Vertex’s pain drug succeeds in late-stage studies, marking major breakthrough in search for new, non-opioid medicines

Vertex’s pain drug succeeds in late-stage studies, marking major breakthrough in search for new, non-opioid medicines
Lei Lei Wu
News Reporter
An experimental non-opioid drug lowered pain levels in a series of pivotal studies, according to the drug’s developer Vertex Pharmaceuticals, which means a new, non-addictive type of pain medicine could soon be on the market.

In two Phase III studies that each had over 1,000 participants, those who received Vertex’s pain pill VX-548 after surgery experienced a greater reduction in pain over two days compared to those who received placebo. Participants in one study had an abdominoplasty, known otherwise as a tummy tuck, and those in the second had bunion surgery.

The data from the late-stage studies have been highly anticipated — if the FDA approves the drug, it would be the first new class of pain medicine to make it to market in over two decades, according to Vertex.

Paul Negulescu
“We’ve been waiting for these results for a very long time,” Vertex SVP of research Paul Negulescu told Endpoints News in an interview. “And this was the big study to indicate whether we had something that could be approved and brought to patients.

“My reaction was, ‘Wow, we’ve made it,’” he added.

The readout comes as drug overdose deaths have continued to match record levels in the US. Fatal overdoses claimed 106,000 lives over a 12-month period as of August 2023, according to the CDC, but the numbers are underreported.

Vertex’s two randomized studies were also designed to test whether VX-548 could beat hydrocodone plus acetaminophen, a common pain treatment combo that’s best known by the brand name Vicodin. While Vertex’s pain drug failed to beat the combo, it did show comparable efficacy over two days in the tummy tuck study, but not in the bunion surgery study, where the comparator arm won.

“This may complicate the prescribing decision in the acute setting, where patients often take a short course of treatment and efficacy is a primary consideration. The implications to chronic pain however — which is more important commercially — may be less significant,” Stifel analyst Paul Matteis wrote in a note Tuesday morning.

Vertex’s shares $VRTX fell about 4% in premarket trading.

In a separate, single-arm Phase III study for acute pain, Vertex also tested the drug in a variety of other surgical and non-surgical conditions for up to two weeks. “It’s important that this drug be used in both the extremely acute setting, such as the hospital, but also we think it’ll have a very important role for those discharged patients who will take something home to manage their pain after they’re released from the hospital,” Negulescu noted.

Vertex hopes to package the data from all three late-stage trials to win a broad label for moderate-to-severe acute pain from the FDA. The big biotech company plans to submit its application to the FDA in the first half of the year.

The company, which established itself with its cystic fibrosis medicines and in December won approval for a sickle cell disease gene therapy, has become one of the few major players developing new non-opioid drugs for pain as the field for years saw repeated failure and retreat from the likes of Biogen, Roche and Pfizer.

“We’ve been working on this approach of selective inhibition of pain signaling via NaV1.8 for over 25 years,” Negulescu added.

The goal of Vertex’s drug is to block a sodium channel called NaV1.8, which is found mainly in neurons that detect pain. Importantly, these channels are found in the peripheral nervous system — all the nerves that branch out to the rest of the body from the brain and spinal cord — but not the brain and spinal cord themselves, meaning that the drug isn’t expected to have addictive potential. Opioids, on the other hand, tap powerful reward centers in the brain, which makes them addictive.

Detailed data from studies will be presented at a future medical conference, according to Vertex.

Editor’s note: This story was corrected to reflect that Vertex’s pain drug showed different levels of efficacy in the two studies compared to hydrocodone plus acetaminophen. A previous version stated that the drug showed comparable efficacy in both studies.

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Lei Lei Wu
News Reporter
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