Dive Brief:
- A new type of pain medication from Vertex Pharmaceuticals succeeded in a Phase 2 study of patients with diabetic peripheral neuropathy, boosting the company’s shares on Wednesday.
- Researchers tested three doses of the drug, dubbed VX-548, along with an approved treatment known as pregabalin that’s sold under the brand name Lyrica. Patients in all four groups reported statistically significant decreases in pain, Vertex said, with the company’s drug showing signs of effects comparable to Lyrica.
- There were no serious side effects related to drug treatment in any patients in the trial, which suggested some advantages for VX-548 in avoiding the dizziness, edema and weight gain sometimes seen with Lyrica. Still, a greater percentage of patients on the Vertex drug had a decrease in a marker of kidney function that will be closely watched in future studies.
Dive Insight:
In a pain market dominated by opioids and their associated addiction risks, the promise of a medicine that works in a new way is enticing. To date, the pharmaceutical industry’s attempts to find alternatives have fallen short.
The Phase 2 trial announced Wednesday is one of a range of ongoing studies in various indications for Vertex’s medication, which analysts have said could bring in as much as $6 billion in sales for chronic pain. The company also plans to release results from three Phase 3 studies of the drug in acute pain in the first quarter of next year.
In the latest study, researchers asked patients to rate their pain on a scale of 0-10. They found that patients given Lyrica had a mean decrease of 2.09 points in pain after 12 weeks, compared with 2.18, 2.11 and 2.26 in the three VX-548 arms. The trial wasn’t designed to directly compare VX-548 with Lyrica, but the similar decreases satisfied analyst expectations for the research.
In addition, Vertex said other measures in the study were encouraging. More than 30% of the patients given VX-548 had at least a 50% reduction in pain and more than 20% of patients given the two highest doses had a pain reduction of at least 70%. Those rates were lower in the Lyrica arm.
Even so, analysts said some questions remain. All of the patients in the trial knew that they would be getting an active treatment, so it’s impossible to measure the “placebo effect,” a common issue in pain drug studies. Also, the overall response among patients varied among the doses of VX-548 instead of increasing with each higher dose.
But pain studies are notoriously hard to conduct, because the problem is so subjective. One person’s 6 rating on the pain scale could easily be another person’s 4 rating. The overall takeaway is positive, analysts said, and Vertex shares rose 9% in trading Wednesday morning.
The drug is a key part of Vertex’s plan to branch out beyond its signature multibillion-dollar cystic fibrosis business. It’s already succeeded in other Phase 2 studies for patients suffering pain after bunion or stomach surgery, which prompted the company to move into Phase 3. Vertex said Wednesday it will also move onto the next stage of research for diabetic peripheral neuropathic pain.
