MMRF PRESS RELEASES
MMRF Announces Launch of Carfilzomib Expanded Access Program for Patients with Relapsed and Refractory Multiple Myeloma in Partnership with Onyx Pharmaceuticals
Norwalk, CT — August 3, 2011
The Multiple Myeloma Research Foundation (MMRF) announced today that through an innovative collaboration with Onyx Pharmaceuticals, it has launched a new initiative to make carfilzomib available immediately through an expanded access program to eligible patients in the United States with relapsed and refractory multiple myeloma who have no available treatment options.
The Carfilzomib Myeloma Access Program (C-MAP), launched today, will enable eligible patients with relapsed and refractory multiple myeloma to receive access to carfilzomib while it is still being evaluated in Phase 3 clinical trials. Carfilzomib is an experimental, selective, next-generation proteasome inhibitor that has shown early promise in patients with relapsed and refractory multiple myeloma.
Working in partnership with Onyx Pharmaceuticals, the manufacturer of carfilzomib, the MMRF is helping to provide new options to patients with advanced multiple myeloma that are no longer responding to available therapies. C-MAP is the first expanded access program for multiple myeloma patients in nearly six years.
“The pre-approval access to carfilzomib is a tremendously important development for the myeloma community that would not be possible without a strong partnership with Onyx, the members of the Multiple Myeloma Research Consortium (MMRC), and other clinical partners,” said Kathy Giusti, Founder and Chief Executive Officer of the MMRF and a multiple myeloma patient. “We are proud of our role in driving this initiative forward on behalf of patients, who are in urgent need of expanded treatment options both today and over the long-term.”
The MMRF and MMRC have been involved in the development of carfilzomib for over 6 years, from early Phase 1 to the most recent Phase 2b studies conducted within the MMRC and at additional sites in the U.S. and Canada. To enable C-MAP, the MMRF developed the clinical platform for this expanded access program and will provide a range of ongoing support to ensure its successful completion, including patient and healthcare provider communications, study site coordination, data management and reporting, and overall project management.
About the Carfilzomib Myeloma Access Program (C-MAP)
Under C-MAP, approximately 40 medical centers across the U.S. will enroll patients with relapsed and refractory multiple myeloma for whom no satisfactory approved treatment alternatives are available and who do not qualify for an existing carfilzomib clinical trial.
The trial is a single-arm study, meaning carfilzomib is not being compared to another anti-cancer therapy. Refractory disease is defined as progression during therapy or within 60 days after completion of therapy. Onyx and the MMRF have made every effort to ensure geographical balance of sites to provide as convenient access as possible to patients.
Eligible patients should ask their physician to call Onyx Medical Information at 1-877-ONYX-1-2-1 (1-877-669-9121) or visit www.onyxtrials.com to learn if they are eligible for the trial.
Patients will receive carfilzomib on 28-day cycles until disease progression, unacceptable toxicity or until the drug is commercially available. Per the treatment protocol, safety and certain efficacy data will be collected.
Upon FDA approval and commercial availability, patients who are receiving carfilzomib through C-MAP will be transitioned to commercially available treatment. Patients without financial means to access carfilzomib commercially will be assessed for eligibility into Onyx assistance programs.
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer. The five-year relative survival rate for multiple myeloma is approximately 34 percent, one of the lowest of all cancers. In 2010, more than 20,000 adults in the United States were estimated to be diagnosed with multiple myeloma and approximately 11,000 people died from the disease.
About the Multiple Myeloma Research Foundation (MMRF)
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 $170 million since its inception to fund nearly 120 laboratories worldwide, including 70 new compounds and approaches in clinical trials and pre-clinical studies and has facilitated more than 30 clinical trials through its affiliate organization, the Multiple Myeloma Research Consortium (MMRC). As exceptional stewards of its donors' investments, the MMRF has been consistently recognized for its sound fiscal management. 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. 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, H. Lee Moffitt Cancer Center & Research Institute, Mayo Clinic, Ohio State University, Mount Sinai School of Medicine, 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 was founded in 2004 by Kathy Giusti, a myeloma patient, and with the help of the scientific community. The MMRC is a sister organization to the Multiple Myeloma Research Foundation (MMRF), the world's leading funder of multiple myeloma research. The MMRC is widely recognized as an optimal research model to rapidly address critical challenges in drug development and to explore opportunities in the today's most promising research areas in genomics, compound validation, and clinical trials. 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.
About Onyx Pharmaceuticals, Inc.
Onyx Pharmaceuticals, Inc. is a biopharmaceutical company committed to improving the lives of people with cancer. The company, in collaboration with Bayer HealthCare Pharmaceuticals Inc., is developing and marketing Nexavar® (sorafenib) tablets, a small molecule drug that is currently approved for the treatment of liver cancer and advanced kidney cancer. Additionally, Nexavar is being investigated in several ongoing trials in a variety of tumor types. Beyond Nexavar, Onyx has established a development pipeline of anticancer compounds at various stages of clinical testing, including carfilzomib, a selective proteasome inhibitor, that is currently being evaluated in multiple clinical trials for the treatment of patients with relapsed or relapsed/refractory multiple myeloma and solid tumors. ONX 0801, an alpha-folate receptor targeted inhibitor of thymidylate synthase, and ONX 0912, an oral proteasome inhibitor, are currently in Phase 1 testing. For more information about Onyx, visit the company’s website at www.onyx-pharm.com.
Eileen Regan, MMRF
Krystle Ficco, Feinstein Kean Healthcare
Lori Melançon, Onyx Pharmaceuticals