Dive Brief:

  • Vertex Pharmaceuticals is moving an experimental pain drug into mid-stage testing after early studies showed it appears to be safe and takes effect in the body as intended.
  • The drug, known as VX-548, is meant to block a sodium channel called NaV1.8 that plays a role pain signaling. Vertex has been developing small molecule drugs that inhibit this channel as a way to create a new kind of pain treatment that doesn't have the addictive potential of opioids. So far, the company has looked at the impact of these drugs on several pain types, including acute, neuropathic and musculoskeletal.
  • For VX-548, which is currently the most advanced of Vertex's pain drugs, Phase 1 testing found that it has a "favorable profile" at lower doses than what was required with the company's earlier inhibitors. Vertex said the planned Phase 2 program will evaluate VX-548 in patients with both visceral and non-visceral types of acute pain.

Dive Insight:

Best known for its work in cystic fibrosis, Vertex has been trying to diversify both the drugs it makes and the markets it targets. The Boston-based company is now developing a wide range of experimental medicines — from a gene-editing therapy for blood disorders, to a cellular medicine for Type 1 diabetes, to small molecule drugs for an inherited form of kidney disease.

Vertex argues that programs like these will further establish it as a major player in specialty medicine. But the company's choices haven't always resonated with investors and analysts, with some questioning how big or impactful the programs could be.

Just last week, for example, Vertex agreed to hand over $900 million to its partner CRISPR Therapeutics in exchange for a 10% greater share of the potential profits on that experimental therapy for blood disorders. To Brian Skorney, an analyst at Baird, the agreement suggests Vertex sees the therapy as a $9 billion opportunity, which is a "steep" valuation, given some of the obstacles in its way.

"Notwithstanding the company's catchphrases of 'cracking the biology' and 'pouring on the chemistry' ... [there are] inconsistencies between their portfolio and their strategy," Geoffrey Porges, an analyst at SVB Leerink, wrote in a February note to clients following Vertex's most recent annual earnings report.

While doubts about the company's strategy may linger, Vertex maintains that it's committed to its areas of focus.

In pain, the company has been working to show that small molecule drugs can block the NaV1.8 channel and provide a non-opioid alternative for pain relief. Two of Vertex's earlier candidates, known as VX-961 and VX-150, ran into challenges during clinical testing, and development of them has since stopped.

VX-548, meanwhile, is the most recent of Vertex's channel-blocking agents to enter clinical testing. Having proved safe and tolerable in Phase 1 testing of healthy volunteers, the drug is now headed to Phase 2 "proof-of-concept" trials.

Carmen Bozic, Vertex's chief medical officer, said in a Monday statement that the company is pushing VX-548 forward "with urgency, as these studies are the key next step toward our ultimate goal of developing transformative medicines for the treatment of pain."

In the U.S., an opioid crisis has created an immense demand for new, non-addictive forms of pain management. According to the Centers for Disease Control and Prevention, opioids were involved in nearly 50,000 overdose deaths in 2019, representing more than 70% of all drug overdose deaths.

While Vertex and a few other large drug companies have their sights on developing better pain drugs, setbacks have been common. In one recent example, advisers to the Food and Drug Administration expressed concerns about the safety plans surrounding a closely watched experimental pain medicine from Pfizer and Eli Lilly. The advisers' feedback led some analysts to believe the medicine now has lower odds of approval.

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