Our multi-modal investigational drug, efruxifermin (EFX), is designed to treat NASH holistically.

EFX has been engineered to mimic the biological activity of fibroblast growth factor 21 (FGF21), which regulates multiple metabolic pathways and cellular processes. By delivering sustained and balanced signaling through FGF21’s receptors in liver and adipose tissue, EFX has the potential to treat NASH by addressing all core drivers of disease progression.

EFX leverages the whole body to improve metabolic balance.

Our Phase 2a clinical trial (the BALANCED study) showed how balanced agonism of FGF21’s receptors has the potential to:

  • Reduce liver fat, with EFX patients achieving 63-72% relative reductions in liver fat, compared to 0% for placebo
  • Improve glycemic control, including reductions in HbA1c of 0.6% to 0.9% among patients with Type 2 diabetes treated with 50mg and 70mg, respectively, compared to 0% for placebo
  • Restore healthier lipoprotein profile, including reductions in triglycerides of 39% to 48%, compared with a 6% increase for placebo

We believe effective treatment of NASH requires the reversal of fibrosis – particularly among patients with the most advanced fibrosis. We believe EFX has the potential to act at each step of the cascade that leads to fibrosis.

Data from the Phase 2a BALANCED study showed EFX’s potential to reduce fibrosis in as little as 16 weeks of treatment:

  • 48% of all EFX-treated patients achieved at least a one-stage improvement in fibrosis without worsening of NASH1
  • 50% of all EFX-treated patients who had fibrosis stage 2 or 3 at the start of the study achieved at least a two-stage improvement in fibrosis by the end of the study
  • 48% of all EFX-treated patients achieved resolution of NASH2 without worsening of fibrosis

EFX was also reported to be generally well tolerated. Gastrointestinal events, which were transient in nature, were the most frequent side effect.

These encouraging results pave the way for continued development of EFX, which we believe has the potential to be a foundational NASH monotherapy.

1 No worsening of NASH mean no increase in NAFLD Activity Score (NAS) for ballooning, inflammation, or steatosis.

2 NASH resolution is defined as having a NAS of 0 or 1 for lobular inflammation and a score of 0 for ballooning, out of a total possible NAS of 8 (0-3 for steatosis, 0-3 for lobular inflammation, and 0-2 for hepatocyte ballooning).

We are exploring additional pipeline programs and assets to complement EFX.