Bayer HealthCare Pharmaceuticals Inc. and Onyx Pharmaceuticals, Inc. have announced that Nexavar (sorafenib) tablets significantly extended overall survival in patients with hepatocellular carcinoma (HCC), or primary liver cancer versus those taking placebo by 44 per cent. Results were presented at the 43rd annual meeting of the American Society of Clinical Oncology (ASCO).

The international, phase III, placebo-controlled Sorafenib HCC Assessment Randomised Protocol (SHARP) trial randomised and evaluated 602 liver cancer patients who had no prior systemic therapy at sites in the Americas, Europe, and Australia/New Zealand. The primary objective of the study was to compare overall survival in patients administered Nexavar versus those administered placebo. Median overall survival was 10.7 months in Nexavar-treated patients compared to 7.9 months in those taking placebo.

"Because there are no therapies that significantly improve survival for the thousands of patients with liver cancer, these findings demonstrate the compelling study results of Nexavar as the new reference standard of care for the first-line treatment of HCC," said Dr Josep M Llovet, co-principal investigator and professor of research, Barcelona clinic liver cancer (BCLC) group, IDIBAPS, liver unit, hospital clinic Barcelona; director of research, HCC Programme, associate professor of medicine, mount Sinai School of Medicine, New York.

Bayer and Onyx halted the Sharp trial in February 2007 when an independent data monitoring committee determined in a pre-scheduled analysis that the overall survival endpoint had been met. There were no significant differences in serious adverse event rates between the Nexavar and placebo-treated groups, with the most commonly observed serious adverse events in patients receiving Nexavar being diarrhoea and hand-foot-skin reaction. Based on the strength of the data, the companies are now in the process of preparing applications to the US Food and Drug Administration (FDA) and European health authorities for a supplemental indication for Nexavar in treatment of patients with liver cancer.

"Although much progress has been made in cancer research, the number of lives lost to liver cancer is increasing," said Dr Jordi Bruix, co-principal investigator and director of the Barcelona Clinic Liver Cancer (BCLC) group; senior consultant, liver unit, Hospital Clinic of Barcelona. "For that reason, these results represent an unprecedented achievement and Nexavar could become the first widely-approved new therapy for this difficult to treat cancer."

Hepatocellular carcinoma is the most common form of liver cancer and is responsible for about 90 per cent of the primary malignant liver tumours in adults. It is the fifth most common cancer in the world and the third leading cause of cancer-related deaths globally. Over 600,000 new cases of HCC are diagnosed globally each year (19,000 in the United States and 32,000 in the European Union), and in 2002 approximately 600,000 people (about 13,000 Americans and 57,000 Europeans) died of HCC. Although overall cancer incidence and mortality are decreasing in the United States, both the incidence and mortality of liver cancer are increasing.

"HCC is the second tumour type in which Nexavar has demonstrated a clinical benefit. We intend to move swiftly with our partner Onyx to file these data for health authority review," said Susan Kelley, MD, Vice President, Therapeutic Area Oncology, Bayer HealthCare Pharmaceuticals. "Our strategy of leveraging the unique attributes of Nexavar, the only approved orally administered anti-angiogenic that targets the Raf pathway, has led to a robust ongoing clinical programme that could bring the potent cancer fighting properties of this oral multi-kinase inhibitor to an even broader number of patients in the coming years."