Therapeutic Areas

We develop novel therapies to address high unmet medical need where metabolic dysregulation is a core disease driver.

Our current focus is on metabolic dysfunction-associated steatohepatitis (MASH), a severe form of metabolic dysfunction-associated steatotic liver disease marked by whole-body metabolic dysregulation with multiple, complex disease drivers that also increase the risk of cardiovascular disease. MASH is caused by excessive caloric overburdening in the liver, which leads to excess storage of fat in the liver and stress on liver cells called hepatocytes. Hepatocyte cell stress, injury and death leads to liver inflammation, which may cause fibrosis (scarring).

Ultimately, liver fibrosis may progress to cirrhosis (severe scarring) and liver cancer. People with MASH and mild liver scarring (fibrosis) may or may not experience symptoms, but severe scarring (cirrhosis) increases risk of liver failure and death.

Effective treatment of MASH requires intervening across stages of disease progression, addressing both the early-stage metabolic drivers and later-stage inflammation and fibrosis.

We believe restoring whole-body metabolic balance is essential to treating MASH. Our lead program, EFX, has yielded promising data showing potential to address the diverse drivers of MASH pathology.

Learn more about the published research supporting efruxifermin (EFX) development.

Learn more about the published research supporting EFX development.