Long-Term Data from Mirum’s LIVMARLI Studies in ALGS and PFIC Highlighted at EASL Congress
Mirum Pharmaceuticals presented data at the EASL Congress showing long-term benefits of LIVMARLI (maralixibat) in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The MERGE study demonstrated sustained clinical benefits for ALGS patients over seven years, including reductions in pruritus and serum bile acids, and improvements in height.
The MARCH/MARCH-ON trials indicated statistically significant growth improvements in PFIC patients over 82 weeks, with better height and weight z-scores. Another analysis from these trials showed that reductions in serum bile acids correlated with improved liver health markers like bilirubin, suggesting potential disease-modifying effects of LIVMARLI.
- Sustained benefits in ALGS patients for up to seven years.
- Statistically significant growth improvements in PFIC patients over 82 weeks.
- Correlations between reduced serum bile acids and improved liver health markers.
- Potential disease-modifying effects of LIVMARLI.
- No new safety data reported, which may raise concerns about long-term safety.
- The results are based on a number of patients.
- The duration of the PFIC study is relatively short (82 weeks) for long-term conclusions.
Insights
The long-term data from Mirum Pharmaceuticals on their drug LIVMARLI (maralixibat) highlights its sustained efficacy in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) over extended periods. For ALGS, the data from the MERGE study revealed that patients experienced durable benefits over seven years, including reductions in pruritus (itching) and serum bile acids, as well as improvements in height. This suggests that LIVMARLI not only manages symptoms but also supports growth, which is important for pediatric patients.
In PFIC, the analyses from the MARCH and MARCH-ON trials demonstrated statistically significant improvements in height and weight z-scores among patients after 82 weeks of treatment. Additionally, reductions in serum bile acids were correlated with decreases in bilirubin levels, indicating potential improvements in overall liver health. These results underscore the potential of LIVMARLI as a disease-modifying treatment for PFIC, offering hope for better long-term outcomes in these challenging conditions.
Overall, these findings provide strong evidence of LIVMARLI's lasting clinical benefits, making it a promising option for patients with ALGS and PFIC.
The new data on LIVMARLI presented at the EASL Congress has significant implications for Mirum Pharmaceuticals' market position. Long-term, sustained clinical benefits from a drug can be a key differentiator in the competitive pharmaceutical market, translating into stronger sales and higher market penetration. By demonstrating the drug's efficacy over seven years, Mirum strengthens its case for both current and potential investors.
Investor confidence is likely to be bolstered by the fact that LIVMARLI not only addresses symptoms like pruritus but also positively impacts growth and key liver health markers. This comprehensive benefit profile can lead to increased adoption by healthcare providers and improved patient adherence, ultimately driving revenue growth.
Moreover, the positive data can support Mirum's ongoing efforts to expand the drug's market approval and reimbursement coverage, which are critical for scaling their business.
Contrast this with the broader biotech sector, where long-term data often reveals diminishing returns or unforeseen side effects and LIVMARLI's results stand out as particularly robust. This could positively influence Mirum's stock performance in both the short and long term.
- Data showcases sustained clinical benefit following seven years of LIVMARLI treatment in patients with Alagille syndrome
- Long-term extension data from LIVMARLI PFIC studies highlight improvement in key liver markers and growth
THU-094: Clinical Benefits with Maralixibat for Patients with Alagille Syndrome are Durable Through 7 Years of Treatment: Data from the MERGE Study
Presented by Doug Mogul, MD, PhD – Mirum Pharmaceuticals, Inc.,
An analysis of ALGS studies evaluating LIVMARLI for up to seven years of treatment were presented to demonstrate the durability of response to LIVMARLI across multiple clinical and laboratory outcomes. Data demonstrated that nearly all patients who remained on LIVMARLI for seven years experienced benefit including clinically meaningful reductions in pruritus and serum bile acids, along with improvements in height.
View the full presentation.
THU-129: Maralixibat Improves Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials
Presented by Professor Richard J. Thompson, MD, PhD – King's College,
This analysis reported on the long-term impact of LIVMARLI on improvements in growth across a variety of PFIC types in 60 patients from the All-PFIC cohort of the MARCH/MARCH-ON clinical trials. Patients treated with LIVMARLI from the All-PFIC cohort had a statistically significant improvement in height and weight z-scores which persisted through 82 weeks of treatment.
View the full presentation.
THU-158: Improvements in Serum Bile Acid Levels are Associated with Improvements in Key Markers of Liver Health After Maralixibat Treatment in Children with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials
Presented by Lorenzo D’Antiga, MD - Papa Giovanni XXIII Hospital,
This analysis aimed to assess correlations between serum bile acid (sBA) improvements and key liver parameters, including bilirubin. These data showed that reductions in sBA after treatment with LIVMARLI are correlated with reductions in bilirubin, a key marker of liver disease. These results indicate the potential for LIVMARLI to have disease modifying effects and improvements in liver health for patients with PFIC.
View the full presentation.
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 European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for
LIVMARLI has received positive opinion by CHMP in
LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.
LIVMARLI can cause side effects, including:
Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.
US Prescribing Information
EU SmPC
Canadian Product Monograph
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.
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.
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
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 regarding patient growth data while on LIVMARLI, the reliability of long-term data, and 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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Media Contact:
Erin Murphy
media@mirumpharma.com
Investor Contact:
Andrew McKibben
investors@mirumphama.com
Source: Mirum Pharmaceuticals, Inc.
FAQ
What are the long-term benefits of LIVMARLI in Alagille syndrome (ALGS) patients?
How does LIVMARLI affect growth in PFIC patients?
What liver health markers improved in PFIC patients treated with LIVMARLI?
What is the potential impact of LIVMARLI on liver health in PFIC patients?
