Mirum Pharmaceuticals to Present New Data at The Liver Meeting®

2023-10-30

临床2期上市批准突破性疗法孤儿药

- Late-breaker poster presentation accepted featuring LIVMARLI® data showing long-term maintenance of response in patients with PFIC - Four poster presentations will highlight LIVMARLI PFIC data and volixibat proof-of-concept trial data in intrahepatic cholestasis of pregnancy FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI and volixibat programs will be presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting, taking place November 10-14, 2023, in Boston, Massachusetts. Presentations during AASLD Late-breaker Poster Presentation November 13, 2023 from 1:00-2:00 p.m. as part of the late-breaker poster presentation session Poster Number 5048-C: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): 2-year data from the MARCH-ON study Poster Presentations Presented November 13, 2023 from 1:00-2:00 p.m. Poster 4602-C: Improvements in pruritus with maralixibat are associated with improved quality of life for patients with progressive familial intrahepatic cholestasis: Data from the MARCH-PFIC trial Poster 4604-C: Maralixibat leads to significant improvement in cholestatic pruritus for children with progressive familial intrahepatic cholestasis due to TJP2 or MYO5B deficiency: Data from the MARCH-PFIC trial Poster 4605-C: Maralixibat leads to significant improvements in cholestatic pruritus for children with progressive familial intrahepatic cholestasis without a genetic diagnosis: Data from the MARCH-PFIC trial Poster 4547-C: Efficacy, safety and tolerability of volixibat in patients with intrahepatic cholestasis of pregnancy: A case series of 4 patients Full data will be available within the Publications and Presentations section on Mirum’s website on November 13. 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 U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome Alagille syndrome (ALGS) three months of age and older. LIVMARLI is also the only approved IBAT inhibitor 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 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 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 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 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 IBAT inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. 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. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme defects 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 three investigational treatments for debilitating liver diseases. The LIVMARLI development program includes the Phase 2b EMBARK study for biliary atresia. Mirum’s second investigational IBAT inhibitor IBAT inhibitor is volixibat, which 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. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter. 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, including the therapeutic potential and/or commercial viability of our product candidates and technologies in various liver disease indications. 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.