Maralixibat Chloride

FOSTER CITY, Calif.--( BUSINESS WIRE )--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present data at the American Association for the Study of Liver Disease’s (AASLD) The Liver Meeting®, taking place November 15-19, 2024, in San Diego, California. Enclosed below are the titles that have been accepted for presentation during the meeting. The abstracts are available via the AASLD website . Full analyses will be available following their presentation within the Publications & Presentation section on Mirum’s website . Presentations Abstract #5038: Volixibat for Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2b Trial (VANTAGE): Interim Results **Late-breaker poster presentation** Monday, November 18 from 1:00-2:00pm, Poster Hall C Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA Abstract #184: Improvements in Pruritus are Associated with Improvements in Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH-ON trial **Oral presentation** Sunday, November 17 from 12:15-12:30pm during the ‘Advances in Pediatric Liver Disease’ session, Ballroom 6C Presented by Dr. Alexander Miethke, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA Abstract #4347: Pilot Study of Volixibat Co-administered with OCA for Primary Biliary Cholangitis (PBC) Treatment: The VLX-602 Trial Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA Abstract #4400: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA Abstract #4419 : Real-world Use of Maralixibat in Biliary Atresia: A Case Series Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA Abstract #4432: The Relationship Between Serum Bile Acids and Event-Free Survival Following the Use of Maralixibat for Progressive Familial Intrahepatic Cholestasis: Data from MARCH/MARCH-ON Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Professor Richard Thompson, King’s College London, United Kingdom Abstract #4436: Bile Acid Subspecies are Correlated with Pruritus and Bilirubin Improvement in PFIC Patients Treated with Maralixibat: Data from MARCH and MARCH-ON Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Professor Henkjan Verkade, University of Groningen, Groningen, Netherlands Alagille Syndrome Patient & Physician Fireside Discussion During the meeting, Mirum will host a session spotlighting a pediatric hepatologist and an adult patient living with ALGS. Dr. Ajay Jain, St. Louis University, will discuss clinical insights and patient, Emma, will share her experience with LIVMARLI. Sunday, November 17 from 1:30-2:00 p.m. in Exhibit Hall TLM Theater #2 About LIVMARLI® (maralixibat) oral solution LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com . 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 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. 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 for both commercial and clinical programs, which may include discussion of the Company’s clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI or other Company product candidates in various liver disease indications and in patient populations that are investigational only. Words such as “will,” “could,” “would,” “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 and the other risks described in Mirum’s filings with the Securities and Exchange Commission. 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. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov .

中国苏州，2024年11月13日—北海康成制药有限公司（以下简称「北海康成」，股票代码「1228.HK」），是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。公司今日宣布，中国国家药品监督管理局（NMPA）已正式受理CAN103（注射用维拉苷酶β）用于治疗成人和儿童I型和III型戈谢病的新药申请（NDA）。 CAN103是北海康成与药明生物（2269.HK）在罕见病领域的首个合作项目，是中国首个针对戈谢病开发的酶替代疗法（ERT）。据弗若斯特沙利文统计，2020年中国约有3,000名戈谢病患者。 国家药监局正式受理CAN103的新药上市申请是我们取得的又一重要里程碑。获批后， CAN103将是我国首个本土自主研发并进入临床应用的戈谢病酶替代疗法药物 , 属一类创新药并可完全替代同类进口产品 。同时，CAN103在全链条研创过程中积累了完整的研发数据和成本数据，将为打通本土罕见病新药研发商业化闭环提供首个案例，最终满足众多患者多年来的治疗需求 。我们预计在2025年将CAN103推向市场，这将是继海芮思和迈芮倍之后北海康成上市的第三个罕见病治疗药物，也是北海康成将进入商业化的首个自主研发产品。 北海康成于今年 8 月公布了 CAN103 针对成人和儿童戈谢病患者关键临床试验的积极顶线数据结果。国家药品监督管理局药品审评中心（CDE）于 9 月授予了 CAN103 优先审评资格。今年 10 月，北海康成顺利通过药品上市许可持有人（MAH）的生产和质量管理体系审核和现场检查，获得《药品生产许可证》B证。 2024年10月22日，国家药监局正式印发《生物制品分段生产试点工作方案》。在北海康成与药明生物的紧密合作下，双方顺利取得了相关生物药制品生产许可证，成为中国首批MAH制度下生物制品分段生产试点的实践者。 CAN103（注射用维拉苷酶β）是国内首个本土自主研发并即将进入临床应用的用于治疗I型和III型戈谢病患者的重组人源脑苷脂酶替代疗法。大多数戈谢病患者为I型和III型，分别为慢性非神经病变型和慢性神经病变型。CAN103通过静脉输注特异性地补充戈谢病患者体内溶酶体中缺乏的葡萄糖脑苷脂酶。 CAN103 的关键性临床试验于2024年8月取得积极顶线数据，是一项随机、双盲、剂量比较研究，旨在评估静脉滴注CAN103每2周一次在初治戈谢病患者中的有效性、安全性和药代动力学，并设有开放标签的扩展期。结果表明，该研究在60U/kg剂量组（P<0.0001）和较低的30U/kg剂量组（P<0.001）都成功达到了其主要疗效终点，即治疗9个月后受试者脾脏体积较基线的平均缩小百分比。该研究方案主要终点的设定已获得国家药品监督管理局药品审评中心(CDE)认可。 戈谢病是一种最常见的溶酶体贮积症之一，为位于1q22号染色体长臂上的葡萄糖脑苷脂酶基因突变引起的常染色体隐性遗传病。该病对男性和女性的影响是同等的。临床上戈谢病包括围产期致死型、I型（非神经病变型）、II型（急性神经病变型）和III型（慢性神经病变型），其中I型和III型戈谢病患者多能活到成年。戈谢病是由葡萄糖脑苷脂酶（酸性β-葡萄糖苷酶）缺乏引起的，这种酶有助于分解溶酶体内一种被称作葡萄糖脑苷脂（葡糖神经酰胺）的细胞膜鞘脂。葡萄糖脑苷脂主要积聚在某些器官内的单核巨噬细胞系细胞中（Gaucher细胞），导致脾肿大，肝肿大，贫血，血小板减少症，骨痛和骨折，严重的戈谢病类型（围产期致死型，II型和III型），早期可出现神经系统症状。30年以来，重组人源葡萄糖脑苷脂酶替代疗法 (ERT) 一直是戈谢病的标准治疗方法，临床试验和真实世界数据表明，患者的主要非神经系统症状和体征以及生活质量得到了显着改善。根据弗若斯特沙利文的报告数据显示，2020年在中国约有3000名戈谢病患者。 向上滑动阅览 北海康成制药有限公司（以下简称“北海康成”，股票代码1228.HK）是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。公司目前拥有11个具有可观市场潜力的药物资产组合，其中包括 3 个已获批上市产品和 8 个在研药物。这些产品均针对一些较普遍的罕见疾病和罕见肿瘤适应症， 比如亨特综合征（黏多糖贮积症II型）和其他溶酶体贮积症、补体介导的疾病、血友病 A、代谢紊乱、罕见的胆汁淤积性肝病、神经肌肉疾病。 北海康成的新一代基因技术研发中心实验室正在开发针对罕见遗传病，包括庞贝氏症、法布雷病、杜氏肌肉营养不良症（DMD）和其他神经肌肉疾病的新型及潜在治愈性的基因疗法，并与世界领先的研究人员和生物技术公司合作。公司的全球合作伙伴包括不限于 Apogenix、GC Pharma、Mirum、药明生物、Privus、华盛顿大学医学部和Scriptr Global 等。 如需了解更多关于北海康成制药有限公司的信息，请访问：http://www.canbridgepharma.com/   前瞻性声明：本文中作出的前瞻性陈述仅与本文作出陈述之日的事件或信息有关。除非法律要求，否则我们不承担公开更新或修改任何前瞻性陈述的义务，无论是由于新信息、未来事件或其他原因，在做出陈述的数据之后或反映意外事件的发生情况。您应完整阅读本文，并了解我们未来的实际结果或表现可能与我们的预期存在重大差异。在本文中，我们或我们的任何董事或我们公司的意图的陈述或引用均在本文发表之日作出。这些意图中的任何一个都可能随着未来的发展而改变。 联系我们： 投资者关系： ir@canbridgepharma.com