The Food and Drug Administration recommended Blueprint Medicines change the ultimate goal of a midstage study on a drug for a rare disorder, leading BPMC stock to crumble Thursday.
Blueprint is testing its drug in patients with indolent systemic mastocytosis. With this disease, a type of cell that lives in connective tissues builds up. This causes a wide array of problems including itching, abdominal pain, bleeding disorders, bone or muscle pain or an enlarged liver. Patients with the indolent form of the disease have a low buildup, so their prognosis is positive.
However, the FDA suggested Blueprint analyze the total change in patients' symptoms. Previously, Blueprint planned to measure the number of patients who responded to treatment with at least a 30% reduction in symptoms, SVB Securities analyst Andrew Berens said in a note to clients. Blueprint will still measure the number of responders, but as a secondary goal of the study.
"Analysis of the delta in magnitude of responses rather than the number of respondents allows all enrolled patients, regardless of baseline disease burden, to contribute to the endpoint, providing results that would be more relevant to the entire patient population," he said.
On today's stock market, BPMC stock toppled 8.9% to 58.77.
BPMC Stock: Expanding Drug's Uses
Blueprint's drug, Ayvakit, is already approved in the advanced form of the same disease.
Further, early-stage testing looked promising, Berens said. In 2020, Blueprint said Ayvakit-treated patients showed a statistically significant decrease in symptoms compared to placebo recipients. But the new FDA requirement does create uncertainty in the test, he said.
"In our view, while we continue to believe in a positive outcome for the study, we note that this increase in granularity likely interjects more statistical uncertainty by considering the depth and variability of responses that are likely to be observed in each group," he said.
Berens kept his market perform rating on BPMC stock.
The news sent shares tumbling below their 50-day moving average, according to MarketSmith.com. BPMC stock has a middling Relative Strength Rating of 39 out of a best-possible 99, IBD Digital shows. This puts the stock's 12-month performance in the lowest 39% of all stocks.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
