Clinical trials
In 2010 ARIAD announced result from a Phase I study of ponatinib in patients with resistant and refractory chronic myeloid leukemia (CML) and Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL). The study demonstrated that in chronic-phase CML patients treated with ponatinib, 66 percent of patients in the trial achieved a major cytogenetic response, including 100 percent of patients who also had a T315I mutation.The PACE (Ponatinib Ph+ ALL and CML Evaluation) pivotal phase II trial started enrolling patients in September 2010 and is designed to provide definitive clinical data for regulatory approval in this setting. Good results were reported in December 2012.The EPIC (Evaluation of Ponatinib versus Imatinib in CML) phase-III trial began in June 2012  and was halted on October 18, 2013. Mechanism of action
The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is the hallmark of CML and Ph+ ALL. CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to more aggressive phases such as accelerated or blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.
Cost, coverage and availability
[US] Oncologists have complained that many patients cannot afford the "astronomical" cost of $138,000 a year, which makes it one of the most expensive drugs in medicine, and [in their view] far more expensive than what is needed to pay the development costs.In 2015 ponatinib is available in England for the treatment of CML (chronic phase, accelerated phase or blast phase) and Ph+ ALL in patients with documented T315I mutation under the Cancer Drugs Fund, and has not been appraised by the National Institute for Health and Care Excellence (NICE), who noted the small expected patient population. NICE estimated that ponatinib would cost approximately £61,000 per year, but the price paid under the Cancer Drugs Fund is confidential and may be different. See also
Bcr-Abl tyrosine-kinase inhibitor, especially the sections on Ponatinib development
 and binding

References
External links
"Ponatinib". Drug Information Portal. U.S. National Library of Medicine.