A sign hangs in front of Vertex Pharmaceuticals’ global headquarters in Boston on October 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 reduced pain in an intermediate trial.
These positive results for diabetes patients suffering from chronic nerve disease support the biotech company’s hopes of developing a drug that can provide powerful pain relief without the addictive potential of opioids. Many other similar painkillers it never reached the market.
Analysts said the painkiller, called VX-548, could become a blockbuster drug if it wins regulatory approval, meaning its annual sales could top $1 billion.
Vertex said in a statement that it is “working urgently” to advance the drug into a late-stage trial, which would bring it one step closer to winning regulatory approval.
Vertex is also testing the drug in closely watched late-stage studies for acute pain, with data expected in the first quarter of next year. Acute pain is caused by injury, surgery, disease, trauma, or painful medical procedures.
VX-548 has the potential to be a multi-billion dollar product for both acute pain and chronic nerve pain in patients with diabetes, Vertex executives said on a call Wednesday.
Vertex stock closed 13% higher after the mid-stage test data was released. The company’s shares are up nearly 40% this year and rallied last week after US regulators approved the first gene-editing therapy for sickle cell disease from Vertex and its partner CRISPR Therapeutics.
The second phase tested the drug for 12 weeks in about 160 patients with diabetic peripheral neuropathy, a long-term complication of diabetes that damages peripheral nerves, such as those in the hands and feet, due to high blood sugar levels.
The condition can cause mild to debilitating pain, numbness and, in more severe cases, problems with digestion, bladder and heart rate control. One estimated 50% of the approximately 40 million patients in the US 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, medium, 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 side effects were mild or moderate.
The trial also followed a separate group of patients treated with pregabalin, a non-opioid treatment 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 painkiller Vertex show efficacy “at least in part” with pregabalin, noting Wednesday’s results “clearly support that.”
Fye also pointed out that patients appeared to have an easier time tolerating VX-548 compared to pregabalin in the trial. The rate of adverse events associated with treatment with the pain reliever Vertex was lower than that of pregabalin, he noted.
In a note on 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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