Clinical Trial Of New Drugs For Children With Liver Disease Opened
Gemphire Therapeutics is a biopharmaceutical company that develops therapies for cardiovascular and metabolic diseases. Focusing on the development and commercialization of treatments for diseases such as dyslipidemia and non-alcoholic steatohepatitis, the company recently announced the launch of a phase 2a clinical trial of proof-of-concept, aiming to evaluate the efficacy of gemcabene as a treatment for nonalcoholic's fatty liver disease in children. The trial is part of a larger plan to assess gemcabene in patients with NAFLD/NASH and will be conducted in parallel with the study of gemcabene of the recent development of familial partial fat metabolism disorders. The pedigree results of the pediatric NAFLD trial are expected to be published in early 2019.
NAFLD is becoming the most common chronic liver disease in many countries. With about 75 million to 100 million NAFLD patients in the United States, it's the leading cause of chronic liver disease in children and adolescents in the United States. It is also a major burden of chronic liver disease in China. It ranges from mild liver fat infiltration to simple fatty degeneration to more severe NASH and liver fibrosis. Its treatment is very limited.
Gemphire's candidate drug, gemcabene, is expected to change that. It's first-in-class, once a day oral therapy. Gemcabene may be applicable to patients with currently approved therapies (mainly statins) who are still unable to reach normal levels of low density's lipoprotein cholesterol (LDL-C) or triglycerides. Gemcabene is intended to improve the clearance of VLDLs in plasma. It also inhibits the production of cholesterol and triglyceride in livers.
This open-label clinical trial will enroll approximately forty 12-to-17-year-old children. They were diagnosed with NAFLD and abnormal liver function after assessment of liver transaminase. Patients will receive gemcabene once a day at a dose of 300 mg. The main endpoints were the changes of alanine aminotransferase (alt) from baseline to 12 weeks. Alt is a biomarker of liver function. The secondary endpoint includes measuring the changes of liver fat denaturation by non-invasive magnetic resonance imaging with proton density fat fraction MRI-PDFF, investigating the score changes of liver inflammation and liver fibrosis (LIF) by noninvasive MRI, changes of AST, insulin sensitivity, blood lipids (including triglyceride), apolipoprotein and inflammatory markers (including hsCRP), and drug safety and tolerance. It is believed that gemcabene will have a significant competitive advantage based on its good safety and a new mechanism for dyslipidemia and inflammation.
Reference: Wuxi App Tec Summary of Recent Advances in the Field of Inventory of Diabetes (issue 33). February 9th, 2018