Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI
- None.
- None.
Insights
The recent publication in Hepatology regarding Mirum Pharmaceuticals' LIVMARLI® treatment for Alagille syndrome (ALGS) marks a significant milestone in the management of this rare liver disease. The reported 70% risk reduction in long-term clinical outcomes, such as the need for liver transplantation, is a substantial improvement over the natural history control group. This figure is particularly notable given the rarity of ALGS and the limited treatment options historically available.
From a research perspective, the use of the Global Alagille Alliance (GALA) clinical database as a control is a robust methodological choice, ensuring a comprehensive comparison to untreated patients. The statistical significance (p<0.0001) indicates a low probability that the results are due to chance, reinforcing the potential of LIVMARLI as a transformative treatment option for ALGS patients.
The implications for healthcare systems and insurers are considerable. Potentially reducing the need for liver transplants, which are complex and costly procedures, could lead to significant cost savings and improved patient quality of life. Furthermore, the data could influence future research directions and healthcare policies regarding the management of cholestatic liver diseases.
The reported findings have profound economic implications for the healthcare industry, particularly in the context of rare diseases such as ALGS, where treatment advancements can drastically alter the cost landscape. The reduction in the need for liver transplants could translate into lower long-term healthcare costs, despite the initial investment in pharmacological intervention with LIVMARLI®.
Moreover, the improved transplant-free survival rate could decrease the economic burden on patients and their families, who often face significant indirect costs such as loss of income, travel for treatment and long-term care expenses. The ripple effect of this could be seen in reduced insurance premiums and a shift in resource allocation within healthcare systems.
It is important to analyze the cost-effectiveness of LIVMARLI® in the context of ALGS treatment. The long-term savings from avoided transplants and improved quality of life for patients must be weighed against the drug's pricing and the cost of managing any associated side effects. Such an analysis would be essential for payers and policymakers when considering the inclusion of LIVMARLI® in treatment protocols and insurance coverage.
The positive outcomes associated with LIVMARLI® could have a substantial impact on Mirum Pharmaceuticals' market position. The rarity of ALGS and the apparent efficacy of LIVMARLI® position it as a potential 'orphan drug,' which often commands premium pricing due to the lack of alternative treatments and the high value placed on disease mitigation.
Investors and stakeholders should closely monitor the adoption rate of LIVMARLI® post-publication of these results. Market penetration will depend on factors such as physician awareness, patient access and reimbursement policies. The success of LIVMARLI® could also pave the way for Mirum Pharmaceuticals to invest in additional research and development for similar cholestatic liver diseases, potentially expanding their portfolio and market share in the niche of rare liver disorders.
Lastly, the impact on Mirum Pharmaceuticals' stock could be significant, as groundbreaking treatment options like LIVMARLI® often lead to increased investor confidence and can drive stock prices upward. However, it is essential to consider the broader market context, including competitor activities and the overall performance of the biotech sector.
- Results demonstrate
- Data are the first to demonstrate a six-year transplant-free survival benefit in patients with ALGS using a pharmacological interventional therapy
“Patients with Alagille syndrome often undergo liver transplantation for complications related to cholestasis,” said Binita M. Kamath, MBBChir, Staff Physician and Senior Associate Scientist, The Hospital for Sick Children (SickKids),
“We are grateful to the GALA Study Group for this important academic contribution and are thrilled about the recognition of this analysis by Hepatology,” said Pam Vig, PhD, chief scientific officer and head of research at Mirum. “We would like to thank the patients who participated in the LIVMARLI studies as well as the Alagille Syndrome Alliance for their continued partnership and dedication to advancing research in this rare liver disease.”
The pre-specified statistical analysis was conducted independently by the lead author Dr. Bettina Hansen, Professor of Clinical Biostatistics at Erasmus MC,
Data from the analysis demonstrated a significant improvement in six-year event-free survival with a p-value of <0.0001 (HR: 0.305,
The full publication including additional data from the analysis is available on the Hepatology website.
About Alagille Syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys, and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports,
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the
Mirum has also submitted LIVMARLI for approval in the
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
LIVMARLI can cause side effects, including:
Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.
Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.
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 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.
Forward-Looking Statements
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 for LIVMARLI to exceed real world outcomes of patients with ALGS and the clinical significance of reduction of serum bile acids on liver transplantation. 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. 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 Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240229510785/en/
Media Contact:
Erin Murphy
media@mirumpharma.com
Investor Contacts:
Andrew McKibben
ir@mirumpharma.com
Sam Martin
Argot Partners
ir@mirumpharma.com
Source: Mirum Pharmaceuticals, Inc.
FAQ
What is the significance of the results for patients with Alagille syndrome (ALGS) treated with LIVMARLI?
What is the comparison made in the analysis published by Hepatology?
Who is the senior author of the manuscript mentioned in the PR?
What was the key finding of the analysis regarding clinical outcomes in patients with ALGS?
What is the ticker symbol for Mirum Pharmaceuticals, Inc.?
