Foster City, California, United States, October 11, 2026
Mirum Pharmaceuticals, Inc. announced plans to host an investor conference call to share topline results from its Phase 3 EXPAND clinical study evaluating LIVMARLI® (maralixibat) in patients with ultra-rare cholestatic liver diseases. The announcement, issued on October 11, 2026, highlighted the company’s continued efforts to develop treatments for rare liver conditions associated with significant unmet medical needs. The investor call was scheduled for October 12, 2026, at 8:30 a.m. Eastern Time, providing investors and other stakeholders with an opportunity to hear the latest findings from the study. The trial evaluates maralixibat for cholestatic pruritus, a persistent and distressing itching symptom associated with impaired bile flow. The study includes patients aged six months and older with ultra-rare cholestatic liver diseases, including biliary atresia. The announcement marked an important communication milestone for Mirum’s rare disease development program, with the topline findings expected to inform further clinical and regulatory planning.
EXPAND Study Targets Cholestatic Pruritus
The Phase 3 EXPAND study was designed to investigate the potential of maralixibat to reduce cholestatic pruritus in patients with ultra-rare liver diseases. Cholestatic pruritus can substantially affect patients’ daily lives, disrupting sleep, increasing discomfort and creating additional challenges for caregivers, particularly when the condition affects infants and children. Biliary atresia is a rare condition in which bile ducts are blocked or absent, preventing normal bile drainage from the liver. The resulting cholestasis can contribute to progressive liver damage and other complications. Maralixibat is an ileal bile acid transporter (IBAT) inhibitor designed to reduce the reabsorption of bile acids in the intestine and increase their elimination through the feces. By modifying bile acid circulation, the treatment aims to address biological processes associated with cholestatic disease symptoms. The EXPAND program extends the investigation of maralixibat into additional ultra-rare cholestatic liver disease populations, complementing its established development and approved uses in other eligible patient groups. The trial’s topline results are important for understanding whether the medicine could provide a treatment option for patients whose conditions may be associated with persistent itching and limited therapeutic alternatives.
Investor Call Highlights Clinical Development Milestone
Mirum scheduled the investor conference call for October 12, 2026, at 8:30 a.m. Eastern Time, with access available through telephone and webcast. The company announced that an archived webcast would be available for replay through the investor section of its corporate website. The call was intended to communicate the topline findings from the Phase 3 EXPAND study to investors and other interested stakeholders. Topline clinical results generally provide an initial summary of a study’s principal findings, including whether its primary endpoint was achieved and the overall direction of the results. Detailed clinical interpretation requires consideration of the study design, patient population, statistical analysis, secondary endpoints and safety findings. For pharmaceutical companies developing treatments for rare diseases, these updates can inform decisions about regulatory submissions, further clinical development and future investment in research. Mirum’s announcement established the timing and purpose of the call but did not itself provide the complete clinical results. The company subsequently announced on October 12 that the study had met its primary endpoint, providing a separate update on the clinical outcome.
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Mirum Advances Its Rare Disease Treatment Portfolio
Mirum Pharmaceuticals focuses on developing and commercializing treatments for rare liver and rare genetic diseases. Its portfolio includes LIVMARLI (maralixibat) for eligible patients with Alagille syndrome and progressive familial intrahepatic cholestasis, alongside other medicines for rare conditions. The company also has investigational programs targeting diseases with substantial unmet medical needs. The EXPAND study reflects its strategy of evaluating existing therapeutic approaches in additional rare disease populations where further treatment options may be needed. Progress in these programs depends on demonstrating clinically meaningful benefits, establishing an acceptable safety profile and meeting regulatory requirements. Further clinical evidence will be important in determining the potential role of maralixibat in additional ultra-rare cholestatic liver diseases. The investor call and subsequent results announcement provide stakeholders with updates on this development pathway, while additional regulatory review will determine whether the treatment can be approved for new indications. The study represents a relevant development for rare liver disease research and the broader pharmaceutical industry, particularly in the pursuit of treatments for patients with limited therapeutic choices.
Source: Mirum Pharmaceuticals press release
