Blueprint Medicines unveiled the "long-awaited" results of its study in indolent systemic mastocytosis on Wednesday, and the biotech stock crashed.
Indolent systemic mastocytosis is a condition in which a type of white blood cells, known as mast cells, build up in the body. This can lead to rashes, bone pain and diarrhea. On Wednesday, Blueprint said its drug, Ayvakit, met every goal in a pivotal-stage study.
However, the results paled from an earlier test and Ayvakit's benefit over standard drugs diminished in this test, SVB Securities analyst Andrew Berens said in a report to clients. He called the results of the study "long-awaited."
"While we think these data are approvable, they do represent a deterioration in the absolute improvement in the system score," he said. "Conversely, the control arm of best supportive care showed a larger reduction in total system score from baseline."
In response, biotech stock Blueprint plummeted 10.8% to 61.17 on today's stock market.
Biotech Stock Hammered On Results
Blueprint's Ayvakit is already approved for the aggressive form of systemic mastocytosis. The indolent disease is characterized by fewer mast cells and less severe symptoms.
After 24 weeks of treatment with Ayvakit, patients showed a 15.6-point reduction on a 110-point scale measuring symptoms. But an earlier study showed a 19.7-point reduction, Berens said. Patients who received standard treatments had a 9.2-point reduction in symptoms vs. 3.2 points in the Phase 1 test.
He kept his market perform rating on the biotech stock. But he questioned the ultimate market for the drug in patients with indolent disease. He expects Ayvakit to cost more than $350,000 per year.
"We think some physicians, payers and patients may balk at the anticipated cost given that Ayvakit appears to add about a 10% improvement in symptoms vs. best supportive care," he said.
More than nine in 10 patients experienced side effects. Serious side effects occurred in 5% of Ayvakit recipients vs. 11.3% of patients who received standard treatments. Bullishly for the biotech stock, few Ayvakit recipients dropped out of the study due to side effects.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
