Learn about clinical trials that are currently recruiting.

— Phase 3 NASH Study
— Phase 4 PBC Study
— Phase 2 Biliary Atresia Study


At Intercept, our work is motivated by our desire to help patients and families who struggle with chronic liver diseases and need better treatment options.


Overview of NASH

  • Nonalcoholic fatty liver disease (NAFLD) is associated with excessive fat in the liver (steatosis) and can progress to NASH, which is defined by the histologic hallmarks of inflammation, cell death, and fibrosis
  • Primary NASH is associated with insulin resistance
  • Secondary NASH is rare in adults and is caused by medical or surgical conditions, or drugs such as tamoxifen

Natural history of NASH

  • Histologically, NASH is similar to alcohol-induced steatohepatitis, and is associated with factors that cause an increase in oxidative stress and promote expression of proinflammatory cytokines
  • NASH is the most common cause of fibrosis and cirrhosis in patients with unexplained increased alanine aminotransferase

Epidemiology of NASH

  • NASH is one of the most common liver diseases
  • NASH is now the 2nd leading cause of liver transplant in the US, surpassing alcoholic liver disease, and is expected to be the leading cause by 2020
  • Since 2001, liver transplants attributed to NASH have increased tenfold
  • NASH is estimated to affect 3–5% of the US adult population
  • Studies show that 5% to 25% of the growing number of patients with NASH develop cirrhosis within 7 years of follow-up

Identifying patients with NASH

  • Highest prevalence is in men between 40 and 65 years of age
    • Higher prevalence in Hispanics
    • Lower prevalence in African Americans
  • Family members are also at increased risk
  • Factors associated with NASH and cirrhosis are
    • Increasing age
    • More extensive obesity
    • Glucose intolerance or type 2 diabetes

Symptoms of NASH

  • Usually a silent disease with minimal symptomatology
    • Fatigue, weight loss, and weakness appear in later stages as disease progresses and cirrhosis develops

Potential complications of NASH

Outcomes may include, but are not limited to

  • Fibrosis
  • Cirrhosis
  • Insulin resistance
  • Variceal hemorrhage
  • Sepsis
  • Hepatocellular carcinoma
  • Liver failure

Diagnosis of NASH

  • Any of the following factors, especially with a history of abnormal levels of aspartate aminotransferase (AST) and alanine aminotransferase (ALT), should lead to a workup for NASH:
    • Presence of obesity, especially morbid obesity (BMI >35)
    • Diagnosis of type 2 diabetes mellitus
    • Diagnosis of metabolic syndrome
    • History of obstructive sleep apnea
    • Presence of insulin resistance
    • Chronic elevation of AST/ALT, otherwise unexplained
    • Detailed patient history of alcohol consumption—threshold <20 g/day in women, <30 g/day in men
  • A liver biopsy is considered necessary to diagnose NASH

Treatment of NASH

Currently, no therapies are approved for the treatment of NASH.

  • To prevent disease progression of NASH, physicians recommend
    • Lifestyle changes
    • Exercise to reduce body weight
    • Treatment of concomitant diabetes and dyslipidemia
  • All are accepted as the standard of care, but none are conclusively shown to prevent disease progression
  • American Association for the Study of Liver Diseases guidelines recommend vitamin E for first-line therapy in nondiabetic patients with NASH

Development of OCA for NASH

  • OCA is currently being evaluated in an international phase 3 trial for the treatment of NASH
  • In accordance with harmonized advice from the FDA and European Medicines Authority (EMA), the Randomized Global Phase 3 Trial to Evaluate the Impact on NASH with Fibrosis of Obeticholic Acid Treatment (REGENERATE) has been designed as a double-blind, placebo-controlled pivotal Phase 3 clinical trial
  • REGENERATE will assess the potential benefit of OCA treatment on liver-related clinical outcomes

NASH resources for in-depth clinical information

  1. Prevalence of NAFLD and NASH Among a Largely Middle-Aged Population Utilizing Ultrasound and Liver Biopsy: A Prospective Study (Williams, Gastroenterology, Jan 2011)
  2. Farnesoid X receptor targeting to treat nonalcoholic steatohepatitis (Adorini, Drug Discovery Today, September 2012)
  3. Efficacy and Safety of the Farnesoid X Receptor Agonist Obeticholic Acid in Patients With Type 2 Diabetes and Nonalcoholic Fatty Liver Disease (Mudaliar, Gastroenterology, September 2013)
  4. Bile acid receptors in non-alcoholic fatty liver disease (Li, Biochemical Pharmacology, December 2013)
  5. Liver fibrosis and repair: immune regulation of wound healing in a solid organ (Pellicoro, Nature Reviews Immunology, March 2014)
  6. Farnesoid X receptor ligand obeticholic acid for non-cirrhotic, non-alcoholic steatohepatitis (FLINT): a multicentre, randomised, placebo-controlled trial (Neuschwander-Tetri, et al, AASLD November 8, 2014)

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