Vertex claims success on its non-opioid pain drug, pushing candidate into pivotal trials
Vertex may soon have three irons in the fire in its bid to repeat the scientific and financial success of its blockbuster cystic fibrosis medicines.
The company on Thursday released positive results from a pair of Phase II trials for its non-opioid program, announcing that the experimental pill significantly reduced patients’ pain compared to placebo in two post-operative trials. One trial looked at patients who had just received a bunionectomy, the other in patients who received abdominoplasty — the medical term for a “tummy tuck.”
Now, Vertex executives said they will push the drug into pivotal trials later this year. That will likely make it the third program in the Vertex pipeline to move into Phase III trials, after its sickle cell and beta-thalassemia gene therapy and a treatment for genetically driven kidney disease.
Although none have quite the sales potential of CF, Vertex argues they can each be curative or disease-modifying in areas — like non-opioid pain relief — where progress has historically been slow.
“Our high expectations of achieving therapeutic pain control … have been met with these results from the two acute pain studies,” said Vertex CMO Carmen Bozic. “The remarkable consistency in the safety, tolerability and efficacy results in these two studies demonstrates the potential of VX-548 to be a first-in-class non-opioid treatment for acute pain.”
Pain, however, is a notoriously difficult area for drug development, in part because of the profound placebo response many patients can experience. And the Phase II trials left at least some question about how well it will perform in Phase III trials.
The company said that within 48 hours of surgery, bunionectomy patients who received the high dose of the Vertex pill experienced significantly less pain than patients who received placebo. The same was true on tummy tuck patients in the high and mid-level dose, when compared to tummy tuck patients on placebo.
But one clear sign researchers look for in interpreting early studies is whether there’s a dose response: Do patients experience more benefit as the dose increases?
In the bunionectomy trial, there wasn’t. Bunionectomy patients who received the mid-level dose actually experienced more pain than those on placebo. And bunionectomy patients who received the lowest dose saw some improvement over placebo, although it was not statistically significant.
At the same time, few companies have two statistically significant trials before entering Phase III trials. And it marks a milestone in a more than decades-long industry-wide effort to develop alternatives to opioids by targeting nerve receptors called Nav1.8 or Nav1.7 — a place where Pfizer, Roche and Biogen have all failed.
“While there are nuances that imply some outstanding clinical risk (i.e. pain is hard, only the high dose separated), ultimately we feel confident saying this mechanism works,” Stifel’s Paul Matteis wrote in a note to investors.