Mirum Pharmaceuticals’ LIVMARLI Data Showcased at ESPGHAN Annual Meeting By Investing.com

– Seven summaries highlighting ALGS and PFIC data, including three oral presentations

– Long-term extension data from the phase III PFIC study were presented in plenary and selected from among the highest-scoring abstracts

FOSTER CITY, Calif.–( BUSINESS WIRE )–Mirom Pharmaceuticals (Nasdaq: MIRM) today announced data presented at the 56th Annual Meeting of the European Society of Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN). this year. A week in Milan, Italy. Data from clinical studies of LIVMARLI ® (maralixibat) oral solutions and real-world settings for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) were presented in oral and poster presentations during the meeting.

“We continue to build on the strong body of evidence demonstrating LIVMARLI's ability to provide long-term benefit to PFIC patients across key quality of life and liver disease parameters, as well as improvements in diverse genetic types of PFIC,” said Pam Feig, Ph.D., president. Scientific Officer and Head of Research at Merom. Furthermore, we are pleased to present data showing that children treated with LIVMARLI reduce or stop taking certain anti-itch medications and nutritional supplements.

Summary 587: Maintaining long-term response and improving liver health with maralexbat in patients with advanced familial intrahepatic cholestasis: 2-year data from the MARCH-ON study
Plenary session: summaries of the highest scores in liver disease
Presented by Professor Richard J. Thompson, MD, King's College London, UK
Patients with PFIC demonstrated significant and sustained improvements in itch severity, serum bile acid (sBA) levels, total bilirubin and growth after up to 2 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.

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Summary 594: Maralexpat results in a significant reduction in bilirubin in patients with advanced familial intrahepatic cholestasis: data from the MARCH trial.
Submitted by Lorenzo D'Anteja, MD, Pope Giovanni XXIII Hospital, Bergamo, Italy
Patients treated with LIVMARLI experienced a significant reduction in both total and direct bilirubin compared to placebo. 40% of patients with abnormal bilirubin at baseline who were treated with LIVMARLI achieved normal versus none in the placebo group. Furthermore, these reductions in bilirubin were consistent with reductions in sBAs.

Summary 600: The effect of maraloxib on the use of concomitant medications for the treatment of cholestatic pruritus in Alagille syndrome: a real-world experience
Submitted by Jolan Turner Rosenthal, Ph.D., Merom Pharmaceuticals (NASDAQ:), Inc., Foster City, CA, USA
Real-world evidence from 116 patients treated with LIVMARLI for at least one year showed that more than a third of patients were able to discontinue 1¥¥ of concomitant antipruritic medications, and one in five patients discontinued 2¥¥ of medications. Reductions in the use of concomitant medications were observed across all drug classes and suggest that LIVMARLI may reduce the burden of polypharmacy during the first year of treatment.

Other presentations given during ESPGHAN include:

Summary 592: Maralexpat results in significant improvements in cholestatic pruritus in children with advanced familial intrahepatic cholestasis without a genetic diagnosis: data from the MARCH trial
Presented by Professor Richard J. Thompson, MD, King's College, London, UK

Summary 599: Maralexpat leads to improvements in cholestatic pruritus in children with advanced familial intrahepatic cholestasis due to MDR3 deficiency: data from the MARCH/ON trials.
Presented by Professor Richard J. Thompson, MD, King's College, London, UK

Summary 595: Improvements in pruritus with maralexbate are associated with improved quality of life for patients with familial progressive intrahepatic cholestasis: data from the MARCH trial.
Submitted by Douglas P. Mogul, MD, PhD, Merom Pharmaceuticals, Inc., Foster City, CA, USA

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Summary 597: Maraloxibate can improve cholestatic pruritus in children with familial progressive intrahepatic cholestasis who have previously undergone surgical biliary diversion: data from the MARCH/ON trial
Submitted by Lorenzo D'Anteja, MD, Pope Giovanni XXIII Hospital, Bergamo, Italy

To view full presentations, please visit the Publications section on the Merom website.

About LIVMARLI ® (maralixibat) Oral Solution

LIVMARLI ® (maralixibat) is a once-daily oral solution, a bile acid transporter (IBAT) inhibitor approved by the FDA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older and for cholestasis. Progressive familial intrahepatic intrahepatic (PFIC) age five years and older.

LIVMARLI is also the only IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS for 2 months or more, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for US residents, please visit LIVMARLI.com.

Mirum has also filed for approval of LIVMARLI in Europe in PFIC for patients 2 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 the Mirum Clinical Trials section of the company's website.

Important safety information

Limitations of use: LIVMARLI is not for use in patients with type 2 PFIC who have a severe defect in the bile salt export pump protein (BSEP).

Levmarle can cause side effects, including:

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Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but may worsen during treatment. These changes may be a sign of liver injury. In the case of PFIC, this can be serious or may lead to liver transplantation or death. Your healthcare provider should do blood tests and physical examinations before starting and during treatment to check your liver function. Tell your health care provider right away if you have any signs or symptoms of liver problems, including nausea or vomiting, yellowing of the skin or white part of the eye, dark or brown urine, or pain on the right side of the eye. Stomach (abdomen). Bloating in the stomach area, loss of appetite, or bleeding or bruising more easily than usual.

Stomach and intestine (digestive) problems. Levmarle 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 are more severe than usual for you.

condition called Deficiency of fat-soluble vitamins (FSV). Caused by low levels of certain vitamins (vitamins 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 that have been reported as common side effects.

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US prescription information
EU SmPC
Canadian product study

About Miram Pharmaceutical Company

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications available: LIVMARLI ® (maralixibat) Oral Solution, CHOLBAM ® (cholic acid) Capsules, and CHENODAL ® (Chenodiol) Tablets.

LIVMARLI, an IBAT inhibitor, is approved to treat two rare liver diseases that affect children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States (three months and older), in Europe (two months and older), and in other regions worldwide. It is also approved in the United States for the treatment of cholestatic pruritus in PFIC patients five years of age and older. Mirum has applied for approval in Europe to treat PFIC in patients 2 months of age and older. CHOLBAM is approved by the FDA for the treatment of disorders of bile acid synthesis due to single enzyme deficiency and the adjuvant treatment of peroxisomal disorders in patients who develop signs, symptoms, or liver disease. CHENODAL has received recognition of medical necessity by the US Food and Drug Administration (FDA) for the treatment of patients with cerebrotendinomatous xanthomatosis (CTX).

Merom's late-stage pipeline includes two investigational therapies for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two prospective registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and the Phase 2b VANTAGE study for primary cholangitis. Finally, CHENODAL has been evaluated in a phase III clinical study, RESTORE, for the treatment of patients with CTX, with positive results reported in 2023.

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To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook (NASDAQ:), LinkedInAnd Instagram Twitter (X).

Forward-looking statements

this The press release includes forward-looking statements regarding the Company's planned participation in a scientific conference, including the title and abstract of the data presentation, which may include a discussion of the Company's clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI in various areas. Indications for liver disease and in patient populations under investigation 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, target, 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 not limited to, the following factors: the uncertainties inherent in research and development; Uncertainties inherent in business and financial planning, including, but not limited to, risks related to Mirum's business and outlook, adverse developments in our focused markets, or adverse developments in the U.S. or global regulatory environment or economies generally; the ongoing impact of Coronavirus (COVID-19) on our business, operations and financial results; and competitive developments. Other factors that could cause such a difference include those discussed in the Company's filings with the Securities and Exchange Commission. All forward-looking statements in this press release speak only as of the date they are made and are based on management's assumptions and estimates as of that date. Mirum undertakes no obligation to update these statements to reflect events that occur or circumstances that exist after the date on which they are made, except as required by law.

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Media Contact:
Erin Murphy
media@mirumpharma.com

Investor Contact:
Andrew McKibben
investors@meruvama.com

Source: Miracle Pharmaceuticals, Inc.

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