Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Participants With Cholestatic Pruritus

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.

开始日期2024-10-14

申办/合作机构

Mirum Pharmaceuticals, Inc.

NCT06413368

/ Not yet recruiting临床2/3期IIT

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Chronic constipation is a feature of children with cystic fibrosis (CF). This is postulated to be a result of inhibition of secretory activity of the gastrointestinal luminal cells due to ineffective chloride channel function. Typical laxatives that work as osmotic agents fail to produce adequate relief in this population.
Maralixibat is a non-systemic bile acid transport inhibitor (IBATi) that acts by interrupting bile acid reabsorption in the ileum thus interrupting the normal enterohepatic circulation. This interruption results in a larger volume of bile acids reaching the colon and being excreted in stool. Bile acids are known to decrease bowel transit time, increase mucosal permeability and secretions, as well as alter gut microbiota resulting in diarrhea.
The overarching hypothesis of the study is that Maralixibat will improve stool consistency in children (Age <18 years) with cystic fibrosis and constipation (Bristol Stool Scale <4). Specifically, we aim to test the hypothesis that IBATi improves the consistency of stool to Bristol scale >4 in children with CF and constipation.
We will recruit a total of 20 patients with CF and constipation (defined as Bristol Stool Scale <4 for 1 week prior to enrollment while on a stable laxative regimen for at least 4 weeks.) Design is a 'Within-Subjects' study by which each enrolled patient will take Maralixibat for 2 weeks total in addition to their stable laxative regimen during the study. Stool consistency & ease of defecation will be recorded before and during the study period by families of enrolled patients via materials provided by the investigators. Stool consistency and ease of defecation will be compared before and after initiation of Maralixibat.
The primary endpoint: Improvement in stool consistency to Bristol scale >4 in children with CF and constipation. The secondary endpoint: Improvement in ease of defecation in children with CF and constipation. This will be measured via survey using a standardized scale (Bristol Stool Scale) and questionnaires developed by the research team. Analysis will involve comparison of pre-intervention to post-intervention stool consistency & survey

开始日期2024-07-01

申办/合作机构

Children's Hospital Los Angeles

NCT06193928

/ RecruitingN/A

Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

开始日期2023-09-21

申办/合作机构

Mirum Pharmaceuticals, Inc.

100 项与氯马昔巴特相关的临床结果

登录后查看更多信息

100 项与氯马昔巴特相关的转化医学

登录后查看更多信息

100 项与氯马昔巴特相关的专利（医药）

登录后查看更多信息

项与氯马昔巴特相关的文献（医药）

2025-09-01·JOURNAL OF HEPATOLOGY

Use of maralixibat in severe and recurrent intrahepatic cholestasis of pregnancy with concurrent benign recurrent intrahepatic cholestasis

Letter

作者: Katzenstein, Cecilia ; Kushner, Tatyana ; O'Kula, Susanna ; Rodriguez, Nina

2025-09-01·METABOLIC ENGINEERING

Improved biosynthesis of C4 derivatives by engineered thiolase

Article

作者: Ma, Zhiping ; Xu, Bing ; Zhao, Jing ; Nie, Mengzhen ; Chen, Zeyao ; Zhang, Changxi ; Mao, Yaping ; Zhang, Kechun

Ethylene glycol (EG), a major product of the enzymatic degradation of polyethylene terephthalate (PET), provides a promising feedstock for sustainable biomanufacturing. Herein, we developed a novel metabolic pathway using Escherichia coli(E. coli) as a host for the biosynthesis of four-carbon compounds such as 1,4-butanediol (1,4-BDO), 1,2,4-butanetriol (1,2,4-BTO), and succinate, from two-carbon substrates such as glycolate and EG. This represents an efficient strategy of using C2 precursors for these high-value chemicals. Through directed evolution of the β-ketoacyl thiolase B of Cupriavidus necator (CnBktB), one of the rate-limiting enzymes in the pathway, via an established growth-coupled screening platform, we identified the L89S mutant, which exhibits significantly enhanced catalytic efficiency in assimilating glycolyl-CoA and acetyl-CoA. Using glycolate and glucose as substrates, the route achieves production titers of >200 mg/L for 1,4-BDO, 266 mg/L for 1,2,4-BTO, and 9.22 g/L for succinate. Furthermore, integrating an upstream module for EG conversion to glycolate allows direct utilization of PET-derived EG, yielding 11.4 g/L succinate with 93 % conversion efficiency from EG. This work bridges the fields of synthetic biology and plastic waste recycling, demonstrating a sustainable and scalable route for converting PET-derived EG into valuable four-carbon compounds. The novel biosynthetic pathways developed in this study offer a foundation for advancing circular bioeconomy strategies and reducing the environmental impact of plastic waste.

2025-07-22·Cureus Journal of Medical Science

Treatment of Pruritus With Maralixibat in Early-Stage Chronic Graft Dysfunction of a Child With Alagille Syndrome

Article

作者: Weigert, Alexander ; Ganschow, Rainer ; Katzer, David

Alagille syndrome (ALGS) is a rare, autosomal dominant disorder characterized by cholestasis and progressive liver disease that can lead to liver transplantation or death. Liver transplant recipients with ALGS can face chronic graft dysfunction and refractory pruritus that significantly impairs quality of life. We present the case of a 16-year-old male patient with ALGS who developed severe pruritus 13 years after receiving a liver transplant. Despite conventional therapies, his symptoms persisted, accompanied by elevated serum bile acids. Treatment with maralixibat, an ileal bile acid transporter inhibitor, resulted in complete resolution of pruritus within two months as well as a substantial reduction in serum bile acids. No adverse events were reported. This case offers real-world evidence of the effectiveness of maralixibat in managing cholestatic pruritus in a patient with ALGS and early-stage chronic graft dysfunction, highlighting its potential as a therapeutic option in this challenging patient population.

262

项与氯马昔巴特相关的新闻（医药）

2025-09-08

·ir.mirumpharma.com

Mirum Pharmaceuticals Announces Enrollment Completion in the Phase 2b VISTAS Study

- Topline data announcement expected second quarter of 2026 FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the completion of enrollment in the Phase 2b VISTAS study of volixibat, an investigational oral IBAT inhibitor, for the treatment of cholestatic pruritus in patients with primary sclerosing cholangitis (PSC). The trial previously met its pre-specified efficacy and safety thresholds at the blinded interim analysis for dose selection conducted in 2024 and topline results are expected to be announced in the second quarter of 2026. “Completing enrollment in VISTAS is a major milestone for the PSC community and for Mirum,” said Joanne Quan, M.D., Chief Medical Officer. “This progress reflects the dedication of patients, investigators, and our team working together toward the shared goal of addressing the daily challenges of PSC, including persistent itch and fatigue. We believe the data generated from VISTAS will be an important step toward bringing meaningful medicines to patients who currently have no approved therapies.” Ricky Safer, CEO and Founder of PSC Partners Seeking a Cure, a patient-led advocacy organization dedicated to supporting those affected by PSC, added “PSC Partners is thrilled to see Mirum reach this important milestone with the VISTAS study. Completing enrollment marks a significant step forward in advancing a possible treatment for itch in PSC, bringing tremendous hope to our community. We are grateful to Mirum for their commitment and to all the patients and families who made this kind of progress possible. Together, we are moving closer to new possibilities and a brighter future for those impacted by PSC.” Volixibat is also being evaluated in the Phase 2b VANTAGE study for primary biliary cholangitis (PBC), which is expected to complete enrollment in 2026 and announce topline results in the first half of 2027. About the VISTAS Phase 2b Study The VISTAS study (NCT04663308) is a global, randomized, double-blind, placebo-controlled Phase 2b trial evaluating the efficacy and safety of volixibat in patients with primary sclerosing cholangitis and cholestatic pruritus over a 28-week period. The primary endpoint is a reduction in pruritus as measured by the Adult ItchRO, a pruritus numerical rating scale ranging from 0 (no itch) to 10 (worst possible itch). Secondary endpoints include assessments of fatigue, serum bile acids, and safety. About Primary Sclerosing Cholangitis (PSC) Primary sclerosing cholangitis (PSC) is a rare, chronic, progressive liver disease in which the bile ducts inside and outside the liver become inflamed, scarred, and narrowed over time. This bile duct damage leads to bile accumulation, liver injury, and eventually liver failure. Patients with PSC experience a heavy symptom burden, with pruritus (severe itching), fatigue, and abdominal pain that may be debilitating and life-altering. Beyond symptom burden, PSC is associated with increased risk of bile duct cancer and need for liver transplantation. There are currently no approved therapies for PSC, leaving patients with limited treatment options focused on symptom management and supportive care. About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach to 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 is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2024, 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. The Phase 2 VISTAS study met its pre-specified efficacy and safety thresholds at the blinded interim analysis for dose selection conducted in 2024. Volixibat has been granted breakthrough therapy designation for the treatment of cholestatic pruritus in patients with PBC. 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/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (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. for 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 conducting 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b 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. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X. Forward-Looking Statements 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, the results and progress of our ongoing and planned studies for our product candidates, the timing and results of topline data for our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. 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,” “continue” 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, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. 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. Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Bryan Blatstein Spectrum Science Communications BBlatstein@spectrumscience.com

临床2期上市批准突破性疗法临床结果临床3期

2025-09-02

·研发客

商保创新药目录初成，进院品种赔付可能性有多大？

// • 当前的关注点是商保创新药目录如何落地。公立医院考核体系该如何优化，为商保支付留出空间。 • 对于医院，应推动医院与商保合作，建立商保专员制度，为患者提供一站式商保服务。 • 商保创新药目录“应建立退出机制，对疗效不佳、安全性存在问题或经济性不合理的药品及时进行调整和退出” 。 8月28日，国家医保局正式公布《通过2025年国家基本医疗保险、生育保险和工伤保险药品目录及商业健康保险创新药品目录调整形式审查的申报药品名单》的结果：商业健康保险创新药目录（下简称“商保创新药目录”）申报信息141份，涉及药品通用名141个，121个通过形式审查，其中同时申报基本医保目录和商保创新药目录的有79个。来源|研发客整理多款备受关注的“明星药物”此前通过了商保创新药目录的初步形式审查，包括多款PD-1、国内获批上市的5款CAR-T疗法和全球首个靶向Trop-2的ADC药物戈沙妥珠单抗等。“只有通过评审、谈判等全部环节的药品，才能最终被纳入目录，期间仍存在诸多不确定性。”多家PD-1和CAR-T企业表示。复旦大学附属肿瘤医院肿瘤内科主任医师张剑教授表示，商保在支付创新药时，常常面临目录不统一、支付标准不一致等问题，这使得患者在报销过程中遭遇诸多困难。而商保创新药目录的出台，有望有效解决这些问题，推动商保支付朝着更加规范化和系统化的方向发展。目前更多的讨论，集中在商保创新药目录如何真正地落地。“只有提供具体进院品种和赔付项目，才对消费者更具购买实用价值。”前RDPAC执行总裁康韦说。尽管有观点认为此类商业保险更易在民营医院落地，然而不少公立医院也已表态，将积极筹划并引入商保项目。此外，对于患者而言，能否事前、事中赔付而非事后报销也很关键。拓展阅读商保创新药品目录来了，丙类目录呢？商保创新药目录官宣！与医保目录同步申报商保创新药目录出台时间线 7月《2025年国家医保药品目录调整工作方案》及相关文件正式发布。 7月企业通过国家医保服务平台进行网上申报。 8月国家医保局对申报药品进行形式审查，并公示初步审查结果。 8~9月组织专家进行评审 9~10月医保谈判/竞价及商保价格协商 10~11月公布新版目录探索医院与商保衔接康韦最近正在谋划成立广州市粤港澳大湾区生物医药创新联合会，其中，探索商业健康保险创新实践是该联合会的使命之一。最近，她在广州参加了“穗新保”之创新药械商业健康险可持续发展研讨会。研发客从珠江人寿保险相关人士了解到，广州今年3月推出的“穗新保”已可提供具体保障与服务。例如，“穗新保”纳入“港澳药械通”、广州本地及其他高值自费创新药械，保障种类更全，以及实现在22家医院一站式结算服务等等。 “实现在22家医院一站式结算服务，这实际上是在积极推动相关医疗保障体系的优化。”康韦说，一站式结算不仅涉及硬件设施的升级，如医院系统的改造，更体现了政府，尤其是广州市医保局在此方面的努力。前RDPAC执行总裁康韦此外，作为医疗保障改革先锋的上海、深圳，在医药商业保险领域的成果也非常明显。上海“沪惠保” 2025年新增海外特药等先进疗法，累计参保超2600万人次、赔付超22亿元，还整合12项健康管理服务促转型，依托“随申办”实现“一站式”理赔，参保率稳定在38%以上。深圳“深圳惠民保”聚焦罕见病与特药，2025年罕见病药品报销额提至60万元/年，新增15种抗癌新药，年赔付上限达300万元，通过降免赔等措施，2024年参保率达51%。然而，康韦提醒说：“多层次医疗保障体系实施需要多层次医疗服务供给支撑。然而，公立医院主要承担基本医疗服务，受到预算和考核指标的限制，对商保的推进缺乏积极性。” “因此，公立医院考核体系如何优化，给商保支付留够空间，从而满足不同层次的医疗服务需求值得研究。”康韦说，“只有提供具体进院和赔付项目，才对消费者更具购买实用价值。” 康韦认为，当前大家关注的焦点在于商保创新药目录对惠民保目录的影响。惠民保续保率面临死亡螺旋的挑战，叠加商保创新药目录的影响，惠民保今后如何健康持续发展，值得关注。针对商保创新药目录的未来发展，张剑教授也认为商保品种能进院非常重要，对于医院而言，应该推动医院与商保合作，建立商保专员制度，为患者提供一站式的商保服务。同时，医院应优化进药流程，减少集采、医保考核指标和药占比等因素对商保药品进院的影响。复旦大学附属肿瘤医院肿瘤内科主任医师张剑教授张剑还提出了以下几点建议：一是建立动态调整机制。“国家应建立商保创新药目录的动态调整机制，每年或每两年根据药品疗效、安全性和经济性等因素进行评估和调整。”他说，这样，有助于确保目录内的药品符合临床需求和患者利益。二是要商保体系应加强对患者数据的规整与利用，建立真实世界数据平台。这有助于收集创新药的临床使用数据，为药品评价、医保谈判和商保支付提供科学依据。对于企业，商保创新药目录应发挥引导作用。张剑鼓励企业研发针对罕见病、高值药品和基因治疗等领域的创新药。通过商保支付，这些药品得以进入市场，为企业提供了新的盈利模式。 “政府部门应加强对商保创新药目录的监管与评估，确保目录内的药品符合临床需求和患者利益。同时，应建立退出机制，对疗效不佳、安全性存在问题或经济性不合理的药品及时进行调整和退出。”张剑说。定价：“协商”而非“谈判” 整体上，企业对此持审慎乐观态度，并期待通过商保创新药目录的建立，进一步拓宽创新药物的支付渠道，提高患者可及性。北海康成CEO薛群博士告诉研发客，此次商保创新药目录将多款罕见病药物作为重点，是对创新药物支付破冰的一个重要举措。“无论未来的落地情况如何，都将是破冰之举。” 北海康成的3款产品戈芮宁（注射用维拉苷酶β）、迈芮倍（氯马昔巴特口服溶液）、海芮思（艾度硫酸酯酶β注射液）于8月12日通过初审。北海康成CEO 薛群薛群认为，罕见病药物纳入商保目录的利好就是“加速市场准入与支付闭环”。高价值罕见病药物的支付问题解决极为艰难，对政府相关部门、商业保险公司、药企都是一个任重道远的课题。但商保创新药目录的出台无疑是对创新药物支付的破冰之旅。 “由于商保创新药目录药品不计入医疗机构医保自费率考核、不纳入集采替代监测范围、不纳入按病种付费范围，因此，罕见病药物使用门槛将会降低。”薛群说。在支付体验方面，薛群认为“一站式结算”破解垫付难题：北京、上海等12家医院试点医保商保同步结算，商保理赔周期从7天缩短至30分钟，罕见病患者无需垫付高额药费。当然，他也看到即使药品进入目录并由商保覆盖，患者通常仍需承担一定比例的自付费用（如惠民保的免赔额和报销比例）。对于一年花费数十万元甚至上百万元的药品，这笔自付费用对普通家庭而言仍是重负。“患者看到有药品进目录是很高兴，但更关心最终自己需要付多少钱，是不是真的用得起。”他说。关于后续流程，目录通过初步审查后，将进入谈判竞价环节。他提出罕见病药物的创新价值量化应当引入多维度评估体系，包括：临床需求紧迫性（如无替代疗法的罕见病）、疗效突破性（如显著延长生存期或改善生活质量）、社会效益（如减少长期护理成本）。这样才能使优秀的罕见病药物能真正的惠及中国患者。从支付角度来看，商保目录采用“协商”而非医保的“谈判”模式，药品的定价依然是关键。他认为协商的目标是让药价降到商保能承受、患者可及、企业也有合理回报的水平，这需要非常精细的设计。薛群认为，对商保公司而言，纳入高价药必然推高保险产品的赔付成本。这需要探索风险分担机制，例如引入再保险、建立罕见病专项赔付池，开发更多元化、分层的健康险产品。但他同时指出，罕见病的优势在于，相较于肿瘤药品而言，每个罕见病种的患者数量是可预估的，可以较为清晰的算出资金池的规模大小。保险公司不仅可以对药品价格进行商议，也可以商磋“资金池封顶”设计，对于超出部分的赔付由药企兜底，从而避免高价值罕见病药物击穿整个保险项目的资金池。最后，薛群表示，如果罕见病药物进入商保创新药目录，同时目录在落地过程中也扩大了市场的覆盖面，罕见病的商业化进展就可以推动研发可持续性增强，使得整个中国的罕见病药物发展进入良性循环。编辑 | 姚嘉 yao.jia@PharmaDJ.com 总第2562期访问研发客网站，深度报道和每日新闻抢鲜看 www.PharmaDJ.com

抗体药物偶联物免疫疗法细胞疗法

2025-08-28

·精准药物

2025创新药涨幅榜单

PREFACE 前言 2025年是中国创新药产业的爆发之年。在国家密集出台医药支持政策、企业业绩普遍增长、对外授权交易活跃的多重利好下，创新药板块迎来了前所未有的上涨行情。据数据显示，2024年国产创新药出海交易总额在523亿美元，这已经是历史新高了。而今年上半年，我国药企海外授权总金额超去年全年，达608亿美元。突破1000亿美元大关实现100%的暴增也很有可能。这一数字背后是中国创新药质量的全面提升和国际认可度的飞跃。从"跟跑"到"并跑"再到部分领域实现"领跑"，中国创新药正在完成从量变到质变的华丽转身。出海的超预期，让资金普遍认为今年创新药板块极大概率要迎来业绩拐点，这就是本轮行情的核心驱动力。随着创新药企半年报披露逐渐接近尾声，bioSeedin柏思荟也盘点了从2025年初至今创新药药企涨幅TOP榜单。数据肯定不够严整，涨幅也不代表企业实力排名。只希望通过这个线索，窥见数据背后的产业逻辑，探寻那些引领行业变革的标杆企业，观察中国医药创新的未来走向。图源：根据公开数据整理 01 北海康成断层第一纵观该榜单，北海康成（01228.HK）以接近2000%的涨幅位居2025年至今的创新药涨幅榜首位，成为了市场上最引人注目的“罕见病第一股”。这家曾经股价连续三年下跌、甚至沦为“仙股”的企业，从2025年5月份开始实现了惊人逆转。其上涨逻辑主要基于三大因素： 1、首款自研罕见病药物戈芮宁获批。今年5月，北海康成的注射用维拉苷酶β（商品名：戈芮宁）获得批准，这是中国本土首款自主研发的罕见病创新药，也是国家药监局《生物制品分段生产试点工作方案》发布后，首个通过分段生产检查的创新生物药。要知道罕见病药在国内放量销售的最大难点，其实是“能不能付得起”和“能不能找到病人”的问题。药再好，没有合适的支付渠道，医生开处方、患者买单都会有障碍。而这一大障碍也随着以下两点的利好，开始扭转北海康成的命运。 2、政策红利支持。2025年，在国家医保局指导下，商业保险机构首次联合设立商保创新药目录，并于7月11日正式启动申报工作。北海康成的3款产品戈芮宁、迈芮倍、海芮思于8月12日通过初审，有望加速市场准入。通过初审名单这一动作，一方面意味着这3款产品的创新性和临床价值得到了官方认可，为其未来的市场推广奠定了坚实基础；另一方面，药品如能够获得商业保险的支持，患者用药的经济负担有望减轻，从而提高产品的可及性。 3、战略投资与合作。8月13日，北海康成宣布与百洋医药达成股份认购协议，百洋医药将斥资1亿港元认购北海康成股份，双方还签订了独家商业化服务协议。此次战略股权融资，对于北海康成而言意义重大。一方面，能够优化其商业化业务模式，增强商业运营实力；另一方面，加速已上市产品的市场渗透，进一步提升市场竞争力。毕竟百洋医药凭借其在创新品牌商业化平台的深厚实力，在医药商业化领域有着广泛的渠道资源和强大的营销能力，此次合作无疑是优势互补的绝佳典范。 02 德琪医药德琪作为年初至今涨幅第二的创新药企，俨然成为创新药板块的一匹黑马。总体来看，德琪医药正从 “低调蓄力” 走向 “全面爆发”。这一转变看似是资本市场的热烈追捧，实则是企业发展深层蜕变的外在显现。其中，两大核心要素推动着这场蜕变： 1、核心产品塞利尼索在亚太地区的快速放量，为企业发展筑牢现金流根基； 2、Claudin 18.2 ADC 药物ATG-022，成为公司实现价值跃升的关键引擎其中塞利尼索作为全球首个全新机制的口服选择性XPO1抑制剂，已在亚太市场的10个国家和地区获批用于治疗多项适应症，并在其中5个市场实现了医保收录。市场表现更是锦上添花，2024年其销售收入达0.92 亿元，同比增长37%，其中中国地区收入0.72 亿元（同比+ 27%），亚太地区收入0.20 亿元（同比+ 86%）。据太平洋证券预测，随着塞利尼索适应症的不断拓展，其未来销售峰值有望接近8亿元。此外，其管线下的Claudin 18.2 ADC药物ATG-022则是公司实现价值飞跃的关键引擎。在2025年美国临床肿瘤学会胃肠道肿瘤学术会议（ASCO GI 2025）上，德琪医药公布的I/II期CLINCH研究最新数据显示：ATG-022在Claudin 18.2不同表达水平的患者中均显示出显著疗效。非头对头比较数据显示，后线治疗CIDN18.2中低表达胃癌/胃食管交界处(GC/GEJ)患者，ATG-022具有更高的反应率和安全性优势。图源：太平洋证券值得一提的是，2025年5月20日，德琪医药宣布与默沙东达成全球临床合作，共同评估ATG-022联合默沙东PD-1抑制剂K药用于治疗晚期实体瘤患者的效果。与默沙东的合作是对ATG-022临床价值的高度认可，也为该药物未来的全球商业化铺平了道路。随着2025年下半年ATG-022关键注册临床的启动，德琪有望在2026年提交新药上市申请（NDA），2027年获批胃癌及胰腺癌适应症，成为全球首个覆盖广泛表达水平的Claudin 18.2靶向药物。 03 和铂医药和铂医药（02142.HK）作为当之无愧的BD之王，从2022年开始，和铂医药便集中资源，进行战略调整，截至目前，和铂医药已达成合作40多项，潜在合作总金额超百亿美元。仅今年上半年，和铂医药就已达成6次合作。近日，和铂医药发布正面盈利预告：预计2025上半年盈利6800万美元（约5.32亿港元）至7400万美元（约5.79亿港元）。这一数字较2024年同期约140万美元增长超47倍，而且轻松越过其2024年全年277.8万美元的盈利规模。业绩的稳健增长，源于和铂医药已经实践出了一套契合自身商业模式——基于独有的Harbour Mice®抗体发现平台，持续不断输出BD合作，回补现金流，同时赋能自身研发管线向商业化不断迈进。凭借连续三年盈利，和铂医药印证了技术平台授权模式的可持续性。在持续创新的驱动下，和铂医药未来有望继续依托这一模式实现稳健盈利。当然，若能如再生元一般，成功打造出自主的重磅产品，公司发展将再上一层楼。 04 加科思 2025年，作为加科思持续创收的第一年，随着其在KRAS等领域的深耕，注定了其基本面的蜕变。今年5月份，加科思靶向肺癌药物KRAS G12C抑制剂戈来雷塞获NMPA批准上市。由此事件会解锁两个收入来源：第一，来自艾力斯的里程碑款；第二，两位数比例的净销售额提成。由于是净销售的分成，因此会是加科思稳定的现金奶牛，让加科思成为一家能够持续产生收入的公司。与此同时，加科思不仅着眼于肺癌的治疗，还积极拓展其他癌症适应症的注册临床试验，包括胰腺癌、胆道癌等领域。公司正逐步实现“上市一代、临床一代、储备一代”的产品管线战略，下一代KRAS抑制剂如JAB-23E73现已进入临床试验，展现出更大的市场潜力。 05 荣昌生物所有人都不看好你，但属你最争气，这句话用于荣昌生物再合适不过。过去的2024年，荣昌生物充满争议，这在它的股票波动上完全反映了出来，从巅峰坠落低谷，然后再从低谷急速而上。争论背后的原因，无非两大点：现金流紧张、产品授权不及预期。然而，荣昌生物并未就此沉沦。2025年开始，公司通过加强商业化能力、成本费用控制、引入战略投资者以及BD等多项措施，成功扭转局面。根据荣昌半年报，半年内营收突破了10亿元，同比增长48.02%，净利润上，同比减亏3.3亿元。值得注意的是，营收上的10亿元还没计入今年BD收入。从商业化层面来看，已商业化的泰它西普和维迪西妥单抗不断拓展新适应症、市场渗透加深，推动销售收入强劲增长。而在研管线中，8月8日，荣昌宣布PD-1/VEGF双抗RC148获得FDA新药IND许可，在美国开展针对多种晚期恶性实体肿瘤的Ⅱ期临床研究。从进度上来说，荣昌生物RC148已经处于第一梯队。 8月13日，泰它西普干燥综合征三期临床达到主要终点，即将申请上市。总的来看，今年荣昌共达成泰它西普、RC28-E眼科药物两个大BD，叠加两个国内申请上市，一个海外申请上市，还有一个海外里程碑回款的彩蛋。明年8个新适应症密集上市。未来三年，荣昌生物各种利好将连绵不绝。 SUMMARY 小结本次涨幅排行榜不仅反映了市场资金的流向，更揭示了中国医药创新的新趋势：罕见病药物、肿瘤治疗、自身免疫疾病等细分领域正成为价值挖掘的新高地；从"me-too"到"me-better"再到"first-in-class"，创新层级不断攀升；license-out也已成为企业价值实现的重要路径。声明：发表/转载本文仅仅是出于传播信息的需要，并不意味着代表本公众号观点或证实其内容的真实性。据此内容作出的任何判断，后果自负。若有侵权，告知必删！长按关注本公众号粉丝群/投稿/授权/广告等请联系公众号助手觉得本文好看，请点这里↓

引进/卖出申请上市

100 项与氯马昔巴特相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16226

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
进行性家族性肝内胆汁淤积症	欧盟	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	冰岛	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	列支敦士登	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	挪威	Mirum Pharmaceuticals, Inc.	2024-07-08
阿拉吉欧综合症	欧盟	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	冰岛	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	列支敦士登	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	挪威	Mirum Pharmaceuticals, Inc.	2022-12-09
胆汁淤积性瘙痒	美国	Mirum Pharmaceuticals, Inc.	2021-09-29

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
胆汁淤积性肝病	临床3期	美国	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	巴西	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	加拿大	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	法国	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	德国	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	意大利	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	黎巴嫩	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	墨西哥	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	波兰	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	沙特阿拉伯	Mirum Pharmaceuticals, Inc.	2024-10-14

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03905330 (FDA_CDER) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	64	Maralixibat	積網廠製繭艱艱糧窪顧(構鏇積夢艱憲積範蓋觸) = 繭簾遞遞艱顧衊遞鏇鏇鏇鏇糧餘蓋選製鹹窪顧 (糧夢顧膚壓鬱衊艱憲夢, -2.2 ~ -1.4)	积极	2025-04-10
				Placebo	積網廠製繭艱艱糧窪顧(構鏇積夢艱憲積範蓋觸) = 憲範鑰鏇製繭鏇鑰糧壓鏇鏇糧餘蓋選製鹹窪顧 (糧夢顧膚壓鬱衊艱憲夢, -1.0 ~ -0.2)
NCT02160782 (FDA_CDER) 人工标引	临床2期	阿拉吉欧综合症 \| 瘙痒	29	Maralixibat	製壓鑰願築觸鹹網廠鹹(壓鏇範願夢廠願網遞願) = 網餘膚艱餘網顧艱遞觸壓糧鑰製憲範鏇襯鹽衊 (夢遞淵製顧蓋夢獵鑰積, -0.3 ~ 0.7)	积极	2025-04-10
				Placebo	製壓鑰願築觸鹹網廠鹹(壓鏇範願夢廠願網遞願) = 壓齋艱膚構積遞壓壓壓壓糧鑰製憲範鏇襯鹽衊 (夢遞淵製顧蓋夢獵鑰積, 1.2 ~ 2.1)
NCT05543174 (CTgov)	临床3期	阿拉吉欧综合症	7	TAK-625	顧鏇膚醖觸鬱顧獵糧鹹(齋膚積壓鏇糧餘鹽衊夢) = 獵齋餘餘艱範鏇鬱簾築範襯網簾簾醖鏇衊衊憲 (衊蓋願構衊艱繭遞簾簾, 鏇膚築製壓製鹽鹹餘廠 ~ 積遞築鑰襯廠築醖築鹽) 更多	-	2024-10-15
NCT05543187 (CTgov)	临床3期	进行性家族性肝内胆汁淤积症	5	TAK-625 (Primary Cohort: TAK-625)	餘獵鏇淵鏇鑰獵糧餘選(餘廠築鏇遞齋憲襯艱窪) = 襯蓋艱範顧憲選鹽繭壓願簾構齋壓醖壓鏇願網 (糧鹹觸壓餘膚顧鹹夢築, 願鑰憲選壓獵選選願鬱 ~ 築鏇膚淵簾築繭簾範醖) 更多	-	2024-09-24
				TAK-625 (Supplemental Cohort: TAK-625)	餘獵鏇淵鏇鑰獵糧餘選(餘廠築鏇遞齋憲襯艱窪) = 衊選醖艱築網襯築製選願簾構齋壓醖壓鏇願網 (糧鹹觸壓餘膚顧鹹夢築, 觸憲壓簾蓋鑰膚糧積遞 ~ 願窪積鹹醖餘製製製襯) 更多
NCT04168385 (MERGE, NEWS)	临床2期	阿拉吉欧综合症	76	马昔巴特	構壓製廠製襯製壓積夢(壓鏇壓築鏇鏇鑰壓壓淵) = 膚構蓋鹹襯齋觸衊壓網艱獵鏇壓製膚遞憲簾鑰 (齋鹹願遞顧齋廠膚範憲 ) 更多	积极	2024-06-07
MARCH-ON (Biospace) 人工标引	N/A	进行性家族性肝内胆汁淤积症	-	LIVMARLI	鹹餘獵壓鏇憲鏇夢衊廠(衊襯憲廠醖顧積蓋鬱廠) = 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. 簾製構蓋壓繭觸膚簾蓋 (壓蓋憲願壓淵鑰艱簾衊 )	积极	2024-05-18
NCT03905330 (MARCH-PFIC, Pubmed) 人工标引	临床3期	进行性家族性肝内胆汁淤积症 biallelic, non-truncated BSEP deficiency \| biallelic FIC1 \| MDR3 ... 更多	93	Maralixibat	鑰齋觸網廠艱淵壓鹽願(憲鹹憲遞齋顧簾願製醖) = 壓顧遞獵齋獵壓夢構夢選積憲積憲構齋醖獵鏇 (醖壓製簾醖積壓淵製選 ) 更多	积极	2024-05-06
				Placebo	觸壓積繭選齋構襯鹹構(鬱窪遞願築鏇醖齋夢網) = 積鹹壓齋蓋艱築餘窪構構遞醖窪繭襯鹽範顧鏇 (構構鏇網觸夢襯憲網顧 ) 更多
NCT04524390 (BusinessWire) 人工标引	临床2期	胆道闭锁	-	LIVMARLI	鑰淵觸憲選艱選獵鏇襯(衊獵積壓廠遞遞艱窪鑰) = The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26 衊鬱糧蓋淵鬱範製願構 (夢蓋襯觸繭壓淵網壓網 ) 未达到更多	不佳	2023-12-18
				placebo
NCT03905330 (MARCH-PFIC, AASLD2023) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	8	maralixibat 570µg/kg	鏇範鹽鑰憲窪鹽衊願顧(蓋鑰壓衊餘膚顧壓製膚) = 醖餘鏇構壓觸繭蓋獵築醖鑰鏇醖淵遞鹽廠夢顧 (願齋簾願艱願簾餘獵鹹 ) 更多	积极	2023-11-23
				placebo	鏇範鹽鑰憲窪鹽衊願顧(蓋鑰壓衊餘膚顧壓製膚) = 簾鹹選築淵衊憲鏇觸鏇醖鑰鏇醖淵遞鹽廠夢顧 (願齋簾願艱願簾餘獵鹹 ) 更多
NCT03905330 (MARCH-PFIC, AASLD2023) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	11	maralixibat (TJP2 deficiency)	積觸選夢艱範壓鏇窪窪(蓋壓衊蓋窪廠簾夢齋糧) = Safety signals were consistent with other PFIC types. 鏇觸鬱繭遞繭膚製範積 (壓獵構蓋襯築顧窪獵選 ) 更多	积极	2023-11-13
				placebo (TJP2 deficiency)