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▎药明康德内容团队报道9月4日，北海康成发布了2023年半年报。根据报告，该公司下一步里程碑包括：预计于2024年第一季度在中国推出用于治疗罕见肝病阿拉杰里综合征（ALGS）患者的氯马昔巴特口服溶液；预计于2023年底之前，在中国台湾地区及香港地区获得氯马昔巴特口服溶液治疗1岁及以上ALGS患者的新药申请（NDA）批准；将继续推进治疗脊髓性肌萎缩症（SMA）的创新第二代scAAV基因疗法等等。北海康成是一家专注于罕见疾病领域全球化的生物医药公司，致力于创新疗法的研究、开发和商业化。该公司目前拥有14个药物资产组合，其中包括4种已获批上市产品和10个在研药物。这些产品均针对一些较普遍的罕见疾病和罕见肿瘤适应症，比如亨特综合征和其他溶酶体贮积症、补体介导的疾病、血友病A、代谢紊乱、罕见的胆汁淤积性肝病、神经肌肉疾病以及胶质母细胞瘤。此外，北海康成的新一代基因技术研发中心实验室正在开发针对罕见遗传病，包括庞贝氏症、法布雷病、脊髓性肌萎缩症（SMA）和其他神经肌肉疾病的新型及潜在“治愈性”基因疗法，并与全球范围内研究人员和生物技术公司合作。2023年上半年，北海康成的产品进展包括：海芮思（CAN101）：酶替代疗法CAN101是一款针对黏多糖贮积症II型（MPS II/亨特综合征）的酶替代疗法。该产品于2021年5月在中国大陆推出。随着新患者识别的加速，截至2023年6月30日已诊断识别739例患者。迈芮倍（氯马昔巴特口服溶液，CAN108）：IBAT抑制剂迈芮倍是一款用于治疗罕见胆汁淤积性肝病的口服液药物，是一种几乎不被吸收的回肠胆汁酸转运蛋白（IBAT）抑制剂。北海康成已获得迈芮倍口服液大中华区对罕见肝病阿拉杰里综合征（ALGS）、进行性家族性肝内胆汁淤积症（PFIC）、胆道闭锁（BA）以及其他可选择的适应症的开发，商业化以及在特定条件下生产的独家授权 。2023年5月，中国国家药监局（NMPA）批准迈芮倍上市，治疗1岁及以上ALGS患者的胆汁淤积性瘙痒。北海康成计划于2024年第一季度在中国推出迈芮倍。2023年5月在中国完成迈芮倍治疗胆道闭锁（BA）的2期研究患者招募。该临床试验由Mirum Pharmaceuticals公司主导进行，并由北海康成根据与Mirum公司订立的许可协议提供支持。预计于2023年底前，迈芮倍在中国台湾地区及香港地区获得治疗1岁及以上ALGS患者的新药申请批准。预计于2023年下半年在中国大陆和台湾地区提交用于治疗进行性家族性肝内胆汁淤积症（PFIC）的新药申请。CAN106：一种新型长效单克隆抗体CAN106拟开发用于治疗包括阵发性睡眠性血红蛋白尿症（PNH）、重症肌无力（MG）及其它多种C5补体介导相关疾病。在中国正在进行的CAN106针对PNH患者的1b期研究取得积极初步数据。研究数据表明，CAN106能对补体C5进行有效阻断，具有良好的安全性和耐受性。基于1b期研究的正面结果，北海康成将于2023年下半年开始在中国进行CAN106治疗PNH的关键性临床试验，并于2024年下半年开始收集潜在数据。CAN008：CD95-Fc糖基化融合蛋白CAN008是一款用于治疗胶质母细胞瘤（GBM）的药物，是一种CD95-Fc糖基化融合蛋白。2023年上半年，独立数据监测委员会完成CAN008正在中国进行的、针对新诊断GBM患者2期研究的中期分析及审查，并建议在不改变当前的试验设计的情况下继续进行该研究。CAN103：用于治疗戈谢病的酶替代疗法CAN103完成正在中国进行的1/2期临床试验的A部分，并于2023年第一季度启动B部分，该部分将作为潜在的可用于中国上市申请的关键性试验。基因治疗（CAN203）：北海康成的基因疗法平台专注于腺相关病毒（AAV）作为基因递送载体，有望成为多种基因遗传疾病一次性持久治疗方案。CAN203在2023年美国基因与细胞治疗学会（ASGCT）年会上公布的临床前数据显示，这一新型第二代scAAV对于治疗脊髓性肌肉萎缩症（SMA）具有潜力，从内源性hSMN1启动子表达了co-hSMN1。基因治疗的低剂量脑室内输送在SMA小鼠数据中显示了比基准载体更强的效力、有效性和安全性。此基准载体与美国FDA批准用于治疗SMA基因疗法的载体类似。北海康成创始人、董事长兼首席执行官薛群博士表示，今年上半年，北海康成取得了非常显著的成果，在多个临床数据和药政事务方面实现了重要里程碑。在最新临床数据方面，北海康成取得了多个令人鼓舞的的数据，包括正在开发治疗阵发性睡眠性血红蛋白尿症（PNH）的CAN106，以及治疗脊髓性肌肉萎缩症（SMA）的候选基因疗法CAN203。在药政事务方面，很高兴治疗罕见肝病阿拉杰里综合征（ALGS）胆汁淤积性瘙痒的迈芮倍（氯马昔巴特口服溶液）在中国获批上市，这对北海康成而言是一个关键性的里程碑。北海康成会进一步将迈芮倍的适应症扩展至其他罕见肝病。展望下半年，他们期待在开发罕见病创新疗法这一领域取得持续的进展。了解更多罕见病相关知识及研究进展请点击访问我们的小程序参考资料：[1]北海康成发布截至2023年6月30日止六个月中期业绩公告. Retrieved Sep 4，2023. From https://mp.weixin.qq.com/s/Qqn7PTZCmxkq2zyBadqWHQ本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

BEIJING and CAMBRIDGE, Mass., Aug. 31, 2023 /PRNewswire/ -- CANbridge Pharmaceuticals, Inc. ("CANbridge," stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare diseases and oncology indications, today announced its financial results for the six months ended June 30, 2023 plus corporate updates. "On the product front, it had been a very productive first half year for CANbridge. We achieved multiple data and regulatory milestones that we believe will continue to drive both near- and long-term value for our stockholders," said James Xue, Ph.D., CANbridge founder, chairman, and CEO. "Regarding new data, we have encouraging updates on several programs, notably CAN106 for treating paroxysmal nocturnal hemoglobinuria (PNH), as well as CAN203, our gene therapy candidate for SMA. On the regulatory front, we are pleased to have received approval for LIVMARLI® (maralixibat/CAN108) in China for treating Cholestatic Pruritus in patients with the rare liver disease Alagille syndrome (ALGS). Livmarli is the first and only approved product in China for the disease, thus marked a pivotal milestone for CANbridge, given the sizeable commercial opportunity and the potential to expand the Livmarli label to cover multiple other rare liver disease indications. In the second half year, we look forward to achieving continuous progress in developing our pipeline of leading therapies for rare diseases." Upcoming Milestones CAN108 – Plan to launch Livmarli for patients with ALGS in China in Q1 2024. CAN108 – Anticipate New Drug Application (NDA) approval for Livmarli's use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023. CAN108 – Data from the Mirum-sponsored EMBARK Phase 2 trial in biliary atresia patients is expected in H2 2023. CAN008 – Plan to report data from the Phase 2 clinical trial in H1 2024. CAN106 – Begin enrolling a Phase 2 clinical trial in PNH in China that will commence in H2 2023 with data potentially ready in H2 2024. Gene Therapy – Advance novel second-generation scAAV gene therapy for the treatment of spinal muscular atrophy (SMA) Financial Highlights Revenue increased by RMB8.4 million, or 24.2%, to RMB43.1 million for the six months ended 30 June 2023 from RMB34.7 million for the same period last year, mainly attributable to the increase in sales of Hunterase® and Livmarli®. Research and development ("R&D") expenses decreased by approximately RMB15.3 million, or 9.7%, to RMB143.0 million for the six months ended 30 June 2023 from RMB158.3 million for the same period last year, primarily attributable to the decrease in upfront and milestone payments made to CANbridge's licensing partners and in technical service fees, partially offset by the increase in depreciation and amortization costs. Loss for the Reporting Period decreased by approximately RMB30.8 million, or 12.4%, to RMB218.2 million for the six months ended 30 June 2023 from RMB249.0 million for the same period last year, primarily attributable to the increase in revenue and decrease in selling and distribution expenses, R&D expenses and administrative expenses. Recent Highlights Hunterase (CAN101) , an enzyme replacement therapy for treating MPS II, also known as Hunter syndrome, has been on the "First National List of Rare Diseases" since May 2018. It was launched in May 2021 in mainland China as the first and only ERT for MPS II, and since then more new patients have been identified, up to 739 as of 30 June 2023. Expanded relevant commercial insurance programs (Huiminbao) to 109 cities, covering a population of 586 million in China. LIVMARLI is an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) being developed for treating rare cholestatic liver diseases. CANbridge has the exclusive rights to develop, commercialize, and under certain conditions, manufacture LIVMARLI in Greater China. On May 29, 2023, the Chinese National Medical Products Administration (NMPA) approved CAN108, making Livmarli the first and only approved product in China to be marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged one year and older. CANbridge plans to launch Livmarli for ALGS in Q1 2024. Completed enrollment of the EMBARK Phase 2 study of Livmarli in biliary atresia (BA) in China in May 2023. The clinical trial in BA is conducted by Mirum and supported by CANbridge under the license agreement with Mirum. Expected to obtain NDA approval for Livmarli's use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023. Plan to submit NDA for Livmarli's use in PFIC patients in Taiwan and mainland China in the second half of 2023. CAN106 is a novel, long-acting monoclonal antibody for treating complement-mediated diseases, including PNH, myasthenia gravis (MG) and various other complement-mediated diseases that are targeted by anti-C5 antibodies. PNH has been included on the "First National List of Rare Diseases" in China since May 2018. Reported positive preliminary top-line Phase 1 data from the ongoing Phase 1b study of CAN106 in China for PNH. The results suggest complete blockade of complement function at safe and well-tolerated doses. Based on the positive results from the Phase 1b study, CANbridge will begin enrolling a registrational clinical trial in PNH in China that will commence in the second half of 2023 with data amassed potentially in the second half of 2024. CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM). The IDMC (independent data monitoring committee) completed an interim analysis and review of the ongoing Phase 2 study of CAN008 in China in patients with newly diagnosed GBM and recommended the study to continue without any changes to the current trial design. CAN103 is an ERT for treating Gaucher Disease (GD) and GD has been included on the "First National List of Rare Diseases" in China since May 2018. Completed Part A of the ongoing Phase 1/2 clinical trial in China and initiated Part B in the first quarter of 2023, with the latter to serve as a potential registrational trial. CAN103 is the first clinical stage ERT being developed for GD in China. In Gene Therapy, CANbridge advanced its world-class gene therapy platform, focusing on adeno-associated virus (AAV) as a gene delivery vehicle, with potential as a one-time durable therapy for many genetic diseases. Programs in Fabry disease and SMA have been included on the "First National List of Rare Diseases" since May 2018. Appointed Jason West, Ph.D., as Vice President, Head of Gene Therapy Research. Dr. West possesses expertise in areas such asgene therapy development, platform innovation and clinical candidate development. Presented preclinical data at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. The data shared evidenced the potential of the novel, second-generation scAAV in treating SMA as it expresses a codon optimized (co)-hSMN1 from an endogenous hSMN1 promoter. Low-dose intracerebroventricular delivery of the gene therapy was able to achieve superior potency, efficacy and safety in mice with SMA, compared to the benchmark vector, scAAV-CMVen/CB-hSMN1, which is similar to the FDA-approved gene therapy vector for SMA. CANbridge will also host a conference call to discuss the 2023 interim results and recent corporate progress. To register for the English session, please click here. To register for the Chinese session, please click here. This webcast will be English, and translation will be provided on the Chinese channel. About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with 4 approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: . Forward-Looking Statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. SOURCE CANbridge Pharmaceuticals, Inc.