Event-Free Survival Data from Mirum’s LIVMARLI Alagille Syndrome Program Published in Hepatology

• Improvement in pruritus, serum bile acid levels and bilirubin were highly predictive of six-year event-free and transplant-free survival in patients with cholestatic pruritus in Alagille syndrome treated with LIVMARLI
  
  
• Analysis highlights for the first-time potential markers associated with better outcomes to help drive clinical care in patients with Alagille syndrome

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from LIVMARLI® (maralixibat) oral solution clinical studies evaluating patients with Alagille syndrome (ALGS) were published in Hepatology. LIVMARLI is the first and only approved treatment for cholestatic pruritus in patients with Alagille syndrome three months of age and older.

The overall findings from the retrospective analysis suggest that patients with ALGS treated with LIVMARLI who meet the identified prognostic parameters may have improved six-year survival and clinical outcomes. In the analysis, 46 pre-specified variables were evaluated as potential predictors of event-free and transplant-free survival (EFS and TFS) from the LIVMARLI ALGS clinical program. Patients were assessed for up to six years; EFS was defined as absence of liver transplant (LT), surgical biliary diversion (SBD), hepatic decompensation, or death; TFS was absence of LT or death.

Results from the analysis:

• Improvement in pruritus from baseline to week 48 with LIVMARLI was predictive of six-year EFS and TFS compared to patients with minimal to no improvement in pruritus; 88% vs 57% (p = 0.0046) and 93% vs 57%, (p = 0.0007), respectively.
• Week 48 serum bile acids (sBA) of <200μmol/L was predictive of six-year EFS and TFS compared to those with higher values; 85% vs. 49% (p = 0.0010) and 90% vs. 49% (p = 0.0001), respectively. Similarly, 50% reduction in sBA demonstrated increased EFS and TFS; 86% vs. 39% (p < 0.0001) and 90% vs 39% (p < 0.0001).
• Week 48 bilirubin <6.5 mg/dL was predictive of six-year EFS and TFS, compared with those with higher values; 90% vs. 43% (p < 0.0001) and 94% vs. 42%; (p < 0.0001).
• The overall predictive ability of these variables was stable over several years, with all three variables being correlated.

“Complications of cholestasis, including refractory pruritus, are among the main indications for liver transplantation in patients with Alagille syndrome and many children will unfortunately require a liver transplant before adulthood,” said Ronald J. Sokol, MD, chief scientific officer, child health, Children’s Hospital Colorado. “This six-year analysis provides important insight into which markers predict transplant-free and event-free survival in those receiving LIVMARLI, and therefore can help guide medical management of patients receiving LIVMARLI.”

“This robust statistical analysis from the LIVMARLI studies demonstrates that by reducing cholestasis and the associated symptomatic burden, outcomes can be significantly improved for Alagille syndrome patients,” said Pam Vig, PhD, head of research and development at Mirum. “We are grateful for the collaboration of our academic partners who share our goal to improve the lives and clinical care for patients living with Alagille syndrome.”

For further details on the analysis, read the article published in Hepatology.

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. LIVMARLI is also approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older. For more information for U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older).

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. 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.

US Prescribing Information

EU SmPC

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI^® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and in Europe for the same indication in patients two months of age and older.

Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.

Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.

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Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s publication of clinical data, including article title and synopsis, which may include discussion of the Company’s clinical and research data, including the therapeutic potential of Company products and the potential for reducing cholestasis and the associated symptomatic burden over a period of time. 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.

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