News Feature | December 12, 2014

Ariad Announces Safety And Efficacy Data For Ponatinib In Leukemia Trials

By Cyndi Root

Ariad Pharmaceuticals has announced safety and efficacy data from Phase 1 and 2 trials of Iclusig (ponatinib). The company announced the findings in press releases, stating that it is presenting the data at the annual meeting of the American Society of Hematology (ASH) in San Francisco, CA. The kinase inhibitor was Food and Drug Administration (FDA) approved in 2012 to treat chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Ariad is currently studying the agent for Ph+ ALL and CML patients with the T315I mutation.

Phase I and Phase II PACE Trials

Ariad announced in a press release that follow-up data on patients in Phase 1 and Phase 2 PACE trials of Iclusig showed meaningful clinical responses. Eleven of the 12 (92 percent) patients with the T315I mutation showed major cytogenetic response (MCyR) in the Phase 1 trial. In the Phase 2 trial, 46 of 64 patients showed MCyR (72 percent); a 75 percent rate overall. Ariad stated that safety was achieved by a careful benefit-risk evaluation, especially in patients at risk for arterial thrombotic events.

The company released further details on the Phase 1 trial in another press release, stating that 65 percent of patients in the Phase 1 trial had a complete cytogenetic response (CCyR) and 56 percent had a major molecular response (MMR). Moshe Talpaz, MD, from the University of Michigan Comprehensive Cancer Center, commented on the study, “With nearly six years of treatment for some patients in this study, ponatinib continues to demonstrate anti-leukemic responses in this heavily pre-treated patient population.”

In the Phase 2 trial, investigators followed patients a median of three years and found that the agent continued to demonstrate anti-leukemic activity. The company characterizes the responses as “deep and durable.” Patients who underwent a dose reduction achieved maintenance of response.


Iclusig (ponatinib) blocks proteins implicated in cancer cell proliferation. Patients take the tablet once daily to treat chronic, accelerated, and blast phases of CML and Ph+ ALL. In December 2013, the FDA issued a safety announcement regarding Iclusig. The federal agency warned of life-threatening blood clots and severe narrowing of blood vessels. The FDA asked Ariad to change the label, narrow the indication, and provide warnings on blood clots and blood vessel narrowing.