Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data highlighting quality of life and sleep improvements from its pivotal ICONIC study evaluating LIVMARLI® (maralixibat) oral solution in patients with Alagille syndrome (ALGS), were published in the Journal of Pediatrics.

ALGS is a rare genetic disease caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause retention of bile acids in the liver leading to cholestasis and ultimately inflammation and liver injury which prevents the liver from working properly. Cholestasis in ALGS leads to a debilitating pruritus impacting the quality of life so severely that pruritus is a leading indication for liver transplantation.

The analysis evaluated 27 patients with ALGS who received 380 µg/kg per day of maralixibat and who remained in the pivotal ICONIC study through week 48. The analysis compared health-related quality of life (HRQoL) outcomes from baseline and week 48 based on treatment response to maralixibat, defined as a ≥1-point reduction in pruritus (measured by Itch-Reported Outcome Observer (ItchRO[Obs]) where a 1-point reduction is clinically meaningful). The Pediatric Quality of Life (PedsQL™) questionnaires (Generic Core PedsQL, Family Impact Scale, and Multidimensional Fatigue Scale) were prospectively collected via a caregiver proxy report and analyzed retrospectively.

Pruritus treatment response at week 48 was associated with clinically meaningful improvement in HRQoL from baseline to week 48. Across all domains evaluated, the average increase in responders’ HRQoL scores from baseline to week 48 exceeded the minimal clinically important difference (MCID), compared to non-responders. Responders experienced a significantly greater change in Family Total Impact Scale Score and Multidimensional Fatigue Total Scale Score, and a numerically higher increase in PedsQL Generic Core Total Scale Score compared with non-responders (where higher scores indicates a better HRQoL). Importantly, six sleep-related items demonstrated significantly larger changes from baseline to week 48 in responders (p<0.05).

The analysis concluded that the significant improvements in pruritus seen with maralixibat at week 48 of the ICONIC study were clinically meaningful and associated with improved HRQoL and sleep-related parameters.

“The data published in Journal of Pediatrics underscore not only the burden of disease in patients with ALGS, which significantly affects the way these children are able to live and function, but also how the reduction of pruritus with LIVMARLI provides a significant improvement to their sleep as well as improvements to their quality of life which exceed the MCID,” said Pam Vig, PhD, head of research and development at Mirum. “The improvements observed demonstrate the tremendous impact LIVMARLI can have for these children by targeting cholestasis directly and reducing cholestatic pruritus.”

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) one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and 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 bone fractures and gastrointestinal bleeding.

Prescribing Information

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare 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 one year of age and older. Please view the warnings and precautions available in the Prescribing information or, for more information, visit LIVMARLI.com. LIVMARLI is also approved in the same indication in Israel. Mirum recently announced that the CHMP adopted a positive opinion of LIVMARLI for the treatment of cholestatic pruritus in patients with ALGS two months of age and older. EC decision is expected by year-end 2022.

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study 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 three potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis, the OHANA Phase 2b clinical trial for pregnant women with intrahepatic cholestasis of pregnancy, and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.

Learn more about Mirum by visiting www.mirumpharma.com. Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s regulatory approval pathways and products or therapies as presented in a scientific journal, which may include discussion of the Company’s clinical and research data, including the discovery, development and commercialization of our product candidates and technologies, and the therapeutic potential thereof. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading “Risk Factors” included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements, except as required by law.

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