Mirum Pharmaceuticals Highlights Commitment to Rare Diseases at J.P. Morgan Healthcare Conference

Mirum Pharmaceuticals Expands Focus on Rare Diseases with Upcoming Presentation

Mirum Pharmaceuticals, Inc. is set to showcase its commitment to addressing rare and orphan diseases at the 43rd Annual J.P. Morgan Healthcare Conference on January 15, 2025. The San Francisco event, a hallmark gathering for biopharmaceutical companies, will highlight Mirum's innovative approaches to developing therapies for challenging medical conditions. The presentation, scheduled for 9:45 a.m. PT, will be available via live webcast, allowing broader access to stakeholders and investors interested in the company's advancements and pipeline.

Mirum's current portfolio includes three FDA-approved medications: LIVMARLI® (maralixibat), CHOLBAM® (cholic acid), and CHENODAL® (chenodiol). LIVMARLI, an IBAT inhibitor, is specifically designed to treat cholestatic pruritus in patients suffering from Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). This medication is approved for use in children as young as three months in the U.S. and two months in Europe. Mirum is also actively pursuing further label expansion through its Phase 3 EXPAND study, demonstrating the company's commitment to enhancing treatment options for patients with these rare conditions.

In addition to its existing therapies, Mirum is advancing its late-stage pipeline with investigational treatments. Notably, Volixibat, which has received Breakthrough Therapy Designation for cholestatic pruritus in patients with primary biliary cholangitis (PBC), is undergoing evaluation in registrational studies for both primary sclerosing cholangitis (PSC) and PBC. Furthermore, the company recently reported positive topline results from the Phase 3 RESTORE study for CTX, leading to a new drug application to the FDA for chenodiol approval. Plans are also in place for a Phase 2 study for MRM-3379, a PDE4D inhibitor targeting Fragile X syndrome, demonstrating Mirum's ongoing dedication to addressing significant unmet medical needs in the rare disease space.

As Mirum Pharmaceuticals prepares for its forthcoming presentation, the biopharmaceutical landscape continues to evolve, with a growing emphasis on specialized therapies for rare diseases. The company’s strategic initiatives underscore its potential to make a meaningful impact in this niche market, fostering hope for patients who face limited treatment options. Further information regarding the company’s developments can be accessed through its website and social media channels.