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
“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
Ariad fell less than 1 percent to $15.63 at the close in
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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