Antipodean Pharmaceuticals,
Inc. announced, at the European Meeting for the Study of the Liver
(EASL), the positive results of a Phase 2 trial of its lead compound
MitoQ(R) (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 40mg/day, MitoQ(R) 80mg/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 40mg dose group, and 28% (p<0.05) for patients in the 80mg
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 (NAFLD)," said Ken Taylor, Ph.D., 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(R)
MitoQ(R) 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 Non-alcoholic Fatty Liver Disease (NAFLD)
Symptoms of NAFLD include inflammation of the liver, often associated
with alcoholic livers, but occurring in nonalcoholic patients, fat in the
liver and liver damage. As with Hepatitis C, oxidative stress is involved
in disease progression in NAFLD, which affects approximately 20% of the
world's population.
There are no satisfactory treatment options currently available for
NAFLD, and the incidence is growing rapidly as obesity rates rise. MitoQ(R)
is a potent, targeted antioxidant that limits oxidative stress and cell
degeneration by protecting the mitochondria from oxidative damage, and the
Company believes it offers the potential to treat the oxidative stress
observed in NAFLD patients.
About Antipodean
Antipodean is a clinical-stage pharmaceutical company developing
targeted molecules that prevent oxidative damage to endothelial, epithelial
and liver cells leading to apoptosis and fibrosis. The Company is
developing a mitochondria-targeted antioxidant, MitoQ (mitoquinone
mesylate), for the treatment of hepatic inflammatory disorders caused by
oxidative stress such as nonalcoholic fatty liver disease (NAFLD) and
nonalcoholic steatohepatitis (NASH). The Company's business plan is to
develop drugs to the point of proof of principle and then partner further
development. Antipodean has research collaborations with pre-clinical and
clinical investigators in Cambridge, UK, Auckland, New Zealand, and several
centers in the US to identify and develop lead compounds through to
clinical proof-of-principle. Currently the Company's lead compounds target
liver and skin diseases. Antipodean is located in San Francisco,
California. Further information is available at
http://www.antipodeanpharma.com
Antipodean Pharmaceuticals, Inc.
http://www.antipodeanpharma.com
