Carfilzomib 

Carfilzomib is a next generation proteasome inhibitor that selectively targets the proteasome with minimal affinity for off-target proteases. To date, carfilzomib has generated a positive signal in multiple early-stage studies with an encouraging safety profile, including low rates of neuropathy, a known side effect of some approved multiple myeloma therapies.

Onyx’s lead proteasome inhibitor product candidate, carfilzomib, is being evaluated in multiple clinical settings.

An ongoing 250 patient Phase 2b trial in patients with relapsed and refractory multiple myeloma, known as the 003 trial, has completed enrollment with data anticipated in the second half of 2010 to support a potential new drug application (NDA) filing by year-end 2010.

As well, carfilzomib is being evaluated in a companion Phase 2 trial, known as the 004 trial, in patients with relapsed and/or refractory multiple myeloma who have relapsed after one to three, prior therapies.  For this trial, patients are divided into two populations:  patients with relapsed and/or refractory multiple myeloma who had not received prior bortezomib (Velcade®) treatment, or patients with relapsed and/or refractory disease following bortezomib treatment. 

Carfilzomib is also being evaluated in a Phase 1b/2 study in patients with solid tumors.

A Phase 1b/2 combination study of carfilzomib with lenalidomide and dexamethasone in patients with relapsed multiple myeloma, known as the 006 trial, has currently completed the dose escalation phase, achieving the safe combination of full dose carfilzomib with full dose lenolidomide and low dose once weekly dexamethasone.

A Phase 3 international randomized trial evaluating the efficacy of carfilzomib in combination with lenalidomide and dexamethasone versus lenalidomide and dexamethasone as a potential treatment option for patients with relapsed multiple myeloma is expected to begin in 2010.