A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.

开始日期2021-09-22

申办/合作机构

Mirum Pharmaceuticals, Inc.

NCT04718961

/ Terminated临床2期

A Phase 2a/2b Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in Adult Women With Intrahepatic Cholestasis of Pregnancy and Elevated Serum Bile Acid Concentrations (OHANA).

Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat. Part 2 is a double-blind, placebo controlled, study designed to evaluate the safety and efficacy of a selected volixibat dose.

开始日期2021-01-04

申办/合作机构

Mirum Pharmaceuticals, Inc.

NCT04663308

/ Active, not recruiting临床2期

A Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Sclerosing Cholangitis

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

开始日期2020-12-18

申办/合作机构

Mirum Pharmaceuticals, Inc.

100 项与 Volixibat 相关的临床结果

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100 项与 Volixibat 相关的转化医学

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100 项与 Volixibat 相关的专利（医药）

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项与 Volixibat 相关的文献（医药）

2022-05-01·ALIMENTARY PHARMACOLOGY & THERAPEUTICS

Meta‐analysis: analysis of mechanistic pathways in the treatment of non‐alcoholic steatohepatitis. Evidence from a Bayesian network meta‐analysis

Article

作者: Nicholas Chew ; Eunice Tan ; Jie Ning Yong ; Xin‐Hui Pan ; Mazen Noureddin ; Phoebe Tay ; Mark D. Muthiah ; Daniel Q. Huang ; Arun J. Sanyal ; Cheng Han Ng ; Mohammad Shadab Siddiqui ; Darren Jun Hao Tan ; Jieling Xiao ; Rohit Loomba ; Wen Hui Lim ; Yip Han Chin ; Grace Lim ; Jeffery Wei Heng Koh ; Nicholas Syn

Summary:

Background and Aims:

Non‐alcoholic steatohepatitis (NASH) is the most common cause of liver disease. However, there is lack of comparison of efficacy between different NASH drug classes. We conducted a network meta‐analysis evaluating drug classes through comparing histological outcomes and targets of drugs.

Approach and Results:

Medline, EMBASE and CENTRAL were searched for randomised controlled trials evaluating NASH drugs in biopsy‐proven NASH patients. Primary outcomes included NASH resolution without worsening of fibrosis, at least 2‐point reduction in Non‐alcoholic fatty liver disease Activity Score (NAS) without worsening of fibrosis and at least 1‐point reduction in fibrosis. Treatments were classified into inflammation, energy, bile acid and fibrosis modulators. The analysis was conducted with Bayesian network model and surface under the cumulative ranking curve (SUCRA) analysis. Among 49 included trials, treatments modulating energy (Risk ratio (RR): 1.92, Credible intervals (Crl): 1.59‐2.34) were most likely to achieve NASH resolution followed by treatments modulating fibrosis (RR 1.66, Crl: 0.65‐4.50), bile acids (RR: 1.37, Crl: 0.99‐1.92) and inflammation (RR: 1.00, Crl: 0.75‐1.33). Energy and bile acids modulation were effective in at least 2‐point NAS reduction without worsening of fibrosis (RR: 1.52, Crl 1.30‐1.77; RR: 1.69, Crl 1.41‐2.03) and at least 1‐point reduction in fibrosis (RR: 1.26, Crl:1.05‐1.49; RR: 1.54, Crl: 1.20‐1.97).

Conclusions:

This network analysis demonstrates the relative superiority of drugs modulating energy pathways and bile acids in NASH treatment. This guides the development and selection of drugs for combination therapies.

2020-08-01·Journal of hepatology1区 · 医学

Volixibat in adults with non-alcoholic steatohepatitis: 24-week interim analysis from a randomized, phase II study

1区 · 医学

Article

作者: Aithal, Guruprasad P ; Sarles, Harry ; Sheikh, Aasim ; Herring, Robert ; Sheikh, Muhammad Y ; Freilich, Bradley ; Hassanein, Tarek ; Newsome, Philip N ; Mantry, Parvez ; Lee, Hak-Myung ; Palmer, Melissa ; Kayali, Zeid

BACKGROUND & AIMS:

Volixibat is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT) that has been hypothesized to improve non-alcoholic steatohepatitis (NASH) by blocking bile acid reuptake and stimulating hepatic bile acid production. We studied the safety, tolerability and efficacy of volixibat in patients with NASH.

METHODS:

In this double-blind, phase II dose-finding study, adults with ≥5% steatosis and NASH without cirrhosis (N = 197) were randomized to receive volixibat (5, 10 or 20 mg) or placebo once daily for 48 weeks. The endpoints of a predefined interim analysis (n = 80), at week 24, were: ≥5% reduction in MRI-proton density fat fraction and ≥20% reduction in serum alanine aminotransferase levels. The primary endpoint was a ≥2-point reduction in non-alcoholic fatty liver disease activity score without worsening fibrosis at week 48.

RESULTS:

Volixibat did not meet either interim endpoint; the study was terminated owing to lack of efficacy. In participants receiving any volixibat dose, mean serum 7-alpha-hydroxy-4-cholesten-3-one (C4; a biomarker of bile acid synthesis) increased from baseline to week 24 (+38.5 ng/ml [SD 53.18]), with concomitant decreases in serum total cholesterol (-14.5 mg/dl [SD 28.32]) and low-density lipoprotein cholesterol (-16.1 mg/dl [SD 25.31]). These changes were generally dose-dependent. On histological analysis, a greater proportion of participants receiving placebo (38.5%, n = 5/13) than volixibat (30.0%, n = 9/30) met the primary endpoint. Treatment-emergent adverse events (TEAEs) were mainly mild or moderate. No serious TEAEs were related to volixibat. Diarrhoea was the most common TEAE overall and the most common TEAE leading to discontinuation.

CONCLUSIONS:

Increased serum C4 and decreased serum cholesterol levels provide evidence of target engagement. However, inhibition of ASBT by volixibat did not elicit a liver-related therapeutic benefit in adults with NASH.

LAY SUMMARY:

A medicine called volixibat has previously been shown to reduce cholesterol levels in the blood. This study investigated whether volixibat could reduce the amount of fat in the liver and reduce liver injury in adults with an advanced form of non-alcoholic fatty liver disease. Volixibat did not reduce the amount of fat in the liver, nor did it have any other beneficial effect on liver injury. Participants in the study generally tolerated the side effects of volixibat and, as in previous studies, the main side effect was diarrhoea. These results show that volixibat is not an effective treatment for people with fatty liver disease.

CLINICAL TRIAL IDENTIFIER:

NCT02787304.

2020-05-01·Biomeditsinskaia khimiia

The inhibitors of the apical sodium-dependent bile acid transporter (ASBT) as promising drugs

Review

作者: Saveleva, E.E. ; Salmina, A.B. ; Tyutrina, E.S. ; Nakanishi, T. ; Tamai, I.

Inhibition of the apical sodium-dependent bile acid transporter (ASBT, also known as IBAT — ileal bile acid transporter, SLC10A2) leads to disruption of the enterohepatic circulation of bile acids and their excretion with fecal masses. This is accompanied by cholesterol utilization for synthesis of new bile acids. ASBT inhibitors are promising drugs for the treatment of such diseases as non-alcoholic fatty liver disease, non-alcoholic steatohepatitis, type 2 diabetes mellitus, necrotic enterocolitis, chronic constipation, atherosclerosis. To date the most known chemically synthesized inhibitors are: A3309, SHP626, A4250, 264W94, GSK2330672, SC-435. All of them are at different stages of clinical trials, which confirm the high efficacy and good tolerance of these inhibitors. Current trends in this field also include directed chemical synthesis of ASBT inhibitors, as well as their search among substances of plant origin.

175

项与 Volixibat 相关的新闻（医药）

2025-12-30

·GlobeNewswire-Health Care

XOMA Royalty and Takeda Execute Strategic Royalty Sharing Transaction and Amend Existing Agreement for Mezagitamab

– Takeda regains a majority of XOMA Royalty’s royalty interest in mezagitamab (TAK-079) –– XOMA Royalty will be entitled to payments based on a share of milestones and royalties associated with nine development-stage assets held within Takeda’s externalized assets portfolio – EMERYVILLE, Calif., Dec. 30, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has amended its collaboration, originally established in 2006, with Takeda through a strategic royalty share transaction. Takeda’s royalty and milestone payment obligations to XOMA Royalty related to mezagitamab will be reduced, and XOMA Royalty will receive payments based on low to mid-single-digit royalties and milestones across a basket of nine development-stage assets that are held within Takeda’s externalized assets portfolio. “We continue to find ways to expand and diversify our royalty and milestone portfolio through creative transactions that are beneficial to both parties,” stated Brad Sitko, Chief Investment Officer of XOMA Royalty. “Takeda and XOMA Royalty have a long history of collaboration. By amending our collaboration, we are able to return a portion of our mezagitamab economics to Takeda, while also expanding and diversifying XOMA Royalty’s portfolio across several interesting early- and late-stage programs.” MezagitamabPrior to amending the collaboration, XOMA Royalty held a mid-single digit royalty and $16.25 million in potential milestones associated with mezagitamab. Going forward, XOMA Royalty will retain a low single-digit royalty entitlement on mezagitamab and up to $13.0 million in milestones. Development-Stage Assets from Takeda’s Externalized Assets Portfolio Osavampator Neurocrine Biosciences is developing osavampator, a potential first-in-class, investigational alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) positive allosteric modulator (PAM) for patients who have inadequate response to treatment for major depressive disorder (MDD). Volixibat Mirum Pharmaceuticals is developing volixibat, a minimally absorbed, orally administered investigational therapy designed to selectively inhibit ileal bile acid transporter (IBAT), for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). OHB-607 and five early-stage Oak Hill Bio assets Oak Hill Bio and their partner are developing OHB-607, a recombinant human IGF-1/IGFBP-3 for the prevention of bronchopulmonary dysplasia in premature infants. Additional Oak Hill Bio assets that have the potential to generate royalties address other high unmet need or rare disease areas. REC-4881 Recursion Pharmaceuticals is developing REC-4881, an investigational MEK1/2 inhibitor for familial adenomatous polyposis, a rare tumor predisposition syndrome affecting approximately 50,000 people in the U.S., France, Germany, Italy, Spain, and the UK1. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the sellers receive non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. XOMA Royalty has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about XOMA Royalty and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn. XOMA Royalty Forward-Looking Statements/Explanatory NotesCertain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the timing and amount of potential commercial and milestone payments to XOMA Royalty and other developments related to mezagitamab, osavampator, volixibat, OHB-607 and the Oak Hill Bio assets, and REC-4881. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will”, “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-Q and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. XOMA Royalty Investor Contact:XOMA Royalty Media Contact:Juliane SnowdenKathy VincentXOMA Royalty CorporationKV Consulting & Management+1-646-438-9754kathy@kathyvincent.com juliane.snowden@xoma.com 1 https://ir.recursion.com/news-releases/news-release-details/preliminary-phase-1b2-data-rec-4881-familial-adenomatous

引进/卖出

2025-12-25

·雪球

Mirum制药：罕见病赛道的“三级火箭”，能否在18个月内点燃百亿市值引擎？

$Mirum(MIRM)$在投资界追逐减肥药与抗癌神药的喧嚣中，Mirum选择了一条截然不同的道路——将药物研发的“矛尖”刺向那些患者仅有数千人的极致罕见病，却在七年内构建起了一套足以挑战百亿市值的商业模型。2018年，当MirumPharmaceuticals在美国加州悄然成立时，几乎没有人会预料到，这家专注于“罕见病中的罕见病”的公司，将在七年后成长为年销售额超5亿美元、手握近4亿美元现金的细分领域巨头。在生物制药这个以“十亿级患者市场”为梦想的行业，Mirum反其道而行之。它的成功，不是依靠一款面向亿万大众的“重磅炸弹”，而是通过一套精密如瑞士钟表般的“三级火箭”增长模型，在罕见病这条狭窄却深邃的赛道中，实现了令人惊叹的商业跃迁。从挽救罕见肝病儿童生命的“奇迹药物”，到瞄准数十万成人患者的“明日之星”，再到通过一场豪赌式并购切入的全新战场，Mirum的故事远不止于医学突破，更是一部关于战略聚焦、风险杠杆与时机博弈的商业教科书。而此刻，这家公司正站在其发展史上最具决定性意义的转折点上——未来18个月内，4项关键的临床试验数据将陆续揭晓。这些结果，将直接决定其“三级火箭”能否成功点燃下一级引擎，进入一个价值呈数量级增长的崭新轨道。01罕见病的“逆袭”：一家小而美巨头的诞生逻辑要理解Mirum的商业奇迹，首先要破除一个迷思：罕见病药物真的是“商业价值有限”的选择吗？传统观点认为，患者群体小意味着市场规模天花板低。然而，以Mirum为代表的现代罕见病药企，却发现了其中隐藏的结构性优势：1.极高的定价权与支付意愿：当一种疾病无药可治，而新药能显著改善生存或生活质量时，支付体系（如保险公司、政府医保）往往愿意承担极高的费用。2.加速的审批通道：FDA（美国食药监局）等机构为罕见病药物设有“孤儿药资格认定”，提供快速审批、税费减免和长达7年的市场独占期，极大缩短了商业化进程。3.极低的营销成本与极高的医患粘性：目标医生群体高度集中，患者社群紧密，使得市场教育效率极高，客户忠诚度远超普通药物。4.深厚的科学护城河：针对罕见病的药物机制往往独特，且成功后可横向拓展至患者更多的相关常见病，形成“从罕见病到常见病”的杠杆效应。Mirum正是这一新逻辑的完美实践者。它没有在肿瘤、自免等“红海”中与巨头血战，而是选择了胆汁淤积性肝病这一存在巨大未满足需求的垂直领域。这里的患者虽少，但痛苦极深——尤其是那种“从血液里透出来的”、令人绝望的全身性瘙痒，足以摧毁一个家庭。公司自成立之初就确立了清晰的战略：通过“自主研发+战略收购”双轮驱动，快速构建一个覆盖不同阶段、不同患者群体的产品矩阵，最终成为一个罕见肝病领域的平台型领导者。02解码“三级火箭”：Mirum增长模型的深度剖析Mirum的成功，并非源于运气，而是其精心设计的“三级火箭”商业模型系统运作的结果。每一级火箭都承担着特定的战略使命，并为下一级的点燃提供至关重要的动力和稳定性。第一级火箭（基石级）：现金流“压舱石”与已验证的科学平台使命：产生稳定、可预测的现金流，证明核心科学平台（IBAT抑制）的有效性，建立商业化和监管信誉。核心资产：Livmarli：公司的“心脏”与“王牌”。作为口服IBAT抑制剂，它通过抑制肠道对胆汁酸的重吸收，直接排出多余胆汁酸，从而缓解瘙痒和肝损伤。它已获批用于治疗两种毁灭性的儿童罕见肝病：Alagille综合征和进行性家族性肝内胆汁淤积症。数据的力量：在Alagille综合征（ALGS）患者中，Livmarli治疗使84%的患者瘙痒得到临床意义的改善；在进行性家族性肝内胆汁淤积症（PFIC）患者中，62%的患者达到了几乎无痒的状态。更惊人的是，长期数据显示，它对延长患儿无需肝移植的生存期有显著贡献。商业成果：凭借无可替代的疗效，Livmarli上市后迅速放量，成为年销售数亿美元的“孤儿药重磅炸弹”。其专利保护更延长至2040年以后，构成了长期、深厚的竞争护城河。胆汁酸产品组合（Cholbam/Chenodal）：这是Mirum通过收购获得的“隐形冠军”。它们是胆汁酸替代疗法，用于治疗因基因缺陷完全无法自身合成胆汁酸的患者。这类疾病患者更少，但药物是“生存必需品”，因此业务呈现高利润率、高稳定性的特点，与Livmarli共享肝病专科营销网络，产生强大的协同效应。小结：第一级火箭的成功，为Mirum提供了赖以生存的“血液”（现金流），赢得了FDA的信任（快速审批经验），更重要的是，彻底验证了“靶向胆汁酸通路”这一核心科学策略在真实世界中的巨大价值。这为第二级火箭的发射，铺平了道路。第二级火箭（增长级）：市场规模的数量级跨越使命：将已验证的科学机制（IBAT抑制），应用于患者基数扩大数十至上百倍的疾病领域，实现公司营收和市值的跃迁式增长。核心武器：Volixibat可以将其视为Livmarli的“成人优化版”。同为IBAT抑制剂，但剂型经过改良，目标是攻克两大成人胆汁淤积性肝病：1.原发性硬化性胆管炎（PSC）：这是一种目前在美国完全无药可治的疾病，病因不明，最终多导致肝衰竭或癌症。约65%的患者遭受着中重度瘙痒的折磨。Volixibat的III期注册临床试验（VISTAS研究）的顶线数据，将于2026年第二季度公布。这是其成人征程的第一场、也是最重要的一场大考。CAMEO研究旨在验证IBAT抑制剂Volixibat对PSC患者的治疗潜力。数据显示，在基线到第14周治疗后，患者的瘙痒症状（Pruritus）平均下降70%，血清胆汁酸水平（BileAcids）下降40%。瘙痒评分从基线约40分降至约12分，统计学显著（p=0.0078）。胆汁酸水平从约70μmol/L降至约40μmol/L，虽未达显著性差异（p=0.078），但趋势明显。2.原发性胆汁性胆管炎（PBC）：这是最常见的成人自身免疫性肝病之一，全球患者众多。现有疗法仅对部分患者有效，且无法解决瘙痒这一核心症状。Volixibat的II期研究（VANTAGE）数据惊艳，已获得FDA突破性疗法认定，这几乎是为其未来的快速获批铺上了红毯。其III期研究正快速推进。II期研究结果显示在28周内，经过安慰剂校正后，VLX20mg组瘙痒评分下降2.4分（P=0.0039），VLX80mg组下降2.6分（P=0.0010），差异具有统计学意义。市场预期：PSC和PBC的患者总数，是ALGS和PFIC的百倍以上。公司内部预估，Volixibat在这两个适应症上的峰值销售潜力合计超过100亿美元。一旦成功，Mirum将从一个“超罕见病公司”真正蜕变为“肝病领域的重要玩家”。第三级火箭（想象级）：突破边界的战略豪赌使命：突破现有疾病领域的边界，通过前沿研发和战略并购，开辟全新的增长曲线，将公司提升至平台型生物制药企业的维度。战略并购：Brelovitug2025年底，Mirum宣布以首付6.2亿美元（现金+股票）+最高2亿美元里程碑付款的价格，收购BluejayTherapeutics。这笔交易的核心，是一款名为Brelovitug的抗丁型肝炎病毒单克隆抗体。为何这是一场“豪赌”？1.绝对的蓝海市场：丁肝由乙肝病毒共感染引起，是最严重的病毒性肝炎，进展为肝硬化、肝癌的速度极快。然而，美国市场至今没有批准任何治疗药物。这是一个完全的、空白的、亟待满足的百亿级市场。2.颠覆性的临床数据：在II期临床试验中，Brelovitug展示了100%的病毒学应答率，如此出色的数据使其同时获得了FDA的“突破性疗法”和欧盟的“PRIME”优先药物资格。3.决定性的时间窗口：支持其在美国上市申请的两项关键III期研究，顶线数据都将在2026年下半年读出。若结果积极，2027年即可提交上市申请并可能获批。这笔收购，让Mirum从“遗传性肝病”领域，一举杀入了“病毒性肝病”这个更大的战场。前沿探索：MRM-3379与此同时，Mirum并未停止内部创新的脚步。其自主研发的MRM-3379，是一款靶向脆性X综合征（一种导致智力障碍和自闭症谱系障碍的常见遗传病）的PDE4D抑制剂。该药物已进入II期临床研究。这标志着Mirum的战略触角，首次伸向了罕见神经疾病领域，展现了其将科学能力平台化的野心。03财务解码：从“烧钱”到“造血”的健康跃迁对于生物科技公司，财务健康与否是生存与发展的生命线。Mirum的财务表现，清晰地印证了其“三级火箭”模型正在有效运转。爆炸性的营收增长：公司预计2025年净产品销售额将达到5.0-5.1亿美元，这意味着在短短四年内，其营收规模增长了近7倍。这主要得益于Livmarli在核心适应症上的持续渗透，以及胆汁酸产品组合的稳定贡献。关键的财务拐点：公司明确指引，预计在2025年实现经营性现金流转正。这是一个里程碑式的信号，标志着Mirum已经跨越了生物科技公司最危险的、持续“烧钱”的阶段，进入了具备自我造血能力的可持续发展新周期。充足的“军火库”：截至2025年第三季度，公司资产负债表上躺着3.78亿美元的现金、现金等价物及投资。这笔雄厚的资金，足以覆盖其包括推进Volixibat关键临床试验、完成对Bluejay的收购以及运营开支在内的所有战略需求，无需在资本市场低迷时进行可能稀释股权的融资。未来的盈利路径：随着Livmarli销售规模扩大带来的规模效应，以及高毛利的胆汁酸产品占比提升，公司的毛利率和运营效率将持续优化。当第二级火箭（Volixibat）成功商业化后，其利润水平有望迎来又一次飞跃。0418个月，四场战役：决定命运的“临床大考”所有宏大的战略和精妙的模型，最终都需要在严谨、残酷的临床试验中得到验证。对于Mirum而言，未来18个月就像一场决定性的“期末考试”，四门主科的成绩将决定其未来十年的发展轨迹。1.2026年第二季度：Volixibat的PSC终局之战（VISTAS研究）这是最不容有失的一战。PSC在美国无药可治，临床需求急切。阳性数据将直接支撑其新药申请，并确立其在成人胆汁淤积领域的领导地位。若数据不佳，其向成人市场扩张的叙事将受到严重质疑。2.2026年下半年：Brelovitug的丁肝双星试验（AZURE-1&AZURE-4）这是检验8亿美元并购是否物有所值的时刻。两项III期研究需同时取得积极结果，才能支撑其在美国的上市申请。考虑到II期数据的卓越表现，市场期望极高。成功则打开百亿市场大门，失败则意味着巨额收购款可能面临减值风险。3.2027年上半年：Volixibat的PBC巩固之战（VANTAGE研究）与Livmarli的边界拓展（EXPAND研究）这将是扩大战果和夯实基础的战役。PBC数据将进一步证明Volixibat在更广大患者中的价值。而Livmarli在“其他超罕见胆汁淤积症”中的数据，则旨在挖掘其现有王牌药物的剩余潜力，延长其生命周期。

2025-12-22

·BioSpace

Mirum Pharmaceuticals Announces Additional Private Placement Financing With TCGX

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that it has entered into a subscription agreement with entities associated with TCGX, an institutional investor, for a private placement of 1,000,000 shares of its common stock (the “Common Stock”). The Common Stock issued to the private placement investors at closing will be priced at $68.48 per share for approximately $68.5 million in gross proceeds. The proceeds from the private placement, together with Mirum’s $200 million private placement announced earlier this month, are intended to fund clinical development and commercial activities following the previously announced proposed acquisition of Bluejay Therapeutics (“Bluejay”), a privately held biotechnology company focused on viral and liver diseases. Both private placements are expected to close concurrently with the proposed acquisition with Bluejay, anticipated in the first quarter of 2026, subject to regulatory approval and other customary closing conditions. The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended (the “Securities Act”), and may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. Concurrently with the execution of the subscription agreement, Mirum and the investors entered into a registration rights agreement, which agreement will become effective upon the closing of the private placement, pursuant to which Mirum has agreed to registration statement with the Securities and Exchange Commission registering the resale of the shares of Common Stock sold in the private placement. This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep expertise with strong connections to the rare disease community. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at 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 closing of the proposed acquisition, including the anticipated receipt of regulatory approval and the satisfaction or waiver, as applicable, of customary closing conditions; the anticipated occurrence, manner and timing of the closing of the private placement; the gross proceeds expected to result from the private placement; the anticipated use of proceeds from the private placement; and statements about Mirum’s future financial performance. 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 “anticipate,” “expected,” “will,” “could,” “would,” “potential,” “continue,” “plans,” 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 inherent with the acquisition of companies, including the potential risks that regulatory approval will not be timely received (if at all) and the satisfaction or waiver, as applicable, of closing conditions; the risks and uncertainties inherent with private placements, including the successful and timely completion of the private placement; the 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 . 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. Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

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100 项与 Volixibat 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	Volixibat	-	DB13914

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
原发性胆汁性胆管炎	临床2期	美国	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	中国	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	日本	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	阿根廷	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	比利时	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	巴西	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	加拿大	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	法国	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	德国	Mirum Pharmaceuticals, Inc.	2021-09-22
原发性胆汁性胆管炎	临床2期	以色列	Mirum Pharmaceuticals, Inc.	2021-09-22

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05050136 (VANTAGE, Company_Website) 人工标引	临床2期	胆汁淤积性瘙痒 \| 原发性胆汁性胆管炎	31	Volixibat	醖選壓鏇顧遞構膚構淵(襯鑰繭願鏇衊餘蓋艱淵) = 鬱顧憲蓋遞憲壓鑰醖積鏇憲製顧衊鏇製觸製觸 (糧網艱網齋願鹹鹹獵築 ) 更多	积极	2025-05-09
				Volixibat 20 mg	醖選壓鏇顧遞構膚構淵(襯鑰繭願鏇衊餘蓋艱淵) = 繭蓋製齋夢鹹獵選製觸鏇憲製顧衊鏇製觸製觸 (糧網艱網齋願鹹鹹獵築 )
NCT04718961 (OHANA, CTgov)	临床2期	妊娠期肝内胆汁淤积	4	Placebo	鏇襯積顧繭繭範鬱淵簾(構憲鬱構製遞壓鹹夢蓋) = 鹽膚遞網糧醖製願醖鏇願顧夢憲遞鏇範膚憲廠 (製積鹽簾蓋繭觸積膚餘, 餘醖淵膚廠選餘鏇構構 ~ 選餘糧築蓋夢膚鑰構鹽) 更多	-	2024-08-06
NCT05050136 (VANTAGE, Biospace) 人工标引	临床2期	原发性胆汁性胆管炎	30	volixibat	壓夢簾衊夢願築糧廠憲(構觸鹹製顧鏇鏇構鏇衊) = 衊壓窪膚網鹽築膚顧願憲鑰醖簾獵繭觸窪選醖 (鏇艱齋廠襯憲艱獵構簾, 0.414) 更多	积极	2024-06-17
				volixibat (20mg)	壓夢簾衊夢願築糧廠憲(構觸鹹製顧鏇鏇構鏇衊) = 憲壓糧鏇簾艱網選糧築憲鑰醖簾獵繭觸窪選醖 (鏇艱齋廠襯憲艱獵構簾, 0.609)
NCT04718961 (OHANA, AASLD2023) 人工标引	临床2期	妊娠期肝内胆汁淤积	4	Volixibat	醖鏇積餘鑰餘選遞艱醖(築鏇衊獵襯糧鑰膚願齋) = 糧範獵積鹽選鏇製鹹鏇鏇醖淵簾廠構選鹹蓋淵 (構網窪繭蓋鬱繭夢糧齋 ) 更多	积极	2023-11-13
NCT02787304 (Pubmed \| J Hepatol) 人工标引	临床2期	代谢功能障碍相关脂肪性肝炎	197	volixibat	築觸蓋簾蓋壓淵衊製鏇(艱繭鹽顧蓋製鑰鹹艱製) = 齋襯憲鹽憲襯鑰製顧醖鏇餘獵蓋鏇鏇糧壓窪膚 (遞顧廠觸觸齋鏇鬱淵壓 ) 更多	不佳	2020-08-01
				Placebo	築觸蓋簾蓋壓淵衊製鏇(艱繭鹽顧蓋製鑰鹹艱製) = 顧獵淵鏇選製獵簾餘構鏇餘獵蓋鏇鏇糧壓窪膚 (遞顧廠觸觸齋鏇鬱淵壓 )
NCT02787304 (CTgov)	临床2期	代谢功能障碍相关脂肪性肝炎	197	SHP626 (SHP626 5 Milligram (mg))	壓鏇構壓簾醖構願鹹廠 = 鬱壓窪觸顧選製鹹築願製鹽繭顧壓獵艱餘築夢 (醖齋鑰製衊網積願願壓, 夢淵艱顧顧壓糧選獵壓 ~ 鏇膚艱遞築膚範築廠蓋) 更多	-	2019-11-25
				SHP626 (SHP626 10 Milligram (mg))	壓鏇構壓簾醖構願鹹廠 = 鏇壓構壓鏇夢製鏇醖艱製鹽繭顧壓獵艱餘築夢 (醖齋鑰製衊網積願願壓, 鏇遞選願膚廠築鹽簾築 ~ 範鹽製憲衊壓衊觸醖獵) 更多
NCT02287779 (Pubmed \| BMC Pharmacol Toxicol) 人工标引	临床1期	代谢功能障碍相关脂肪性肝炎	84	volixibat	膚糧憲餘憲鬱鹹鑰鏇繭(繭願範築鬱構憲築鏇餘) = no serious adverse events 鑰憲範構淵顧積鹹製鬱 (壓觸築鹽鬱築獵鑰構憲 )	积极	2018-03-16
				Placebo
NCT02571192 (Pubmed \| Eur J Drug Metab Pharmacokinet) 人工标引	临床1期	-	8	[ 14 C]-Volixibat	築壓範餘鬱繭憲壓網範(艱憲襯夢構襯衊積齋構) = No radioactivity was observed in plasma or whole blood. The percentage (mean ± standard deviation) of total radioactivity in urine was 0.01 ± 0.007%. The vast majority (92.3 ± 5.25%) of volixibat was recovered in feces (69.2 ± 33.1% within 24 h). 鏇選壓窪繭製廠獵襯餘 (憲糧顧齋選餘鑰醖網顧 ) 更多	积极	2018-02-01
NCT02287779 (CTgov)	临床1期	代谢功能障碍相关脂肪性肝炎 \| 肥胖	84	Placebo	窪壓繭膚鑰鹽築糧鏇觸 = 獵築築夢糧醖獵憲憲艱觸淵窪襯淵餘遞選憲構 (鬱淵蓋遞獵廠壓憲夢衊, 窪糧鬱鑰鑰淵觸鏇壓齋 ~ 構選積餘獵膚鏇簾繭構) 更多	-	2016-12-07