Phase 3 Data Show Bayer's Investigational Compound Regorafenib (BAY 73-4506) Met Primary Endpoint Showing Statistically Significant Improvement in Overall Survival in Patients with Metastatic Colorectal Cancer Refractory to Standard Approved Therapies

SOUTH SAN FRANCISCO, Calif., Jan. 17, 2012 /PRNewswire/ -- Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced results from the Bayer HealthCare Phase 3 CORRECT (Colorectal cancer treated with regorafenib or placebo after failure of standard therapy) trial of Bayer's investigational compound regorafenib (BAY 73-4506). The study met its primary endpoint, showing statistically significant improvement in overall survival (OS) by 29% (HR=0.77, p=0.0052, median OS: 6.4 months vs. 5.0 months for the placebo group) in patients with metastatic colorectal cancer (mCRC) whose disease had progressed after approved standard therapies.[i] This late-breaking abstract, with these updated data, will be presented by Axel Grothey, MD, Professor of Oncology, Mayo Clinic and co-principal investigator of the CORRECT study, in an oral abstract session (LBA No. 385, January 21, 2012 from 2:30 p.m. – 4:00 p.m. PT, Level 3 Ballroom, Moscone Center West) at the 2012 Gastrointestinal Cancers Symposium of the American Society of Clinical Oncology (ASCO-GI), in San Francisco, CA.

Additionally, findings from the secondary endpoints of the CORRECT study showed statistically significant improvement in progression-free survival (PFS) (HR=0.49, p<0.000001, median PFS: 1.9 months vs. 1.7 months) and an improvement in disease control rate (44.8% vs. 15.3%) in patients treated with regorafenib compared to those treated with placebo. The difference in objective response rate between the two arms (1.0% vs. 0.4%) did not reach statistical significance.[i]

The most common drug-related treatment-emergent adverse events included fatigue (47.4% vs. 28.1%), hand-foot skin reaction (46.6% vs. 7.5%), diarrhea (33.8% vs. 8.3%), anorexia (30.4% vs. 15.4%), hypertension (27.8% vs. 5.9%), oral mucositis (27.2% vs. 3.6%) and rash/desquamation (26.0% vs. 4.0%) for patients receiving regorafenib as compared to placebo.[i]

Per the recommendation from an independent Data Monitoring Committee, the CORRECT study was unblinded in late 2011 following a pre-planned interim analysis that determined that the regorafenib arm showed significant improvement in overall survival; patients on the placebo arm were unblinded and offered treatment with regorafenib.

Bayer plans to submit regorafenib for marketing authorization in mCRC in 2012.

In 2011, Bayer and Onyx entered into an agreement under which Onyx will receive a royalty on any future global net sales of regorafenib in oncology.

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, which must have included fluoropyrimidine, oxaliplatin, irinotecan, bevacizumab and cetuximab or panitumumab.[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.[i]

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

About Colorectal Cancer

Colorectal cancer (CRC) 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.[ii]

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.[iii]  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.[iv] 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.[v]

About Regorafenib

Regorafenib is an investigational oral multi-kinase inhibitor targeting angiogenic, stromal and oncogenic kinases[vi] 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.

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 and other serious diseases.  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-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, the expanded access program, safety, regulatory processes, 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 submission, review, potential approval of the NDA, development 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, 2010, 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] 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.

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

[v] 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.

[vi] S. Wilhelm, et al. Regorafenib (Bay 73-4506): A New Oral Multikinase Inhibitor of Angiogenic, Stromal, and Oncogenic Receptor Tyrosine Kinases with Potent Preclinical Antitumor Activity. Online International Journal of Cancer. December 17, 2010.

SOURCE Onyx Pharmaceuticals, Inc.