ARIAD Pharmaceuticals recently announced the publishing of results from a phase I dose-escalation study of ponatinib in the New England Journal of Medicine. Ponatinib, a tyrosine kinase inhibitor, is being developed for use in heavily pretreated patients, suffering from resistant and refractory chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
ARIAD enrolled 81 patients with resistant hematologic cancers for the study. Of these, 60 patients had CML and five of them were suffering from Ph+ ALL. After 72 weeks, 72% of the chronic-phase CML patients (31 of 43), who enrolled for the study, experienced a major cytogenetic response (MCyR).
ARIAD reported some dose-limiting toxicities in the study, which included elevated lipase or amylase levels and pancreatitis. The study also had adverse events, such as rash, thrombocytopenia and arthralgia among others.
We note that ponatinib is currently under US Food and Drug Administration (FDA) review for patients suffering from resistant or CML or Ph+ ALL. A decision on ponatinib is expected from the US regulatory body by March 27, 2013.
In August this year, ARIAD submitted a Marketing Authorization Application (MAA) for ponatinib for the same indication to the European Medicines Agency (EMA). ARIAD had requested accelerated assessment of the MAA for the candidate, which was granted by the Committee for Medicinal Products for Human Use (CHMP).
ARIAD also intends to commercialize ponatinib beyond Europe and the US. In August this year, the company initiated a multi-center phase I/II clinical trial with ponatinib in Japan, one of the largest CML markets in the world.
The Japanese trial is evaluating ponatinib in patients with CML, who have failed treatment with Bristol-Myers Squibb Company’s (BMY - Free Report) Sprycel (dasatinib) or Novartis’ (NVS - Free Report) Tasigna (nilotinib) or with Ph+ ALL, who have failed treatment with tyrosine kinase inhibitors.
We currently have a Neutral recommendation on ARIAD. The stock carries a Zacks #3 Rank (Hold rating) in the short run.