Onyx Pharmaceuticals Receives Fast Track Designation for Carfilzomib; Company Initiates Rolling NDA Submission for Accelerated Approval

EMERYVILLE, Calif., Jan. 31, 2011 /PRNewswire/ -- Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) announced today that the U.S. Food and Drug Administration (FDA) has granted fast track designation for carfilzomib, a next generation proteasome inhibitor for the potential treatment of patients with relapsed and refractory multiple myeloma. The Fast Track designation process was developed by the FDA to facilitate the development, and expedite the review of drugs to treat serious or life-threatening diseases and address unmet medical needs.  Positive complete results from the Phase 2b 003-A1 study evaluating single-agent carfilzomib in patients with relapsed and refractory multiple myeloma were recently announced at the American Society of Hematology (ASH) meeting in December 2010.

Based on these results, Onyx has initiated a rolling submission of a New Drug Application (NDA) for potential accelerated approval of carfilzomib in the U.S. Through the Fast Track designation, Onyx is eligible to submit the carfilzomib NDA on a rolling basis, allowing Onyx to begin the NDA filing process immediately and giving the FDA an opportunity to review the completed sections of the registration application. Onyx has now submitted the non-clinical section of the carfilzomib NDA with the FDA and commenced its rolling NDA process. Onyx intends to complete its submission of the NDA for potential accelerated approval of carfilzomib in the U.S. as early as mid-2011.

"The FDA's Fast Track designation recognizes the significant need for new treatment options for patients," said Ted Love, M.D., executive vice president, research and development and technical operations at Onyx. "We look forward to continuing to work with the FDA on the regulatory review and further development of carfilzomib, which has demonstrated encouraging response rates and tolerability in patients with advanced multiple myeloma. We are committed to making carfilzomib available to these very ill patients as quickly as possible."

Carfilzomib was granted orphan drug designation by the FDA for the treatment of multiple myeloma in 2008.  Orphan drug designation is granted to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the designation, the sponsor may be eligible for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA.

About the Carfilzomib Development Program

Carfilzomib is a selective, next generation proteasome inhibitor that has shown encouraging results in a broad clinical trial program in multiple myeloma.  

The carfilzomib development program includes a large, randomized international Phase 3 clinical trial, known as the ASPIRE trial, studying the combination of lenalidomide and low dose dexamethasone with or without carfilzomib in patients with relapsed multiple myeloma. The company has an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) and received Scientific Advice from the European Medicines Agency (EMA) on the design and planned analysis of the ASPIRE trial. A second Phase 3 clinical trial, called the FOCUS trial, is evaluating carfilzomib in patients with relapsed and refractory myeloma in Europe. Carfilzomib is also being evaluated in a broad investigator sponsored trial program including 1st line multiple myeloma, combination studies, lymphoma and other malignancies.

About Multiple Myeloma

Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow. In the United States, more than 50,000 people are living with multiple myeloma and approximately 20,000 new cases are diagnosed annually.(i) Worldwide, more than 180,000 people are living with multiple myeloma and approximately 86,000 new cases are diagnosed annually.(ii)

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 in various settings 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.

Forward Looking Statements

This news release contains "forward-looking statements" of Onyx within the meaning of the federal securities laws.  These forward-looking statements include without limitation, statements regarding the progress and results of the clinical development, safety, regulatory processes, including FDA review, and commercialization efforts or commercial potential of carfilzomib.  These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including risks related to the development, regulatory approval and commercialization of pharmaceutical products.  Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements.  Reference should be made to Onyx's Annual Report on Form 10-K for the year ended December 31, 2009, filed with the Securities and Exchange Commission under the heading "Risk Factors" and Onyx's Quarterly Reports on Form 10-Q for a more detailed description of such factors.  Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date of this release.  Onyx undertakes no obligation to update publicly any forward-looking statements to reflect new information, events, or circumstances after the date of this release except as required by law.  

(i)  National Cancer Institute, Surveillance Epidemiology and End Results, 2007 Facts and Figures

(ii)  International Agency for Research on Cancer, GLOBOCAN 2002 database

SOURCE Onyx Pharmaceuticals, Inc.