Mirum Pharmaceuticals to Present LIVMARLI® Efficacy Data at NASPGHAN Annual Meeting 2024

Mirum Pharmaceuticals Set to Showcase LIVMARLI® Efficacy at NASPGHAN Annual Meeting

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) announces its active participation in the upcoming NASPGHAN annual meeting, taking place from November 6-9, 2024, in Hollywood, Florida. The company plans to present crucial clinical data on LIVMARLI® (maralixibat) oral solution, specifically targeting its effectiveness in treating pediatric patients suffering from Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). This platform provides an opportunity for Mirum to highlight significant advancements in the management of these complex liver conditions, while also reinforcing its commitment to enhancing pediatric healthcare.

The presentations will feature key findings from various clinical studies that underline the therapeutic benefits of LIVMARLI. Among the notable presentations is Abstract 77, which showcases improvements in growth metrics for PFIC patients derived from the MARCH/MARCH-ON trials. This data is pivotal as it illustrates the tangible impact that maralixibat can have on the physical development of children suffering from this challenging condition. Additionally, Abstract 361 will present long-term data from the MERGE study, affirming that the clinical benefits for ALGS patients persist even after seven years of treatment, emphasizing the sustained efficacy of LIVMARLI over time.

Further insights will be shared through Abstract 637, which evaluates improved liver health markers in children treated with maralixibat for PFIC. This finding is crucial as it connects treatment outcomes with quality of life improvements, a theme echoed in Abstract 638, which links reduced pruritus to enhanced overall well-being for patients with cholestatic liver disease. Finally, Abstract 664 will offer real-world evidence regarding the long-term effects of maralixibat on concomitant medication use for managing cholestatic pruritus in Alagille syndrome cases. Collectively, these presentations underscore the integral role of LIVMARLI in transforming the treatment landscape for pediatric patients with liver diseases.

In addition to the clinical data, Mirum Pharmaceuticals emphasizes its ongoing dedication to advancing treatment options for patients with ALGS and PFIC. LIVMARLI has received FDA approval for use in pediatric patients aged three months and older with ALGS and 12 months and older with PFIC in the U.S., demonstrating the company's focus on addressing unmet medical needs in this vulnerable population. For more information about LIVMARLI, residents in the U.S. can visit LIVMARLI.com.

As Mirum Pharmaceuticals prepares for this important event, the company not only aims to showcase its innovative therapeutic solutions but also highlights the importance of ongoing research in the pediatric liver disease space, advocating for better treatment protocols and quality of life for affected families.