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 is not going to advance the drug, VX-993, to Part 3 testing as a monotherapy for treating acute ache, the corporate introduced after Monday’s market shut.
The ache strategy 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 strategy 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 known as NaV1.8, the identical goal as Journavx, Vertex’s twice-daily ache capsule authorised in January. With VX-993, Vertex was creating each oral and intravenously administered formulations. The IV model had supplied the potential for increased dosing and for offering a substitute for 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 examine that evaluated an oral model of VX-993 in sufferers who’ve acute ache after bunionectomy surgical procedure. The 367 examine contributors have been assessed in keeping 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 examine was powered to check whether or not VX-993 would lead to increased medical efficacy than beforehand demonstrated with the NaV1.8 pathway,” Carmen Bozic, govt vp, international medicines growth and medical affairs, and chief medical officer at Vertex, stated in a ready assertion. “Primarily based on these outcomes, in addition to the totality of preclinical information and outcomes from our earlier bunionectomy medical research, VX-993 just isn’t anticipated to be superior to our present NaV1.8 inhibitors and due to this fact we is not going to be advancing it as monotherapy in acute ache.”
The Part 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 at the moment. Consequently, Vertex is not going to a begin a Part 3 take a look at of the capsule in lumbosacral radiculopathy, a standard decrease again ache situation also called sciatica.
The corporate stated it is going to prioritize growth of Journavx in diabetic peripheral neuropathy; a Part 2 examine is ongoing. A separate Part 2 take a look at of VX-993 is enrolling sufferers with diabetic peripheral neuropathy. Vertex expects to finish enrollment in each mid-stage exams by the tip of 2026. The plan is to advance Journavx to a pivotal take a look at in diabetic peripheral neuropathy. Vertex stated it is going to proceed to work with the FDA to increase the diabetic peripheral neuropathy indication over time to incorporate different neuropathic ache circumstances and to evaluate potential methods to safe a broad label in the sort of 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 stated practically 150 million folks have coated entry to the product, representing practically half of U.S. coated lives. Vertex reported $13.3 million in income from Journavx for the primary half of 2025.
Picture: David L. Ryan/The Boston Globe, through Getty Photographs
