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BASEL, Switzerland I October 23, 2024 I AMYRA Biotech AG (“AMYRA”), a company developing novel, oral digestive enzyme therapeutics for gastrointestinal diseases announced the peer-reviewed publication of its clinical proof-of-principle study with its lead product AMYNOPEP in Frontiers in Immunology – Nutritional Immunology ( https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2024.1425982/full ). AMYNOPEP is the first and only gluten-digesting enzyme combination that supports and enhances the activity of critical endogenous enzymes on the lining of the small intestine. AMYNOPEP is designed to break down hard-to-digest gluten peptides into harmless and absorbable single amino acids and dipeptides, thereby supporting individuals with gluten-related health disorders in avoiding exposure to inflammatory peptides. AMYRA’s first-in-human clinical study assessed gluten-digesting enzyme efficacy using high-resolution analytics and demonstrated in near-real-time that AMYNOPEP swiftly and significantly enhanced the digestion of proteolytically-resistant, immunogenic gluten peptides in healthy volunteers. A team of researchers from AMYRA and the University of Greifswald (Germany) evaluated AMYNOPEP’s digestive efficacy on the degradation of a long, proteolytically resistant gluten peptide known as the 33-mer, which has been associated with an adverse immune response in celiac disease. The digestion efficacy of AMYNOPEP on the 33-mer was first assessed in vitro , demonstrating end-to-end degradation of the gluten immunogenic peptide into single amino acids and dipeptides. These in vitro results were then validated in a crossover study of 14 healthy volunteers who received stable isotope-labelled 33-mer with and without AMYNOPEP. The study clearly showed that AMYNOPEP enhanced 33-mer digestion kinetics within minutes, significantly increasing levels of stable isotope-labelled amino acids detected in blood. Strikingly, AMYNOPEP improved 33-mer digestion even under “stress-test” conditions that included competing food substrates such as hydrolyzed whey protein and wheat gluten, revealing up to 3 times higher maximum concentrations of labelled amino acids in blood with AMYNOPEP compared to control. “The positive results of this proof-of-principle study pave the way for a new therapeutic enzyme paradigm leveraging specific exopeptidase combinations that support, enhance, or even replace the activity of critical endogenous enzymes lining the small intestine,” said Dr. Sulay Mourabit, CSO of AMYRA and co-lead author on the study. In the human gastrointestinal tract, most exopeptidases are found at the intestinal brush border membrane, which is sensitive to inflammatory damage. These exopeptidases are essential for completing protein digestion by cleaving peptides from end-to-end, generating absorbable amino acids. He added: “As single agents, exopeptidases are inefficient as they have limited substrate range, and we see this with DPP-IV supplements currently on the market. AMYRA specializes in developing unique combinations of exopeptidases with complementary and enhanced activity, designed to thoroughly break down proteolytically resistant food peptides. Ultimately, complex digestive diseases such as celiac may require a combination of treatment approaches. Given the critical role of the exopeptidase machinery in the human GI tract, and the vulnerability of the intestinal brush border membrane to chronic inflammatory damage, we’re excited to see the role that AMYNOPEP will play in digestive disease therapeutics.” “This is an encouraging and far-reaching proof-of-principle study demonstrating the validity and therapeutic relevance of our approach,” said Dr. Werner Tschollar, CEO and founder of AMYRA and senior author of the study. “As a next step, AMYRA is planning further clinical studies to investigate AMYNOPEP’s enzyme efficacy, immune response suppression, and symptom relief in patients with celiac disease. We strongly believe that supplementing the body with specific exopeptidases will prove to be a valuable adjunct therapy to the gluten-free diet, which alone represents a suboptimal solution.” About AMYRA Biotech AG AMYRA Biotech AG is a privately held biotech company developing novel therapeutic solutions for patients suffering from dietary peptide-driven disorders including celiac disease, gluten sensitivity, and other gastrointestinal disorders. The Company’s proprietary enzyme combinations have the unique capability of rapidly and systematically digesting proteolytically resistant gluten peptides from one end to the other, generating harmless and absorbable degradation products in the small intestine.  AMYRA is based in Basel, Switzerland. For further information, please visit www.amyra.com. About AMYNOPEP Several digestive enzyme supplements on the market and therapeutics under development seek to help the body break down hard-to-digest gluten peptides, which are known to trigger adverse reactions in individuals with gluten-related health disorders. The vast majority of these are endopeptidases that target gluten digestion in the stomach and generate smaller protein fragments (peptides) that may retain the ability to trigger an inflammatory response. In contrast, AMYNOPEP is a novel therapeutic enzyme principle based on specific combinations of exopeptidases that thoroughly degrade gluten peptides from end-to-end in the small intestine, generating individual, easily absorbable, and harmless amino acids and dipeptides that are incapable of triggering an immune response. AMYRA’s lead exopeptidase combination, AMYNOPEP, is capable of digesting proline-rich, hard-to-digest peptides found in foods such as gluten, which is notoriously linked to gastrointestinal distress. SOURCE: AMYRA Biotech

近日，上海医药集团股份有限公司控股子公司上海禾丰制药有限公司收到国家药品监督管理局颁发的关于硫酸阿托品注射液的《药品补充申请批准通知书》（通知书编号：2024B03849），该药品通过仿制药质量和疗效一致性评价。 01 基本情况 药品名称： 硫酸阿托品注射液 剂    型： 注射剂 规    格： 1ml:0.5mg 注册分类： 化学药品 申 请 人： 上海禾丰制药有限公司 原批准文号： 国药准字H31021172 审批结论： 本品通过仿制药质量和疗效一致性评价 02 相关信息 硫酸阿托品注射液主要用于各种内脏绞痛，如胃肠绞痛及膀胱刺激症状；全身麻醉前给药、严重盗汗和流涎症；迷走神经过度兴奋所致的窦房阻滞、房室阻滞等缓慢型心律失常，继发于窦房结构功能低下而出现的室性异位节律；抗休克；以及解救有机膦酸酯类中毒。该药品由田边三菱研发，于1947年在日本上市。2023年3月，上药禾丰就该药品仿制药一致性评价向国家药监局提出申请并获受理。 根据国家相关政策，通过一致性评价的药品品种在医保支付及医疗机构采购等领域将获得更大的支持力度。因此上药禾丰的硫酸阿托品注射液通过仿制药一致性评价，有利于扩大该药品的市场份额，提升市场竞争力，同时为公司后续产品开展仿制药一致性评价工作积累了宝贵的经验。 来源：【信谊良药】微信公众号

2024年8月28日，由中国儿童药物研发与产业化生态圈联合中国儿科人群临床试验协作网共同主办，国家儿童医学中心首都医科大学附属北京儿童医院与中国医药工业信息中心共同承办的“儿童用中成药创新发展研讨会”成功举办。会议邀请了政产学研医媒体等多方代表，吸引了上万人次产业同仁直播参会。 中医药作为中华民族的瑰宝，历经数千年而不衰，它独特的理论体系和治疗方法，在现代医学快速发展的今天，依然焕发着勃勃生机。特别在儿童健康领域，许多历史悠久的中药制剂因其显著的临床疗效，护佑着孩子们的成长。本次研讨会携手多位中医药领域专家，共话中成药的传承与创新，共谋儿童用中成药的美好未来。 以传世名药王氏保赤丸为例，介绍了它的非凡历程，从诞生到创新发展，每一步都凝聚着中医中药人的智慧和汗水。在传承和荣耀背后，是不断的探索，从创新到药理学的研究，从循证医学研究到国际化的探索，始终走在了中药发展的前沿，既保留了传统中药的精髓，又在现代科学的指引下焕发着新的光芒。中医药学这一源远流长博大精深的文化瑰宝，历尽沧桑而愈显其独特的魅力。 中成药如何精准定位西医病种，在临床中如何合理且准确的使用中成药，这是一个很大的课题。能达到精准的使用中成药来治疗儿科疾病，相关循证医学的研究之路还很长。精准治疗包括三个方面：第一是精准定位；第二是精准评价；第三是精准研发。除此之外，儿童中成药如何与其他药物配伍使用也是今后需要研究与探讨的重要课题。 当前我国中成药市场稳步增长，发展动力强劲，尤其儿童中成药备受关注，在临床治疗儿童疾病中占据重要地位。中成药精准定位西医病种及循证医学研究，有助于临床大夫更好地使用药物。中成药对肠道菌群稳态的调控与毒理学作用研究，为中成药在世界范围内的临床应用提供了可能。中成药的生命力在于临床疗效，既需要通过询证方法展现它的疗效和安全性，也需要通过基础研究方法增强中药自信和他信力。 功能性胃肠病是与年龄相关的慢性反复发作的胃肠道功能异常，包括婴儿反流综合征、周期性呕吐、婴儿肠绞痛、功能性腹泻和功能性便秘等七个病种。我国婴儿功能性消化不良综合征的患病率为38%，经过随机双盲安慰剂对照的多中心临床研究，验证王氏保赤丸治疗应用功能消化不良综合征的疗效是确切的，能有效消除和缓解消化不良等相应症状，是一个疗效突出又安全的药物。 中药的发展历史悠久。王氏保赤丸的临床应用超过100年，安全性得到临床验证，可适用于不同年龄段的患者。虽然中药成分复杂，研究难度较大，但生物学活性可能不弱于单一成分的西药，且疗效优异，适应症广泛，如王氏保赤丸能双向调节肠道蠕动，同时治疗腹泻和便秘。中药的治疗方案与西药不同，能针对患者而非特定病症，提高人体生命力，这也是中药优于西药的优势。 很多中药还维持传统的剂型和给药方式，基于这一点，在中药领域针对给药方式和给药途径的创新较多，希望在中药制剂研究中，针对临床需求开发更多更适合临床使用的中药。在传承临床有效的儿童药时，还要注意对传统工艺进行合理改造，以便更好的通过质量控制来发挥药效，进一步提升传统中药的有效性和安全性。 研究儿童药，特别是儿童专用中成药的难度较大：能够完成儿童药的临床研究，特别是儿童专用中成药的研究单位数量不足；一些临床合并症也缺乏临床技术指导原则；临床方案的制定过程也充满挑战。对此，希望药监局和药审中心从政策方面更好的推动相关工作，鼓励和支持儿童药企业和儿科专家更好的开展新药研发。 数据技术和人工智能的发展给人们生活和用药带来不同的变化，中医对自然和人体的认识也需要适应新的变化。患者对用药提出更快捷、更方便、更高效的需求，促进了以传统汤药为主的治疗过程向新的制剂、新的临床治疗方法转变，这种创新包括组方创新、剂型创新、口感创新、给药方式创新、老药新用以及研究方法的创新。 目前儿童外用制剂较少，在儿童外用药研究中发现，儿童依从性较差，可以尝试儿童经鼻吸入给药或经皮渗透给药。对于已有药物来说改为外用制剂后可以扩大应用范围和适应症。医院儿科住院体量虽小，但使用的实验技术如清洗、敷贴等方法，为治疗提供了另一条途径和渠道，希望临床医生可以为企业在外用制剂开发中贡献方法和技术。 王氏保赤丸是经典的儿童用药，在消化和呼吸系统上有广泛的临床应用，服用方便，儿童用药剂量也小。但其也存在不足之处，如适应症宽泛，医生用药有时不能明确定位，质量控制也不够严谨。在传承过程中针对这些不足，通过工艺标准化和质量控制方法如指纹图谱，保留传统工艺并提升质量标准。未来计划进行更多临床机制研究，以提升儿童中成药的品质，更好地服务于临床。 从企业角度来看，儿童药的研发，最难的还是临床阶段。今年上市的两个品种在二期临床试验中遇到各种问题，得到了各方专家的指导。另外，还存在一些难点，如剂量探索研究，或者开展临床试验时可备选机构数量不足等。希望行业专家可以联合药监部门，出台相应政策或指导原则，便于企业新药研发项目的推进。 葫芦娃专注于儿童医药开发，尤其是儿科中成药领域。企业在选择一类新药开发时，会从中药的22个优势病种中选择对应的处方，在选择新药时，关注药材的多元性、可及性和毒性，对于儿童用药要特别注意避免选择含毒性的药材。对于二类中药，企业主要关注适应症的改变和儿童用药剂型的增加，也在探索通过真实世界研究来改善儿童用药的口感。 我国儿童用药市场不成熟，品种少，剂量模糊，规格缺乏，主要集中在抗生素、感冒、解热镇痛、清热等常见病用药。2023年以来，国家发布了十项鼓励儿童用药的研发政策，但在中成药方面，过去十年儿童中成药获批上市及获批临床的药品数量很少。对此，人民日报健康客户端开设了儿童健康频道，重点关注儿童创新药和中成药，发布了约200篇关于儿童用药的报道，旨在呼吁解决儿童用药短缺问题。 感谢精华制药集团股份有限公司对本次活动的大力支持！ 会议预告 2024年（第41届）全国医药工业信息年会将于9月6日-9日在成都召开，届时儿童药物创新发展大会将在9月8日同期举办，期待在成都与各位相聚！ END 如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001