ARIAD’s second oncology product candidate, ponatinib, is an internally discovered oral multi-targeted kinase inhibitor that we believe has broad potential applications in cancer. Ponatinib has fully enrolled its pivotal Phase 2 registration trial in patients with resistant or intolerant chronic myeloid leukemia (CML) and Philadelphia positive acute lymphoblastic leukemia (Ph+ ALL). The PACE (Ponatinb Ph+ ALL and CML Evaluation) trial is designed to provide definitive clinical data for regulatory approval in this setting. Read recent press release on ponatinib.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is the hallmark of CML and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). Ponatinib was designed using ARIAD’s computational and structure-based drug design platform to inhibit the enzymatic activity of BCR-ABL with very high potency and broad specificity. Ponatinib was intended to target not only native BCR-ABL, but also its isoforms that carry mutations that confer resistance to treatment with existing tyrosine kinase inhibitors, including especially the T315I mutation for which no effective therapy exists.