Hengrui Medicine announced that it has resubmitted the biological product license application for Bartoli monoclonal antibody treatment of systemic severe myasthenia gravis to the National Medical Products Administration.

PR Newswire · Jun 27 07:18

中国苏州、美国马萨诸塞州剑桥和荷兰鹿特丹2024年6月27日 /美通社/ -- 和铂医药（股票代码：02142.HK），一家专注于肿瘤及免疫领域创新抗体疗法发现、开发及商业化的全球生物医药公司今日宣布，已向国家药品监督管理局（NMPA）重新递交公司在研药物巴托利单抗（HBM9161）治疗全身型重症肌无力（gMG）的生物制品许可申请（BLA）。

根据临床试验方案，和铂医药已顺利完成III期临床试验的扩展研究，在未招募新患者的情况下收集了更多长期安全性数据。因此，公司自愿纳入这批安全性数据并按计划重新递交巴托利单抗的BLA。

和铂医药创始人、董事长兼首席执行官王劲松博士表示："很高兴能按计划重新递交BLA，我们将继续与NMPA保持密切沟通，以推动这项创新疗法的审评工作。III期临床试验的结果证明了巴托利单抗在主要及次要终点的疗效，我们相信这款创新药有望进一步丰富全身型重症肌无力的治疗手段，使更多患者受益。"

巴托利单抗于2021年获得中国国家药品监督管理局授予的"突破性治疗认证"，并于同年8月完成针对中国全身型重症肌无力患者的概念验证研究。2023年3月，公司宣布其III期临床试验取得积极研究结果。

2022年10月，和铂医药与石药集团全资子公司恩必普药业有限公司达成协议，在大中华区共同开发巴托利单抗，和铂医药负责巴托利单抗在中国针对全身型重症肌无力的全完整临床实验的设计与执行, 并将根据产品年度净销售额获得分层销售提成。

关于全身型重症肌无力（gMG）

重症肌无力（MG）是一种由抗乙酰胆碱受体（AChR）免疫球蛋白G（IgG）和抗肌肉特异性酪氨酸激酶（Anti-MuSK）IgG等抗体介导的获得性自身免疫性疾病，该疾病导致神经肌肉接头传递障碍，表现为骨骼肌收缩无力。患者常有眼睑下垂和复视等眼外肌无力表现，绝大多数患者会出现眼肌以外的症状，发展为全身型重症肌无力（gMG），明显影响患者的工作状态和生活质量，严重者发生肌无力危象而危及生命。

目前MG的主要治疗方法包括胆碱酯酶抑制剂、糖皮质激素以及其他免疫抑制剂，但各类药物的疗效及安全性尚不能满足各类患者的临床需求。从病理生理机制出发，减少致病性自身抗体（IgG）是治疗MG最直接的解决方案，如血浆置换和静脉注射免疫球蛋白等，然而，该类治疗方案由于可及性、安全性和经济成本等问题导致临床仍存在大量未被满足的需求。

关于巴托利单抗（HBM9161）

巴托利单抗（HBM9161）是一种全人源单克隆抗体（mAb），可阻断FcRn-IgG相互结合，加速体内IgG的清除，从而达到有效治疗致病性IgG介导的自身免疫性疾病的效果。在重症肌无力的III期研究中，巴托利单抗取得积极研究结果，可快速、显著地缓解临床症状，改善患者的生活质量，且安全性良好。

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：

Suzhou, China, Cambridge, Massachusetts in the United States and Rotterdam in the Netherlands, June 27, 2024 / US PRNewswire/ - Harbour BioMed (stock code: 02142. HK), a global biopharmaceutical company dedicated to discovering, developing and commercializing innovative antibody therapies in the fields of oncology and immunology, announced today that it has resubmitted the Biological License Application (BLA) for its investigational drug Batoclimab (HBM9161) for the treatment of generalized myasthenia gravis (gMG) to National Medical Products Administration (NMPA). In accordance with the clinical trial protocol, Harbour BioMed has successfully completed the extension study of Phase III clinical trial and collected more long-term safety data without enrolling new patients. Therefore, the company voluntarily incorporated these safety data and resubmitted the BLA for Batoclimab as planned. Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, said: "We are pleased to resubmit the BLA as planned and will continue to communicate closely with NMPA to advance the review of this innovative therapy. The results of the Phase III clinical trial have demonstrated the efficacy of Batoclimab in the major and secondary endpoints. We believe that this innovative drug has the potential to improve the treatment of generalized myasthenia gravis, enriching treatment options for more patients." Batoclimab received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China in 2021 and completed a proof-of-concept study for generalized myasthenia gravis patients in China in August of the same year. In March 2023, the company announced positive results from its Phase III clinical trial. In October 2022, Harbour BioMed and CSPC Pharma, a wholly-owned subsidiary of CSPC Pharmaceutical Group Limited, reached an agreement to jointly develop Batoclimab in Greater China, with Harbour BioMed responsible for the design and execution of the complete clinical trial for Batoclimab in China for the treatment of generalized myasthenia gravis, and receiving tiered sales royalties based on the annual net sales value of the products.

According to the clinical trial protocol, Harbour BioMed has successfully completed the extension study of Phase III clinical trial and collected more long-term safety data without enrolling new patients. Therefore, the company voluntarily incorporated these safety data and resubmitted the BLA for Batoclimab as planned.

Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, said: "We are pleased to resubmit the BLA as planned and will continue to communicate closely with NMPA to advance the review of this innovative therapy. The results of the Phase III clinical trial have demonstrated the efficacy of Batoclimab in the major and secondary endpoints. We believe that this innovative drug has the potential to improve the treatment of generalized myasthenia gravis, enriching treatment options for more patients."

Batoclimab received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China in 2021 and completed a proof-of-concept study for generalized myasthenia gravis patients in China in August of the same year. In March 2023, the company announced positive results from its Phase III clinical trial.

In October 2022, Harbour BioMed and CSPC Pharma, a wholly-owned subsidiary of CSPC Pharmaceutical Group Limited, reached an agreement to jointly develop Batoclimab in Greater China, with Harbour BioMed responsible for the design and execution of the complete clinical trial for Batoclimab in China for the treatment of generalized myasthenia gravis, and receiving tiered sales royalties based on the annual net sales value of the products.

About Generalized Myasthenia Gravis (gMG)gMGMazdutide (IBI362) is a GLP-1R/GCGR dual agonist jointly developed by Innovent Bio and Eli Lilly and Co. As a somatostatin analogue similar to human gastric inhibitory polypeptide (OXM), Mazdutide not only promotes insulin secretion, lowers blood sugar, and reduces weight by stimulating GLP-1R, but also increases energy consumption and enhances weight loss by stimulating GCGR, while improving liver fat metabolism. Mazdutide has shown excellent weight loss and hypoglycemic effects, as well as multiple metabolic benefits in reducing waist circumference, blood lipids, blood pressure, uric acid, liver enzymes, and liver fat content, as well as improving insulin sensitivity, in multiple clinical studies. Currently, five Phase III clinical studies of Mazdutide are underway in Chinese overweight or obese participants (GLORY-1 and GLORY-2) and type 2 diabetes participants (DREAMS-1, DREAMS-2, and DREAMS-3). Among them, the GLORY-1 and DREAMS-2 studies have achieved their primary endpoints.

Myasthenia gravis (MG), a acquired autoimmune disease mediated by antibodies such as immunoglobulin G (IgG) against nicotinic acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (Anti-MuSK) IgG, is characterized by impairment of neuromuscular transmission at the neuromuscular junction, resulting in skeletal muscle weakness. Patients often demonstrate extraocular muscle weakness that leads to myriad ocular manifestations such as ptosis and diplopia, and most go on to develop symptoms beyond just the ocular muscles, progressing to generalized myasthenia gravis (gMG), and significantly impacting patients' working status and quality of life with life-threatening cases developing myasthenic crisis. Current treatments for MG mainly include cholinesterase inhibitors, glucocorticoids, and other immunosuppressive drugs. However, the efficacy and safety of various drugs cannot meet the clinical needs of various patients. From the perspective of pathophysiological mechanisms, reducing pathogenic autoantibodies (IgG) is the most direct solution to treat MG, such as plasma exchange and intravenous injections of immunoglobulin, etc. However, the clinical demand for this class of treatment programs remains largely unmet due to issues such as accessibility, safety, and economic costs.

About Batoclimab (HBM9161)

HBM9161Batoclimab (HBM9161) is a fully human monoclonal antibody (mAb) that can block FcRn-IgG interaction, accelerate the clearance of IgG in the body, and effectively treat pathological IgG-mediated autoimmune diseases. In the Phase III study of severe myasthenia gravis, Batoclimab demonstrated positive results, rapidly and significantly alleviating the clinical symptoms and improving the quality of life of patients, with good safety profile.Mazdutide (IBI362) is a GLP-1R/GCGR dual agonist jointly developed by Innovent Bio and Eli Lilly and Co. As a somatostatin analogue similar to human gastric inhibitory polypeptide (OXM), Mazdutide not only promotes insulin secretion, lowers blood sugar, and reduces weight by stimulating GLP-1R, but also increases energy consumption and enhances weight loss by stimulating GCGR, while improving liver fat metabolism. Mazdutide has shown excellent weight loss and hypoglycemic effects, as well as multiple metabolic benefits in reducing waist circumference, blood lipids, blood pressure, uric acid, liver enzymes, and liver fat content, as well as improving insulin sensitivity, in multiple clinical studies. Currently, five Phase III clinical studies of Mazdutide are underway in Chinese overweight or obese participants (GLORY-1 and GLORY-2) and type 2 diabetes participants (DREAMS-1, DREAMS-2, and DREAMS-3). Among them, the GLORY-1 and DREAMS-2 studies have achieved their primary endpoints.

Bartoli monoclonal antibody (HBM9161) is a fully human monoclonal antibody (mAb) that can block FcRn-IgG binding and accelerate the clearance of IgG in the body, thus effectively treating pathogenic IgG-mediated autoimmune diseases. In the phase III study of myasthenia gravis, Bartoli monoclonal antibody achieved positive research results and can quickly and significantly relieve clinical symptoms, improve patients' quality of life, and have good safety.

About Harbour BioMed

Harbour BioMed (stock code: 02142. HK) is a global biopharmaceutical company specializing in the research and commercialization of innovative drugs for cancer and immune diseases. The company quickly expands its pipeline of innovative drugs through independent research and development, joint development, and diversified cooperation models.

Harbour MiceHarbour Mice is a technology platform for generating fully human monoclonal antibodies (mAbs) in bispecific, bivalent (H2L2) and heavy-chain-only (HCAb) formats. The dual immune cell engager (HBICE) bispecific technology developed based on the HCAb antibody platform can achieve an anti-tumor effect that cannot be achieved by traditional drug combination therapy. Harbour Mice, HBICE and the single-B-cell clone screening platform together form the next generation innovative therapeutic antibody development engine at Harbour BioMed. For more information, please visit.Bispecific technology can achieve anti-tumor efficacy that traditional drug combination therapy cannot. Harbour MiceHBICETogether with the single B cell clone screening platform, it forms the next-generation innovative therapeutic antibody research and development engine of Hebo. For more news, please visit: news

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和铂医药宣布向国家药品监督管理局重新递交巴托利单抗治疗全身型重症肌无力的生物制品许可申请

Hengrui Medicine announced that it has resubmitted the biological product license application for Bartoli monoclonal antibody treatment of systemic severe myasthenia gravis to the National Medical Products Administration.

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