Kyprolis™ (carfilzomib) for Injection Receives Accelerated Approval from FDA for Advanced Multiple Myeloma

Multiple Myeloma Research Foundation (MMRF) and Multiple Myeloma Research Consortium (MMRC) Played Integral Role in Clinical Development of First New Treatment for Multiple Myeloma in Five Years

NORWALK, Conn.--()--The Multiple Myeloma Research Foundation (MMRF) today announced that its partner, Onyx Pharmaceuticals, Inc., received U.S. Food and Drug Administration (FDA) approval for Kyprolis™ (carfilzomib) for the treatment of patients with multiple myeloma who have received at least two prior therapies, including Velcade (bortezomib) for Injection and an immunomodulatory agent, such as Thalomid (thalidomide) or Revlimid (lenalidomide), and have demonstrated disease progression on or within 60 days of completion of the last therapy. The approval of Kyprolis is based on response rate.

The MMRF’s affiliate organization, the Multiple Myeloma Research Consortium (MMRC), a clinical trials network of 16 leading multiple myeloma centers in North America, has bolstered the clinical development of Kyprolis for the past six years, providing clinical trial support and resources, including driving accrual to the pivotal Phase IIb trial (003-A1) that supported the accelerated approval of the treatment.

“The approval of Kyprolis is an immensely important milestone for the multiple myeloma patient community, which continues to face significant unmet need in terms of safe and effective treatments for advanced disease. While we have seen tremendous progress in the past decade, multiple myeloma remains incurable,” stated Kathy Giusti, Founder and CEO of the MMRF and MMRC and a multiple myeloma patient. “We commend the FDA and Onyx for their dedication to enabling patient access to Kyprolis, and are proud of our long-term and continued partnership with Onyx throughout the development of this critical new treatment.”

In 2006, the MMRF identified Kyprolis as a promising new treatment for advanced multiple myeloma during its development by Proteolix, Inc. (acquired by Onyx in 2009), and entered into a collaboration to support the therapy’s development. Since that time, the MMRF has provided integral support to advance its clinical development, including:

  • Facilitating early Phase I clinical trials and three Phase II trials with Kyprolis within the MMRC network, including: the pivotal Phase IIb trial (003-A1) that supported its approval by the FDA, the original Phase II trial (003), and the Phase II trial (004) in patients who had relapsed after one to three prior therapies. Today, three combination trials involving Kyprolis are ongoing within the MMRC, including a Phase II trial for newly diagnosed patients and two Phase I trials evaluating the drug in combination with other investigational agents.
  • Providing Onyx access to the MMRC network of clinical experts, securing trial investigators, supplying centralized contracts, providing guidance on site selection and protocol design, and accruing patients to ensure that clinical work was done in the most high-quality and efficient way possible to benefit patients.
  • Raising awareness and driving enrollment to ASPIRE, Onyx’s pivotal Phase III clinical trial with Kyprolis, which is now fully enrolled, and informing the entire multiple myeloma community on emerging data from clinical trials, involving Kyprolis.
  • Partnering with Onyx on the Carfilzomib Myeloma Access Program (C-MAP), an Expanded Access program that made carfilzomib available to hundreds of eligible patients in the U.S. before FDA approval.

“Over the past five years, through C-MAP and trials facilitated by the MMRC, in partnership with Proteolix and Onyx, the MMRF facilitated access to Kyprolis to more than 500 patients, many of whom had exhausted all available treatment options,” stated Walter M. Capone, Chief Operating Officer of the MMRF and MMRC. “Beyond these studies and programs, Onyx has shown a deep commitment to the multiple myeloma community through its extensive clinical development program. We are very much looking forward to the opportunity for a much broader group of patients, particularly those with the greatest unmet need, to access this breakthrough therapy.”

About the Multiple Myeloma Research Foundation (MMRF)

The Multiple Myeloma Research Foundation (MMRF) was established in 1998 as a 501(c)(3) non-profit organization by twin sisters Karen Andrews and Kathy Giusti, soon after Kathy's diagnosis with multiple myeloma. The mission of the MMRF is to relentlessly pursue innovative means that accelerate the development of next-generation multiple myeloma treatments to extend the lives of patients and lead to a cure. As the world's number-one private funder of multiple myeloma research, the MMRF has raised over $200 million since its inception and directs 90% of total budget to research and related programming. As a result, the MMRF has been awarded Charity Navigator’s coveted four-star rating for nine consecutive years, the highest designation for outstanding fiscal responsibility and exceptional efficiency. For more information about the MMRF, please visit www.themmrf.org.

About the Multiple Myeloma Research Consortium

The Multiple Myeloma Research Consortium (MMRC) is a 509(a)3 non-profit organization that integrates leading academic institutions to accelerate drug development in multiple myeloma. It is led from MMRC offices in Norwalk, Conn., and comprises 16 member institutions: Barbara Ann Karmanos Cancer Institute, Baylor Charles A. Sammons Cancer Center at Dallas, City of Hope, Dana-Farber Cancer Institute, Emory University's Winship Cancer Institute, the John Theurer Cancer Center at Hackensack University Medical Center, Mayo Clinic, Mount Sinai School of Medicine, Ohio State University, Sarah Cannon Research Institute, University Health Network (Princess Margaret Hospital), University of California-San Francisco, University of Chicago, University of Michigan, Virginia Cancer Specialists, and Washington University in St. Louis.

The MMRC is the only consortium to join academic institutions through membership agreements, customized IT systems, and an integrated tissue bank. For more information, please visit www.themmrc.org.

Contacts

MMRF
Anne Quinn Young, 203-536-8691
QuinnYoungA@themmrf.org

Release Summary

The Multiple Myeloma Research Foundation (MMRF) today announced that its partner, Onyx Pharmaceuticals, Inc., received U.S. Food and Drug Administration (FDA) approval for Kyprolis™ (carfilzomib).

Contacts

MMRF
Anne Quinn Young, 203-536-8691
QuinnYoungA@themmrf.org