The big story at Cambridge, MA-based Ariad Pharmaceuticals ($ARIA) is its leukemia drug ponatinib, despite the fact that its lead experimental cancer drug is ridaforolimus (which is advancing in partnership with Merck). Ponatinib is now in a pivotal Phase II clinical trial as a treatment for patients with chronic myeloid leukemia or Philadelphia positive acute lymphoblastic leukemia whose cancers are resistant to Bristol-Myers Squibb's Sprycel or Novartis's Tasigna.
Ponatinib is designed to target multiple mutant forms of BCR-ABL fusion proteins, making it a potentially potent next-generation inhibitor of tyrosine kinases. In December the company reported that in a Phase I trial 66 percent of patients on the drug showed a major cytogenetic response, evidence that the drug was weakening their cancer (and such responses were shown in 100 percent of patients with a T315I mutation). The firm is interested in partnering on the development of ponatinib in certain markets outside of the U.S; so another pharma deal might be in the offing for the company.