- Vertex Pharmaceuticals on Tuesday reported more positive clinical data for its most advanced pain drug, reinforcing the biotech's stance that its scientific capabilities go beyond cystic fibrosis.
- The drug, VX-150, significantly outperformed placebo at reducing pain caused by small fiber neuropathy. After six weeks of treatment, the Phase 2 study's experimental arm had a mean within-group change of -2.02 points, as measured by an 11-point scale, with regard to their weekly average of daily pain intensity. Conversely, the mean within-group reduction for the placebo arm was a little less than 1 point.
- Results also found the treatment generally well-tolerated. Six patients discontinued treatment due to adverse events — two from the experimental arm and four from the placebo arm — while all three cases of serious adverse events were seen in the latter group. Overall, adverse events occurred in 63% of patients with treatment versus 56% of patients on placebo.
Vertex is far-and-away the leader in cystic fibrosis drugmaking. Three approved therapies collectively raked in more than $2.1 billion this year, and the biotech is rapidly progressing with triple-combination regimens that executives expect will open 90% of the cystic fibrosis market to the company's portfolio.
But even as their confidence in Vertex grows, investors have pushed for more details on what the biotech's next targets will be.
CEO Jeff Leiden said in an earnings call at the beginning of the year that Vertex's first objective would be wrapping up its journey in cystic fibrosis — work that involves further development of triplets and gene therapies. Beyond that, the company has its hand in several therapeutic areas, including pain, hemoglobinopathies and a rare disorder known as alpha-1 antitrypsin deficiency that causes lung and liver problems, among other things.
In pain, VX-150 had already demonstrated positive effects through a pair of Phase 2 studies in patients with acute pain following bunionectomies and with osteoarthritis pain. Vertex contended in a Tuesday statement that the new data in small fiber neuropathy patients showcases the drug's strong efficacy across multiple pain conditions.
Vertex also touted how VX-150 works by inhibiting a type of sodium ion channel called NaV1.8 rather than through an opioid, an attribute executives argue will ultimately secure reimbursement even against inexpensive generics.
"A large part of the acute market has been genericized and, as you know, a large part of that market is actually opioids for acute post-surgical pain, post-dental pain, etcetera," Leiden said on Vertex's most recent earnings call in October, adding that VX-150 could help meet the high demand for pain medicines that work well but don't have the abuse potential of opioids.
"[W]e feel that VX-150 is a very, very competitive drug to a generic opioid and with ... reasonable pricing, that market is a multi-billion-dollar opportunity even if you only capture a portion of it," he said.
Moving forward, Vertex recently kicked off a Phase 2b dose-ranging study of VX-150 in patients with acute pain following bunionectomies, with the goal of later supporting pivotal development. The company is working on preclinical pain assets as well, and expects the first of these to enter the clinic next year.
Vertex shares opened Tuesday at $168.06 apiece, up a little more than 1% from close of market Monday.