Published On: 6/21/2023

World-Leading Real World Data Registry, TARGET-NASH, Surpasses 7,000+ Enrolled Patients and 500,000+ Months of Patient Follow-Up

New Data from Target RWE to Also Be Presented at the European Association for the Study of the Liver (EASL) Congress 2023

DURHAM, N.C., June 21, 2023 /PRNewswire/ -- Real world evidence (RWE) leader, Target RWE, today shared updates from its nonalcoholic steatohepatitis (NASH) longitudinal registry of more than 7,000 enrolled patients, representing more than 500,000 patient months of follow up, making TARGET-NASH the most mature dataset following patients with nonalcoholic fatty liver disease (NAFLD) and NASH in the world. Target RWE will be presenting new data from its TARGET-NASH and TARGET-HCC cohorts at the annual EASL Congress in Vienna, Austria June 21-24th, including disease progression and results from a post-authorization safety study.

Non-alcoholic steatohepatitis disease progression in participants from the United States TARGET-NASH real world longitudinal observational study (Presentation OS-089) has been accepted as an oral presentation by Philip N. Newsome, MD, PhD, Director of National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre at University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham; Director of Centre for Liver & Gastrointestinal Research, Institute of Immunology and Immunotherapy, University of Birmingham, UK.

Estimating fibrosis progression in patients with NAFLD/NASH is essential for understanding the comprehensive burden of disease, risk of future liver-related morbidity and to inform future clinical trial designs. This study analyzed demographic and clinical characteristics, rate of occurrence of disease progression outcomes, and identified factors associated with and probabilities of progression outcomes for different risk factors among noncirrhotic NASH F2-F3, NASH F4 compensated cirrhosis (with no clinically significant portal hypertension (CSPH) and with CSPH), and NASH F4 decompensated cirrhosis.

Progression outcomes included all-cause mortality, progression from non-cirrhosis to cirrhosis and from compensated to decompensated cirrhosis, and liver-related and cardiovascular events. Incidence rates and incidence proportion for outcomes were estimated in each group, univariate survival models evaluated the association between risk factors and time-to-event outcomes, and multivariable survival models were used to estimate adjusted Hazard Ratios.

A prospective, pragmatic post-authorisation safety study of early recurrence of hepatocellular carcinoma in hepatitis C virus-infected patients after direct-acting antiviral (DAA) therapy: DAA-PASS (Poster THU-185) has been accepted as a poster presentation by Bruno Sangro, MD, PhD, Cancer Center Clinica Universidad de Navarra, Madrid-Pamplona, Spain. Early reports of DAA therapy for chronic hepatitis C (HCV) after successful treatment of hepatocellular carcinoma (HCC) raised concerns about an increased risk of early and aggressive HCC recurrence. Although limited by a small sample size, this prospective pragmatic study suggests DAA therapy is not associated with increased HCC recurrence risk among HCV patients with a previous successfully treated HCC.

"The impact of NASH over the past several years has been troubling to researchers as they seek to better understand this complex patient population, who often live with other comorbid conditions. While there are no approved therapies to date, there are multiple promising therapies currently under review with the FDA," said Michael W. Fried, M.D., FAASLD, Co-Founder and Chief Medical Officer, Target RWE.

"Identifying patient profiles in the real world who may benefit from these novel treatments will be critical. TARGET-NASH has been the cornerstone of important analyses surrounding disease classification and progression. This provides unique insights for stakeholders in the development of novel therapeutics for NASH that are unmatched in the RWE space."

Sponsored by Target RWE, TARGET-NASH provides unique insights for stakeholders in the development of novel therapeutics for NASH. TARGET-NASH is a longitudinal, observational cohort of 7,000+ adult and pediatric participants with NAFLD and/or NASH receiving usual care from academic and community centers in the U.S. and Europe. TARGET-HCC has additionally enrolled more than 3,000 patients with HCC to date.

Real world evidence from the TARGET-NASH cohort has been presented and published in numerous peer-reviewed journals and major academic conferences. Most recently, leading researchers validated a non-interventional risk-based classification score to predict risk for adverse outcomes in NAFLD/NASH, considered monumental research in the field of liver disease toward better understanding optimal patient populations. In late 2022, Target RWE further enriched its NASH real world dataset with the Cirrhosis Quality Collaborative partnership with the American Association for the Study of Liver Diseases (AASLD).

TARGET-NASH has an extensive biorepository of 100,000+ whole blood, plasma, and serum samples, combined with lengthy clinical patient follow-up, are ideal for pharmaceutical industry partners to identify biomarkers associated with progression in NASH.

Target RWE recently announced the launch of 35 new disease state registries, which includes the entire spectrum of immune-mediated inflammatory conditions to capture valuable real world patient data to better understand these complex conditions and how treatment approaches may overlap. Of these new communities, TARGET-LD (Liver Disease), partnered with AASLD, and TARGET-Metabolic will be sister registries to TARGET-NASH.