administered formulations. The IV model had provided the potential for greater dosing and for offering an alternative choice to IV opioid drugs. The corporate additionally hoped VX-993 could possibly be a part of a future mixture therapies alongside a drug that goes after a special sodium channel, NaV1.7.
The outcomes reported Monday are from a placebo-controlled dose-ranging research that evaluated an oral model of VX-993 in sufferers who’ve acute ache after bunionectomy surgical procedure. The 367 research contributors had been assessed based on a scale used to measure ache depth. Outcomes confirmed numerical enchancment in scores for the center and excessive doses, however not sufficient to be statistically important.
“This proof-of-concept research was powered to check whether or not VX-993 would lead to greater medical efficacy than beforehand demonstrated with the NaV1.8 pathway,” Carmen Bozic, government vp, world medicines improvement and medical affairs, and chief medical officer at Vertex, mentioned in a ready assertion. “Primarily based on these outcomes, in addition to the totality of preclinical knowledge and outcomes from our earlier bunionectomy medical research, VX-993 isn’t anticipated to be superior to our current NaV1.8 inhibitors and due to this fact we won’t be advancing it as monotherapy in acute ache.”
The Section 2 outcomes for VX-993 had been reported concurrent with Vertex’s launch of second quarter 2025 monetary outcomes. In that report, Vertex mentioned its discussions with FDA indicated the regulator doesn’t see a path to broaden Journavx’s label to peripheral neuropathy presently. Consequently, Vertex won’t a begin a Section 3 check of the capsule in lumbosacral radiculopathy, a typical decrease again ache situation also called sciatica.
The corporate mentioned it would prioritize improvement of Journavx in diabetic peripheral neuropathy; a Section 2 research is ongoing. A separate Section 2 check of VX-993 is enrolling sufferers with diabetic peripheral neuropathy. Vertex expects to finish enrollment in each mid-stage checks by the tip of 2026. The plan is to advance Journavx to a pivotal check in diabetic peripheral neuropathy. Vertex mentioned it would proceed to work with the FDA to increase the diabetic peripheral neuropathy indication over time to incorporate different neuropathic ache situations and to evaluate potential methods to safe a broad label in any such ache.
Since Vertex launched Journavx in early March, the corporate has reported greater than 110,000 prescriptions written and stuffed for the product, in each hospital and retail settings, for several types of acute ache. The corporate additionally mentioned almost 150 million individuals have coated entry to the product, representing almost half of U.S. coated lives. Vertex reported $13.3 million in income from Journavx for the primary half of 2025.
Photograph: David L. Ryan/The Boston Globe, by way of Getty Photographs
