July 26, 2010

MMRC Plays Critical Role in Advancing Development of Carfilzomib, Kathy Giusti, CEO

Despite incredible odds of bringing a new treatment to market — just 11 new cancer drugs have been approved by the FDA in the last three years – multiple myeloma patients may have another treatment option available by the end of next year, thanks in part to the efforts of the Multiple Myeloma Research Consortium (MMRC) and its Member Institutions.

Earlier today, Onyx Pharmaceuticals announced promising top-line results from the Phase II trial of carfilzomib, a next-generation proteasome inhibitor in the same class as Velcade®, in patients with relapsed and refractory multiple myeloma. We could not be more proud that the MMRC played such a critical role in advancing this potential new treatment, particularly because carfilzomib shows promise in treating even the most heavily pre-treated patients, including those whose disease no longer responds to neither Velcade® nor Revlimid®.

Over the last four years, the MMRC has worked tirelessly to fast-track the development of carfilzomib:

  • We first introduced Proteolix in 2006 to our experts from the MMRC, and then streamlined the start of the trial by providing the company with access to centralized contracts and guidance on site selection and protocol design — saving the company critical time and resources.
  • When Onyx acquired Proteolix late last year, our strong leadership ensured that the trial's momentum continued and that research efforts were not delayed.
  • MMRC Member Institutions enrolled a majority — 60 percent — of patients into the trial while representing just over one-third of the sites. This was essential in completing the trial's enrollment, often the most time-consuming part of the drug development process.

In the results announced today, an incredible 36 percent of patients demonstrated at least a minor response to carfilzomib. These same patients had previously relapsed following a median of five types of multiple myeloma treatments — representing 13 different agents — and did not respond to their last treatment. Based on these impressive results in a population that has so few treatment options available outside of clinical trials, Onyx is in continuing discussions with the FDA, and anticipates submitting for potential accelerated approval by the end of the year. This means that patients would have access to carfilzomib before the Phase III clinical trial is complete.

As a patient, I am grateful to Onyx for demonstrating such remarkable commitment to the myeloma community and to all of our MMRC Members and the patients who participated in this trial. Final results from the trial are expected by year-end — stay tuned!