Monday, December 12, 2011

Ariad Blood-Cancer Drug’s 46% Success Rate May Prompt FDA Approval Boost

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By Ryan Flinn - Dec 12, 2011 9:46 PM GMT+0700

Ariad Pharmaceuticals Inc. (ARIA)’s experimental drug ponatinib spurred a positive response in 47 percent of chronic myeloid leukemia patients, a pivotal study the company said may help the medicine gain approval as a new therapy for people without treatment options.

The patients in the trial, the second of three usually required for U.S. regulatory approval, had an untreatable mutated version of the disease, or had failed to respond to Bristol-Myers Squibb Co.’s Sprycel, also known as dasatinib, or Novartis AG ‘s Tasigna, also known as nilotinib, according to the study released yesterday at the American Society of Hematology meeting in San Diego.

“This is a very hard disease to treat as patients become resistant to earlier lines of therapy,” Harvey Berger, chairman, president and chief executive officer of Cambridge, Massachusetts-based Ariad, said in an interview. “Even in these very advanced patients, we’re seeing what most physicians would say is a very important and clinically beneficial response.”

The trial included 449 patients, some of whom have a mutation called T315I, for which no drugs are currently approved. About 65 percent of the patients with the mutation had a major cytogenetic response to ponatinib, the goal for the study, meaning the drug had reduced their disease.

Ariad fell less than 1 percent to $11.61 at 9:45 a.m. New York time. The shares had more than doubled this year before today.

Ponatinib’s data “could be a highlight of the conference,” Howard Liang, an analyst at Leerink Swann & Co. in Boston, wrote in a note to clients last week before the study was released.

Ariad will submit the drug for regulatory approval in mid- 2012 based on the results, Berger said. The company may seek a partner to help develop the treatment by selling some of its international rights, though he said Ariad isn’t for sale.

Side Effects

In November, Ariad declined 8 percent, the most in a month, after reporting that 17 percent of patients in its ponatinib trial had serious side effects. Berger said the side effects were typical for patients with chronic myeloid leukemia, a slow and progression disease in which the bone marrow makes too many white blood cells.

The cancer usually occurs during or after middle age and will be diagnosed in about 5,150 people in the U.S. this year, killing 230, according to the National Cancer Institute.

About 6 percent of patients in the trial developed pancreatitis, although none dropped out because of it, the study said. Four patients died, “possibly or probably” related to ponatinib, according to investigators. The patients, all with advanced stages of the disease, died from pneumonia, fungal pneumonia, gastric hemorrhage and cardiac arrest.

The deaths “were likely linked to the advanced nature of their disease, including the myelosuppressive effects of ponatinib on already badly damaged bone marrows,” said Frank Haluska, Ariad’s chief medical officer, in a company statement.

“In all of the drugs to treat chronic myeloid leukemia, you see side effects,” Berger said. “What’s important is the drug is very well tolerated.”

To contact the reporter on this story: Ryan Flinn in San Francisco at

To contact the editor responsible for this story: Reg Gale at

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