Polaris begins phase III HCC trial

Polaris Group Doses First Patient in Pivotal Phase 3 Clinical Trial of Lead Therapeutic Candidate ADI-PEG 20 for the Treatment of Hepatocellular Carcinoma Company Advances Into Late-Stage Development a Biologic That Systemically Depletes Circulating Arginine Needed by Certain Cancer Cells to Survive SAN DIEGO, July 28, 2011 /PRNewswire/ -- Polaris Group (Polaris) today announced that it has dosed the first patient in a pivotal Phase 3 clinical trial evaluating its lead cancer therapeutic, pegylated arginine deiminase (ADI-PEG 20), in hepatocellular carcinoma (HCC) patients. Over 600 patients who have failed prior systemic therapy will be enrolled in the randomized, double-blind, placebo-controlled study at sites in China, Taiwan, the United States, Italy and the U.K.. Patients will receive weekly intramuscular injections of ADI-PEG 20 until disease progression.  The primary endpoint of the study is overall survival, and secondary endpoints will include progression-free survival and time to progression. "Based on our recently approved Special Protocol Assessment from the FDA, this pivotal Phase 3 study is significant as it will hopefully serve as the basis for approval in the U.S.," said John Bomalaski, M.D., executive vice president, medical affairs, of Polaris. "We believe ADI-PEG 20 is a promising cancer therapeutic not only for HCC, but also other arginine-dependent cancers such as leukemia, lymphoma, sarcoma and prostate cancer. We look forward to continuing clinical development in all of these areas to understand the full potential for this therapeutic."Ghassan Abou-Alfa, M.D., of Memorial Sloan-Kettering Cancer Center in New York, the principal investigator of the study, said, "We are delighted to be involved in a pivotal study for ADI-PEG 20, which has the potential to become a much needed treatment option for HCC patients. There is no currently approved therapeutic for HCC patients who have failed first line therapy. Thus there is a significant unmet need in this patient population."Additional information about the trial can be found online at www.clinicaltrials.gov/ct2/show/NCT01287585?term=polaris+group&rank=1 .

About Hepatocellular Carcinoma Hepatocellular carcinoma (primary liver cancer) is one of the most common cancers worldwide, resulting in approximately 700,000 deaths annually. Infection with hepatitis B or C and alcohol consumption are highly associated with the development of hepatocellular carcinoma. The prognosis for patients with this disease is poor because chemotherapy often does not result in prolonged survival, and liver transplantation only increases the survival of selected patients. Despite all forms of current treatment, life expectancy for most patients is less than one year post-diagnosis.

About ADI-PEG 20 ADI-PEG 20 is a biologic being developed by Polaris to treat cancers carrying a major metabolic defect that renders them, unlike normal cells, unable to make arginine internally. Because arginine is one of the 20 amino acids that are essential for protein synthesis and survival of cells, these cancer cells become dependent upon the external supply of arginine to survive and grow. ADI-PEG 20 works by systemically depleting the external supply of arginine, which causes these arginine-dependent cancer cells to die while leaving the normal cells unharmed.Multiple cancers have been reported to have a high degree of arginine-dependency. Phase 2 clinical trials of ADI-PEG 20 have yielded positive results in patients with hepatocellular carcinoma or metastatic melanoma, and Phase 2 trials for small cell lung cancer and mesothelioma are currently ongoing. Polaris also plans to initiate clinical studies in prostate cancer, pancreatic cancer, leukemia, lymphoma and sarcoma this year.

About Polaris Group Polaris Group is a privately held multinational biopharmaceutical company that specializes in the research and development of protein drugs to treat cancer and other debilitating diseases. The company's lead therapeutic, ADI-PEG 20, is currently being evaluated in a pivotal Phase 3 trial for hepatocellular carcinoma. Polaris is also investigating ADI-PEG 20 as a treatment for other arginine-dependent cancers, such as melanoma, prostate cancer, leukemia, lymphoma, sarcoma and pancreatic cancer. In addition to the ADI-PEG 20 project, Polaris is researching and developing other biotherapeutic agents and has a small molecule drug program that utilizes a rational structure-based approach to design novel compounds that inhibit the biological function of cancer-related protein targets.