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posted on 24/04/2008
Full Results Presented at European Association for the Study of the Liver Annual Meeting
SAN FRANCISCO, April 24 /PRNewswire/ — 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 40mg/day, MitoQ® 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.
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 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® 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.
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
Source: PR Newswire
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The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.