FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the 56th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting which took place this week in Milan, Italy. Data from LIVMARLI® (maralixibat) oral solution clinical studies and real-world settings in Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) were presented in oral and poster presentations during the meeting..

加利福尼亚州福斯特城（商业新闻短讯）--Mirum Pharmaceuticals，Inc.（纳斯达克：MIRM）今天宣布了本周在意大利米兰举行的第56届欧洲儿科、胃肠病学、肝病学和营养学会（ESPGHAN）年会上提供的数据。会议期间，LIVMARLI®（maralixibat）口服液临床研究和Alagille综合征（ALGS）和进行性家族性肝内胆汁淤积症（PFIC）的现实环境的数据在口头和海报上进行了介绍。。

“We continue to build upon the strong body of evidence demonstrating LIVMARLI’s potential to provide long-term benefit to PFIC patients across key quality of life and liver disease parameters, as well as improvements in varied genetic types of PFIC,” said Pam Vig, PhD, chief scientific officer and head of research at Mirum.

Mirum首席科学官兼研究负责人Pam Vig博士说：“我们继续以大量证据为基础，证明LIVMARLI有潜力在关键的生活质量和肝病参数方面为PFIC患者提供长期益处，并改善PFIC的各种遗传类型。”。

“Further, we are pleased to demonstrate data that shows children treated with LIVMARLI are reducing or discontinuing some antipruritic medications and nutritional supplements.”.

“此外，我们很高兴能够证明数据显示，接受利夫玛利治疗的儿童正在减少或停止服用一些止痒药物和营养补充剂。”。

Abstract 587: Long-term Maintenance of Response and Improved Liver Health with Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis: 2-year Data From the MARCH-ON Study

摘要587:Maralixibat对进行性家族性肝内胆汁淤积症患者的长期维持反应和改善肝脏健康：MARCH-ON研究的2年数据

*Plenary Session: Hepatology Highest Scoring Abstracts*

*全体会议：肝病学得分最高的摘要*

Presented by Professor Richard J. Thompson, MD, King’s College London, United Kingdom

由英国伦敦国王学院医学博士Richard J.Thompson教授介绍

Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two 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..

PFIC患者在利夫马利治疗两年后，瘙痒严重程度，血清胆汁酸（sBA）水平，总胆红素和生长均显着持续改善。在开放标签研究中，最初随机接受安慰剂治疗的患者在瘙痒和sBA方面也有类似的改善。。

Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial

摘要594：马利昔巴可显着降低进行性家族性肝内胆汁淤积症患者的胆红素：来自MARCH试验的数据

Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy

由意大利贝加莫Papa Giovanni XXIII医院医学博士Lorenzo D'Antiga介绍

Patients treated with LIVMARLI experienced significant decreases in both total and direct bilirubin compared to placebo. 40% of the patients with abnormal bilirubin at baseline treated with LIVMARLI achieved normalization versus none in the placebo group. Further, these reductions in bilirubin were consistent with reductions in sBAs..

与安慰剂相比，接受利夫玛利治疗的患者总胆红素和直接胆红素均显着降低。用利夫玛利治疗的基线胆红素异常患者中有40%达到了正常化，而安慰剂组则没有。此外，胆红素的这些降低与SBA的降低是一致的。。

Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience

摘要600：马拉利昔巴特对治疗Alagille综合征胆汁淤积性瘙痒症的伴随药物使用的影响：现实世界的经验

Presented by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA

由美国加利福尼亚州福斯特市Mirum制药公司Jolan Terner Rosenthal博士介绍

Real-world evidence from 116 patients treated with LIVMARLI for at least one year showed that more than one-third of patients were able to discontinue ≥1 concomitant antipruritic medication, and one in five patients discontinued ≥2 medications. Reductions in concomitant medication usage were seen across all medication types and suggest that LIVMARLI may reduce the polypharmacy burden within the first year of treatment..

来自116名接受利夫玛利治疗至少一年的患者的现实证据表明，超过三分之一的患者能够停用≥1种伴随的止痒药物，五分之一的患者停用≥2种药物。所有药物类型的伴随药物使用都有所减少，这表明利夫马利可能会在治疗的第一年内减轻多种药物的负担。。

Other presentations featured during ESPGHAN include:

ESPGHAN期间的其他专题介绍包括：

Abstract 592: Maralixibat Leads to Significant Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Without a Genetic Diagnosis: Data from the MARCH Trial

摘要592：Maralixibat可显着改善无基因诊断的进行性家族性肝内胆汁淤积症儿童的胆汁淤积性瘙痒症：来自MARCH试验的数据

Presented by Professor Richard J. Thompson, MD, King’s College, London, United Kingdom​

由英国伦敦国王学院医学博士Richard J.Thompson教授介绍​

Abstract 599: Maralixibat Leads to Improvements in Cholestatic Pruritus for Children with Progressive Familial Intrahepatic Cholestasis Due to MDR3 Deficiency: Data From the MARCH/MARCH-ON Trials

摘要599:Maralixibat可改善MDR3缺乏引起的进行性家族性肝内胆汁淤积症儿童的胆汁淤积性瘙痒症：来自MARCH/MARCH-ON试验的数据

Presented by Professor Richard J. Thompson, MD, King’s College, London, United Kingdom

由英国伦敦国王学院医学博士Richard J.Thompson教授介绍

Abstract 595: Improvements in Pruritus with Maralixibat are Associated with Improved Quality of Life for Patients with Progressive Familial Intrahepatic Cholestasis: Data From the MARCH Trial

摘要595：马利昔布改善瘙痒症与改善进行性家族性肝内胆汁淤积症患者的生活质量有关：来自MARCH试验的数据

Presented by Douglas B. Mogul, MD, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA

由美国加利福尼亚州福斯特城Mirum Pharmaceuticals，Inc.医学博士Douglas B.Mogul介绍

Abstract 597: Maralixibat Can Improve Cholestatic Pruritus in Children with Progressive Familial Intrahepatic Cholestasis Who Previously Underwent a Surgical Biliary Diversion: Data From the MARCH/MARCH-ON Trials

摘要597：马利昔巴可改善进行性家族性肝内胆汁淤积症儿童的胆汁淤积性瘙痒症，这些儿童先前曾接受过手术胆汁分流：来自MARCH/MARCH-ON试验的数据

Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy

由意大利贝加莫Papa Giovanni XXIII医院医学博士Lorenzo D'Antiga介绍

To view the full presentations, please visit the Publications section of Mirum’s website.

要查看完整的演示文稿，请访问Mirum网站的出版物部分。

About LIVMARLI® (maralixibat) oral solution

关于LIVMARLI®（马利昔巴特）口服液

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 and for progressive familial intrahepatic cholestasis (PFIC) five years of age and older..

LIVMARLI®（maralixibat）口服溶液是一种口服给药的回肠胆汁酸转运蛋白（IBAT）抑制剂，每日一次，经美国食品和药物管理局批准，用于治疗三个月及以上的Alagille综合征（ALGS）患者的胆汁淤积性瘙痒症以及五岁及以上的进行性家族性肝内胆汁淤积症（PFIC）。。

LIVMARLI is also the only approved 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..

LIVMARLI也是唯一被欧盟委员会批准用于治疗两个月及以上ALGS患者胆汁淤积性瘙痒症的IBAT抑制剂，也是加拿大卫生部批准用于治疗ALGS胆汁淤积性瘙痒症的IBAT抑制剂。有关美国居民的更多信息，请访问LIVMARLI.com。。

Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older.

Mirum还向两个月及以上的患者提交了LIVMARLI在欧洲PFIC的批准。

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.

LIVMARLI已获得ALGS和PFIC 2型的突破性治疗指定以及ALGS和PFIC的孤儿指定。要了解有关LIVMARLI正在进行的临床试验的更多信息，请访问该公司网站上的Mirum临床试验部分。

IMPORTANT SAFETY INFORMATION

重要安全信息

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.

使用限制：LIVMARLI不适用于胆汁盐输出泵（BSEP）蛋白严重缺陷的PFIC 2型患者。

LIVMARLI can cause side effects, including:

利夫马利会引起副作用，包括：

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.

肝损伤。某些肝脏检查的变化在Alagille综合征和PFIC患者中很常见，但在治疗过程中可能会恶化。这些变化可能是肝损伤的迹象。在PFIC中，这可能很严重，也可能导致肝移植或死亡。您的医疗保健提供者应该在开始治疗前和治疗期间进行血液检查和身体检查，以检查您的肝功能。

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..

如果你有任何肝脏问题的迹象或症状，包括恶心或呕吐、皮肤或眼睛白色部分变成黄色、黑色或棕色尿液、胃（腹部）右侧疼痛、胃部腹胀、食欲不振或出血或比正常情况更容易瘀伤，请立即告诉你的医疗保健提供者。。

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..

胃和肠（胃肠）问题。利夫马利会引起胃部和肠道问题，包括腹泻和胃痛。您的医疗保健提供者可能会建议您监测新的或恶化的胃部问题，包括胃痛、腹泻、大便出血或呕吐。如果您的这些症状比正常情况更频繁或更严重，请立即告诉您的医疗保健提供者。。

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..

在患有Alagille综合征和PFIC的患者中，由于体内脂肪中某些维生素（维生素A，D，E和K）含量低而导致的脂溶性维生素（FSV）缺乏症很常见，但在治疗过程中可能会恶化。您的医疗保健提供者应在开始治疗前和治疗期间进行血液检查，并可能监测骨折和出血，据报道这是常见的副作用。。

US Prescribing Information

美国处方信息

EU SmPC

欧盟SmPC

Canadian Product Monograph

加拿大产品专著

About Mirum Pharmaceuticals, Inc.

关于Mirum Pharmaceuticals，股份有限公司。

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.

Mirum Pharmaceuticals，Inc.是一家生物制药公司，致力于改变影响儿童和成人的罕见疾病的治疗方法。Mirum有三种批准的药物：LIVMARLI®（maralixibat）口服溶液，CHOLBAM®（胆酸）胶囊和CHENODAL®（chenodiol）片剂。

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.

LIVMARLI是一种IBAT抑制剂，被批准用于治疗影响儿童和成人的两种罕见肝病。在美国（三个月及以上），欧洲（两个月及以上）和全球其他地区，它被批准用于治疗Alagille综合征患者的胆汁淤积性瘙痒症。

It is also approved in the U.S. in cholestatic pruritus in PFIC patients five years of age and older. Mirum has submitted for approval in Europe for the treatment of PFIC in patients two months of age and older. 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.

它在美国也被批准用于五岁及以上PFIC患者的胆汁淤积性瘙痒症。Mirum已提交欧洲批准，用于治疗两个月及以上患者的PFIC。CHOLBAM被FDA批准用于治疗由于单酶缺乏引起的胆汁酸合成障碍，以及对表现出体征或症状或肝病的患者进行过氧化物酶体疾病的辅助治疗。

CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX)..

CHENODAL已获得FDA的医疗必要性认可，用于治疗脑腱黄瘤病（CTX）患者。。

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 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis.

Mirum的晚期管道包括两种用于治疗衰弱性肝病的研究性治疗方法。IBAT抑制剂Volixibat正在两项潜在的注册研究中进行评估，包括原发性硬化性胆管炎的2b期VISTAS研究和原发性胆汁性胆管炎的2b期VANTAGE研究。

Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023..

最后，CHENODAL已在一项3期临床研究RESTORE中进行了评估，以治疗CTX患者，2023年报告的结果为阳性。。

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

要了解有关Mirum的更多信息，请访问mirumpharma.com，并在Facebook、LinkedIn、Instagram和Twitter（X）上关注Mirum。

Forward-Looking Statements

前瞻性声明

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 relating to the therapeutic potential and/or commercial viability of LIVMARLI in various liver disease indications and in patient populations that are investigational only.

本新闻稿包括与该公司计划参加科学会议有关的前瞻性声明，包括数据介绍标题和概要，其中可能包括讨论该公司的临床和研究数据，这些数据与利夫马利在各种肝病适应症和仅用于研究的患者人群中的治疗潜力和/或商业可行性有关。

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.

由于此类报表存在风险和不确定性，因此实际结果可能与此类前瞻性报表明示或暗示的结果存在重大差异。诸如“意志”、“目标”、“潜力”等词语以及类似的表达方式旨在识别前瞻性陈述。此类报表的准确性受到许多风险、不确定性和假设的影响，包括但不限于以下因素：研究和开发固有的不确定性；业务和财务规划中固有的不确定性，包括但不限于与Mirum业务和前景相关的风险、我们重点市场的不利发展或美国的不利发展。

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.

或全球监管环境或总体经济；新型冠状病毒肺炎对我们的业务、运营和财务业绩的持续影响；和竞争发展。可能导致这种差异的其他因素包括公司向美国证券交易委员会提交的文件中讨论的因素。本新闻稿中包含的所有前瞻性声明仅在做出之日起生效，并基于管理层截至该日期的假设和估计。

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..

除法律要求外，Mirum没有义务更新此类报表，以反映报表编制日期后发生的事件或情况。。