Vertex nonopioid painkiller posts constructive midstage trial outcomes

A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston on Oct. 23, 2019.

Brian Snyder | Reuters

Shares of Vertex Pharmaceuticals jumped Wednesday after the company’s painkiller, which is being tested as an alternative to opioids, significantly decreased pain in a midstage trial.

Those positive results for diabetes patients suffering from a chronic nerve condition support the biotech company’s hopes to develop a drug that can provide strong pain relief without the addictive potential of opioids. Plenty of other similar painkillers never reached the market.

Analysts have said that the painkiller, called VX-548, could become a blockbuster drug if it wins approval from regulators, meaning its annual sales could exceed $1 billion.

Vertex said in a release that it is “working with urgency” to advance the drug to a late-stage trial, which would bring it one step closer to winning approval from regulators. 

Vertex is also testing the medication in closely watched late-stage studies for acute pain, with data due in the first quarter of next year. Acute pain is caused by injury, surgery, illness, trauma or painful medical procedures. 

VX-548 has the potential to be a multibillion-dollar product for both acute pain and the chronic nerve pain in diabetes patients, Vertex executives said in a call Wednesday. 

Vertex’s stock closed 13% higher following the release of the midstage trial data. Shares of the company are up nearly 40% this year and got a boost last week after U.S. regulators approved the first-ever gene-editing therapy for sickle cell disease from Vertex and its partner CRISPR Therapeutics

The phase-two trial tested the drug over 12 weeks in roughly 160 patients with diabetic peripheral neuropathy, a long-term complication from diabetes that damages peripheral nerves, such as those in the arms and legs, due to high blood sugar levels. 

The condition can cause mild to debilitating pain, numbness and, in more severe cases, issues with digestion, bladder and heart rate control. An estimated 50% of the roughly 40 million U.S. patients with diabetes have some peripheral neuropathy. 

The trial specifically measured pain intensity using an 11-point scale, with 10 being the “worst pain imaginable.” High, mid and low doses of the drug reduced average pain intensity by 2.26, 2.11 and 2.18 points, respectively.

The company said the drug was generally well-tolerated, and that the majority of adverse events were mild or moderate.

The trial also followed a separate group of patients treated with pregabalin, a nonopioid therapy approved nearly two decades ago to block nerve pain and treat seizures. Pregabalin reduced average pain intensity by 2.09 points over 12 weeks. 

JPMorgan analyst Jessica Fye said investors likely wanted to see Vertex’s painkiller show efficacy “at least on part” with pregabalin, noting that Wednesday’s results “clearly support that.”

Fye also highlighted that patients appeared to have an easier time tolerating VX-548 compared to pregabalin in the trial. The rate of adverse events related to treatment with Vertex’s painkiller was lower than that of pregabalin, she noted.

In a note Wednesday, Jefferies analyst Michael Yee wrote that the data overall “looks at least as good as or better than investor expectations.”

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