Mirum Pharmaceuticals to Present Maralixibat Data and Host Symposium at the 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) Annual Meeting 2021

Rhea-AI Summary

Mirum Pharmaceuticals (Nasdaq: MIRM) announced that data from maralixibat studies will be presented at the 6th Annual World Congress of Pediatric Gastroenterology from June 2-5, 2021. Notable findings include an oral presentation by Professor Richard Thompson on the association between serum bile acid control and liver survival in children with PFIC. A poster presentation on gastrointestinal tolerability will also occur. A symposium discussing treatments for pediatric cholestasis will be held on June 3. Maralixibat is under investigation for rare cholestatic liver diseases, with significant results in ongoing trials.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from maralixibat studies in patients with Alagille syndrome and progressive familial intrahepatic cholestasis type 2 will be presented in oral and poster sessions during the 6^th Annual World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) taking place virtually June 2-5, 2021.

Data featured during the congress include:

Oral Presentation
Abstract number: H-O-012: Serum bile acid control in long-term maralixibat treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency.
Presenter: Professor Richard Thompson, M.D., Ph.D., Professor of Molecular Hepatology at King’s College London and principal investigator for the INDIGO study
Session: Highest Scoring Abstracts
Date and time: Thursday, June 3, 2021 – 3:55-4:05 p.m. CEST
This abstract has been nominated for the prestigious Annual Alex Mowat Prize for best oral presentation in hepatology. Results to be announced during the congress closing ceremony on June 5, 2021 at 1:30 p.m. CEST.

Poster Presentation
Abstract number: H-ePwP-030: Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment.
Presenter: Rakesh K. Raman, M.D., Mirum Pharmaceuticals, Inc.
Session: ePoster Session Hepatology
Date and time: Saturday, June 5, 2021 – 10:40-11:30 a.m. CEST

Satellite Symposium
Pediatric Cholestasis: Itching for an alternative to liver transplantation.
Date and time: Thursday, June 3 at 5:30 p.m. CEST
Featured Speakers: Dr. Binita M. Kamath (The Hospital for Sick Children (SickKids) Toronto, Ontario, Canada), Dr. Emmanuel Gonzales (Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France), Dr. Richard Thompson (King’s College, London, UK)

Mirum will be sponsoring a virtual symposium featuring leading experts who will discuss the current treatment landscape for pediatric cholestasis, including the high unmet medical need and the potential for pharmacologic treatments as an alternative to surgical options. This symposium will highlight diagnostic challenges related to Alagille syndrome and progressive familial intrahepatic cholestasis as well as the burden of disease on children and their caregivers. Data supporting the potential rationale for apical sodium-dependent bile acid transporter inhibition as an alternative to surgical treatment options will also be explored.

WCPGHAN registered attendees can view the live symposium via the WCPGHAN website within the calendar of events in the Scientific Programme At-A-Glance section.

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent tre

FAQ

What is the significance of the maralixibat studies presented by Mirum Pharmaceuticals at WCPGHAN 2021?

The studies focus on the effects of maralixibat in patients with Alagille syndrome and PFIC, highlighting its potential for improving liver survival and gastrointestinal tolerability.

When will Mirum Pharmaceuticals present data on maralixibat at the World Congress of Pediatric Gastroenterology?

Oral and poster presentations are scheduled during the congress, taking place from June 2-5, 2021.

What were the findings regarding maralixibat's impact on children with PFIC?

Findings suggest that serum bile acid control through maralixibat treatment is linked to improved liver survival in children with PFIC.

What does the satellite symposium sponsored by Mirum Pharmaceuticals focus on?

The symposium will discuss the treatment landscape for pediatric cholestasis and explore pharmacologic alternatives to liver transplantation.

What is Maralixibat and its role in treating cholestatic liver diseases?

Maralixibat is an investigational drug that inhibits bile acid absorption, potentially reducing liver damage in cholestatic liver disease patients.