A Phase 1 clinical trial to evaluate oral ponatinib in patients with refractory hematological cancers, including patients with CML and AML is complete; patients are continuing to be observed. This multi-center, sequential dose-escalation study was designed to determine the safety and tolerability of oral ponatinib, as well as its pharmacokinetics (behavior of the drug in patients) and its pharmacodynamics (the effects of the drug on patients’ cells). Since all participants had well-characterized malignancies, the trial provided us with initial information describing ponatinib’s anti-tumor activity. We plan to explore the potential of ponatinib in specific solid tumors through additional clinical studies.
In December 2010, we announced updated clinical data from the ongoing Phase 1 study in patients with resistant and refractory CML. The data confirmed strong clinical evidence of hematologic, cytogenetic and molecular anti-cancer activity in heavily pretreated patients with CML, including those with the T315I mutation of the target protein, BCR-ABL. The updated results were presented at the 51st Annual Meeting of the American Society of Hematology (ASH). Full Press Release
In September 2011, updated long-term results of the Phase 1 study of ponatinib were presented at the international Chronic Myeloid Leukemia foundation (iCMLf) conference sponsored by the European Society of Hematology (ESH) at its 13th Annual Meeting held in Estoril, Portugal. With the trial fully enrolled and all patients evaluable, 72% of chronic-phase CML patients treated with ponatinib achieved a major cytogenetic response (MCyR), including 92% of patients who also had a T315I mutation. Full Press Release