Maralixibat Chloride

罕见病制药巨头以2.686亿美元收购罕见肝病单克隆抗体药物 Mirum制药2.686亿美元收购Bluejay Therapeutics，  扩大其罕见疾病领域的全球领导地位。 Mirum制药公司是全球领先的罕见病制药公司，其专为被忽视的罕见病人群带来突破性药物。 Mirum制药，看见罕见，研发治疗罕见，罕见之光。 进入HDV治疗市场，扩大Mirum罕见疾病领域的全球领导地位 Mirum制药宣布完成对Bluejay Therapeutics的收购，新增添了一个晚期肝脏疾病项目到其研发管线，并使Mirum有望进入慢性肝炎病毒HDV治疗领域，这是一种罕见且严重的疾病，在美国尚无批准的疗法。 Mirum Pharmaceuticals收购Bluejay Therapeutics，将Bluejay Therapeutics研发的处于III期临床试验的单克隆抗体Brelovitug纳入其研发管线，此在Ⅱ期研究中对HDV治疗显示出有希望的结果。Brelovitug现处于III期临床试验，  此收购，为Mirum可能成为首个获批治疗HDV慢性肝炎病毒的疗法奠定基础，同时加强其对晚期罕见肝病研发的关注,和有机会进入HDV治疗市场，扩大其罕见疾病领域的全球领导地位。 HDV 是一种严重的乙型肝炎患者合并感染，在部分乙型肝炎病毒感染者中发生的共感染，由于HDV可能迅速发展为肝硬化、肝癌以及与肝脏损害相关的死亡，因此是最严重的病毒性肝炎形式。死亡率较高。 HDV这种疾病影响了估计约23万人，分布于美国和欧洲，估计超过50%的HDV患者将在诊断后10年内死于与肝脏损害相关的病因。 Mirum计划在2027年提交生物制剂许可申请，具体进展如何，则取决于其临床试验结果。 Mirum表示，此收购符合其在罕见病领域的核心优势，并基于其在罕见肝脏疾病领域的深厚专业知识。Mirum的全球发展和商业协同效应能够为HDV患者提供良好服务。 Mirum获得了brelovitug的全球权利此次收购使Mirum获得了brelovitug的全球权利，brelovitug是一种目前处于III期临床开发的单克隆抗体，反映了Mirum通过收购潜在注册的资产在罕见肝病领域扩张的战略。Brelovitug目前正在进行HDV治疗的评估，由于美国和世界大多数国家尚无获批的HDV治疗方法，这一适应症标志着对显著未满足的临床医疗需求的一个极大的令人心动的希望。Mirum表示，收购完成后，重点工作将转向执行层面，才华横溢的Bluejay团队会加入Mirum，同时完成HDV的AZURE III期项目，并为潜在的注册和上市做准备。 Brelovitug研发进展Brelovitug已经获得了FDA的突破性疗法认证和欧洲药品管理局的PRIME认证，表明其潜力基于早期临床数据获得了监管机构的认可。Brelovitug目前正在全球AZURE三期注册项目中进行研究，预计2026年下半年将公布主要数据。AZURE项目包括多项开放标签研究，旨在评估病毒学和生化反应的综合效果，主要目标是支持美国和欧洲的监管申请。在二期临床研究中，Brelovitug在HDV中表现出强烈的抗病毒活性，包括完整的HDV RNA反应、肝酶水平改善以及主要以注射部位红斑为特征的安全性特征。 虽然三期临床数据将决定该药物的获批能力，但其现有数据集仍支持其进入注册研究的进程。预计生物制剂许可申请的提交和上线将于2027年进行，具体取决于试验结果。收购财务交易要点 根据收购主要财务条款，Mirum 将通过现金和 Mirum 普通股的组合收购 Bluejay Therapeutics 的所有流通股份，并可能根据销售额的分阶段里程碑支付额外款项。根据此次收购，Mirum 还完成了此前宣布的针对现有和新医疗保健投资者的私募融资，筹集的总收入超过 2.685 亿美元。这笔资金预计将有助于支持与 brelovitug 相关的持续临床开发和未来的商业活动。 Mirum制药公司专为被忽视的罕见病人群带来突破性药物Mirum制药公司，于2018年在美国加州成立，其已经发展成为一家领先的罕见病制药公司。2026年1月26日宣布成功完成对Bluejay Therapeutics的收购，Bluejay Therapeutics是一家专注于病毒和肝脏疾病的生物技术公司。Mirum作为全球领先的罕见病制药公司，拥有全球认证产品和广泛的试验性药物管线。 Mirum专为罕见病人群，被忽视疾病的人群带来突破性药物而打造治疗罕见病的药物，其结合了深厚的罕见病专业知识和罕见病患者群体的紧密联系。 Mirum公司的商业产品组合包括用于Alagille综合征ALGS和进行性家族性肝内胆汁淤积PFIC的LIVMARLI®maralixibat，用于胆汁酸合成障碍的CHOLBAM®胆酸，以及用于脑叶突性黄肿病CTX的CTEXLI®chenodiol，等。Mirum的临床阶段研发流程包括volixibat，一种IBAT抑制剂，正处于原发性硬化性胆管炎PSC和原发性胆管炎PBC晚期开发阶段。Brelovitug是一种针对慢性肝炎病毒HDV晚期开发的全人单克隆抗体，以及MRM-3379一种PDE4D抑制剂，正在评估用于脆弱X综合征FXS。Mirum的成功源于一支致力于通过战略开发、纪律执行和在罕见病生态系统中明确协作，推动高影响力药物发展的团队。

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a leading rare disease company, today announced the successful completion of its acquisition of Bluejay Therapeutics, a privately held biotechnology company focused on viral and liver diseases. “With the acquisition now complete, our focus shifts to execution – adding the talented Bluejay team to Mirum as we complete the AZURE Phase 3 program for HDV and prepare for potential registrations and launches,” said Chris Peetz, CEO of Mirum. With the completion of the acquisition, Mirum adds worldwide rights to brelovitug, a late-stage, fully human monoclonal antibody for chronic hepatitis delta virus (HDV), a rare and severe liver disease with no FDA-approved therapies in the United States. Brelovitug has received Breakthrough Therapy designation from the FDA and PRIME designation from the European Medicines Agency and is currently being evaluated in the global AZURE Phase 3 registrational program, with topline data expected in the second half of 2026. “With the acquisition now complete, our focus shifts to execution – adding the talented Bluejay team to Mirum as we complete the AZURE Phase 3 program and prepare for potential registrations and launches,” said Chris Peetz, Chief Executive Officer of Mirum Pharmaceuticals. “This program fits squarely within our core strengths in rare disease and builds on our deep expertise in rare liver conditions. We believe Mirum’s global development and commercial synergies position us well to deliver for patients living with HDV.” Under the terms of the transaction, Mirum acquired all outstanding shares of Bluejay Therapeutics for a combination of cash and Mirum common stock, with potential additional tiered sales-based milestone payments. Concurrently with the acquisition, Mirum completed the previously announced private placement financings, including investments from existing and new healthcare investors, resulting in aggregate gross proceeds of approximately $268.5 million. The proceeds are intended to support clinical development and commercial activities following the acquisition of brelovitug in HDV. Mirum is evaluating strategic options for Bluejay’s additional investigational programs. About Brelovitug Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the chronic hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of HDV and PRIME and Orphan designations from the European Medicines Agency. In Phase 2 studies, brelovitug demonstrated strong antiviral activity in HDV, achieving a 100% HDV RNA response, along with improvements in liver enzyme levels and a favorable safety profile, with the most common adverse event being injection-site erythema. Mirum owns worldwide rights to brelovitug. About the AZURE Clinical Program The AZURE program is a global, registrational Phase 3 clinical development program evaluating brelovitug for the treatment of chronic hepatitis delta virus (HDV). The program includes multiple open-label studies designed to assess the primary endpoint of combined virologic and biochemical response. Together, the studies are intended to support regulatory filings in the United States and Europe. Topline data from the program are expected in the second half of 2026 with a potential BLA submission and launch in 2027. About Chronic Hepatitis Delta Virus (HDV) HDV, a coinfection that occurs in some people infected with the hepatitis B virus, is the most severe form of viral hepatitis due to the potential for rapid progression to liver cirrhosis, liver cancer and liver-related death. HDV affects approximately 230,000 people in the United States and Europe. It is estimated that more than 50% of individuals with HDV will die of liver-related causes within 10 years of diagnosis. There are currently no approved treatments for HDV in the United States and most countries worldwide. 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 rare disease expertise with strong connections to patient communities. 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), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), 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 www.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 implementation of post-acquisition initiatives, including integrating the Bluejay team to Mirum; the anticipated use of proceeds from the private placements; the prospective benefits of the acquisition, including expectations that it will strengthen Mirum’s rare disease portfolio and will be a strong, strategic fit for Mirum; the expectation that brelovitug will be synergistic with and advance Mirum’s leadership in rare disease and benefit from Mirum’s existing expertise, programs and relationships; expectations regarding brelovitug’s ongoing development, including the potential completion of and successful results from the pivotal Phase 3 studies for HDV, the potential benefits of brelovitug, the anticipated timing for data from the Phase 3 studies, the anticipated costs of developing brelovitug, the estimated prevalence of HDV in the United States and Europe and its effects on patients, and the potential regulatory BLA submission and launch of brelovitug in 2027; and plans for additional brelovitug or other clinical programs. 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,” “intended,” “believe,” 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 associated with the development of acquired product candidates, including the failure of any expected synergies to be realized; risk and uncertainties arising from the integration of an acquired company, its employees and its assets with Mirum’s business; risks associated with evaluating companies and assets for acquisition, including that the perceived benefits of the acquisition are not realized; risks and uncertainties with the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies and the potential that estimated prevalences are materially inaccurate; 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 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.