Vertex Pharma now has a pair of studies backing up its new drug VX-150, suggesting it can provide opioidlike pain relief without the addiction risks.
The positive phase 2 data comes right on the heels of the FDA approval for Vertex’s two-drug cystic fibrosis combination Symdeko and according to analysts at Jefferies is further evidence that a “new pipeline” is emerging at the Boston-based biotech beyond its core CF focus.
With the U.S. currently in the grip of an opioid addiction epidemic with an estimated 11.5 million people misusing these drugs at last count, it’s clear that the potential rewards for a company that brings a nonaddictive, potent painkiller to market would be off the charts.
In its phase 2 trial in 243 patients undergoing bunionectomy surgery, Vertex’s orally active drug—a sodium ion channel blocker targeting the NaV1.8 channel—outperformed placebo in alleviating pain using the Sum of the Pain Intensity Difference measure at 24 and 48 hours. It also seemed to provide a similar benefit to an active control of hydrocodone plus acetaminophen, although the study wasn’t powered to make a direct comparison between the active arms.
Vertex has previously reported encouraging phase 2 data in the treatment of osteoarthritis pain, showing significant pain relief compared to placebo in the 14-day trial. The new study is the first to assess the drug’s effects on acute pain, and Vertex says it will now start a phase 1 trial later this year of an intravenous formulation of the drug for acute pain indications.
The biotech is also waiting on the results of a third phase 2 trial of the oral formulation, this time looking at the drug’s effects on hard-to-treat neuropathic pain in subjects with small fiber neuropathy, which are due in early 2019.
Having two positive trials is “de-risking the asset and making it more valuable—and perhaps attractive for a future partner,” says Jefferies, which puts Vertex ahead of rivals such as Biogen among companies with pain drugs targeting voltage-gated sodium channels. The analysts also note that the biotech has started a phase 1 trial in healthy subjects of a second NaV1.8 inhibitor, VX-128, which seems to be more potent than VX-150.
While a lot of the buzz about Vertex is focusing on its CF therapies and particularly the start of pivotal trials for new three-drug regimens, the analysts think other portions of its pipeline are underappreciated. That includes not just VX-150 but also its CRISP/Cas9 program for sickle cell disease and beta thalassemia heading for the clinic, but also early-stage projects in AAT liver disease, muscular dystrophy, blood diseases and gene therapy.