New Drug Application for Regorafenib for the Treatment of Metastatic Colorectal Cancer Submitted by Bayer

SOUTH SAN FRANCISCO, Calif., May 23, 2012 /PRNewswire/ -- Onyx Pharmaceuticals, Inc. today announced that Bayer HealthCare has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval for the oral multi-kinase inhibitor regorafenib for the treatment of patients with metastatic colorectal cancer (mCRC).  Regorafenib is a Bayer compound, and Onyx will receive a royalty on global net sales of regorafenib in oncology.

The submission is based on the results of the pivotal, global Phase 3 CORRECT (Colorectal cancer treated with regorafenib or placebo after failure of standard therapy) trial. Results from the study were first presented at the Annual Gastrointestinal Cancers Symposium of the American Society of Clinical Oncology (ASCO-GI) in January 2012 and will be presented at the upcoming ASCO annual meeting in Chicago, IL (USA) in early June 2012.

Bayer has also submitted an application for European marketing authorization for regorafenib for the treatment of patients with mCRC.

About the CORRECT Study
The CORRECT study was an international, multicenter, randomized, double-blind, placebo-controlled Phase 3 study that enrolled 760 patients with mCRC whose disease had progressed during or within 3 months following last administration of approved standard therapies.[i]

Patients who had withdrawn from standard treatment due to unacceptable toxicity warranting discontinuation of treatment and precluding retreatment with the same agent prior to progression of disease were also allowed into the study.

Patients were randomized to receive either regorafenib plus best supportive care (BSC) or placebo plus BSC. Treatment cycles consisted of 160 mg of regorafenib (or matching placebo) once daily for three weeks on / one week off plus BSC. [ii]

The study was conducted in North America, Europe, China, Japan and Australia.

About Colorectal Cancer
Colorectal cancer is a disease in which malignant (cancer) cells form in the tissues of the colon or rectum. The majority of cancers occurring in the colon and rectum are adenocarcinomas, which account for more than 90 percent of all large bowel tumors.[iii]

CRC is the third most commonly diagnosed cancer and the third leading cause of cancer death in the United States, in both men and women.[iv]  It is estimated that more than 140,000 people will be diagnosed with CRC in 2011, and nearly 50,000 people will die from the disease.^[v] Approximately 50 percent of colon cancer patients will be diagnosed with metastases (most commonly to the liver) either at the time of diagnosis or due to recurrent disease.[vi]

About Regorafenib
Regorafenib is an investigational oral multi-kinase and is currently being investigated in clinical trials for its potential to treat patients with various tumor types.

Regorafenib is an investigational agent and is not approved by the FDA, EMA or other health authorities. 

Regorafenib is one of several cancer compounds in Bayer's development pipeline. In 2011, Bayer entered into an agreement with Onyx Pharmaceuticals, Inc., under which Onyx will receive a royalty on any future global net sales of regorafenib in oncology.

About Onyx Pharmaceuticals, Inc.
Based in South San Francisco, California, Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer. The company is focused on developing novel medicines that target key molecular pathways. For more information about Onyx, visit the company's website at www.onyx.com.

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 expansion of Onyx's product portfolio, the possibility of royalty payments to Onyx from the sale of regorafenib, and the timing, progress and results of clinical development and the regulatory approval process. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: Nexavar being our only approved product; we may never receive marketing approval for Kyprolis (carfilzomib) or regorafenib; competition; failures or delays in our clinical trials or the regulatory process; dependence on our collaborative relationship with Bayer; if approved, we or Bayer, as the case may be, may be unsuccessful in launching, maintaining adequate supply of or obtaining reimbursement for Kyprolis or regorafenib; market acceptance and the rate of adoption of our products; pharmaceutical pricing and reimbursement pressures; serious adverse side effects, if they are associated with Nexavar, regorafenib or Kyprolis; government regulation; possible failure to realize the anticipated benefits of business acquisitions or strategic investments; protection of our intellectual property; the indebtedness incurred through the sale of our 4.0% convertible senior notes due 2016; and product liability risks. Reference should be made to Onyx's Annual Report on Form 10-K for the year ended December 31, 2011 filed with the Securities and Exchange Commission, as updated by Onyx's subsequent Quarterly Reports on Form 10-Q, under the heading "Risk Factors" for a more detailed description of these and other risks. 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] A. Grothey, et al. Results of a randomized Phase 3 trial (CORRECT) of regorafenib plus best supportive care (BSC) versus placebo plus BSC in patients with mCRC who have progressed after standard therapies [January 17, 2012 ASCO-GI Presscast Presentation]. 2012 Gastrointestinal Cancers Symposium; January 19-21, 2012. San Francisco, CA.

[ii] American Cancer Society. Colorectal Cancer Guide. Available from: http://www.cancer.org/acs/groups/cid/documents/webcontent/003096-pdf.pdf. Accessed on October 14, 2011.

[iii] American Cancer Society. Colorectal Cancer Facts & Figures 2011-2013. Available at: http://www.cancer.org/acs/groups/content/@epidemiologysurveilance/documents/document/acspc-028323.pdf. Accessed on October 24, 2011. Accessed on October 14, 2011.

[iv] American Cancer Society. Colorectal Cancer Facts & Figures 2011-2013. Available at: http://www.cancer.org/acs/groups/content/@epidemiologysurveilance/documents/document/acspc-028323.pdf. Accessed on October 24, 2011.

[v] National Cancer Institute. Colon and Rectal Cancer. Available at: http://www.cancer.gov/cancertopics/types/colon-and-rectal. Accessed on October 24, 2011.

[vi] National Cancer Institute. Stage IV and Recurrent Colon Cancer. Available at: http://www.cancer.gov/cancertopics/pdq/treatment/colon/HealthProfessional/page9. Accessed on October 24, 2011.

SOURCE Onyx Pharmaceuticals, Inc.