Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals start Phase 3 trial to evaluate Nexavar tablets

Bayer HealthCare Pharmaceuticals, Inc. and Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced the companies have begun enrolling patients in an international Phase 3 trial to evaluate Nexavar® (sorafenib) tablets for the treatment of patients with radioactive iodine-refractory, locally advanced or metastatic differentiated thyroid cancer. This Phase 3 trial was started based on the results from Phase 2 clinical trials evaluating Nexavar in patients with advanced thyroid cancer.

"Patients with thyroid cancer who failed to respond to surgical or radiotherapies, have limited treatment options to help them manage their disease," said Dimitris Voliotis, vice president, Nexavar clinical development, Bayer HealthCare Pharmaceuticals. "Recognizing this unmet need, we are evaluating Nexavar in this special patient population."

Phase 3 Trial Design

The DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial is an international, multicenter, randomized, placebo-controlled study that will enroll approximately 400 patients with locally advanced or metastatic, radioactive iodine-refractory, differentiated thyroid cancer (papillary, follicular and Hurthle cell) who have received no prior systemic therapy.

Patients will be randomized to receive 400 mg of oral Nexavar twice daily or matching placebo. Patients will continue on treatment until disease progression, toxicity, non-compliance or withdrawal of consent. At the time of progression, patients receiving placebo will have an option to cross over to Nexavar at the discretion of the investigator, based on the patient's clinical status. The primary endpoint of the study is progression-free survival as defined by Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints include overall survival, time to progression and response rate. The safety and tolerability of the two treatment groups will also be compared.

The study will be conducted at sites in the United States, Europe, Asia, and Japan. For information about enrolling in the study, please visit www.clinicaltrials.gov.

Phase 2 Trial Results

Updated results from a single institution, investigator sponsored Phase 2 open-label study in 55 patients with metastatic, iodine refractory, thyroid cancer treated with Nexavar 400 mg twice daily were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting, May 29-June 3, 2009 Orlando, FL, by Marcia Brose, M.D., Ph.D., an assistant professor of Hematology/Oncology and Otorhinolaryngology in the Abramson Cancer Center at the University of Pennsylvania, Philadelphia, PA, U.S.A. In 50 evaluable patients, 18 (36 percent) had a partial response per RECIST criteria. Updated survival results on the first 30 patients enrolled into the study demonstrated that across all histologies the median progression-free survival (PFS) was 63 weeks and the median overall survival was 140 weeks. The most common adverse events (AE) seen in the trial were hand-foot skin reaction, rash, fatigue, stomatitis/mucositis, weight loss, and musculoskeletal pain, and were predominantly grade 1 or 2. Dr. Brose and Martin J. Schlumberger, Institut Gustave-Roussy, Villejuif, France, are the lead investigators on the Phase 3 trial.

"Based on the positive signal generated in the Phase 2 trial, the initiation of this Phase 3 represents progress in exploring the full potential of Nexavar in a variety of treatment settings and tumor types," said Todd Yancey, M.D., vice president of clinical development at Onyx. "Building on our successful foundation of treating unresectable liver cancer and advanced kidney cancer, we are hopeful that this Phase 3 trial will lead to a new treatment option for patients with non-responsive thyroid cancer."