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Ponatinib – Top 10 Late-Stage Cancer Drugs – 2012

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Developer: Ariad Pharmaceuticals
Lead indication: Chronic myeloid leukemia
Key date: NDA expected in Q3

Ariad Pharmaceuticals' ($ARIA) CEO Harvey Berger has set his company on a course to move from a developer to a provider of targeted cancer drugs, and leading the way in the company's pipeline is ponatinib. The drug has won over analysts and investors during its development, as punctuated last year with kudos for interim pivotal data and a recent $258 million stock offering.

With the proceeds of the stock sale, Berger seems confident that his company can take the drug to market as a new treatment for chronic myeloid leukemia (CML) without a big partner to foot the bill. Ponatinib could become the Cambridge, MA-based company's first targeted cancer drug to win an approval--especially in light of an FDA advisory panel's rejection of its experimental sarcoma drug partnered with Merck ($MRK) last month. The FDA action date for the drug, ridaforolimus, is June 5.

Ponatinib is now in the pivotal PACE trial in patients with (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ALL) with resistance to previous therapies. Ariad plans to have data from the studies packaged for review and submitted to U.S. and European regulators this summer. Interim data at the American Society of Hematology (ASH) meeting in December showed that almost half of CML patients in the PACE trial had a "major" response to the drug, building confidence on Wall Street that the company is on track to meet its ambitious goals for the program.

Berger wants Ariad ready to commercially launch ponatinib in the first quarter of next year, and the company is already laying the foundation necessary for leading sales and marketing efforts. On the clinical side of the house, the company is advancing plans to test the drug as a first line of attack against CML, expanding on the use in treatment-resistant patients.