New Fatty Liver Disease Therapy Receives FDA Fast Track Designation

Patricia Silva, PhD avatar

by Patricia Silva, PhD |

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NuSirt Biopharma, a company focused on therapies for chronic metabolic diseases, recently announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s development program to treat non-alcoholic steatohepatitis (NASH) and non-alcoholic fatty liver disease (NAFLD).

Mitochondrial dysfunction in the liver plays a key role in the development of fatty liver disease and also NASH, a liver disorder characterized by fat accumulation, inflammation and damage. NASH occurs in individuals who drink little or no alcohol, and is rapidly becoming a leading cause of cirrhosis and liver failure. It is estimated that 10% to 30% of the individuals with NAFLD develop NASH.

“NAFLD and NASH represent serious unmet medical needs for which there are no approved therapies,” said the president and executive chairman of the board of NuSirt Biopharma, Joseph C. Cook Jr., in a press release. “It is estimated that 75-100 million people in the U.S. have NAFLD, and 10-20 million have progressed to NASH. The NuSirt team is dedicated to improving the lives of those with serious metabolic diseases. We are delighted to have received Fast Track designation, which is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. This designation helps get important new drugs to patients earlier.”

NuSirt has a unique technology that combines leucine, a naturally occurring essential amino acid, with existing human drugs. Pre-clinical studies showed that such therapeutic combinations might be able to prevent and treat metabolic diseases, enhancing the effectiveness of already existing pharmaceuticals. The triple therapeutic combination of leucine, metformin (anti-diabetic medication), and sildenafil (medication to treat erectile dysfunction and pulmonary arterial hypertension), in particular, was shown to reduce NAFLD symptoms, to potentially reverse the disease, and to prevent the onset of both NAFLD and NASH. This triple combination was also reported to reverse obesity-induced liver fat accumulation, fibrosis, and inflammation in mice.

NuSirt is beginning to enroll patients in a 16-week, randomized, placebo-controlled, double-blind Phase 2a clinical trial (TRIPLN, NCT02546609) to assess the impact of its patented triple combination of leucine, metformin, and sildenafil on their liver fat content. Researchers will also assess several liver, inflammatory and metabolic biomarkers.

“Pre-clinical studies have shown the potential of NuSirt technology to target the three pathophysiological aspects of NASH: steatosis, inflammation and fibrosis,” said Dr. Michael Zemel, founder and chief scientific officer of NuSirt Biopharma. “As we begin our first human trial in NAFLD and NASH, we look forward to further exploring how our triple-combination therapy could help the millions affected by this disease.”

For more information on this study and how to participate, contact [email protected] or visit the clinical trial link.