Maralixibat Chloride

北海康成制药有限公司（以下简称“北海康成”，股票代码“1228.HK”），是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。 聚势谋远启新程，领航破浪向未来。10月24日，由中国医药创新促进会（药促会）和香港交易所联合主办的的第十一届医药创新与投资大会在南京扬子江国际会议中心圆满举行。本次大会以“聚势·启新·领航”为主题，汇聚了国内外投资机构、创新药企及顶尖研究院所等百余家机构，两千余名行业大咖共襄盛举。北海康成出席大会并在“上市公司路演专场”发表主题演讲。 大会由中国医药创新促进会驻会副会长冯岚主持，中国工程院院士蒋建东，中国药科大学校长郝海平，中国医药创新促进会首席专家宋瑞霖，中国民生银行副行长李稳狮，以及来自国内外头部药企的核心高管悉数出席。与会大咖围绕原创新药研发、AI制药、BD全球价值兑现、多元化出海路径等核心议题展开了深度探讨。 当前，中国生物医药产业正迎来前所未有的发展机遇，生物医药首次被列入国家新兴支柱产业，“十五五”规划为产业锚定了高质量发展新坐标。在大会主旨报告及系列讨论中，与会专家与企业家达成高度共识：中国创新药正从跟随模仿迈向源头创新与全球价值兑现的2.0阶段。面对全球化竞争，企业需围绕临床未满足需求进行差异化创新，灵活运用License-out、NewCo等多元模式融入全球产业链。同时，AI技术正加速重构药物研发范式，在靶点发现与分子设计等环节显著提效，叠加港股18A等资本通道与“耐心资本”的赋能，中国医药创新正蓄势待发，有望实现从并跑到领跑的跨越。 北海康成首席运营官沈菊平先生发表主题演讲 作为港股18A创新药企代表，北海康成受邀深度参与了此次盛会。北海康成首席运营官沈菊平先生发表演讲：“北海康成始终坚守‘难而正确’的罕见病赛道。尽管罕见病领域面临研发投入大、早期收益小的现实挑战，但这却是一个全球增量可观、极具爆发潜力的蓝海市场。作为全球医药价值链的关键贡献者与中外创新桥梁，公司依托已上市产品的全链路商业化能力与基因治疗等前沿管线布局，将积极拥抱AI技术与‘耐心资本’，加速源头创新与全球战略合作，在满足迫切临床需求的同时为股东创造长期稳健回报，展现中国创新药企在全球双循环中的战略定力与自信。” 站在“十五五”规划开局的历史节点，中国创新药正以更加自信、开放的姿态破浪领航。在这场产业变革的浪潮中，北海康成将紧跟国家战略导向，未来继续以原始创新为引擎，以全球视野谋布局，与业内同仁携手，共同谱写中国医药创新高质量发展的新篇章。 向上滑动阅览 北海康成成立于2012年，并于2021年在港交所主板上市。作为中国领先的专注于罕见疾病的全球化生物制药公司，公司致力于创新疗法的研究、开发与商业化。截至2026年6月30日，针对市场潜力庞大的多类常见罕见病适应症，公司已搭建覆盖多类药物资产的完备产品线，包含3款已上市产品（海芮思®、迈芮倍®、戈芮宁®）及1项处于临床后期阶段的候选药物，技术类型全面覆盖生物制剂、小分子药物及基因疗法。在罕见病领域，公司现布局八款生物制剂及小分子产品，对应多类适应症，涵盖黏多糖贮积症II型（亨特综合症）及其他溶酶体贮积症（LSD）、补体介导疾病、A型血友病、代谢紊乱疾病，以及阿拉杰里综合症（ALGS）、进行性家族性肝内胆汁淤积症（PFIC）等罕见胆汁淤积性肝病。 公司积极投入新一代基因治疗技术的研发，针对治疗方案有限的罕见遗传病开发具备潜在治愈性的创新疗法，并持续评估内外部技术与产品线机会，推动管线组合向first-in-class及best-in-class方向转型。为支撑上述产品管线的研发与商业化落地，公司构建了覆盖全球的合作伙伴网络，包括但不限于药明康德、药明生物、百洋医药、GC Pharma、Mirum、Privus和Scriptr Global 等。 如需了解更多关于北海康成制药有限公司的信息，请访问：http://www.canbridgepharma.com/ 前瞻性声明：本文中作出的前瞻性陈述仅与本文作出陈述之日的事件或信息有关。除非法律要求，否则我们不承担公开更新或修改任何前瞻性陈述的义务，无论是由于新信息、未来事件或其他原因，在做出陈述的数据之后或反映意外事件的发生情况。您应完整阅读本文，并了解我们未来的实际结果或表现可能与我们的预期存在重大差异。在本文中，我们或我们的任何董事或我们公司的意图的陈述或引用均在本文发表之日作出。这些意图中的任何一个都可能随着未来的发展而改变。 联系我们： 投资者关系： ir@canbridgepharma.com

- Zilurgisertib reduced total HO lesion volume and improved clinician-assessed joint mobility; no zilurgisertib-treated patients developed new HO lesions - Mirum plans to submit an sNDA in H1 2027 to expand th e ATEBRIOZ indication to pediatric patients aged 6 to <12 years - Safety and efficacy findings consistent with PROGRESS Cohort 1; no new safety signals FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a leading rare disease company, today announced positive results from Cohort 2 of the pivotal PROGRESS study of ATEBRIOZ ™ (zilurgisertib), an oral activin receptor-like kinase 2 (ALK2) inhibitor, in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva (FOP). At Week 24, zilurgisertib reduced total heterotopic ossification (HO) lesion volume and improved clinician-assessed joint mobility. No zilurgisertib-treated patients developed new HO lesions. The results build on data from PROGRESS Cohort 1, which supported the U.S. Food and Drug Administration (FDA) approval of ATEBRIOZ in September 2026 for adult and pediatric patients aged 12 years and older with FOP. Mirum plans a supplemental New Drug Application (sNDA) submission in H1 2027 to expand the indication to pediatric patients aged 6 to <12 years. “These results represent an important advance for younger children living with FOP,” said Rob Myers, M.D., Chief Medical Officer of Mirum. “The reduction in total HO volume and absence of new lesions seen with zilurgisertib build on the positive results previously observed in adolescents and adults in Cohort 1 of PROGRESS. We look forward to discussing these findings with the FDA to expand access to ATEBRIOZ for younger children living with this debilitating disease.” “FOP is a devastating genetic disease that leads to progressive loss of mobility, independence, quality of life, and shortened lifespan,” said Frederick S. Kaplan, M.D., Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania Perelman School of Medicine. “Childhood is a critical window in FOP, so a therapy targeting the underlying cause of the disease is especially important for this young age group. Results such as these offer hope that we may be able to slow disease progression in children before its most serious consequences arise.” PROGRESS Cohort 2 Results Cohort 2 of the PROGRESS study enrolled 25 pediatric patients aged 6 to <12 years with FOP. Patients were randomized 1:1 to receive once-daily oral zilurgisertib 75 mg (n=12) or placebo (n=13) during a 24-week double-blind period, followed by an open-label extension. The mean age at baseline was 8.3 years, with a mean of 5.0 years since initial diagnosis. All patients completed the double-blind period and entered the open-label extension. At Week 24, mean total HO lesion volume decreased by 8.09 cm³ in patients receiving zilurgisertib, compared with an increase of 9.14 cm³ in patients receiving placebo (nominal p=0.003). No zilurgisertib-treated patients developed new HO lesions. Improvements in clinician-assessed joint mobility were also observed. Joint mobility was assessed using the Cumulative Analogue Joint Involvement Scale (CAJIS), a validated measure of physical function in FOP scored from 0 (no impairment) to 30 (maximum impairment). At Week 24, CAJIS scores improved by a mean of 0.80 points in patients receiving zilurgisertib, compared with 0.09 points in patients receiving placebo. In untreated patients with FOP, CAJIS scores are reported to worsen by approximately 0.5 points per year. 1 Key efficacy data from Cohort 2 are shown below in the Efficacy Results table. Efficacy Results Endpoint Zilurgisertib 75 mg QD Placebo Key Finding Mean (SD) change from baseline in total HO lesion volume, cm³ -8.09 (15.58) n=12 9.14 (11.66) n=13 Treatment difference a of -14.0 cm³ favoring zilurgisertib (nominal p=0.003). b Patients with new HO lesions, n (%) 0 (0) n=12 1 (7.7) n=13 No new HO lesions with zilurgisertib. Mean (SD) change from baseline in CAJIS score -0.80 (1.1) n=10 -0.09 (1.0) n=11 Improvement in clinician-assessed joint mobility with zilurgisertib. a Hodges-Lehmann estimate of treatment difference b p-value calculated using a Wilcoxon rank-sum test Zilurgisertib was generally well tolerated, with a safety pro with that observed in PROGRESS Cohort 1. No new safety signals were identified. No patients discontinued treatment during the double-blind period, and no zilurgisertib-treated patients experienced growth plate closure. Additional safety findings included: Most adverse events were mild or moderate in severity. Serious adverse events and Grade ≥3 adverse events were infrequent and occurred less frequently in patients receiving zilurgisertib than in those receiving placebo. Adverse events occurring in more than 10% of zilurgisertib-treated patients and at a higher frequency than with placebo included arthralgia, cough, pyrexia, and vomiting (3 subjects each), and alopecia, epistaxis, fall, iron deficiency, and nasopharyngitis (2 subjects each). ATEBRIOZ is approved by the U.S. FDA to reduce the volume of total new HO in adult and pediatric patients aged 12 years and older with FOP. Total new HO lesion volume includes expansion of baseline HO lesion burden as well as any new discrete HO that developed during the 24-week double-blind period. Full Cohort 2 results will be presented at an upcoming medical meeting. Cohort 3 of the PROGRESS study, which is evaluating zilurgisertib in pediatric patients aged 2 to <12 years, is currently enrolling. ATEBRIOZ is available in the U.S. through Mirum Access Plus (MAP), a patient support program designed to help patients, families, and healthcare providers navigate treatment access. MAP provides insurance coverage and access support, financial assistance for eligible patients, personalized patient support, and educational resources for patients and caregivers. Eligible patients can pay as little as $0 per month. To learn more, call 855-MRM-4YOU (1-855-676-4968) or visit atebrioz.com . About ATEBRIOZ™ (zilurgisertib) tablets ATEBRIOZ™ (zilurgisertib) tablets are a once-daily oral activin receptor-like kinase 2 (ALK2) inhibitor approved by the U.S. Food and Drug Administration (FDA) to reduce the volume of total new heterotopic ossification in adult and pediatric patients aged 12 years and older with Fibrodysplasia Ossificans Progressiva (FOP). In people living with FOP, pathogenic variants in the ACVR1 gene result in abnormal activation of ALK2, leading to the formation of bone in muscles, tendons, ligaments and other soft tissues through a process known as heterotopic ossification (HO). For more information, please visit atebrioz.com . Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. IMPORTANT SAFETY INFORMATION ATEBRIOZ is contraindicated in pregnancy. ATEBRIOZ can cause fetal harm based on data from animal studies. Patients of reproductive potential should use effective contraception and should immediately discontinue ATEBRIOZ and contact their healthcare provider if pregnancy occurs. US Prescribing Information About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled 63 patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Results from Cohort 1 have been reported through Week 48. The PROGRESS study is also evaluating the safety and efficacy of zilurgisertib in younger patients with FOP. Cohort 2 enrolled 25 patients aged 6 to <12 years who were randomized 1:1 to receive zilurgisertib 75 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Cohort 3, which is evaluating zilurgisertib in patients aged 2 to <12 years, is currently enrolling. About Fibrodysplasia Ossificans Progressiva (FOP) Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, progressive genetic disease affecting approximately 300 people in the United States and 900 worldwide. FOP is characterized by heterotopic ossification (HO), a process in which bone forms in muscles, tendons, ligaments and other soft tissues. Symptoms typically become apparent in early childhood, and the number and volume of HO lesions increase over time, progressively restricting movement and limiting mobility, daily function, and independence. About Mirum Pharmaceuticals 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 focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), ATEBRIOZ™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP), 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 and follow Mirum on Facebook , LinkedIn , Instagram and X. Mirum 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, expectations regarding the timing, enrollment or results of clinical trials for zilurgisertib, the potential benefit of zilurgisertib in real world settings versus clinical trial settings, including the potential to slow disease progression in children before FOP’s most serious consequences arise, the timing of regulatory engagement with the FDA and any resulting submission of an sNDA for ages 6 to <12, the importance of ATEBRIOZ™ as an additional therapy for the treatment of FOP, the potential presentation of zilurgisertib clinical results at an upcoming medical meeting, cost to patients and methods of availability. 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 “plan,” “may,” “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 the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies, risks inherent in the regulatory submission process in the U.S., 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 Quarterly Report for the quarter ended June 30, 2026, filed with the Securities and Exchange Commission on August 5, 2026, 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. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. References 1 Pignolo RJ, et al. Orphanet J Rare Dis. 2019;14:113. Contacts Mirum Investor Contact: Andrew McKibben ir@mirumpharma.com Mirum Media Contact: Meredith Kiernan media@mirumpharma.com