FcRn

3月29日，专注于肿瘤及免疫性疾病创新药研发及商业化的全球化生物医药公司—和铂医药宣布2023年度业绩报告。2023年财报业绩截止2023年12月31日，和铂医药全年总盈利2276万美元，这也是公司首次实现年度财务盈利。财务亮点包括：报告期内总收入达8950万美元，同比增长119.9%（2022年总收入为4066万美元），公司的收入构成主要包括产品授权、研究服务和技术授权，总收入的增长主要得益于与辉瑞、Cullinan Oncology和科伦博泰的授权合作；报告期内，全年研发总投入4508万美元，同比下降66.6%（2022年研发总投入为1.35亿美元），研发费用的降低主要受两个因素的综合影响，一是对自有临床项目以及处于早期发现和临床前阶段项目的优化投资，二是高效实施成本控制的措施；报告期内，全年管理费用共计1950万美元，同比下降28.6%（2022年管理费总计2727万美元）。创新及差异化产品管线和铂医疗是与诺纳生物并行的子品牌，独立负责公司产品管线的开发。当前，和铂医疗专注于肿瘤及免疫疾病领域，拥有十多款差异化候选药物，其中四款处于临床阶段。公司的主要产品包括巴托利单抗（HBM9161）、普鲁苏拜单抗（HBM4003）、HBM7008 和 HBM1020。巴托利单抗是大中华区临床开发进度最快的FcRn抑制剂，有望成为治疗多种自身免疫性疾病的突破性药物。公司于2023年3月公布其治疗全身型重症肌无力III期试验积极研究结果，这也是公司自成立以来首个完成III期临床试验并准备商业化的产品，有望造福广大重症肌无力患者。公司于2022年启动开放标签扩展研究，并于2023年3月完成入组。截至于2023年11月的数据分析显示巴托利单抗在疾病长期管理中具有持续的疗效和良好的安全性特征。普鲁苏拜单抗是开发自HCAb平台的新一代全人源仅重链抗CTLA-4抗体，是全球首个进入临床阶段的全人源仅重链抗体。2023年，和铂医疗继续全速推进普鲁苏拜单抗针对多种实体瘤的全球临床开发项目，目前正在进行中的神经内分泌瘤和肝细胞癌试验已在疗效和安全性方面读出积极数据。HBM7008开发自HBICE®平台，是目前全球唯一针对B7H4和4-1BB靶点的创新双特异性抗体，是和铂医疗强大研发能力的另一有力佐证。凭借公司在生物学及抗体工程方面的专业知识以及HBICE®平台的独特性，HBM7008在临床前阶段就已展现出令人欣喜的疗效和安全性特征，其在美国和澳大利亚的I期临床试验于2022年启动。2023年2月，公司与Cullinan Oncology就HBM7008达成区域性合作开发协议，以进一步加速其在美国、欧洲和澳大利亚的研究进程，保持全球领先的开发优势。HBM1020是一款由Harbour Mice® H2L2转基因小鼠平台开发的靶向B7H7的全球首创全人源单克隆抗体。B7H7是一种新型免疫调节分子，属于B7家族，能独立于PD-L1表达从而在肿瘤细胞逃避免疫监管方面发挥关键作用。HBM1020是全球首个正式获批进入临床阶段的靶向B7H7单克隆抗体，凭借其出色的产品设计和靶点特征，有望解决实体瘤治疗领域尚未满足的巨大医疗需求。在公司领先的药物创新和发现引擎的驱动下，新产品不断被推向临床，其中HBM1020、HBM1022、HBM1007和HBM9033于2023年获得美国FDA的IND许可，HBM9027已于2024年第一季度获得美国FDA的IND许可。此外，包括HBM7004、HBM1047 和 HBM9014在内的多款处于临床前阶段的在研产品，展现出巨大的开发潜力。商业合作2023年，和铂医药继续扩大并专注于高效创新的行业合作伙伴及全球领先学术机构的业务合作，合作模式除对外授权外，还包括与学术机构和其他创新行业先锋合作，共同开发和孵化下一代创新疗法的合资企业。展望2024展望未来，和铂医疗将致力于推进管线多项临床试验，加速全球临床开发项目。往期推荐视角 | 纸面之外的药明康德2023年报Claudin18.2ADC赛道群雄逐鹿：3家率先进入III期临床下一个10亿美元重磅炸弹选手"裁员潮”仍在加剧：2月多家药企裁员点击下方“药研网”，关注更多精彩内容

CAMBRIDGE, Mass., ROTTERDAM, Netherlands and SUZHOU, China, March 28, 2024 /PRNewswire/ -- Harbour BioMed ("HBM" or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery, development and commercialization of novel antibody therapeutics focusing on immune-oncology and immunology, released annual financial results of full year 2023. "We made progress in two significant undertakings in 2023, the third financial year since listing on the Main Board of the Stock Exchange of Hong Kong: preparing Harbour Therapeutics to operate as a faster, more focused clinical-stage next-generation therapeutics company and initiating Nona Biosciences to leverage our unique global patent-protected technology platforms to empower global therapeutic innovation. We recorded a significant increase in our revenue, demonstrating the Company's excellent global business development capabilities," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "We are navigating a fiercely competitive world that is going through rapid changes, facing human being's fundamental quest for longevity and quality of life, and having a greater demand for biotechnological breakthroughs and innovative therapeutics. We've been well-positioned in the new era to achieve results that will propel the Company to new heights and create robust business value." FULL YEAR 2023 FINANCIAL HIGHLIGHTS Harbour BioMed recorded the profit of US$22.8 million for the year ended 31 December 2023. This is the first time that the Company has recorded a net profit on its annual financial statements. Other financial highlights include: Revenue: The revenue for the full year 2023 is US$89.5 million, which increased significantly by US$48.8 million, or 119.9%, compared with US$40.7 million for the year ended 31 December 2022. Our revenue primarily consists of molecule license fee, research service fee and technology license fee. The increase is primarily attributed to license out and collaboration agreement with Pfizer, Cullinan Oncology and Kelun-Biotech. Research and development costs: The research and development expenses decreased by 66.6%, from US$135.1 million in 2022 to US$45.1 million in 2023. This decrease was primarily attributable to the combined impact of (i) optimized investments in our clinical programs and our molecule assets in discovery and pre-clinical stages; and (ii) the efficient implementation of cost control measures. Administrative expenses: The administrative expenses decreased by 28.6%, from US$27.3 million in 2022 to US$19.5 million in 2023. ROBUST PORTFOLIO AND DIFFERENTIATED PIPELINE Harbour Therapeutics, a sub-brand parallel to Nona Biosciences, is individually responsible for the development of the Company's product pipeline. Focused on oncology and immunology, Harbour Therapeutics has a robust and diversified pipeline of more than ten potentially differentiated drug candidates, four of which are in clinical development stage. Batoclimab (HBM9161), porustobart (HBM4003), HBM7008 and HBM1020 are the main products. Batoclimab, as the clinically most advanced FcRn inhibitor being developed in Greater China, has the potential to be a breakthrough treatment for a wide spectrum of autoimmune diseases in Greater China. Harbour Therapeutics completed the treatment of patients in early 2023 and announced the positive topline results of the phase III clinical trial of batoclimab for the treatment of generalized myasthenia gravis (gMG) in March, which is also the first positive pivotal trial outcome for batoclimab worldwide. This marks a major milestone as it is the Company's first product to complete phase III clinical trial and be poised for commercialization to benefit the gMG patients. The Company also initiated open-label extension clinical trial in 2022 and completed enrolment in March 2023. As the cut-off of open-label extension clinical trial for gMG in November 2023, the data showed sustainable efficacy and safety of batoclimab in long-term disease management. Porustobart (HBM4003) is the next-generation, fully human heavy chain only anti-CTLA-4 antibody generated from the HCAb platform. It is also the first fully human heavy chain only antibody which has entered into clinical development around the world. In 2023, Harbour Therapeutics conducted the global clinical development program of porustobart for multiple types of solid tumors, and positive data of efficacy and safety profile have been read out in the ongoing trials of neuroendocrine neoplasms and hepatocellular carcinoma. Another example demonstrating Harbour Therapeutics' strong research capabilities is the discovery of HBM7008, a novel product targeting B7H4 and 4-1BB. Developed from the immune cell engager platform HBICE®, HBM7008 is the only bispecific antibody against these two targets globally. Leveraging and integrating the expertise in biology and antibody engineering and the unique characteristics of HBICE® platform, HBM7008 showed exciting performance both in efficacy and safety profile at pre-clinical stage. In 2022, the Company conducted the phase I trials in the U.S. and Australia. In February 2023, to maintain a leading position in the development of this first-in-class asset, the Company entered into a co-development collaboration with Cullinan Oncology, to expand its study process in the U.S., Europe and Australia. HBM1020 is a first-in-class fully human monoclonal antibody generated from Harbour Mice® platform targeting B7H7. As a newly discovered member of the B7 family, B7H7 expression is found non-overlapping with PD-L1 expression in multiple tumor types, which potentially plays a more important role for tumor cells to escape immune surveillance. HBM1020 is the first product against B7H7 in clinical stage globally. With its excellent product design and target features, HBM1020 presents great potential to address huge unmet medical needs on solid tumors. Driven by the Company's leading drug innovation and discovery engine, new assets are continually being developed, of which HBM1020, HBM1022, HBM1007 and HBM9033 obtained the IND clearance from the U.S. FDA to initiate clinical study in the U.S. in 2023, and HBM9027 obtained the IND clearance from U.S. FDA in the first quarter of 2024. In addition, we have a number of pre-clinical stage products in the pipeline, including HBM7004, HBM1047 and HBM9014, which have shown great potential for development. PLATFORM-VALUE-MAXIMIZED BUSINESS COLLABORATIONS In 2023, the Company continued to expand its business collaborations with leading academic institutions and select industrial partners focusing on innovation and efficiency across the world. The business collaboration model is not only limited to out-licensing, but also to engage with academic institutions or other leading innovative pioneers in the industry for co-development and incubation of joint ventures on next-generation innovative therapy. With flexible business models built around proprietary technologies and platforms, the Company can and will maximize its platform value to address global unmet medical needs. Assets Collaboration of Harbour Therapeutics Harbour Therapeutics has entered into several external collaborations in terms of pipeline licensing and collaborations. In 2023, Harbour Therapeutics has granted the regional out-licensing of HBM7008 in the U.S. to Cullinan Oncology, and HBM7022/AZD5863, which was licensed to AstraZeneca in 2022, has entered into clinical stage. Meanwhile, the three assets which have been licensed the Greater China Rights to Hualan Genetic obtained IND approvals from NMPA to initiate clinical study in China. In addition, HBM9378, which was developed in collaboration with Kelun-Biotech, completed its phase I clinical trial. With these multiple collaborations based on the assets generated from HBICE®, Harbour Therapeutics has shown its strength and unique advantages in building a comprehensive portfolio in immune cell engagers. And the co-development and collaboration of the pipeline is not only the recognition of the industry partners for the Company's products and technology platforms, but will also help the Company to improve the efficiency of portfolio advancement, spread the costs and risks, and make the development of the Company more robust. Multiple Collaborations of Nona Biosciences In addition to collaboration through the molecules and pipeline generated from the platforms, the Company is also focusing the vision on more original and innovative collaborations on early stages. By integrating the industry leading Harbour Mice® and HCAb PlusTM platforms with an experienced therapeutic antibody discovery team, Nona Biosciences provides a one-stop solution for therapeutic antibody discovery, engineering and development from I to ITM (Idea to IND) with flexible business models. From the end of 2022, Nona Biosciences achieved big success in its launch as it has landed several international collaborations in multiple innovative formats. It is worth mentioning that Nona Biosciences has granted the global out-licensing of HBM9033, a potential best-in-class MSLN-targeted ADC, to Pfizer. Until now, Harbour Mice® platforms have been validated by over 50 industry and academic partners. Nona Biosciences has established four leading technology units based on HCAb, including protein engineering, conjugation technology, delivery technology and cell therapy to empower the next-generation therapies. With the multiple collaborations based on the assets generated from HCAb PlusTM, Nona Biosciences has demonstrated its robust capabilities in antibody discovery and development, exploring a new path to expand the collaboration network and maximize the value of the platforms. Incubation on Cutting-Edge Collaborations To give full play to the value of the unique platform technologies, the Company has continued to explore the expandability of platform technology application scenarios which generate impactful values and bring us new value growth points with minimal marginal investment. Representative projects include HBM Alpha Therapeutics, in partnership with Boston Children's Hospital, and Shanghai NK Cell Technology Limited. 2024 OUTLOOK: EXTENSIVE GLOBALIZATION AND BREAKTHROUGH INNOVATION Looking to the future, Harbour BioMed will keep driving business growth and accomplishing its mission through two key pillars, Harbour Therapeutics and Nona Biosciences. The former will advance multiple clinical trials of the internal pipeline to fully advance the global clinical development project, and the latter will keep providing integrated discovery solutions for biotechnology and pharmaceutical companies and ultimately create an innovation ecosystem to promote biological advancement. A range of products based on the technology platform and generated from the concept of T-cell engager and NK cell engager, will be pushed forward to clinical stage in the following years. With a combination of in-house development and business collaborations, the Company will continuously form a portfolio of products with a differentiated competitive advantage in immuno-oncology. The platform-valued-maximized business collaborations, driven by Nona Biosciences, will further walk the Company down the path of global development. Positive outcomes have been attained through platform-based collaborations with top institutions around the world and more extensive global collaborations are expected in 2024 as our preclinical products become increasingly mature. About Harbour BioMed Harbour BioMed (HKEX: 02142) is a global biopharmaceutical company committed to the discovery, development, and commercialization of novel antibody therapeutics focusing on immunology and oncology. The Company is building its robust portfolio and differentiated pipeline through internal R&D capability, collaborations with co-discovery and co-development partners, and select acquisitions. The proprietary antibody technology platforms Harbour Mice® generates fully human monoclonal antibodies in two heavy and two light chains (H2L2) format, as well as heavy chain only (HCAb) format. Building upon the HCAb antibodies, the HCAb-based immune cell engagers (HBICE®) bispecific antibody technology is capable of delivering tumor-killing effects unachievable by traditional combination therapies. Integrating Harbour Mice®, and HBICE® with a single B cell cloning platform, our antibody discovery engine is highly unique and efficient for the development of next-generation therapeutic antibodies. For further information, please refer to . SOURCE Harbour BioMed

中国苏州，美国马萨诸塞州剑桥和荷兰鹿特丹 2024年3月28日 /美通社/ -- 和铂医药（股票代码：02142.HK），一家专注于肿瘤及免疫性疾病创新药研发及商业化的全球化生物医药公司，今日公布其2023年度业绩。 和铂医药创始人、董事长兼首席执行官王劲松博士表示："2023年是我们在香港联交所主板上市以来的第三个财年，我们在两项重要工作上取得了进展：一是让和铂医疗更高效、更专注于临床阶段下一代治疗药物的研发，二是全面启动诺纳生物，利用我们全球专利保护的抗体技术平台，助力全球生物药创新。业绩的显著增长彰显了公司卓越的全球业务拓展能力。我们正处于一个竞争激烈且瞬息万变的世界，人类始终对生命和生活质量保持着根本追求，对生物技术突破和创新疗法的需求也随之激增。我们已经做好了充分的准备，以推动公司再上新台阶，创造更强大的商业价值。" 2023 年业绩亮点 截止2023年12月31日，和铂医药全年总盈利2276万美元，这也是公司首次实现年度财务盈利。其他的财务亮点包括： 报告期内总收入达8950万美元，同比增长119.9%（2022年总收入为4066万美元），公司的收入构成主要包括产品授权、研究服务和技术授权，总收入的增长主要得益于与辉瑞、Cullinan Oncology和科伦博泰的授权合作； 报告期内，全年研发总投入4508万美元，同比下降66.6%（2022年研发总投入为1.35亿美元），研发费用的降低主要受两个因素的综合影响，一是对自有临床项目以及处于早期发现和临床前阶段项目的优化投资，二是高效实施成本控制的措施； 报告期内，全年管理费用共计1950万美元，同比下降28.6%（2022年管理费总计2727万美元）。 全球创新及差异化产品管线 和铂医疗是与诺纳生物并行的子品牌，独立负责公司产品管线的开发。当前，和铂医疗专注于肿瘤及免疫疾病领域，拥有十多款差异化候选药物，其中四款处于临床阶段。公司的主要产品包括巴托利单抗（HBM9161）、普鲁苏拜单抗（HBM4003）、HBM7008 和 HBM1020。 巴托利单抗 是大中华区临床开发进度最快的FcRn抑制剂，有望成为治疗多种自身免疫性疾病的突破性药物。公司于2023年 3月公布其治疗全身型重症肌无力III期试验积极研究结果，这也是公司自成立以来首个完成III期临床试验并准备商业化的产品，有望造福广大重症肌无力患者。公司于2022年启动开放标签扩展研究，并于2023年3月完成入组。截至于2023年11月的数据分析显示巴托利单抗在疾病长期管理中具有持续的疗效和良好的安全性特征。 普鲁苏拜单抗 是开发自HCAb平台的新一代全人源仅重链抗CTLA-4抗体，是全球首个进入临床阶段的全人源仅重链抗体。2023年，和铂医疗继续全速推进普鲁苏拜单抗针对多种实体瘤的全球临床开发项目，目前正在进行中的神经内分泌瘤和肝细胞癌试验已在疗效和安全性方面读出积极数据。 HBM7008 开发自HBICE ® 平台，是目前全球唯一针对B7H4和4-1BB靶点的创新双特异性抗体，是和铂医疗强大研发能力的另一有力佐证。凭借公司在生物学及抗体工程方面的专业知识以及HBICE ® 平台的独特性，HBM7008在临床前阶段就已展现出令人欣喜的疗效和安全性特征，其在美国和澳大利亚的I期临床试验于2022年启动。2023年2月，公司与Cullinan Oncology就HBM7008达成区域性合作开发协议，以进一步加速其在美国、欧洲和澳大利亚的研究进程，保持全球领先的开发优势。 HBM1020 是一款由Harbour Mice ® H2L2转基因小鼠平台开发的靶向B7H7的全球首创全人源单克隆抗体。B7H7是一种新型免疫调节分子，属于B7家族，能独立于PD-L1表达从而在肿瘤细胞逃避免疫监管方面发挥关键作用。HBM1020是全球首个正式获批进入临床阶段的靶向B7H7单克隆抗体，凭借其出色的产品设计和靶点特征，有望解决实体瘤治疗领域尚未满足的巨大医疗需求。 在公司领先的药物创新和发现引擎的驱动下，新产品不断被推向临床，其中 HBM1020 、 HBM1022 、 HBM1007 和 HBM9033 于2023年获得美国FDA的IND许可， HBM9027 已于2024年第一季度获得美国FDA的IND许可。 此外，包括 HBM7004 、 HBM1047 和 HBM9014 在内的多款处于临床前阶段的在研产品，展现出巨大的开发潜力。 平台价值最大化的商业合作 2023年，公司继续扩大并专注于高效创新的行业合作伙伴及全球领先学术机构的业务合作，合作模式除对外授权外，还包括与学术机构和其他创新行业先锋合作，共同开发和孵化下一代创新疗法的合资企业。凭借专有技术和平台建立的灵活商业模式，公司有能力也将继续最大化其平台价值，以解决全球未满足的医疗需求。 基于和铂医疗的自有产品合作 2023年，和铂医疗已成功完成了多项管线许可及外部合作：公司将HBM7008的美国权益授予Cullinan Oncology；于2022年授予阿斯利康的HBM7022/AZD5863已进入临床阶段；授权华兰基因大中华区权益的三项资产已获得中国国家药监局IND批准；与科伦博泰共同开发的HBM9378已完成I期临床试验。 通过一系列基于HBICE ® 产品的合作，和铂医疗显示了其在构建免疫细胞衔接器方面的独特实力和优势。产品管线的协同开发及对外授权，是建立在合作伙伴对公司产品和技术平台认可的基础之上，并将有助于提高公司内部产品组合推进的效率，分散成本和风险，使公司的发展更加稳健。 基于诺纳生物的多形态合作 除了针对平台产生的分子及管线进行合作外，公司还聚焦于早期阶段更多的原创性和创新性合作。凭借业界领先的Harbour Mice ® 和HCAb Plus TM 平台与经验丰富的抗体发现团队，诺纳生物以灵活的商业模式，为治疗性抗体发现、工程和开发提供从发现至临床前研发等I to I TM （Idea to IND）一站式解决方案。 自2022年底起，诺纳生物接连在国际合作中取得巨大成功，收获行业领先合作伙伴的青睐。值得一提的是，诺纳生物已将HBM9033（一款潜在同类最佳靶向人间皮素ADC）全球权益授予辉瑞。截至目前，Harbour Mice ® 技术平台已经得到了来自行业和学术界50多家合作伙伴的验证。 诺纳生物已基于HCAb构建形成四大前沿核心技术，包括蛋白质工程、抗体偶联技术、药物递送技术和细胞治疗，通过一系列对外授权、合作，展示了其在抗体发现和开发方面的强大能力，为拓展合作网络和最大化平台价值探索了一条新途径。 前沿领域的孵化与合作 为了充分挖掘并发挥平台技术的价值，公司不断探索平台技术应用场景的可扩展性，以期用最小的投资产生有影响力的价值，为公司带来新的价值增长点。其中具有代表性的项目包括与波士顿儿童医院合作的HBM Alpha Therapeutics和恩凯赛药。 展望 2024 ：深耕全球，突破创新 展望未来，和铂医药将通过和铂医疗和诺纳生物两大支柱持续推动业务增长并最终惠及全球病患。和铂医疗将致力于推进管线多项临床试验，全面加速全球临床开发项目；诺纳生物将继续为生物技术及制药企业提供I to I TM 解决方案，打造创新生态系统，推动生物技术的进步与飞跃。 基于专有的技术平台组合及T细胞、NK细胞衔接器概念衍生的一系列产品将在未来几年推至临床阶段。公司将以和铂医疗的研发和业务合作为支点，不断完善在肿瘤免疫领域具有差异化竞争优势的产品组合。 在诺纳生物的带动下，平台价值最大化的业务合作将进一步迈向全球市场。当前，与全球领先企业及机构的平台合作已为公司带来可观的积极成果，而随着公司临床前产品的日益成熟，预计2024年将迎来更广泛的全球合作。 关于和铂医药 和铂医药（股票代码：02142.HK）是一家专注于肿瘤及免疫性疾病领域创新药研发及商业化的全球化生物制药企业。公司通过自主研发、联合开发及多元化的合作模式快速拓展创新药研发管线。 抗体技术平台Harbour Mice ® 可生成双重、双轻链（H2L2）和仅重链（HCAb）形式的全人源单克隆抗体。基于HCAb抗体平台开发的免疫细胞衔接器（HBICE ® ）双抗技术能够实现传统药物联合疗法无法达到的抗肿瘤疗效。Harbour Mice ® ，HBICE ® 与单B细胞克隆筛选平台共同组成了和铂的下一代创新治疗性抗体研发引擎。更多资讯，请访问： www.harbourbiomed.com