Antipodean Pharmaceuticals Announces Results of Phase 2 Study of Lead Compound MitoQ

April 24, 2008 -- San Francisco, CA -- Antipodean Pharmaceuticals, Inc. announced today, at the European Meeting for the Study of the Liver (EASL), the positive results of a Phase 2 trial of its lead compound MitoQ (mitoquinone) in liver disease. The trial, conducted by Dr. Edward Gane, Associate Professor of Medicine, New Zealand Liver Transplant Unit at Auckland City Hospital, successfully met the primary clinical endpoint, the reduction of elevated liver enzymes.

In the 28-day trial, 30 patients with the hepatitis C virus (HCV) were enrolled to study the effects of MitoQ on elevated liver enzymes. Researchers measured patients' baseline levels of aminotransferase (ALT), an enzyme released into the blood that indicates damage to the liver. The double-blind trial randomized patients to one of three treatment groups: MitoQ 40 mg/day, MitoQ 80 mg/day, or placebo. The primary endpoint was the reduction of levels of ALT.

Patients who received MitoQ showed a significant decrease in ALT levels at the end of the study compared with baseline levels. The decrease from baseline was 26.4% (P<0.002) for patients in the 40 mg dose group, and 28% (P<0.05) for patients in the 80 mg dose group. These results suggest that MitoQ can reduce necroinflammation and may halt disease progression to fibrosis or cirrhosis.

The drug was well tolerated with no significant safety issues. Commonly reported adverse events (AEs) included nausea, headache and vomiting, which were usually mild and well tolerated. Only one patient withdrew from the study due to nausea. There were no significant laboratory or ECG abnormalities observed and no serious adverse events (SAEs) were reported.

"In patients with chronic liver disease, including the two patient populations with the largest unmet need -- patients with chronic hepatitis C, who have failed current standard of care, and patients with non-alcoholic fatty liver disease (NAFLD) -- there are currently no therapeutic options available to prevent progression to cirrhosis, liver failure and liver cancer," stated Dr. Gane. "In the future, these patients may benefit from maintenance therapy with interventions such as MitoQ, which block either hepatic necroinflammation or fibrogenesis."

"In the coming year, Antipodean plans to explore and develop its lead compound for the treatment of hepatological diseases, particularly non-alcoholic fatty liver disease," said Ken Taylor, PhD, Chief Executive Officer of Antipodean Pharmaceuticals. "The company is actively seeking to achieve this goal with the help of a pharmaceutical partner."

A 12-month study in patients in Parkinson's disease showed MitoQ to be well tolerated; however, there was no significant effect on disease progression. Dr. Barry Snow, Department of Neurology, Auckland Hospital, New Zealand, and Principal Investigator of the trial of MitoQ in Parkinson's disease, believes that the lack of efficacy in Parkinson's Disease may have been due to the large number of impaired cells in that disease; these cells may have had limited or no ability to regenerate, and thus could not benefit from MitoQ's antioxidant properties. The company believes MitoQ has therapeutic potential in diseases where cell regeneration occurs, such as hepatological and dermatological disorders.

About MitoQ

MitoQ is a mitochondria-targeted antioxidant that selectively blocks mitochondrial oxidative damage and prevents liver cell apoptosis. MitoQ is based on a novel technology, targeted lipophilic cations that transport and concentrate antioxidants into the mitochondria -- organelles inside cells that provide energy for life processes -- where they accumulate up to a thousand fold. Targeted antioxidants can reduce the hepatic oxidative damage that is induced by viral infection and that is also involved in the progression of NAFLD through to NASH, leading to fibrosis or cirrhosis.

About Antipodean