Data from LIVMARLI (maralixibat) oral solution clinical studies and real-world settings in Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) were presented in oral and poster presentations during the meeting.
'We continue to build upon the strong body of evidence demonstrating LIVMARLI's potential to provide long-term benefit to PFIC patients across key quality of life and liver disease parameters, as well as improvements in varied genetic types of PFIC,' said
Abstract 587: Long-term Maintenance of
Plenary Session: Hepatology Highest Scoring Abstracts
Presented by Professor
Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study.
Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial
Presented by Lorenzo D'Antiga, MD,
Patients treated with LIVMARLI experienced significant decreases in both total and direct bilirubin compared to placebo. 40% of the patients with abnormal bilirubin at baseline treated with LIVMARLI achieved normalization versus none in the placebo group. Further, these reductions in bilirubin were consistent with reductions in sBAs.
Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience
Presented by
Real-world evidence from 116 patients treated with LIVMARLI for at least one year showed that more than one-third of patients were able to discontinue 1 concomitant antipruritic medication, and one in five patients discontinued 2 medications. Reductions in concomitant medication usage were seen across all medication types and suggest that LIVMARLI may reduce the polypharmacy burden within the first year of treatment.
Other presentations featured during ESPGHAN include: Abstract 592: Maralixibat Leads to Significant Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Without a Genetic Diagnosis: Data from the MARCH Trial
Presented by Professor
Abstract 599: Maralixibat Leads to Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Due to MDR3 Deficiency: Data From the MARCH/MARCH-ON Trials
Presented by Professor
Abstract 595: Improvements in Pruritus with Maralixibat are Associated with Improved Quality of Life for Patients with Progressive Familial Intrahepatic Cholestasis: Data From the MARCH Trial
Presented by
Abstract 597: Maralixibat Can Improve Cholestatic Pruritus in Children with Progressive Familial Intrahepatic Cholestasis Who Previously Underwent a Surgical Biliary Diversion: Data From the MARCH/MARCH-ON Trials
Presented by Lorenzo D'Antiga, MD,
About LIVMARLI (maralixibat) oral solution
LIVMARLI (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the
LIVMARLI is also the only approved IBAT inhibitor approved by the
Mirum has also submitted LIVMARLI for approval in
LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC.
About
LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the
Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023.
Forward-Looking Statements
This press release includes forward-looking statements pertaining to the Company's planned participation at a scientific conference, including data presentation title and synopsis, which may include discussion of the Company's clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI in various liver disease indications and in patient populations that are investigational only. 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 'will,' 'goal,' 'potential' and similar expressions are intended to identify forward-looking statements. The accuracy of such statements is subject to a number of risks, uncertainties and assumptions including, but are not limited to, the following factors: the uncertainties inherent in research and development; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Mirum's business and prospects, adverse developments in our focused markets, or adverse developments in the
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