Vertex Pain Pill Could Replace Opioids. It’s an Opportunity for Investors.

Americans filled roughly one opioid pain prescription for every two U.S. adults in 2020—despite a raging opioid epidemic, where 80,000 people a year fatally overdose. Everyone knows the drugs are dangerous, but patients need pain relief, and the alternatives are limited.

Late this year or early next, the company plans to announce the results of four studies of a pain drug it calls VX-548. Three of the studies are testing VX-548 taken over a short period as a treatment for acute pain, while a fourth aims to measure the efficacy of the drug when taken over a longer duration for chronic pain.

Some scientists who study pain, however, aren’t yet convinced by VX-548. They say that while the need for the drug is clear, its efficacy isn’t. In an editorial published in the New England Journal of Medicine in August, Dr. Mark S. Wallace, a professor in the department of anesthesiology at the University of California, San Diego, wrote that the methodology of early trials of VX-548 make it difficult to gauge how effective the drug is at reducing pain.

But Wallace points to potential problems with the trial, suggesting that the opioid used may not have been given frequently enough, and noting that patients were allowed to take ibuprofen along with the medicines being tested but that data on how they used the ibuprofen weren’t presented.

Newsletter Sign-up A Vertex spokesperson told Barron’s that both the tummy-tuck and bunion-removal trials were “unambiguously” successful. The spokesperson said the Phase 2 trials weren’t designed to compare the effect of VX-548 with the effect of the opioid, but that the continuing Phase 3 trials are.

But nonopioid pain drugs have a history of disappointing investors. The most recent example, nerve growth factor inhibitors, were plagued with safety issues: Pfizer (PFE) and Lilly got as far as a Food and Drug Administration rejection of their version of the drug before dropping it in 2021, while Regeneron Pharmaceuticals (REGN) killed its version late last year, despite some positive Phase 3 data.

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