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 signaling through FGF21’s receptors, EFX has the potential to address NASH by reducing liver fat, inflammation and fibrosis.
We believe EFX is both and redirects calories away from the liver, restores lipid metabolism in the liver to a healthy state and protects against hepatocyte stress and death. We also believe it directly suppresses downstream inflammation and fibrosis.
Results from our Phase 2a BALANCED study show EFX’s potential to be a foundational NASH monotherapy. We reported the results of the primary endpoint and multiple secondary endpoints at week 12. All three dose groups treated with AKR-001 saw highly statistically significant absolute reductions in liver fat (12-15%, compared to 0% for placebo), relative reductions in liver fat (63-72%, compared to 0% for placebo) and reduction in the liver enzyme ALT (24-32 U/L, compared to 6U/L for placebo).
We also reported the results of exploratory endpoints after 16 weeks of treatment, including biopsy-based measurements of liver fibrosis and NASH. Specifically, we reported that 48% of all EFX patients achieved at least a one stage improvement in fibrosis without worsening of NASH, with a 62% response rate for patients taking the 50mg dose of EFX. In addition, 28% of all EFX patients achieved at least a two-stage improvement in fibrosis stage. 48% of all EFX patients – and 54% on the 50 mg dose – achieved resolution of NASH without worsening of fibrosis. EFX patients experienced improvements in glycemic control, including reductions of up to 0.9 in HbA1c among EFX patients with Type 2 diabetes, as well as improvements in triglycerides, HDL cholesterol and non-HDL cholesterol, with no increases in LDL cholesterol. EFX patients in the top two dose groups also lost, on average, about 2-4 kg over the course of the study.
These highly encouraging results pave the way for continued development of EFX, which we believe has the potential to be a foundational NASH monotherapy.