A Vertex Prescribed drugs drug considered as a successor to a non-opioid capsule that received 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.
Based mostly 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 indicators. By blocking these indicators within the periphery, this method is meant to keep away from the dependancy dangers posed by opioid medicine 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 accepted in January. With VX-993, Vertex was growing each oral and intravenously administered formulations. The IV model had provided the potential for larger dosing and for offering a substitute for IV opioid drugs. The corporate additionally hoped VX-993 might be a part of a future mixture therapies alongside a drug that goes after a distinct 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 members have been assessed in line with 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 vital.
“This proof-of-concept research was powered to check whether or not VX-993 would end in larger medical efficacy than beforehand demonstrated with the NaV1.8 pathway,” Carmen Bozic, govt vice chairman, world medicines improvement and medical affairs, and chief medical officer at Vertex, stated in a ready assertion. “Based mostly on these outcomes, in addition to the totality of preclinical information and outcomes from our earlier bunionectomy medical research, VX-993 is just not anticipated to be superior to our present 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 have been reported concurrent with Vertex’s launch of second quarter 2025 monetary outcomes. In that report, Vertex stated its discussions with FDA indicated the regulator doesn’t see a path to broaden Journavx’s label to peripheral neuropathy right now. Consequently, Vertex won’t a begin a Section 3 check of the capsule in lumbosacral radiculopathy, a standard decrease again ache situation also called sciatica.
The corporate stated it’ll 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 assessments by the top of 2026. The plan is to advance Journavx to a pivotal check in diabetic peripheral neuropathy. Vertex stated it’ll proceed to work with the FDA to broaden the diabetic peripheral neuropathy indication over time to incorporate different neuropathic ache circumstances and to evaluate potential methods to safe a broad label in one of these ache.
Since Vertex launched Journavx in early March, the corporate has reported greater than 110,000 prescriptions written and crammed for the product, in each hospital and retail settings, for various kinds of acute ache. The corporate additionally stated almost 150 million individuals have lined entry to the product, representing almost half of U.S. lined lives. Vertex reported $13.3 million in income from Journavx for the primary half of 2025.
Photograph: David L. Ryan/The Boston Globe, through Getty Pictures
