ARIAD Pharmaceuticals (ARIA - Free Report) recently announced positive data from the pivotal PACE study on ponatinib. ARIAD is evaluating the candidate for use in heavily pretreated patients, suffering from resistant and refractory chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
The data from the study showed that 57% of accelerated-phase CML patients, including 50% of patients with the T315I mutation, were successful in achieving a major hematologic response (MaHR). We note that MaHR was the primary objective of the PACE study with patients in an advanced stage of the disease.
Ponatinib is currently under US Food and Drug Administration (FDA) review for patients suffering from resistant or CML or Ph+ ALL. The FDA’s response should be out 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). Both the US and the EU application of the candidate were based on positive data from the PACE trial.
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.
We remind investors that companies like Novartis (NVS - Free Report) already have a presence in the CML market.
We expect investor focus to stay on the FDA’s decision on the approval of the candidate. We currently have a Neutral recommendation on ARIAD. The stock carries a Zacks #3 Rank (Hold) in the short run. Other pharma stocks like Infinity Pharmaceuticals (INFI - Free Report) and Questcor Pharmaceuticals carry a #2 Rank (Hold).