Ariad's ponatinib scores well in key leukemia study

Ariad Pharmaceuticals' ($ARIA) key cancer drug ponatinib didn't disappoint at the ASCO meeting, with new data from the company's pivotal "PACE" trial trumping those revealed late last year. And now the Cambridge, MA-based drug developer is on track to turn in applications for U.S. and European approvals in the third quarter.

Cambridge, MA-based Ariad reported that 54% of leukemia patients on the targeted drug had a major cytogenetic response, besting the figure released during the ASH meeting in December. The pivotal trial involves more than 400 patients with chronic myeloid leukemia (CML) or Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL), who have built up resistance or have intolerance to the marketed kinase inhibitors dasatinib, sold as Sprycel by Bristol-Myers Squibb ($BMY), or Novartis' ($NVS) nilotinib (Tasigna) or who have the T315I mutation.

Seventy percent of patients on ponatinib with the T315I mutation had a major cytogenetic response, meaning that a certain percentage of patients' bone marrow tissue tested normal, up from 65% revealed in December, TheStreet's Adam Feuerstein reported. (Ariad aims to advance a genetic test to spot patients with the mutation via a partnership with a diagnostics company.) As Reuters reported, some of the side effects seen in the study were rashes, low platelet counts and dry skin. And 6% of patients suffered pancreatic inflammation.

"Clinical responses to ponatinib were observed in patients regardless of their mutation status or disease stage," Dr. Jorge Cortes, of the University of Texas MD Anderson Cancer Center, said in Ariad's press release. "Of particular importance, responses to ponatinib appear to be durable, with 93 percent of chronic-phase CML patients projected to remain in major cytogenetic response at one year, clearly highlighting the potency of ponatinib."

Ponatinib is perhaps the top drug in Ariad's pipeline, yet not the most advanced. The company is partnered with Merck ($MRK) on the experimental ridaforolimus, to which FDA advisers gave the thumbs-down as a new treatment for sarcomas in March.

- here's the release
- see Feuerstein's article
- and Reuters' report

Special Report: Ponatinib – Top 10 Late-Stage Cancer Drugs – 2012