FOSTER CITY, Calif.--(

加利福尼亚州福斯特城--(

BUSINESS WIRE

商业热线

)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2024 net product sales, year-end cash balance, corporate updates, and full-year 2025 outlook.

)--Mirum Pharmaceuticals，Inc.（纳斯达克：MIRM）今天提供了其2024年全年净产品销售额、年末现金余额、公司最新情况和2025年全年前景的初步和未经审计的估计。

“2024 marked a significant year for Mirum as we accelerated our commercial business and achieved significant development milestones,” said Chris Peetz, chief executive officer of Mirum. “We continued our leadership in cholestatic disease with a label expansion for LIVMARLI and positive interim analyses for volixibat in PSC and PBC.

Mirum首席执行官克里斯·佩茨（ChrisPeetz）表示：“2024年对Mirum来说是重要的一年，因为我们加快了商业业务的发展，并取得了重大的发展里程碑。”。“我们在胆汁淤积性疾病方面继续保持领先地位，扩大了LIVMARLI的标签，并在PSC和PBC中对volixibat进行了积极的中期分析。

Additionally, we advanced our rare genetic neurology efforts with the NDA submission for chenodiol and the in-licensing of MRM-3379 for Fragile X syndrome. With proven commercial execution in ultra-rare disease and a compelling pipeline in larger indications, we believe we are well-positioned for sustained growth in the years ahead.”.

此外，我们通过提交藜芦醇的NDA和针对脆性X综合征的MRM-3379的in许可证，推进了我们罕见的遗传神经学研究。凭借在超罕见疾病方面的成熟商业执行以及在更大适应症方面的引人注目的渠道，我们相信我们在未来几年的持续增长中处于有利地位。”。

Future Expectations and Milestones

未来的期望和里程碑

2025 Guidance: expect continued revenue growth with global net product sales of approximately $420 million to $435 million and positive cash flow from operations

2025年指南：预计收入将持续增长，全球净产品销售额约为4.2亿美元至4.35亿美元，运营产生正现金流

Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in second half 2025; topline data expected in 2026

原发性硬化性胆管炎（PSC）的Volixibat远景研究预计将于2025年下半年完成登记；预计2026年的topline数据

Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026

原发性胆汁性胆管炎（PBC）的Volixibat VANTAGE研究预计将于2026年完成

LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026

利夫马利扩大了罕见胆汁淤积性瘙痒症的3期研究，预计将于2026年完成登记

FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is March 28, 2025

FDA处方药使用费法案（PDUFA）脑腱黄瘤病（CTX）中藜芦醇的日期为2025年3月28日

Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025

预计在2025年启动脆性X综合征（FXS）MRM-3379的2期研究

2024 Highlights

2024年亮点

Commercial: Continued leadership in rare disease with a franchise in hepatology and genetic neurology

商业：在肝病学和遗传神经病学领域拥有特许经营权，在罕见疾病领域继续保持领先地位

2024 estimated LIVMARLI net product sales of approximately $213 million and CHOLBAM and CHENODAL net product sales of approximately $123 million

2024年，利夫马利的预计净产品销售额约为2.13亿美元，CHOLBAM和CHENODAL的净产品销售额约为1.23亿美元

Total estimated net product sales of approximately $99 million in Q4 2024 including approximately $64 million in LIVMARLI net sales and approximately $35 million in net sales from CHOLBAM and CHENODAL

2024年第四季度，预计产品总净销售额约为9900万美元，其中利夫马利净销售额约为6400万美元，CHOLBAM和CHENODAL净销售额约为3500万美元

Expanded global footprint; 30 countries with commercial access, including successful reimbursement negotiation and launch in the four major European markets

扩大全球足迹；30个具有商业准入的国家，包括成功的报销谈判和在欧洲四大市场的推出

Regulatory and Pipeline: Expanding Mirum’s leadership across multiple rare diseases and larger orphan settings

监管和管道：扩大Mirum在多种罕见疾病和较大孤儿环境中的领导地位

Positive interim results for volixibat in VISTAS PSC and VANTAGE PBC studies

VISTAS PSC和VANTAGE PBC研究中volixibat的中期积极结果

Volixibat granted breakthrough therapy designation for treatment of cholestatic pruritus in PBC by the U.S. Food and Drug Administration (FDA)

Volixibat被美国食品和药物管理局（FDA）授予治疗PBC胆汁淤积性瘙痒症的突破性治疗指定

Volixibat granted orphan drug designation for the treatment of cholestatic pruritus in PBC by the FDA

Volixibat被FDA授予孤儿药称号，用于治疗PBC胆汁淤积性瘙痒症

LIVMARLI approved by the FDA for cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients 12 months and older

LIVMARLI被FDA批准用于12个月及以上的进行性家族性肝内胆汁淤积症（PFIC）患者的胆汁淤积性瘙痒症

LIVMARLI approved in Europe for treatment of PFIC in patients three months and older

LIVMARLI在欧洲被批准用于治疗三个月及以上患者的PFIC

Initiated the LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions

启动了LIVMARLI EXPAND 3期研究，治疗罕见胆汁淤积性瘙痒症

New Drug Application (NDA) submitted for chenodiol in CTX

在CTX中为chenodiol提交的新药申请（NDA）

In-licensed global rights to PDE4D inhibitor MRM-3379 for FXS

在FXS的PDE4D抑制剂MRM-3379的全球许可权中

Corporate and Financial: Sustained financial strength

公司和财务：持续的财务实力

Achieved positive cash flow from operations in Q3 2024

2024年第三季度实现了正的经营现金流

Cash, cash equivalents and investments of approximately $287 million as of December 31, 2024 compared to $286.3 million as of December 31, 2023

截至2024年12月31日，现金、现金等价物和投资约为2.87亿美元，而截至2023年12月31日，现金、现金等价物和投资约为2.863亿美元

Announced the appointment of Joanne Quan, MD as Chief Medical Officer

宣布任命医学博士Joanne Quan为首席医疗官

The foregoing amounts relating to 2024 financial data are unaudited and preliminary and are subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2024..

上述与2024年财务数据有关的金额是未经审计的初步金额，需要完成财务结算程序。为了更全面地了解公司截至2024年12月31日的财务状况和经营成果，需要额外的信息和披露。。

Mirum will present at the 43

Mirum将出席43

rd

rd公司

annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 15, 2025, at 9:45 a.m. PT. The presentation and question and answer session will be webcast live and can be accessed by visiting the

2025年1月15日星期三上午9:45在旧金山举行的摩根大通医疗保健年度会议。演示和问答环节将进行网络直播，您可以访问

Investors and Media

投资者和媒体

section of Mirum’s corporate website. The replay of the webcast will be available for 30 days.

Mirum公司网站的一部分。网络广播的重播将持续30天。

About LIVMARLI

关于利玛丽

®

®

(maralixibat) oral solution

（马拉利昔巴特）口服液

LIVMARLI

利夫马利

®

®

(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.

（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，经美国食品和药物管理局批准用于治疗两种小儿胆汁淤积性肝病。它被批准用于治疗美国Alagille综合征（ALGS）患者的胆汁淤积性瘙痒症。

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.

三个月及以上的患者和两个月及以上的患者在欧洲。它在美国也被批准用于治疗12个月及以上进行性家族性肝内胆汁淤积症（PFIC）患者的胆汁淤积性瘙痒症，在欧洲被批准用于治疗3个月及以上患者的PFIC。

For more information for U.S. residents, please visit .

有关美国居民的更多信息，请访问。

LIVMARLI.com

LIVMARLI.com

.

.

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s .

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。LIVMARLI目前正在胆汁淤积性瘙痒症的其他环境中进行3期EXPAND研究评估。要了解更多有关LIVMARLI正在进行的临床试验的信息，请访问Mirum's。

clinical trials section

on the company’s website.

在公司网站上。

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.

肝损伤。

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.

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，皮肤或眼睛的白色部分变成黄色，黑色或棕色的尿液，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

胃和肠（胃肠）问题

. 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..

利夫马利可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

称为的条件

Fat Soluble Vitamin (FSV)

脂溶性维生素（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..

Alagille综合征和PFIC患者体内脂肪中某些维生素（维生素A，D，E和K）含量低导致的缺乏症很常见，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

EU SmPC

欧盟SmPC

Canadian Product Monograph

加拿大产品专著

About Volixibat

关于Volixibat

Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver.

Volixibat是一种口服，最小吸收的药物，旨在选择性抑制回肠胆汁酸转运蛋白（IBAT）。Volixibat可能通过抑制IBAT阻断胆汁酸的循环，从而全身和肝脏中的胆汁酸减少，从而为治疗成人胆汁淤积性疾病提供了一种新方法。

Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (.

Volixibat目前正在2b期研究中评估原发性硬化性胆管炎（。

VISTAS study

远景研究

), and primary biliary cholangitis (

)，以及原发性胆汁性胆管炎(

VANTAGE study

华帝研究

). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate..

)。6月，Mirum宣布了2b期VANTAGE研究的积极中期结果，显示瘙痒有统计学意义的改善，血清胆汁酸有意义的降低，服用volixibat的患者疲劳有所改善。没有观察到新的安全信号，最常见的不良事件是腹泻，所有病例均为轻度至中度。。

About CHOLBAM

关于CHOLBAM

®

®

(cholic acid) capsules

（胆酸）胶囊

The FDA approved CHOLBAM

FDA批准的CHOLBAM

®

®

(cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM.

（胆酸）胶囊于2015年3月首次获得FDA批准，用于治疗因单酶缺陷引起的胆汁酸合成障碍的儿科和成人患者，以及过氧化物酶体生物发生障碍Zellweger谱系障碍患者的辅助治疗。CHOLBAM的有效性。

®

®

has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy.

已经在胆汁酸合成障碍和过氧化物酶体障碍的辅助治疗的临床试验中得到证实。目前估计有200至300名患者正在接受治疗。

CHOLBAM

胆碱

®

®

(cholic acid) Indication

（胆酸）适应症

CHOLBAM is a bile acid indicated for

CHOLBAM是一种胆汁酸，用于

Treatment of bile acid synthesis disorders due to single enzyme defects.

治疗由单酶缺陷引起的胆汁酸合成障碍。

Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.

对表现出肝病，脂肪泻或脂溶性维生素吸收减少引起的并发症的患者进行过氧化物酶体疾病（包括Zellweger谱系疾病）的辅助治疗。

LIMITATIONS OF USE

使用限制

The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established.

CHOLBAM对由单酶缺陷或过氧化物酶体疾病（包括Zellweger谱系疾病）引起的胆汁酸合成障碍的肝外表现的安全性和有效性尚未确定。

IMPORTANT SAFETY INFORMATION

重要安全信息

WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment

警告和注意事项-肝功能损害加重

Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment.

在治疗期间肝功能恶化的患者中，监测肝功能并停用CHOLBAM。

Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose.

血清γ-谷氨酰转移酶（GGT）和丙氨酸氨基转移酶（ALT）同时升高可能表明CHOLBAM过量。

Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis.

如果有肝功能恶化或胆汁淤积的临床或实验室指标，请随时停止使用CHOLBAM治疗。

ADVERSE REACTIONS

不良反应

The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy.

最常见的不良反应（≥1%）是腹泻，反流性食管炎，不适，黄疸，皮肤病变，恶心，腹痛，肠息肉，尿路感染和周围神经病变。

Please see full

请查看完整信息

Prescribing Information

处方信息

for additional Important Safety Information.

有关其他重要安全信息。

About Cerebrotendinous Xanthomatosis

关于脑腱黄瘤病

Cerebrotendinous xanthomatosis (CTX) is a rare, progressive and underdiagnosed disorder of cholesterol metabolism affecting many parts of the body. In people with CTX, the body is unable to break down cholesterol properly causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time.

脑腱黄瘤病（CTX）是一种罕见的，进行性且诊断不足的胆固醇代谢疾病，影响身体的许多部位。在患有CTX的人中，身体无法正确分解胆固醇，导致毒素（例如胆固醇和胆汁醇）随着时间的推移在全身积聚。

The disorder is inherited in an autosomal recessive genetic manner. Signs and symptoms of CTX include neonatal cholestasis (jaundice or bile flow interruption), chronic diarrhea, the development of bilateral cataracts before the age of 18, development of tendon xanthomas (fatty deposits in the tendons) during teenage years or later, and neurologic deterioration.

该疾病以常染色体隐性遗传方式遗传。CTX的体征和症状包括新生儿胆汁淤积（黄疸或胆汁流动中断），慢性腹泻，18岁之前双侧白内障的发展，青少年或以后肌腱黄瘤（肌腱中的脂肪沉积）的发展以及神经系统恶化。

The types, combinations and severity of symptoms can be different from person to person making diagnosis challenging and often delayed..

症状的类型，组合和严重程度因人而异，这使得诊断具有挑战性，并且经常被延迟。。

About chenodiol tablets

关于藜芦醇片

Chenodiol tablets is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. More recently, the US Food and Drug Administration (FDA) granted chenodiol orphan drug designation for cerebrotendinous xanthomatosis (CTX).

鹅去氧胆酸片是鹅去氧胆酸（CDCA）的另一个名字。CDCA是一种天然存在的胆汁酸，最初被批准用于治疗胆囊中的放射性结石患者。最近，美国食品和药物管理局（FDA）批准了chenodiol孤儿药用于脑腱黄瘤病（CTX）。

CTX is a rare progressive disorder that can affect the brain, spinal cord, tendons, eyes and arteries. Chenodiol is not yet indicated for the treatment of CTX but has received a medical necessity determination in the U.S. by the FDA..

。Chenodiol尚未用于治疗CTX，但已在美国获得FDA的医疗必要性测定。。

About Mirum Pharmaceuticals

关于Mirum Pharmaceuticals

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications:

。Mirum有三种批准的药物：

LIVMARLI

利夫马利

®

®

(maralixibat) oral solution

（马拉利昔巴特）口服液

,

,

CHOLBAM

胆碱

®

®

(cholic acid) capsules,

（胆酸）胶囊，

and

和

CHENODAL

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.

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 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.

它在美国也被批准用于12个月及以上的PFIC患者的胆汁淤积性瘙痒症；在欧洲，它被批准用于三个月及以上的PFIC患者。Mirum还启动了3期EXPAND研究，这是LIVMARLI在胆汁淤积性瘙痒症的其他情况下的标签扩展机会。

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)..

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。。。

Mirum's late-stage pipeline includes investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2

Mirum的晚期项目包括对几种罕见疾病的研究性治疗。Volixibat是一种IBAT抑制剂，正在两项潜在的注册研究中进行评估，包括第二阶段

VISTAS

远景

study for primary sclerosing cholangitis (PSC) and Phase 2b

原发性硬化性胆管炎（PSC）和2b期的研究

VANTAGE

华帝

study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results

原发性胆汁性胆管炎的研究。Volixibat已被授予突破性治疗指定，用于治疗PBC患者的胆汁淤积性瘙痒症。

reported

已报告

in 2023. Mirum has

2023年。Mirum有

submitted

已提交

a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. Lastly, Mirum is planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.

美国食品和药物管理局（FDA）批准了一项新药申请，批准在美国使用chenodiol治疗CTX。最后，Mirum计划进行一项2期研究，评估MRM-3379，一种PDE4D抑制剂，用于治疗脆性X综合征，一种罕见的遗传性神经认知障碍。

To learn more about Mirum, visit mirumpharma.com and follow Mirum on

要了解有关Mirum的更多信息，请访问mirumpharma.com并关注Mirum

Facebook

脸书

,

,

LinkedIn

LinkedIn

,

,

Instagram

Instagram

and

和

Twitter

推特

(X).

（十） 。

Forward-Looking Statements

前瞻性声明

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, commercial results for our approved products, including continued growth in year over year net product sales, being on track to achieve our revised financial guidance, our expected financial results as of December 31, 2024, including our net product sales and cash, cash equivalents and investments, delivering life changing medicines for patients suffering from rare diseases, the results, enrollment, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, including newly in-licensed product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for its product candidates globally, including the anticipated PDUFA date for chenodiol for CTX and additional international launches expected in 2025.

本新闻稿中关于非历史事实的声明是1995年《私人证券诉讼改革法案》所指的“前瞻性声明”。此类前瞻性声明包括以下方面的声明：我们批准的产品的商业业绩，包括产品净销售额同比持续增长，正在按计划实现修订后的财务指导，截至2024年12月31日的预期财务业绩，包括我们的产品净销售额和现金、现金等价物和投资，为患有罕见疾病的患者提供改变生命的药物，Mirum正在进行和计划进行的候选产品研究的结果、注册、实施和进展，包括新获得许可的候选产品，我们正在进行的研究的中期分析的时间和结果，以及全球候选产品的监管批准路径，包括chenodiol for CTX的预期PDUFA日期和预计2025年的其他国际发布是的。

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 “expected,” “will,” “could,” “would,” “guidance,” “potential” and similar expressions are intended to identify forward-looking statements.

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“预期”、“将”、“可能”、“将”、“指导”、“潜在”等词语以及类似的表达方式旨在识别前瞻性陈述。

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,.

这些前瞻性陈述基于Mirum目前的预期，并涉及可能永远不会实现或可能被证明不正确的假设。由于各种风险和不确定性，实际结果可能与此类前瞻性声明中的预期结果存在重大差异，这些风险和不确定性包括但不限于与Mirum总体业务相关的风险和不确定性，。