Onyx Pharma expands Ph III carfilzomib study ahead of EMA filing

USA-based Onyx Pharmaceuticals (Nasdaq: ONXX) says it plans to expand the Phase III European clinical trial FOCUS, which is evaluating the efficacy and tolerability of carfilzomib, a selective next-generation proteasome inhibitor. The FOCUS trial is designed to support the registrational filing with the European Medicines Agency (EMA) in patients with relapsed and refractory myeloma.

Onyx acquired the drug along with its $851 million acquisition of US firm Proteolix (The Pharma Letter October 12, 2009). Onyx subsequently licensed Japanese rights to carfilzomib, as well as ONX 0912, to Ono Pharma for a total consideration of nearly $340 million (TPL September 9, 2010).

The FOCUS (CarFilzOmib for AdvanCed Refractory MUltiple Myeloma European Study) trial modification includes two key enhancements to the study: changing the primary end point to overall survival (OS) from progression-free survival (PFS) and correspondingly increasing patient enrollment to 300 from 84. These modifications are supported by overall survival data from the Phase IIb 003-A1 study evaluating single-agent carfilzomib in patients with relapsed and refractory multiple myeloma announced at the American Society of Hematology (ASH) meeting in December 2010. The company expects to review these data with the EMA and is moving forward with plans to do so.