Carfilzomib has substantial single-agent activity despite several prior treatments with different combination regimensrfilzomib was well-tolerated with chronic administration, even in patients with renal insufficiency. Patients were able to remain on full-dose therapy for more than 12 cycles.

"These data support our ongoing carfilzomib program in multiple myeloma, a disease that has poor long-term survival, and for which there are no alternative courses of therapy for patients who relapse following treatment or become resistant to currently approved therapies," said Michael Kauffman, M.D., Ph.D., interim chief medical officer at Onyx. "There is a clear need to provide new treatment options to patients with multiple myeloma, and we are working to potentially file a New Drug Application for carfilzomib by the end of 2010."

Trial Design

This open-label, single agent ongoing Phase 2 study is being conducted in collaboration with the Multiple Myeloma Research Consortium, and is designed to enroll approximately 150 patients with relapsed and/or refractory multiple myeloma who have received 1-3 prior treatments. Patients included two populations: bortezomib-naive patients with relapsed and/or refractory multiple myeloma and bortezomib-treated patients with relapsed and/or refractory multiple myeloma. Prior therapies include alkylating agents, stem cell transplant, thalidomide, lenalidomide and anthracyclines, and bortezomib in the bortezomib-treated patients. To date, 73 evaluable bortezomib-naive patients (54 who received 20mg/m2 and 19 who received up to 27mg/m2), and 33 evaluable patients receiving previous treatment with bortezomib have been enrolled. The primary endpoint is overall response rate and secondary endpoints include TTP, DoR, overall survival and safety.