Mirum Pharmaceuticals Reports Positive Phase 2b Data for Volixibat in PSC at EASL Congress
Data from the VISTAS study shows volixibat reduces pruritus in primary sclerosing cholangitis, while brelovitug demonstrates efficacy in hepatitis delta virus, supporting upcoming New Drug Application filings.
Mirum Pharmaceuticals Inc. (NASDAQ: MIRM) reported positive results from its rare liver disease programmes at the EASL International Liver Congress 2026 on 30 May. The company presented data highlighting the clinical potential of its pipeline assets, including volixibat for primary sclerosing cholangitis (PSC) and brelovitug for chronic hepatitis delta virus (HDV).
The Phase 2b VISTAS study for volixibat in PSC demonstrated rapid, sustained, and clinically meaningful reductions in pruritus. These findings support the company’s intention to submit a New Drug Application (NDA) for the treatment in the second half of 2026.
Data from the Phase 2b AZURE-1 study of brelovitug in chronic HDV showed strong efficacy, with high rates of virologic response and ALT normalization at Week 24. The results were observed even in patients with advanced disease, reinforcing the potential of the therapy.
Mirum expects topline data from its Phase 3 AZURE trials for brelovitug later in 2026, with a potential Biologics License Application (BLA) submission planned for 2027. This timeline aligns with the company’s strategy to advance its portfolio of novel therapies for rare and orphan diseases.
Real-world evidence for LIVMARLI (maralixibat) in progressive familial intrahepatic cholestasis (PFIC) was also presented. The data showed improved event-free survival compared to an external control cohort, reinforcing the benefits of the established therapy in reducing the risk of surgical biliary diversion, liver transplantation, and death.
Mirum Pharmaceuticals Inc. is a biotech company that develops and commercialises novel therapies for rare and orphan diseases. The company continues to focus on advancing its clinical programmes while managing the regulatory pathways for its key assets.
The presentation at the EASL Congress underscores the company’s progress in addressing unmet medical needs in hepatology. Investors and analysts will be monitoring the upcoming NDA and BLA submissions as critical milestones for Mirum’s growth trajectory.
