Ariad Pharmaceuticals, Inc. Announces Publication of Ponatinib Phase 1 Clinical Trial Results in the New England Journal of Medicine
Published: Nov 29, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced publication of results from the Phase 1 study of ponatinib, its investigational, tyrosine kinase inhibitor in heavily pretreated patients with resistant and refractory chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The paper was published today in the New England Journal of Medicine (Vol. 367, No. 22, Pages 2075-88). The article is accompanied by an editorial by John M. Goldman, D.M., F.R.C.P. of the Department of Haematology, Imperial College London (Pages 2148-49). The Phase 1 dose-escalation study of ponatinib enrolled 81 patients with resistant hematologic cancers, including 60 patients with CML and five patients with Ph+ ALL. With median follow-up at 73 weeks, 72 percent of patients (31 of 43) with chronic-phase CML enrolled in the study had a major cytogenetic response (MCyR), including 92 percent (11 of 12) who had the T315I gatekeeper mutation, which is the most common mutation among resistant patients. It was estimated that 89 percent of patients with chronic-phase CML who had a MCyR would remain in response at 1 year (95% confidence interval, 69% to 96% by Kaplan–Meier analysis). Of 22 patients with accelerated-phase or blast-phase CML or Ph+ ALL, 36 percent (8 of 22) had a major hematologic response, and 32% (7 of 22) had a MCyR.