Mirum Pharmaceuticals Showcases Maralixibat Transplant-Free Survival Data for Progressive Familial Intrahepatic Cholestasis and Long-Term Safety Analyses for Alagille Syndrome during WCPGHAN Annual Meeting
- Maralixibat-treated patients achieving serum bile acid control have improved five-year native liver survival; nominated for prestigious Alex Mowat Prize for best oral presentation in hepatology.
- Safety data from more than five years of maralixibat treatment across three Phase 2 ALGS studies demonstrate that gastrointestinal events were generally mild to moderate and transient in nature, with no discontinuation of maralixibat treatment.
“The breadth of maralixibat data collected for more than five years, coupled with our understanding of the safety and tolerability of long-term use, helps to underscore its importance as a potentially transformative treatment for patients with ALGS and PFIC2,” said
Oral Presentation: 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.
This abstract has been nominated for the Annual Alex Mowat Prize for best oral presentation in hepatology. Results to be announced on
The presentation is available on Mirum’s website in the Publications and Presentations section.
An analysis from the Phase 2 INDIGO open-label study evaluating long-term clinical efficacy and transplant-free survival with maralixibat in patients (n=19) with bile salt export pump (BSEP) deficiency, or progressive familial intrahepatic cholestasis type 2 (PFIC2) was presented during the congress. These data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) response had five-year transplant-free survival. In addition, those patients who achieved sBA response also had significant improvements in pruritus, liver parameters, quality of life and growth. The study showed that maralixibat was generally well-tolerated and the most common adverse events were mild to moderate diarrhea and abdominal pain, which were transient in nature; no gastrointestinal events led to discontinuation of maralixibat.
Poster Presentation: H-ePwP-030: Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment
To be presented on
Poster now available via the WCPGHAN ePoster exhibition section of the website and on Mirum’s website in the Publications section.
Data from more than five years of evaluation across three maralixibat Phase 2 clinical trials and their extension studies were analyzed to assess treatment-emergent adverse events of diarrhea and abdominal pain (GI events) in patients with ALGS. In the 86 patients evaluated, the analysis included incidence, severity, seriousness, time to onset, and duration of events, irrespective of relatedness to treatment with maralixibat, as determined by the investigator.
Findings from the integrated safety study concluded:
- Data from the 13-week placebo group demonstrated that rates of diarrhea were similar between maralixibat and placebo, with a slight difference in abdominal pain.
- The majority of GI events occurred within the first four weeks of treatment and lasted less than one week in duration.
- For patients who experienced GI events, the majority of diarrhea and abdominal pain were mild to moderate in severity and transient in nature.
- There were no GI-related discontinuations of maralixibat over the five years of evaluation.
To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.
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
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential of maralixibat to be a meaningful alternative to the current standard of care for ALGS and PFIC2, and the regulatory approval pathway for maralixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “could,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum’s filings with the