Ariad's ponatinib takes center stage with promising pivotal leukemia data

Ariad Pharmaceuticals ($ARIA) took center stage at the big American Society of Hematology (ASH) meeting over the weekend with interim pivotal data demonstrating that close to half of all chronic myeloid leukemia patients who had failed standard treatment had a "major" response to its experimental drug ponatinib. In what was widely heralded as a promising step toward a potential approval, 39% of the patients who responded to the drug experienced a complete remission. A "major" response was recorded when at least two thirds of their bone marrow returned to normal. The data build on Ariad's first look at the promising interim data back in November.

Ariad has big plans for ponatinib. The biotech plans to hold on to U.S. rights, planning an FDA filing by the middle of next year. And Reuters notes that the company plans to decide in the next few months whether it will line up a European partner. Ariad also plans to begin a study next year in a group of patients at an earlier stage of the disease.

There were some serious side effects noted in the study. Six percent of the patients developed pancreatitis and four of the subjects died. Investigators concluded that the deaths of the four patients with advanced leukemia and co-morbidities could be related to the drug. But the deaths weren't unexpected. "The side effect profile is every bit as good as other drugs in this class," Ariad Chief Executive Officer Harvey Berger told Reuters.

Analysts were clearly impressed with the positive data. Leerink Swann's Howard Liang called it a possible "highlight of the conference."

"This is a very hard disease to treat as patients become resistant to earlier lines of therapy," Berger told Bloomberg. "Even in these very advanced patients, we're seeing what most physicians would say is a very important and clinically beneficial response."

- read the Reuters story
- here's the Bloomberg piece

Special Report: Ponatinib - 10 promising late-stage cancer drugs