Mirum Pharmaceuticals announced that it has finished enrolling patients in its Phase 3 EXPAND study of Livmarli (maralixibat) for rare cholestatic liver diseases beyond Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). The enrollment milestone means the study is now fully staffed and ready to begin the final data‑collection phase, bringing the trial closer to its planned Q4 2026 readout and the possibility of regulatory submissions for a broader indication.
The EXPAND study is a randomized, double‑blind, placebo‑controlled trial that enrolls patients aged six months or older. Its primary endpoint is the change in pruritus severity from baseline to week 20, with secondary endpoints assessing serum bile acids and other cholestatic markers. Completion of enrollment allows the study to move into the final 20‑week observation period, after which topline data are expected in the fourth quarter of 2026.
Livmarli is currently approved in the United States and European Union for cholestatic pruritus associated with Alagille syndrome and PFIC. The EXPAND study seeks to generate data that could support a label expansion to other rare cholestatic liver diseases, potentially enlarging the drug’s addressable market and increasing its revenue potential.
Mirum’s 2025 financial results underscore the commercial momentum behind Livmarli. Net product sales reached $521 million, a 55% increase from 2024, with Livmarli sales alone totaling $360 million, a 69% year‑over‑year rise. The company reaffirmed its 2026 global net product sales guidance of $630 million to $650 million, while reporting a non‑GAAP EPS loss of $0.11 for Q4 2025. These figures illustrate strong demand for Livmarli and a solid cash position of $391.4 million as of December 31 2025.
"Completing enrollment in EXPAND marks an important milestone in our efforts to broaden the reach of LIVMARLI to additional patients living with cholestatic pruritus," said Joanne Quan, Chief Medical Officer at Mirum. "Itch due to cholestasis profoundly affects the daily lives of patients and their families. We aim to address the unmet medical needs of these patients by leveraging our established IBAT mechanism." Analysts upgraded the company following the announcement, citing the strong revenue growth, Livmarli’s sales momentum, and the pipeline’s upcoming data readouts as key drivers of positive market sentiment.
The content on EveryTicker is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.