Onyx Pharmaceuticals Announces Carfilzomib Development and Commercialization Agreement with Ono Pharmaceutical for Japan

EMERYVILLE, Calif., Sept. 8 /PRNewswire-FirstCall/ -- Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced that it has entered into an exclusive agreement with Ono Pharmaceutical Co., Ltd. to develop and commercialize two compounds from Onyx's proteasome inhibitor development program, carfilzomib and ONX 0912. Under the terms of the agreement, Ono has exclusive rights to develop and commercialize both compounds for all oncology indications in Japan. Onyx retains commercialization rights in other countries in the Asia Pacific region, as well as in all other regions of the world, including the U.S. and Europe. The potential value of the transaction, which includes rights to all oncology indications for the two molecules, is estimated to exceed $300 million, plus royalties.

Carfilzomib, a highly selective proteasome inhibitor, is currently being evaluated in multiple clinical trials for the treatment of patients with multiple myeloma and other cancers. ONX 0912, an oral proteasome inhibitor, is currently in Phase 1 testing.

Ono will pay Onyx an upfront payment of Yen 5 billion (Japanese yen, approximately $59 million at current exchange rates). In addition, Onyx will receive development and sales-based payments related to the compounds that could total up to approximately $280 million at current exchange rates.  The agreement also calls for royalty payments in double-digit percentages on net sales in Japan, commensurate with a late-stage asset.  

"This strategically important transaction underscores the growing interest and excitement surrounding carfilzomib.  Ono is an ideal partner in Japan given their focus on highly innovative new pharmaceutical products and their reputation for scientific excellence," said N. Anthony Coles, M.D., president and chief executive officer of Onyx. "Onyx is committed to reaching all patients with multiple myeloma who could potentially benefit from promising therapies such as carfilzomib and ONX 0912, as quickly as possible.  This partnership allows us to benefit from Ono's drug development experience in Japan for the advancement of carfilzomib, while we focus on execution in the U.S. and Europe."

In the U.S., Onyx is preparing a new drug application (NDA) for carfilzomib, which it expects to submit to the Food and Drug Administration (FDA) by year-end 2010 for potential accelerated approval. Given this potential timeline, Onyx is engaged in planning and executing prelaunch activities.

"Ono is actively engaged in the development and the commercialization of innovative drugs in cancer and related areas, including Emend® for chemotherapy-induced nausea and vomiting and an antibody anticancer drug, as well as drugs for cancer anorexia / cachexia and for opioid-induced constipation, and recognizes the tremendous need for new treatment options for multiple myeloma. This agreement facilitates Ono's strategic expansion of its development pipeline in the oncology area with these exciting new compounds from a proven class of drugs. The promising activity and tolerability observed to date indicate that carfilzomib may be an important new treatment option for myeloma patients, and we look forward to collaborating with Onyx to bring this drug to patients in Japan," said Gyo Sagara, Ono's president, representative director and chief executive officer.

About the Proteasome Inhibitor Development Program

The proteasome has been validated as an important clinical target in cancer, and Onyx is developing next-generation proteasome inhibitors with a high degree of selectivity, with the goal of increasing therapeutic efficacy and reducing side effects.

The lead product candidate in this program is carfilzomib. Carfilzomib, a highly selective proteasome inhibitor, has shown encouraging results in a broad clinical trial program in multiple myeloma. Onyx recently announced top-line results from the Phase 2b 003-A1 study of single-agent carfilzomib in heavily pretreated patients with relapsed and refractory multiple myeloma, in which carfilzomib achieved an overall response rate (partial response or greater) of 24 percent and a median duration of response of more than seven months in patients who entered the study after receiving a median five prior lines of therapy (corresponding to a median of 13 anti-myeloma agents) and whose disease was refractory to their last therapeutic regimen.  

Onyx has also initiated a large randomized international Phase 3 clinical trial, known as the ASPIRE trial, studying the combination of carfilzomib, lenalidomide, and low-dose dexamethasone in patients with relapsed multiple myeloma. The company has an agreement with the U.S. FDA on a Special Protocol Assessment (SPA) and received Scientific Advice from the European Medicines Agency (EMA) on the design and planned analysis for the ASPIRE trial. An additional Phase 3 clinical trial, known as the FOCUS trial, is planned to evaluate carfilzomib in patients with advanced myeloma and serve as the basis for a European registration. Carfilzomib is also being studied in advanced solid tumors.

ONX 0912 is distinct from carfilzomib, although the compound is based on the same chemistry that is employed to selectively target the proteasome.  Upon completion of the current Phase 1 study in advanced refractory or recurrent solid tumors, Onyx expects that it will explore ONX 0912 in hematologic malignancies.

The development program also includes ONX 0914, an immunoproteasome inhibitor with activity in preclinical models of autoimmune disorders.

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. Worldwide, more than 180,000 people are living with multiple myeloma and approximately 86,000 new cases are diagnosed annually.(i) In Asia, there are approximately 28,000 people living with multiple myeloma (ii), including approximately 13,000 people living with multiple myeloma in Japan.(iii)  In the United States, more than 50,000 people are living with multiple myeloma and approximately 20,000 new cases are diagnosed annually.(iv)  

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 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.

Nexavar® (sorafenib) tablets is a registered trademark of Bayer HealthCare Pharmaceuticals.

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 potential benefits of the agreement between Onyx and Ono Pharmaceutical Co., Ltd. to develop and commercialize carfilzomib and ONX 0912 and the potential timing, progress and results of the clinical development, safety, regulatory processes, commercialization efforts and commercial potential of carfilzomib and ONX 0912. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including the risks that the Ono collaboration may not be successful and that Onyx may not realize the anticipated benefits of the transaction. 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 these risks and uncertainties. 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) International Agency for Research on Cancer, GLOBOCAN 2002 database

(ii) International Agency for Research on Cancer, GLOBOCAN 2008 database

(iii) Patient Survey 2008 of the Ministry of Health, Labour and Welfare

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

SOURCE Onyx Pharmaceuticals, Inc.