Onyx Pharmaceuticals (Onyx) has reported updated safety data from the pivotal Phase 2b 003-A1 study, known as the 003 trial, demonstrating that Carfilzomib is well-tolerated in heavily pre-treated relapsed and refractory multiple myeloma patients.
Reportedly, the data were presented at the ASH/ASCO Joint Symposium at the 51st annual meeting of the American Society of Hematology (ASH) in New Orleans. Enrollment in the trial is complete, and full results, expected in 2010, could support a potential new drug application (NDA) filing by year-end 2010.
The company said that based on the emerging safety profile, the 003-A0 protocol was amended in 2008 to permit increased dosing of up to 27mg/m. The protocol was also expanded into the 003-A1 Phase 2b trial, enrolling 269 patients with relapsed and refractory myeloma and a dose escalation from 20mg/m to 27mg/m after one cycle.
The primary endpoint for the Phase 2b 003-A1 pivotal study is overall response rate, and secondary endpoints include clinical benefit response, duration of response, progression-free survival, time-to-progression, overall survival and safety.
Sundar Jagannath, lead investigator, chief of the Multiple Myeloma Program, said: “These results show that Carfilzomib is well-tolerated and can be administered at a full dose over a long period of time even in a very sick patient population for whom all available treatment options have been exhausted and who have multiple comorbidities. We look forward to the full data from this trial next year.”