Verification test for non-opia painkiller validation test ends the program for critical research
A Vertex Pharmaceuticals drug, seen as the successor to a landmark FDA-approved non-opia pill earlier this year, failed to conduct mid-term clinical trials, set a setback to the company's strategy to establish a pain drug franchise to diversify its revenue stream.
According to clinical trial results, the company announced after the market shutdown on Monday that VERTEX will not push the drug VX-993 to a Phase 3 test as a monotherapy for acute pain.
Boston-based apex pain approach focuses on blocking sodium channels in the peripheral nervous system that delivers pain signals. By blocking these peripheral signals, this approach aims to avoid the risk of addiction that opioids create targets in the central nervous system.
VX-993 is designed to block a sodium channel called NAV1.8, the same goal as Journavx, the two daily painkillers approved by Vertex in January. Using VX-993, Apex is developing formulas for oral and intravenous administration. Version IV offers the potential for higher doses and provides an alternative to intravenous opioids. The company also hopes that VX-993 can become part of future combination therapy, as well as a drug that follows different sodium channels NAV1.7.
The results reported on Monday were from a placebo-controlled dose range study that evaluated the oral version of VX-993 in patients with acute pain following Banwenctomy. 367 study participants were evaluated based on a scale used to measure pain intensity. The results show that the numerical values of the scores at medium and high doses are not sufficient to be statistically significant.
“This proof-of-concept study has the ability to test whether VX-993 will be more clinically effective than the NAV1.8 pathway than previously demonstrated,” Carmen Bozic, executive vice president of global drug development and medical affairs and chief medical officer of Vertex, said in a prepared statement. “Based on these results, as well as the total number of preclinical data and the results of our previous Bunionsctomy clinical study, VX-993 is not expected to outperform our existing NAV1.8 inhibitors, so we will not advance as a monotherapy in acute pain.”
According to reports, the 2-stage results of VX-993 occur simultaneously with Vertex's second-quarter 2025 financial results. Vertex said in the report that its discussion with the FDA suggests that regulators currently have no pathway to expand the Journavx tag to peripheral neuropathy. Therefore, the apex does not begin the Phase 3 test of the pill in lumbar radiation disease, a common lower back pain condition, also known as sciatica.
The company said it will prioritize the development of journals for diabetes peripheral neuropathy; the 2nd issue of research is underway. The phase 2 test of VX-993 is the recruitment of patients with diabetic peripheral neuropathy. Vertex hopes to complete the enrollment rate of two interim tests by the end of 2026. The program is a key test to push Journavx toward diabetic peripheral neuropathy. Vertex said over time it will continue to work with the FDA to expand indications for diabetic peripheral neuropathy to include other neuropathic pain conditions and to evaluate potential methods to obtain a broad label in this type of pain.
Since Vertex launched Journavx in early March, the company has reported over 110,000 prescriptions written and filled in products in hospital and retail settings for different types of acute pain. The company also said nearly 150 million people covered access to the product, representing nearly half of our lives. Vertex reported that in the first half of 2025, Journavx earned $13.3 million.
Photo: David L. Ryan/Boston Globe, via Getty Images
