By Josh Nathan-Kazis
A new non-opioid pain pill from Vertex Pharmaceuticals performed no better than a placebo in patients with chronic nerve pain, but the company told investors Thursday it was moving the drug into a costly late-stage trial anyhow.
That threw investors for a loop. The stock was down 12% in early trading.
The drug, formerly known as VX-548 and now called suzetrigine, is one of the most-watched medicines across all the drug pipelines in the biotech sector. At best, a highly effective non-opioid pain pill would be a major advance for patients. But the long history of efforts to treat serious pain without resorting to dangerous and addictive opioids is littered with failures.
Early this year, Vertex said that suzetrigine had worked well in a trial in patients recovering from surgeries who needed short-term pain relief. Though the drug worked better than a placebo, it didn't work better than a commonly prescribed generic opioid.
The company is waiting on a Food and Drug Administration decision on whether to approve the drug to treat acute pain, or pain from a procedure or an injury that lasts just a few weeks. That decision is expected early next year.
It is in chronic pain, however, where experts see the larger opportunity. Generic opioids are inexpensive, and the risk of addiction in acute pain is lower. For chronic patients, who are at much greater risk of opioid addiction, an effective alternative would likely meet with enormous demand.
The news on Thursday, however, looked very bad for Vertex. The company tested suzetrigine in a Phase 2 study in patients with painful lumbrosacral radiculopathy, a long-lasting condition created by a pinched nerve in the spine that can cause pain in the back and leg.
Vertex said that patients who received the drug reported a 2.02-point reduction on a measure called the Numeric Pain Rating Scale. Patients on a placebo reported a virtually identical 1.98-point reduction.
Though the trial wasn't designed to measure the difference between the experimental arm and the placebo arm, the lack of any distinction between the two appeared to be a bad sign.
Vertex was undaunted. It said it would proceed to a late-stage trial of suzetrigine in painful lumbrosacral radiculopathy, despite the results.
Highly variable placebo responses are a common issue in pain medicine trials, given the subjective nature of pain and the inherent variability in how it is experienced and reported.
The company's CEO, Dr. Reshma Kewalramani, said it was "unexpected" that the pain reduction was similar in the two arms of the study.
"Our interpretation of this study... is that a high placebo response in this study led to a lack of separation of the suzetrigine and placebo response curves," she said on an investor call early Thursday. "We believe we can innovate in pain clinical trial design to better control the placebo effect, and in so doing, succeed in pivotal development with suzetrigine."
She said that the company would look to design its Phase 3 trial to control better for high placebo response.
In a press release, the company cited a "post-hoc analysis" that showed that some study sites had lower placebo response rates than others
Investors appeared unconvinced by Kewalramani's optimism. The stock opened Thursday at $391, and was on pace for its largest one-day percentage decrease in four years.
"Management sounds positive on this conference call... but we and many investors are debating as to why the Company would continue pumping money into suzetrigine, in a chronic setting," wrote Mizuho Securities healthcare trading desk strategist Jared Holz in a Thursday morning email to investors.
The company is also testing suzetrigine in another form of chronic neuropathic pain, called painful diabetic neuropathy. Vertex is hoping the FDA will approve suzetrigine to treat any form of neuropathic pain, rather than just one specific condition, but would likely need positive data in both painful diabetic neuropathy and painful lumbrosacral radiculopathy to get that nod.
Vertex, which has produced a highly successful set of cystic fibrosis drugs, is hoping the non-opioid pain pill can be its next chapter. Investors appeared to see that hope dimming on Thursday.
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
This content was created by Barron's, which is operated by Dow Jones & Co. Barron's is published independently from Dow Jones Newswires and The Wall Street Journal.
(END) Dow Jones Newswires
December 19, 2024 10:43 ET (15:43 GMT)
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