Mirum Pharmaceuticals’ LIVMARLI Data Showcased at ESPGHAN Annual Meeting

Rhea-AI Summary

Mirum Pharmaceuticals presented data on their oral solution, LIVMARLI (maralixibat), at the 56th ESPGHAN Annual Meeting in Milan. Seven abstracts highlighted LIVMARLI's efficacy in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Key findings from the Phase 3 MARCH-ON PFIC study showed significant and sustained improvements in pruritus severity, serum bile acid levels, total bilirubin, and growth over two years. One-third of patients on LIVMARLI discontinued at least one antipruritic medication. These results underscore LIVMARLI's potential in improving liver health and reducing medication dependency in children with PFIC and ALGS.

Positive

• Seven abstracts presented at the prestigious ESPGHAN Annual Meeting.
• Phase 3 MARCH-ON PFIC study showed significant improvements in pruritus severity, serum bile acid levels, and total bilirubin.
• 40% of patients with abnormal bilirubin levels achieved normalization compared to none in the placebo group.
• One-third of patients on LIVMARLI discontinued at least one antipruritic medication within a year.
• Improvements in pruritus were associated with better quality of life.
• Real-world data supported the clinical trial outcomes.

Negative

• The press release lacks specific financial data or revenue impact from the LIVMARLI studies.
• No new clinical trials or regulatory approvals were announced.
• Potentially high dependency on ongoing clinical and real-world evidence to maintain drug credibility.
• Details on any adverse effects or long-term risks of LIVMARLI treatment were not disclosed.

- Seven abstracts highlighting ALGS and PFIC data, including three oral presentations

- Long-term extension data from Phase 3 MARCH-ON PFIC study presented at plenary session and selected among the highest scoring abstracts

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the 56^th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting which took place this week in Milan, Italy. 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 Pam Vig, PhD, chief scientific officer and head of research at Mirum. “Further, we are pleased to demonstrate data that shows children treated with LIVMARLI are reducing or discontinuing some antipruritic medications and nutritional supplements.”

Abstract 587: Long-term Maintenance of Response and Improved Liver Health with Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis: 2-year Data From the MARCH-ON Study
*Plenary Session: Hepatology Highest Scoring Abstracts*
Presented by Professor Richard J. Thompson, MD, King’s College London, United Kingdom
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, Papa Giovanni XXIII Hospital, Bergamo, Italy
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 Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA
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 Richard J. Thompson, MD, King’s College, London, United Kingdom​

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 Richard J. Thompson, MD, King’s College, London, United Kingdom

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 Douglas B. Mogul, MD, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA

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, Papa Giovanni XXIII Hospital, Bergamo, Italy

To view the full presentations, please visit the Publications section of Mirum’s website.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and for progressive familial intrahepatic cholestasis (PFIC) five years of age and older.

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 U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older.

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 U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients five years of age and older. Mirum has submitted for approval in Europe for the treatment of PFIC in patients two months of age and older. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

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 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 U.S. or global regulatory environment or economies generally; the continued impact of COVID-19 on our business, operations and financial results; and competitive developments. Other factors that might cause such a difference include those discussed in the Company’s filings with the SEC. 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/20240518739027/en/

Media Contact:

Erin Murphy

media@mirumpharma.com

Investor Contact:

Andrew McKibben

investors@mirumphama.com

Source: Mirum Pharmaceuticals, Inc.

FAQ

What data did Mirum Pharmaceuticals present at ESPGHAN 2023?

Mirum Pharmaceuticals presented seven abstracts on LIVMARLI's efficacy in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).

How did LIVMARLI perform in the Phase 3 MARCH-ON PFIC study?

The Phase 3 MARCH-ON PFIC study showed significant and sustained improvements in pruritus severity, serum bile acid levels, total bilirubin, and growth over two years.

What improvements were observed in patients using LIVMARLI?

Patients showed significant reductions in pruritus severity, serum bile acid levels, and total bilirubin. One-third discontinued at least one antipruritic medication within a year.

What was a key finding related to bilirubin levels in the studies?

40% of patients with abnormal bilirubin levels achieved normalization with LIVMARLI, compared to none in the placebo group.

What is the significance of LIVMARLI data presented at ESPGHAN?

The data emphasizes LIVMARLI's potential in improving liver health, quality of life, and reducing medication dependency in children with PFIC and ALGS.