Mirum Pharmaceuticals Showcases Five New LIVMARLI (maralixibat) Presentations at the EASL Congress

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the European Association for the Study of the Liver (EASL) Congress 2023, taking place in Vienna, Austria.

The congress presentations feature new analyses from the LIVMARLI (maralixibat) oral solution MARCH-PFIC study as well as the long-term extension study, MARCH-ON. MARCH-PFIC is the largest Phase 3 trial in progressive familial intrahepatic cholestasis (PFIC), evaluating all types, including genetic types that had not previously been studied. The data underscore long-term maintenance of effect, markers of improved liver health, impact on sleep improvement and long-term safety data. Additionally, data showing LIVMARLI’s benefit in adult patients with Alagille syndrome (ALGS) was presented during EASL.

“We are delighted to see the significant impact LIVMARLI has on multiple measures of health for patients with PFIC, including improvements in key parameters such as sleep, long-term pruritus control and importantly, bilirubin, which is a marker of liver health and disease progression,” said Pam Vig, PhD, head of research and development at Mirum. “We are also very excited to report, for the first time, pruritus control with LIVMARLI in adult patients with Alagille syndrome, providing critical information as children transition to adult care. These data further demonstrate LIVMARLI’s ability to impact lives across these indications.”

Data from Mirum’s studies of LIVMARLI (maralixibat) include oral and poster presentations:

Abstract OS-072: Maralixibat leads to significant reductions in bilirubin for patients with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC

The goal of the analysis was to characterize the impact of maralixibat on bilirubin in patients with PFIC as part of the MARCH Phase 3 study, specifically evaluating the mean change in baseline in total and direct bilirubin. Clinical evidence suggests that elevated serum bilirubin levels may indicate poorer outcomes in patients with PFIC. The data demonstrated:

• Maralixibat resulted in statistically significant improvements in total and direct bilirubin in the All-PFIC cohort versus placebo (total: -18.4 mmol/L vs. 15.9 mmol/L, p=0.047; direct: -12.9 vs. 13.5, p=0.048).
• Of the patients with abnormal total bilirubin values at baseline, 40% of maralixibat-treated patients achieved normalization, versus none in the placebo group.
• Reductions in bilirubin corresponded with reductions in serum bile acids (94.4% with normalized total bilirubin on maralixibat, 19.0% without normalized total bilirubin on maralixibat, 3.3% without normalized total bilirubin on placebo).
• Data suggest that maralixibat may yield clinical improvements in liver health in patients with PFIC.

Maralixibat is the only IBAT inhibitor to demonstrate significant decreases in total and direct bilirubin compared with placebo in patients with PFIC.