Carfilzomib - 10 promising late-stage cancer drugs

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While Onyx Pharmaceuticals ($ONXX) has an approved kidney and liver cancer drug in Nexavar (sorafenib), much of the company's future value growth seems to be tied to its late-stage proteasome inhibitor carfilzomib. 

In multiple myeloma, carfilzomib could offer some advantages over existing treatments for the plasma cancer such as reduced nerve damage, according to Onyx. The drug works by selectively inhibiting a protein complex in cells called the proteasome in order to make cancer cells more vulnerable to cell death. In December the firm revealed some promising Phase II data, showing that 24 percent of patients who took the drug after their cancers failed to respond to other therapies had at least a partial response.   

Onyx is now conducting Phase III trials that aim to support its bids for approval of the drug for multiple myeloma in the U.S. and Europe. The company said it expanded its European trial in March, bringing the number of patients in the study from 84 to 300 and making overall survival the primary goal of the study rather than progression-free survival-a move that analysts at J.P. Morgan Research viewed as a positive because demonstrating survival benefits could differentiate the drug label among those of other myeloma treatments.

Onyx expects to complete submission of its application for FDA approval of the drug by mid-2011.