Non-Alcoholic Steatohepatitis (NASH): Preclinical and Clinical Development and Progress Toward Regulatory Approval Pathways

The prevalence of Non-Alcoholic Steatohepatitis is increasing worldwide in parallel to the expanding obesity epidemic. Insulin resistance and the accumulation of triglyceride-derived toxic lipid metabolites play a key role in its pathogenesis. There are no drugs approved for the treatment of Non-Alcoholic Steatohepatitis at the present time, yet, and given its increasing prevalence and societal health burden, there is an urgency to develop therapeutic strategies. The challenges of developing drugs to treat Non-Alcoholic Steatohepatitis are substantial due to the variable course of the disease and the lack of validated non-invasive markers. Non-Alcoholic Steatohepatitis has a long natural history, typically with alternating periods of progression and regression of the disease. Liver biopsy, the gold standard method for NASH disease diagnosis, grading and staging, has limitations in the assessment of the presence of NASH disease and its severity. In addition to the natural history of the disease, difficulties in developing drugs for NASH disease are compounded by the limitations of predictive preclinical models. The last decade has seen regulatory agencies implement several initiatives to expedite the development of drugs fcor serious conditions such as NASH disease—and these initiatives are especially applicable in cases where there is an unmet medical need (UMN). The FDA has developed several pathways to expedite drug development for an UMN, and the European Medicines Agency has created similar mechanisms known collectively as “adaptive pathways.”

During this webinar, we will discuss the epidemiology and health burden of Non-Alcoholic Steatohepatitis and present contemporary information on new preclinical models that can support drug development for NASH disease. In addition, we will discuss the clinical endpoints and options in support of a successful clinical development plan aimed at major program milestones including proof-of-concept studies and strategies to achieve marketing approval.

Speakers

Claudia Filozof, MD, PhD, Senior Medical Director, Cardiovascular/Metabolic, Covance

Claudia Filozof is a senior medical director in the Phase II-IV Cardiovascular/Metabolic group at Covance where she is responsible for non-alcoholic fatty liver disease/non-alcoholic steatohepatitis projects. Dr. Filozof is board certified in nutrition and metabolism, and has over 20 years of experience in academia and more than 10 years of service in the Pharmaceutical industry. She has broad expertise in diabetes, obesity and related metabolic disorders including non-alcoholic fatty liver disease and steatohepatitis (NAFLD/NASH).

Dr. Filozof was awarded a PhD for her thesis on “Energy Metabolism and Leptin in Offspring of Obese Parents.” She also received additional post-doctoral training in research methodologies and biostatistics. She served as director of the postgraduate course of obesity in the John F Kennedy University in Buenos Aires, Argentina and was the Editor-in-Chief of the Argentine Society of Obesity Journal and a member of the initial editorial board of the prestigious international journal “Obesity Reviews.” She was honored being an IASO (International Association for the Study of Obesity) award Committee member for two consecutive periods and has participated as a member of the first WHO task force in obesity management.

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Arun Sanyal, MD, Executive Director, Education Core, Center for Clinical and Translational Research, Virginia Commonwealth University

Dr. Sanyal is the Executive Director of the Education Core for the Center for Clinical and Translational Research at Virginia Commonwealth University. He has 20 years of experience as an academic Hepatologist and as a trainer of future generations of clinician and investigators. His research program has spanned both basic and clinical aspects of end-stage liver disease and non-alcoholic fatty liver disease and has led to recommendations related to variceal hemorrhage, ascites, hepatorenal syndrome, hepatic encephalopathy and non-alcoholic steatohepatitis in the relevant practice guidelines of various national societies.

Dr. Sanyal has a strong background in national workforce development and training. He served as Secretary, then as President of the American Association for Study of Liver Diseases (AASLD). At the National Institutes of Health (NIH), he chaired the Hepatobiliary Pathophysiology study section and was a founding member of the American Board of Internal Medicine committee for Advanced Hepatology. Dr. Sanyal also served as DDW chair for liver programming and has served on the World Health Organization (WHO) advisory committee for viral hepatitis. He was also one of the drafters for the competency based curriculum for Hepatology training

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Richard Williams, PhD, JD, Senior Director, Global Regulatory Strategy, Covance

Dr. Williams is the Senior Director of Global Regulatory Strategy where he is responsible for advising clients on global, regional or local regulatory strategy associated with preclinical or clinical study design, regulatory agency interactions, as well as for complete drug development programs. He works with, and advises, internal cross-functional product development teams as well as external clients.

In addition to leading and managing successful programs in multiple therapeutic areas, he has also interacted directly with regulatory agencies in the United States, Europe, Japan, and India. He is an advocate for applying contemporary science to strategic drug development and is active member in several scientific societies such as the American Society for Clinical Pharmacology and Experimental Therapeutics, the American Society for Gene & Cell Therapy, and the American Heart Association. Dr. Williams is especially interested in the integration of business with science, particularly with regard to the changing global landscape for product development.

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