Ariad Pharmaceuticals Inc. (ARIA) said its experimental leukemia drug ponatinib helped patients with a mutation resistant to other therapies in a study the company will use to seek approval from U.S. and European regulators.
The trial showed 54 percent of patients with chronic myeloid leukemia experienced a reduction in abnormal cells, tested using the bone marrow to determine the drug’s effect, the Cambridge, Massachusetts-based company said in a statement. The data were presented today at the American Society of Clinical Oncology meeting in Chicago.
The study of about 450 patients included those with chronic myeloid leukemia or another form of blood cancer, called Philadelphia-positive acute lymphoblastic leukemia, for whom Bristol-Myers Squibb Co. (BMY)’s Sprycel and Novartis AG (NOVN)’s Tasigna didn’t work or who have a particularly tricky genetic mutation called T315I. No drugs on the market have shown to work for people with that mutation, said Harvey Berger, Ariad’s chief executive officer.
“There are probably well over 50 mutations that occur clinically in patients with CML,” Berger said in a telephone interview. “Ponatinib works against every one of them.”
The medicine also showed a “favorable” safety profile, the company said. The most-common side effects were thrombocytopenia, or low platelets, rash, dry skin, abdominal pain and headache. Six percent of patients developed pancreatitis.
Ariad fell less than 1 percent to $15.63 at the close in New York.
The company said it expects to file for approval with the U.S. Food and Drug Administration and European Union regulators in the third quarter of this year.
“I can’t find anyone who says it’s not going to get through the FDA,” said Phil Nadeau, an analyst with Cowen & Co. in New York, before the data were released. He estimates ponatinib may draw $350 million in 2016 revenue, and $2.5 billion in annual worldwide sales before it loses patent protection in 2026.
The National Cancer Institute estimates that 5,430 people in the U.S. will be diagnosed with chronic myeloid leukemia in 2012, and that 610 people will die from the disease.
Earlier data on the medicine were presented in December at the American Society of Hematology meeting in San Diego.
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