Clinical Development

Carfilzomib is a late-stage, next-generation proteasome inhibitor that is being developed as a potential treatment for patients with multiple myeloma and solid tumors.  To date, the agent has generated a positive efficacy signal in multiple clinical studies with an encouraging safety profile, including low rates of neuropathy.

A 266-patient Phase 2b trial in patients with relapsed and refractory multiple myeloma, known as the 003-A1 trial, generated positive data in a highly refractory and heavily pre-treated group of patients with multiple myeloma.  Based on these data, we have filed a new drug application (NDA), and it is currently in review with the U.S. Food and Drug Administration (FDA).

Carfilzomib is currently being evaluated in two Phase 3 clinical trials.  The first is a Phase 3 international randomized trial, known as the ASPIRE trial, evaluating the safety and efficacy of carfilzomib in combination with lenalidomide and low-dose dexamethasone as a potential treatment for patients with relapsed multiple myeloma.  The second, known as the FOCUS trial, is a Phase 3 study of single-agent carfilzomib in the relapsed and refractory setting and is designed to support a regulatory filing in Europe.  Our licensee, Ono Pharmaceutical Co., Ltd., has also initiated clinical studies with carfilzomib in Japan, expanding the international development footprint for carfilzomib.

In addition to the ongoing clinical studies in patients with multiple myeloma, carfilzomib is also being evaluated in a Phase 1b/2 study in patients with advanced solid tumors.