North Bethesda, MD, November 15, 2021 – The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium, in a groundbreaking program – Non-Invasive Biomarkers of Metabolic Liver Disease (NIMBLE) – has generated the first set of topline data on the suitability of candidate non-invasive biomarkers for use in clinical trials of treatments for Non-Alcoholic Steatohepatitis (NASH). The data produced by the five-year program was presented yesterday at the American Association for the Study of Liver Disease (AASLD).

Non-alcoholic steatohepatitis (NASH), a serious condition associated with obesity and diabetes and characterized by inflammation and cell damage, is estimated to affect between 9 and 15 million people in the United States. Because NASH does not present symptoms until it has progressed to later stages, patients often remain undiagnosed until they have developed cirrhosis, which may require a liver transplant, or develop liver cancer, which could be fatal. Currently, diagnosing early-stage NASH requires a liver biopsy, which is a painful, invasive, and expensive process for patients, and which carries some risk of mortality.

The FNIH Biomarkers Consortium NIMBLE program has been testing the performance of currently available non-invasive NASH tests. These include blood-based tests, imaging-based tests, and a combination of the two. All of these are being compared to standard measures, like the ALT and FIB-4, with their performance benchmarked against liver biopsy, the current reference standard.

Initial review of blood-based biomarkers involved five tests: the Extended Liver Fibrosis (ELF) test (Siemens), NIS-4 (GENFIT), Pro-C3 (Nordic Biosciences), Fibrometer-VCTE (Echosens), and OWLiver (OWL Metabolomics). These tests were conducted on duplicate samples from more than 1,000 patients previously collected and banked by the NASH Clinical Research Network, which is supported by the National Institute of Diabetes and Digestive and Kidney Diseases, part of the NIH. Criteria for what would and would not be acceptable performance against the current standard of care were established before data analysis, following the highest level of transparency, rigor, and regulatory compliance.

NIS-4 was able to identify patients with “at risk NASH” (patients with NASH at high risk of progressing to serious liver damage and death) better than the frequently used ALT test. ELF and Fibrometer-VCTE were both able to diagnose degrees of significant fibrosis (liver scarring) better than FIB-4 test. PRO-C3 identified clinically significant fibrosis about as well FIB-4. The OWL panel had promising early results for identification of NASH. These data establish the sensitivity and specificity of the biomarkers studied and will inform planning for the next stage of NIMBLE.

Details of these results and key findings can be found here. Final detailed results are expected to be submitted for publication by 1Q 2022.

The ability to rigorously diagnose at-risk NASH and cirrhotic patients with non-invasive tests has important implications for the evolving standard of care for patients and for drug development. NIMBLE’s initial data results offer a potential path for companies developing new therapies for NASH to enrich clinical trial populations and accelerate drug development. Robust non-invasive tests are desperately needed in these areas, as was highlighted in a 2018 FDA Guidance for NASH drug development here.

About NIMBLE: The NIMBLE consortium is a comprehensive multi-year pre-competitive, public-private partnership collaboration conducted under the auspices of the Foundation for the NIH (FNIH) Biomarkers Consortium. The NIMBLE project is supported by multiple entities including AbbVie, Amgen Inc., AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Echosens, GE Healthcare, Genentech, Inc., Gilead Sciences, Inc., Intercept Pharmaceuticals, Inc., Novo Nordisk A/S, Pfizer Inc, Regeneron Pharmaceuticals Inc, and Takeda Development Center Americas Inc. Additionally, many companies have donated their assays, equipment, and services to the NIMBLE consortium, including AMRA Medical, Canon Medical Systems USA, Inc, Echosens, GENFIT SA, GE Healthcare, Nordic Bioscience A/S, OWL Metabolomics, Philips Ultrasound, Inc., P-Value, LLC, Hologic SuperSonic Imagine, Siemens Healthineers, and Siemens Medical Solutions USA, Inc.

About the Biomarkers Consortium: The Biomarkers Consortium (BC) embraces government, industry, patient advocacy groups, and not-for-profit organizations each of which has a stake in the identification, development, and the seeking of regulatory approval for biomarkers. The BC addresses one of the most pressing needs in the diagnosis and treatment of disease: the development and the seeking of regulatory approval for disease biomarkers and surrogates. The core operations of The Biomarkers Consortium are supported through the contributing membership program. Organizations representing private industry (including the pharmaceutical, biotechnology, diagnostics, and information technology industries) and not-for-profit organizations (including associations, advocacy groups, trade organizations, and philanthropic organizations) that wish to support biomarkers development are eligible to become contributing members.

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health (FNIH) creates and manages alliances with public and private Institutions in support of the mission of the NIH, the world’s premier medical research agency. The Foundation, also known as the FNIH, works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe. The FNIH organizes and administers research projects; supports education and training of new researchers; organizes educational events and symposia; and administers a series of funds supporting a wide range of health issues. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization.