Cholestasis

CHICAGO, Oct. 30, 2024 (GLOBE NEWSWIRE) -- COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the first patient has been dosed in its Phase 1b/2a proof-of-concept clinical trial evaluating CNP-106 as a potential treatment for people living with generalized myasthenia gravis (gMG). “Currently approved treatments for gMG work via broad immunosuppression and do not address the underlying autoimmunity of the disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-106 has demonstrated a unique ability to induce tolerance to acetylcholine receptor (AChR), leading to improved muscle function in an experimental model of autoimmune myasthenia gravis. These results indicate that CNP-106 has the potential to be a first-in-class treatment that offers people living with gMG a disease modifying therapy without burdensome immune system suppression.” The Phase 1b/2a double-blind, randomized, placebo-controlled clinical trial (NCT06106672) will enroll up to 54 patients with gMG, aged 18-75, to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106. About CNP-106CNP-106 is a biodegradable nanoparticle that encapsulates acetylcholine receptor (AChR). gMG is primarily driven by the production of antibodies against AChR in the bloodstream. These antibodies either block or destroy the receptors for acetylcholine, which is a crucial neurotransmitter for muscle contraction. As gMG progresses, it may lead to serious health issues, such as myasthenic crisis, tumors of the thymus gland, thyroid imbalances, and other autoimmune diseases. CNP-106 is designed to potentially induce the downregulation of AChR T cell populations and AChR antibodies, leading to improved muscle function. About COUR Pharmaceuticals:COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases. COUR is currently enrolling patients in a Phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human clinical studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. For more information, please visit www.courpharma.com ContactsFor Investor RelationsBrian Bock, Chief Financial Officerbbock@courpharma.com For MediaJason Bracojbraco@lifescicomms.com

中国苏州，2024年10月28日—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。公司今日宣布，已顺利通过药品上市许可持有人（MAH）的生产和质量管理体系审核和现场检查，获得由上海市药品监督管理局颁发的《药品生产许可证》B证。 获得《药品生产许可证》B证标志着北海康成成为了一家具备药品研发、生产和销售的完整功能的生物制药公司；同时，作为生物药分段生产的试点企业，在监管部门的指导和帮助下，成功探索并实践了MAH对生物药分段生产的质量管理体系，为中国生物药分段生产未来的试点工作继续推进做出了我们应有的贡献。 本次B证的获得具有重要里程碑意义，这为后续药物，如治疗成人和儿童I型和III型戈谢病的CAN103（注射用维拉苷酶β ）的NDA申报以及上市后商业化生产提供了重要前提条件，也是CAN103成为首个在中国本土研发和生产的酶替代疗法的重要环节，必将为北海康成持续释放创新活力与潜力提供广阔空间。 《药品生产许可证》B证的取得说明了北海康成在公司组织架构、人员资质、药物生产和质量管理体系等方面已达到国家法律、法规的要求，完全有能力确保药品生产的质量和稳定性。凭借该许可证，我们具备了提交CAN103新药上市申请（NDA）的资质，CAN103有望成为北海康成在中国商业化的第三个罕见病治疗产品，同时也是首个内部研发的产品。 向上滑动阅览 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。公司目前拥有12个具有可观市场潜力的药物资产组合，其中包括3种已获批上市产品和9个在研药物。这些产品均针对一些较普遍的罕见疾病适应症， 比如亨特氏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。 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）、杜氏肌肉营养不良症和其他神经肌肉疾病的新型及潜在治愈性的基因疗法，并与世界领先的研究人员和生物技术公司合作。公司的全球合作伙伴包括不限于 Apogenix、GC Pharma、Mirum、药明生物、Privus、UMass Chan医学院、华盛顿大学医学部和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

Detailed safety and efficacy results to be presented Data show potential of CNP-104 to slow disease progression as demonstrated by statistically significant decrease in liver stiffness compared with placebo Oct. 21, 2024 -- COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for autoimmune diseases, today announced it will present detailed safety and efficacy results from the Phase 2a study of CNP-104 in Primary Biliary Cholangitis (PBC) via a late-breaking poster at The Liver Meeting® 2024, being hosted by the American Association for the Study of Liver Disease (AASLD) from November 15-18 in San Diego, California. Presentation Details: Title: Tolerogenic treatment with CNP-104 results in regulation Th17 cells slowing progression of PBC on liver stiffness Presenter: Paul M. Peloso, M.D., COUR Chief Medical Officer Publication Number: 5039 Poster Session Date and Time: Monday, November 18, 2024, from 1:00 to 2:00 p.m. PT CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic PDC-E2 T-cells that drive inflammation in bile ducts, resulting in clinically improved outcomes in liver health. In January 2022, CNP-104 received Fast Track Designation from the United States Food and Drug Administration (FDA), making it potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met. COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases. COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis. The content above comes from the network. if any infringement, please contact us to modify.