Ponatinib: Updated Phase I data

Ariad reported updated data from 74 patients in an ongoing, open-label, dose-escalation, U.S. Phase I trial. In 38 evaluable patients with chronic phase CML, ponatinib produced 16 major molecular responses (MMR), 7 of which were in patients with the T315l variant of BCR-ABL tyrosine kinase. Ponatinib also produced a major cytogenetic response (MCyR) rate of 66%, with 53% of patients achieving a complete cytogenetic response (CCyR). In 17 evaluable patients with accelerated phase, blast phase or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL), 24% of patients achieved MCyRs.

Complete hematologic responses were observed in 95% of chronic phase CML patients. In 9 evaluable chronic phase CML patients with the T315l variant of BCR-ABL tyrosine kinase, ponatinib led to both a complete hematologic response and a MCyR in all patients, including 8 patients with a CCyR. In 29 evaluable chronic phase CML patients without the T315l variant of BCR-ABL tyrosine kinase, ponatinib produced an MCyR rate of 55%. Additionally, 35% of patients with accelerated phase, blast phase or Ph+ ALL achieved a major hematologic response. Patients received 2, 4, 8, 15, 30, 45 or 60 mg/day oral ponatinib. Data were presented at the American Society of Hematology meeting in Orlando. ...