Multicenter, Open Label Trial to Investigate the Efficacy and Safety of a Single Oral Dose of 1.0 mg/kg Macimorelin Acetate as Growth Hormone Stimulation Test (GHST) in Pediatric Patients With Suspected Growth Hormone Deficiency (GHD)

This research study will find out if a new growth hormone stimulation test is safe and works as well as other tests to diagnose growth hormone deficiency (GHD) in children. The stimulation test will use a new growth hormone stimulating substance called macimorelin. By now, only adults in the USA can get this new stimulation test. The results of this study are expected to help children and teenagers with suspected GHD to get the macimorelin stimulation test.
The macimorelin test will be compared to a clonidine and an arginine test. Both are known standard stimulation tests. Altogether two macimorelin tests are planned to be performed in the study, to show how repeatable macimorelin tests results are (under a set of similar conditions).

开始日期2021-11-16

申办/合作机构

Æterna Zentaris, Inc.

NCT03844217

/ CompletedN/AIIT

Copeptin After an Oral Stimulation With Macimorelin in Healthy Volunteers - The Macicop-Study

This study investigates stimulation of the posterior pituitary gland by oral Macimorelin (a ghrelin receptor Agonist) to be a novel diagnostic test in the polyuria-polydipsia syndrome.

开始日期2019-03-07

申办/合作机构

Universitätsspital Basel

EUCTR2018-001988-23-HU

/ Completed临床2期

Open label, group comparison, dose escalation trial to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of macimorelin acetate after single oral dosing of 0.25 mg/kg, 0.5 mg/kg, and 1 mg/kg in pediatric patients with suspected growth hormone deficiency (GHD)

开始日期2018-11-07

申办/合作机构

Aeterna Zentaris GmbH

100 项与马西瑞林相关的临床结果

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100 项与马西瑞林相关的转化医学

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100 项与马西瑞林相关的专利（医药）

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项与马西瑞林相关的文献（医药）

2025-11-01·ACTA PHARMACOLOGICA SINICA

Molecular recognition of two approved drugs Macimorelin and Anamorelin by the growth hormone secretagogue receptor

Article

作者: Guo, Shi-Meng ; Wang, Yue ; Xu, H Eric ; Zhang, Yu ; Wang, Jiang ; Xie, Xin ; Liu, Hong ; Liu, Heng ; Sun, Jun ; Jiang, Yi ; Zhuang, You-Wen ; Hu, Wen ; Wang, Ruo-Lan

The growth hormone secretagogue receptor (GHSR) plays a critical role in regulating growth hormone release and metabolic homeostasis. Understanding the molecular mechanisms of ligand-GHSR recognition is essential for developing therapeutic interventions. In this study, we investigated the molecular recognition mechanisms of two clinically approved drugs: Macimorelin (used for diagnosing adult growth hormone deficiency) and Anamorelin (approved in Japan for cancer cachexia). Using high-resolution cryo-electron microscopy, we determined the structures of GHSR bound to Macimorelin and Anamorelin in complex with G_q proteins at resolutions of 2.63 Å and 2.52 Å, respectively. We revealed that both drugs occupied a bifurcated binding pocket divided by a conserved salt bridge between E124^3.33 and R283^6.55. Through systematic mutagenesis and functional studies, we identified the key residues underlying the higher binding affinity of Anamorelin compared to Macimorelin. In addition, structural comparison of GHSR in complex with different G protein subtypes elucidated the mechanisms driving G protein selectivity. Our results provide crucial insights into GHSR-drug interactions and offer valuable guidance for designing more selective and potent GHSR agonists.

2025-06-01·Pituitary

Consensus and controversies about diagnosing GH deficiency: a Delphi survey by the GH research society

Review

作者: Grimberg, A ; Karavitaki, N ; Higham, C E ; Bidlingmaier, M ; Giustina, A ; Johannsson, G ; Mauras, N ; Boguszewski, C L ; Choong, C S ; Strasburger, C J ; Schilbach, K ; Ross, R ; van Santen, H M ; Bollerslev, J ; Cianfarani, S ; Melmed, S ; Savendahl, L ; Fleseriu, M ; Juul, A ; Tritos, N A ; Yuen, K C J ; Radovick, S ; Collett-Solberg, P F ; Clayton, P E ; Christ, E ; Höybye, C ; Rosenfeld, R ; Horikawa, R ; Biller, B M K ; Neggers, S J C M M ; Chanson, P ; Dauber, A ; Ho, K K Y ; Jorgensen, J O L ; Hoffman, A R ; Lodish, M ; Miller, K K ; Cohen, P ; Boguszewski, M C S ; Brue, T ; Gebauer, J ; Luo, X ; Arlien-Søborg, M C ; Backeljauw, P

PURPOSE:

Biochemical tests are required for diagnosing GH-deficiency in children and adults, but controversies remain regarding diagnostic criteria and type of biochemical tests. The aim of the study is to map the clinical practices of GHD diagnosis in children and adults.

METHODS:

The Growth Hormone Research Society members initiated a Delphi survey of the diagnosis of GHD in children and adults. Pediatric (n = 18) and adult (n = 25) endocrinologists from 14 countries participated and rated their extent of agreement with 61 statements using a Likert-type-scale (1-7). Consensus was predefined as ≥ 80% of panelists rating their agreement unidirectionally as either ≥ 5 (agreement) or ≤ 3 (disagreement).

RESULTS:

The pediatric panel reached consensus on 17 of 29 (59%) statements on diagnosis in children, whereas the adult panel reached consensus on 28 of 32 (88%) statements on adult patients. There was general agreement to test for GHD in an appropriate clinical context and also on the timing of testing for GHD in both children and adults. A subnormal IGF-I level was considered diagnostic in both children and adults with panhypopituitarism. In children, there was consensus to recommend the arginine stimulation test and the glucagon test. The insulin tolerance test (ITT) was considered gold standard in adults and there was also consensus to recommend the macimorelin test. A stimulated GH cut-off < 5μg/l was consistent with severe GHD in children, whereas test-specific cut-offs were recommended in adults.

CONCLUSION:

Consensus on the GHD diagnosis was lower in pediatric practice, mainly with respect to choice and interpretation of GH stimulation tests.

2025-04-01·Drug Testing and Analysis

Elucidating important factors and corresponding method optimization for sensitive detection of small peptide drugs in human urine by solid‐phase extraction and UPLC‐HRMS: The influence of MS scan modes, protein precipitants, and ammonium formate

Article

作者: Liu, Yunxi ; Chang, Wei ; He, Genye ; Ma, Congcong ; Zhang, Yufeng ; Liu, Lu ; Dong, Tianyu ; Wang, Zhanliang ; Yan, Kuan

Abstract:

Small peptide hormones are widely used in sports as performance‐enhancing substances, making it crucial to develop sensitive analytical methods for their detection in doping control analysis. Various factors significantly affect analytical sensitivity, such as the selection of ultra‐performance liquid chromatography (UPLC) mobile phase, high‐resolution mass spectrometry (HRMS) scanning modes, and extraction solvents for pretreatment. Herein, comparative study approach was utilized to investigate the sensitivity of each peptide analyte under both full scan and parallel reaction monitoring (PRM) modes of HRMS and assess the effects of some protein precipitants as a part of extraction solvents on solid‐phase extraction (SPE). The results showed that full scan should be selected as the primary scan mode of HRMS, and the combination with PRM mode could effectively compensate for the limitations of full scan, and the addition of protein precipitants would adversely affect the detection of certain small peptide analytes. Meanwhile, influences of ammonium formate in reverse UPLC mobile phase on the charge state distribution of small peptides were investigated and elucidated. Based on these findings, a sensitive and reliable UPLC‐HRMS analytical method combining full scan and PRM mode was validated for screening and confirmation of 63 small peptide analytes after SPE, with limits of detection (LODs) ranging between 0.010 and 0.473 ng/ml and limits of identification (LOIs) ranging between 0.015 and 1.512 ng/ml. Additionally, suggestions were provided for the detection of [Arg8]‐vasopressin, dermorphin, and its analogues.

项与马西瑞林相关的新闻（医药）

2025-12-25

·上海冷冻电子显微镜

2025年XuLab年终汇总|一年27篇SCI不是我们的高峰，是我们走过的路!

点击蓝字关注我们 2025年终成果汇总 THE LATEST RESULTS 2025年已渐近尾声，在这一年里，徐Lab凭借着不懈的努力与执着追求，在科研领域持续深耕，不断收获累累硕果，书写着属于自己的精彩篇章。 01 论文发表成果斐然在课题组组长的高瞻远瞩与精心引领下，全体学生和工作人员齐心协力、奋勇拼搏，共同营造出积极向上、勇于创新的科研氛围。本年度，团队在学术期刊发表方面成绩卓著，已在正刊及子刊级累计发表论文达27余篇。这一数字不仅是对课题组过去一年辛勤付出的有力肯定，更是其在科研道路上不断攀登高峰的有力见证。 02 重大突破再谱新篇值得一提的是，继在《Nature》（2024）上首次揭示NET同源二聚化机制及抗抑郁药物识别机制后，2025年10月24日，临港实验室蒋轶研究员团队联合中国科学院上海药物研究所徐华强研究员、杨德华研究员团队，以及临港实验室王震研究员团队，在《Cell》上在线发表了题为“Unveiling conformation-selectivity regulation of the norepinephrine transporter”的研究论文。Cell | 蒋轶/徐华强/杨德华/王震团队提出去甲肾上腺素转运体构象选择性变构调控模型，为新一代抗抑郁药开发提供了新思路! 除了这一重大突破，课题组还有26篇子刊级文章发表，研究范围从G蛋白偶联受体解析到人工智能驱动下的产出，涵盖了多个前沿领域，充分展示了课题组广泛的科研视野和强大的科研实力。 03 黄金法则：高效科研，创新驱动揭秘徐华强课题组高效科研背后的“黄金法则”，犹如揭开一层神秘的面纱，让我们看到了其成功的秘诀。结构决定功能，解析驱动创新 G蛋白偶联受体（GPCR）是重要的药物靶点，其结构解析长期依赖传统X射线晶体学方法，存在诸多限制。冷冻电镜技术的突破使得科学家能在近生理状态下解析GPCR与其配体及G蛋白的复合物结构，极大地推动了相关功能机制的研究。基于结构的药物设计（SBDD）能够帮助开发更具选择性、副作用更小的药物分子。此外，针对GPCR信号通路的特异性调控，设计偏向性激动剂或拮抗剂，也为疾病个性化治疗提供了新方向。机制深度解析，靶向精准干预去甲肾上腺素转运体（NET）等神经递质转运体是抗抑郁药、兴奋剂等多种精神神经疾病药物的关键靶点。在对其研究中，课题组遵循多层次机制剖析的法则。在原子水平上，结合冷冻电镜和分子动力学模拟，如同显微镜下的精细观察，揭示底物识别、结合和转运的原子细节，让我们对 NET 的工作机制有了更深入的了解。研究转运体从向外开放到向内开放的完整构象变化周期，如同追踪一场精彩的舞蹈表演，了解其在不同状态下的变化规律，构建起一个完整的调控网络，为精准干预提供了理论依据。数据智能驱动，颠覆传统范式在当今科技飞速发展的时代，人工智能为科研带来了新的机遇和挑战。AlphaFold 革命准确预测蛋白质结构，如同拥有一本神奇的预言书，极大加速了靶点识别和验证阶段，节省了大量的时间和资源。生成式 AI 模型能够设计具有理想理化性质和结合特的全新分子结构，为药物研发提供了更多的可能性。强化学习优化通过循环的“设计 - 合成 - 测试”反馈，如同一个智能的优化大师，快速优化先导化合物，提高药物研发的效率和质量。 04 文化传承：火炬传递，突破无限随着科研工作的深入推进，从基础研究到结构解析，再到临床新药验证，一代代的科研人如同接力赛中的运动员，举起手中的火炬进行“代帮式”的传递。这种传承不仅传递了知识和技能，更传递了科研的精神和理念，使得徐Lab喜报频传，形成了课题组隐形的文化力量。文章的发表不应该仅仅是数字的积累，更是团队科研理念与方法论的胜利。在这种理念的指引下，课题组不断实现“从0到1”的科研突破，为推动科研事业的发展做出了重要贡献。 2025年即将结束，2026年的脚步正徐徐走来，祝愿徐Lab以及上海市高峰电镜中心继往开来，再攀高峰！ 06 2025年度徐华强课题组已发表论文汇总 1： Meng X, Li Y, Xu J, Wu K, Hu W, Wu C, Xu HE, Xu Y. Structural insights into the activation of the human prostaglandin E2 receptor EP1 subtype by prostaglandin E2. Proc Natl Acad Sci USA. 2025May20;122(20):e2423840122. doi: 10.1073/pnas.2423840122. Epub 2025May14. PMID: 40366695; PMCID: PMC12107139. 2: Wang RL, Sun J, Liu H, Guo SM, Zhang Y, Hu W, Wang J, Liu H, Zhuang YW, Jiang Y, Xie X, Xu HE, Wang Y. Molecular recognition of two approved drugs Macimorelin and Anamorelin by the growth hormone secretagogue receptor. Acta Pharmacol Sin. 2025 Nov; 46(11): 2998-3008. doi: 10.1038/s41401-025-01606-7. Epub 2025 Jun 20.PMID: 40542284; PMCID: PMC12552520. 3: He XH, Li JR, Xu J, Shan H, Shen SY, Gao SH, Xu HE. AI-driven antibody design with generative diffusion models: current insights and future directions. Acta Pharmacol Sin. 2025 Mar;46(3):565-574. doi: 10.1038/s41401-024-01380-y. Epub 2024 Sep 30. PMID: 39349764; PMCID: PMC11845702. 4: Gu YM, Yuan QN, Li X, He Q, Xu HE, Zhao LH. Structural and mechanistic insights into dual activation of cagrilintide in amylin and calcitonin receptors. Acta Pharmacol Sin. 2025 Aug 22. doi: 10.1038/s41401-025-01635-2.Epub ahead of print. PMID: 40847076. 5: Jiang M, Xu Y, Luan X, Wu K, Li Z, Xu HE, Zhang S, Jiang Y, Yin W. Structural basis of the cysteinyl leukotriene receptor type 2 activation by LTD4. Proc Natl Acad Sci U S A. 2025 Apr 15;122(15):e2417148122. doi: 10.1073/pnas.2417148122.Epub 2025 Apr 7. Erratum in: Proc Natl Acad Sci U S A. 2025 Jun 24;122(25):e2512181122. doi: 10.1073/pnas.2512181122. PMID: 40193607; PMCID:PMC12012480. 6: Xia R, Yuan Q, Wang N, Hou L, Abe J, Song J, Ito Y, Xu HE, He Y. Structural insight into GPR55 ligand recognition and G-protein coupling. Cell Res. 2025 Jan;35(1):76-79. doi: 10.1038/s41422-024-01044-w. Epub 2024 Oct 31. PMID:39482404; PMCID: PMC11701112. 7: Shen S, He X, Liu H, Hu W, Xu HE, Duan J. Cryo-EM structures of GnRHR: Foundations for next-generation therapeutics. Proc Natl Acad Sci U S A. 2025 Jun 24;122(25):e2500112122. doi: 10.1073/pnas.2500112122. Epub 2025 Jun 16. PMID:40523184; PMCID: PMC12207466. 8: Zhang H, Zhang T, Wang D, Dai A, Mao J, Chen Q, Du T, Lu X, Hao Y, Zhang C,Yin YL, Hu W, Pan B, Jin S, Jiang M, Si Y, Yuan Q, Wang MW, Zheng M, Wang Z,Yang D, Xu HE, Jiang Y. Unveiling conformation-selective regulation of the norepinephrine transporter. Cell. 2025 Nov 26;188(24):6861-6872.e14. doi: 10.1016/j.cell.2025.10.002. Epub 2025 Oct 24. PMID: 41138730. 9: Xu J, Wu Y, Xu Y, Li Y, He X, Zhang H, Wang JJ, Hou J, Li J, Hu W, Wu K, Yuan Q, Wu C, Xu HE. Molecular basis for ligand recognition and receptor activation of the prostaglandin D2 receptor DP1. Proc Natl Acad Sci U S A. 2025 Jun 3;122(22):e2501902122. doi: 10.1073/pnas.2501902122. Epub 2025 May 29. PMID:40440061; PMCID: PMC12146711. 10: You C, Jiang M, Gao T, Zhu Z, He X, Xu Y, Gao Y, Jiang Y, Xu HE. Decoding the structural basis of ligand recognition and biased signaling in the motilin receptor. Cell Rep. 2025 Mar 25;44(3):115329. doi: 10.1016/j.celrep.2025.115329.Epub 2025 Feb 22. PMID: 39987561. 11: You C, Guo S, Zhang T, He X, Gao T, Xin W, Zhu Z, Lu Y, Xu Y, Li Z, Zhang Y, Cheng X, Jiang Y, Xie X, Xu HE. Molecular mechanism of pH sensing and activation in GPR4 reveals proton-mediated GPCR signaling.Cell Discov. 2025 Jun 25;11(1):59.doi: 10.1038/s41421-025-00807-y. PMID: 40555728; PMCID:PMC12187918. 12: Sun D, Li X, Yuan Q, Wang Y, Shi P, Zhang H, Wang T, Sun W, Ling S, Liu Y, Lai J, Xie W, Yin W, Liu L, Xu HE, Tian C. Molecular mechanism of the arrestin-biased agonism of neurotensin receptor 1 by an intracellular allosteric modulator. Cell Res. 2025 Apr;35(4):284-295. doi:10.1038/s41422-025-01095-7.Epub 2025 Mar 21. PMID: 40118988; PMCID: PMC11958688. 13: Wu C, Zhang C, Jin S, Wang JJ, Dai A, Xu J, Zhang H, Yang X, He X, Yuan Q, Hu W, Xu Y, Wang M, Jiang Y, Yang D, Xu HE. Molecular mechanisms of urate transport by the native human URAT1 and its inhibition by anti-gout drugs. Cell Discov. 2025 Apr 1;11(1):33. doi: 10.1038/s41421-025-00779-z. PMID: 40169562;PMCID: PMC11962085. 14: Zhang H, Cui N, Ma X, Xu HE. Structural basis of augmenting taurine uptake by the taurine transporter in alleviating cellular senescence. Cell Res. 2025 May;35(5):385-388. doi: 10.1038/s41422-025-01090-y. Epub 2025 Mar 20. PMID:40108449; PMCID: PMC12012219. 15: Zhu X, Ping M, Liu H, Yu T, Jiang Z, Liu Z, Li C, Hou X, Chu Q, Li S, Mao C, Luo T, Kang C, Wang F, Yang C, Tang M, Jiang Z, Gao Z, Liu H, Xu HE, Tang B, Cheng X, Yin W, Zhou Y, Li P. Structural insights into the activation of TMEM175 by small molecule. Neuron. 2025 Nov 5;113(21):3567-3581.e9. doi:10.1016/j.neuron.2025.07.029. Epub 2025 Aug 26. PMID: 40865534. 16: He XH, Li JR, Shen SY, Xu HE. AlphaFold3 versus experimental structures: assessment of the accuracy in ligand-bound G protein-coupled receptors. Acta Pharmacol Sin. 2025 Apr;46(4):1111-1122. doi: 10.1038/s41401-024-01429-y. Epub 2024 Dec 6. PMID: 39643640; PMCID: PMC11950431. 17: Zhang T, Liu H, You C, Zhang Y, Xu Y, Pan B, Wu C, Jin S, Yin YL, Wu K, Chen Y, Sun H, Si Y, Tan Y, Yin W, Xu HE, Guo D, Jiang Y. Structural insights into antagonist recognition by the vasopressin V2 receptor. Nat Commun. 2025 Nov 4;16(1):9734. doi: 10.1038/s41467-025-64735-x. PMID: 41188237; PMCID:PMC12586607. 18: Li X, Zhang H, Hu W, Wu K, Li S, Jin S, Yin Y, Yuan Q, Xu HE, Pan B, Jiang Y. Structural basis of peptide recognition and modulation for neuropeptide FF receptors. Cell Rep. 2025 Sep 23;44(9):116160. doi:10.1016/j.celrep.2025.116160. Epub 2025 Aug 19. PMID: 40839429. 19: Luo P, Xin W, Guo S, Li X, Zhang Q, Xu Y, He X, Wang Y, Fan W, Yuan Q, Wu K, Hu W, Zhuang Y, Xu HE, Xie X. Structural insights into the agonist activity of the nonpeptide modulator JR14a on C3aR. Cell Discov. 2025 Jan 10;11(1):7. doi:10.1038/s41421-024-00765-x. PMID: 39788969; PMCID: PMC11718183. 20: Li J, Li C, Zhou Q, Han W, Fang M, Xu Y, Mai Y, Zhang Y, Cui J, Xu HE, Zhang Y, Yin W, Wang MW. Divergent activation patterns of BRS3 revealed by two Chinese herb-derived agonists. Acta Pharm Sin B. 2025 Oct;15(10):5231-5243. doi:10.1016/j.apsb.2025.06.025. Epub 2025 Jun 30. PMID: 41132856; PMCID:PMC12541581. 21: Cao Y, Zhang C, Yuan Q, Zhao F, Liu Q, Wu K, Hu W, He J, Li H, Xu HE, Yang D, Duan J. Mechanisms of chemokine recognition and receptor activation of chemokine receptor CCR7. Cell Rep. 2025 Nov 25;44(11):116582. doi:10.1016/j.celrep.2025.116582. Epub 2025 Nov 17. PMID: 41252250. 22: Jin SS, Zhang H, Yan JH, Wu CR, Cai XQ, Wu K, Wang MW, Xu HE, Yang DH, Jiang Y. Decoding ligand recognition and constitutive activation of histamine H3 and H4 receptors. Acta Pharmacol Sin. 2025 Aug 28. doi: 10.1038/s41401-025-01633-4. Epub ahead of print. PMID: 40877594. 23: Gao T, You C, Cao Y, Xu X, Yuan Q, Shen S, Xu HE, Duan J. Structural insights into hormone recognition and G-protein coupling of the urotensin-II receptor. J Biol Chem. 2025 Nov;301(11):110794. doi: 10.1016/j.jbc.2025.110794.Epub 2025 Oct 6. PMID: 41062066; PMCID: PMC12630362. 24: Wu Y, Zhang H, Xu J, Wu K, Hu W, He X, Wang G, Wu C, Xu HE. Structural insights into selective and dual antagonism of EP2 and EP4 prostaglandin receptors. EMBO J. 2025 Dec;44(23):7242-7262. doi: 10.1038/s44318-025-00611-0. Epub 2025 Oct 29. PMID: 41162752; PMCID: PMC12669672. 25: Shen SY, Li JR, Wang YS, Li SN, Xu HE, He XH. An update for AlphaFold3 versus experimental structures: assessing the precision of small molecule binding in GPCRs. Acta Pharmacol Sin. 2025 Dec;46(12):3355-3364. doi:10.1038/s41401-025-01617-4. Epub 2025 Jul 9. PMID: 40634710; PMCID: PMC12644867. 26： Li Y, Xing Z, Hu W, Wu K, Xu HE, Zhao LH. Structural insights into nonpeptide antagonist inhibition of somatostatin receptor subtype 5. Proc Natl Acad Sci U S A. 2025 Dec 23;122(51):e2522515122. doi: 10.1073/pnas.2522515122. Epub 2025 Dec 19. PMID: 41417603. 27：Li S, Yuan Q, Wu W, Chen Z, Chen D, Shan H, Chu Q, Hu W, Wu K, Liu T, Zhu Y, Hou L, Zhou J, Duan J, Duan J, Xu HE, Ma H.Structural Pharmacology of Bufotenine Derivatives in Activating the 5-HT1A Receptor for Therapeutic Potential in Depression and Anxiety. Research. 23 Dec 2025 Vol 8 Article ID: 0987 07 博后招聘我们长期招药学、化学、生物学、结构生物学、生物物理学、计算机科学等相关专业的博后，博后工作地址可以在上海、广州、深圳和中山等多家单位招聘启事｜广医徐有伟团队招聘博后，年薪23-30万+绩效奖励6-100万招聘启事｜中山药创院尹万超课题组诚聘博士后。 ---- 文字图片｜上海市高峰电镜中心编辑排版｜张书凤审核｜袁青宁 ---- 扫码关注我们获取更多资讯微信号｜SH_Cryo_EM

2025-05-26

·EINPresswire

COSCIENS Biopharma Inc. Acknowledges Receipt of Intended Director Nominations from Goodwood for the Upcoming Annual and Special Meeting of Shareholders

TORONTO, ONTARIO, May 26, 2025 (GLOBE NEWSWIRE) -- COSCIENS Biopharma Inc. (NASDAQ: CSCI) (TSX: CSCI) (“COSCIENS” or the “Company”), a life sciences company developing and commercializing a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products, today confirmed that its Board of Directors has received a letter from Goodwood Fund and Puccetti Funds Management Inc.(“Goodwood”) regarding six (6) director nominations for the Company’s upcoming Annual and Special Meeting of Shareholders. “We respectfully acknowledge receipt of the statement regarding the intentions from Goodwood and are carefully reviewing its contents,” said Ronnie Miller, Chair of the Board of COSCIENS Biopharma. “With this in hand, the Board will evaluate and determine its path forward and remains fully committed to acting in the best interests of COSCIENS and our shareholders.” The Company will continue to provide updates to shareholders related to this matter. About COSCIENS Biopharma Inc. COSCIENS is a life sciences company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products. Our technology includes proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt's Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. Our consolidated portfolio also includes macimorelin (Macrilen ® ; Ghryvelin ® ), the first and only U.S. FDA and European Medicines Agency approved oral test indicated for the diagnosis of adult growth hormone deficiency (“AGHD”). The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol "CSCI". For more information, please visit COSCIENS' website at www.cosciensbio.com . Forward-Looking Statements Certain statements in this news release, referred to herein as "forward-looking statements", constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended, and "forward-looking information" under the provisions of Canadian securities laws. All statements, other than statements of historical fact, that address circumstances, events, activities, or developments that could or may or will occur are forward-looking statements. When used in this news release, words such as "anticipate", "assume", "believe", "could", "expect", "forecast", "future", "goal", "guidance", "intend", "likely", "may", "would" or the negative or comparable terminology as well as terms usually used in the future and the conditional are generally intended to identify forward-looking statements, although not all forward-looking statements include such words. Forward-looking statements in this news release include, but are not limited to, statements relating to: our plans for the Phase 2a Clinical Efficacy Study of its Avenanthramides tablets, our goal to commercialize our nutraceutical products (including our OBG chewable bar and YBG powder), the potential of our PGX technology and our goals and expectations regarding our other plans related to the development, manufacture or commercialization of our products. Forward-looking statements are necessarily based upon a number of factors and assumptions that, while considered reasonable by the Company as of the date of such statements, are inherently subject to significant business, economic, operational and other risks, uncertainties, contingencies and other factors, including those described below, which could cause actual results, performance or achievements of the combined Company to be materially different from results, performance or achievements expressed or implied by such forward-looking statements and, as such, undue reliance must not be placed on them. Forward-looking statements involve known and unknown risks and uncertainties which include, among others: the combined Company's present and future business strategies; operations and performance within expected ranges; anticipated future cash flows; local and global economic conditions and the environment in which the combined Company operates; anticipated capital and operating costs; uncertainty in our revenue generation from our marketed products, product development and related clinical trials and validation studies; results from our products under development may not be successful or may not support advancing the product; the failure of the DETECT-trial to achieve its primary endpoint in children (CGHD) may impact the market for macimorelin (Macrilen ® ; Ghryvelin ® ) in adults (AGHD) and the existing relationships we have for that product; ability to raise capital and obtain financing to continue our currently planned operations; our now heavy dependence on sales by and revenue from our main distributor of our legacy Ceapro products and its customers, the continued availability of funds and resources to successfully commercialize our products; the ability to secure strategic partners for late stage development, marketing, and distribution of our products; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to protect and enforce our patent portfolio and intellectual property; and our ability to continue to list our common shares on the NASDAQ Capital Market. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those discussed in our Annual Report on Form 20-F and MD&A filed under the Company's profile on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov . We disclaim any obligation to update any such risks or uncertainties or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this news release. Issuer: Anna Biehn Chief Executive Officer E: ABiehn@cosciensbio.com Investor Contact: Jenene Thomas JTC Team T: (US): +1 (908) 824-0775 E: csci@jtcir.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

临床2期

2025-01-01

·GlobeNewswire-Health Care

COSCIENS Biopharma Inc. Provides Corporate Update and Highlights Pipeline Prioritization

Company leveraging core competencies to develop and commercialize natural-based products to improve health and wellness Streamlined efficiencies and cost cutting measures taken to refine operations and development pathway forward New corporate presentation outlining key development areas to be available in January 2025 Dec. 23, 2024 -- COSCIENS Biopharma Inc. (NASDAQ: CSCI) (TSX: CSCI) (“COSCIENS” or the “Company”), a specialty biopharmaceutical company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products, today provided a corporate update and reiterated its pipeline prioritization. “Over the course of the last several months following the merger, we have taken strategic and deliberate steps to evaluate and prioritize our combined pipeline of products and programs. Through this effort, we have thoroughly evaluated the potential of our assets and prioritized the areas that we believe are high-value opportunities, and we plan to move forward focusing our resources on advancing a pipeline of natural-based products leveraging our core technologies. We believe that this focus, spanning cosmeceuticals, nutraceuticals and pharmaceuticals, positions us with near and long-term life science growth drivers and the potential to become a global leader in natural-based products for health and wellness,” stated Gilles Gagnon, M.Sc., MBA, President and CEO of COSCIENS. “In addition, we have continued to make progress driving revenues and growing our active ingredients base business. We are looking to kick off 2025 with a clear line of sight towards execution of our business plan and believe we are well-positioned for a transformational year.” Pharmaceuticals: Avenanthramides Tablets (Avs) in Development as an Anti-Inflammatory: The Phase 1-2a clinical trial being conducted at the Montreal Heart Institute has fully completed the Phase 1 part of the study with 72 subjects tested. No significant side effects have been observed from ascending doses ranging from 30 mg to 960 mg. The Company expects the Phase 2a efficacy study will be initiated in Q1 2025, with expected completion in Q3 2025. Cosmeceuticals: Enriched Oat Flour with High Concentration of Avenanthramides: We have successfully produced and sold small batches for inclusion in cream formulations. JuventeDC Product Line: New website, juventedc.com, was launched on December 19, 2024. Nutraceuticals: Yeast Beta Glucan (YBG) Powder - Immune Booster: Our YBG product has been successfully manufactured as part of our PGX scale up project in Edmonton, Alberta. Our YBG product is being finalized in capsule form with the goal to commercialize it as an immune booster in Q2 2025. Oat Beta Glucan (OBG) Chewable Bar – Cholesterol Reduction: We have successfully developed a unique, standardized formulation for a healthy confection which includes a high concentration of OBG with daily dosage according to approved claims in 10 developed countries. The substantive claims include reduction of LDL cholesterol, supporting cardiovascular health reduction of Type 2 diabetes risks, source of fiber for the maintenance of general good health and support of a healthy digestive system. COSCIENS’ team anticipates the official commercial launch of its chewable bar OBG natural health product in Q1 2025, marking an important chapter in the Company's journey toward promoting wellness while expanding its business model. Macimorelin: On August 27, 2024, the Company announced that the Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD") had failed to meet its primary endpoints according to the definitions in the study protocol. Based on the results of the study, we are prioritizing our pipeline moving forward and repositioning the Company as a pure play Life Science’s business offering natural-based products. Therefore, while maintaining the current commercial indication for the use of macimorelin for the diagnosis of Adult Growth Hormone Deficiency (“AGHD”), the Company has decided to not make any future investments in macimorelin for the diagnosis of CGHD. Consequently, in order to further ensure the streamlining of operations and reduce costs, the roles of some additional members of management have been terminated, including Dr. Nicola Ammer, Chief Medical Officer, which will be ending on March 31, 2025. “On behalf of the entire Board and Company, we are grateful to the members of the team for their years of dedicated service. Nicola has been a long-standing, valued member of our team and provided years of expertise for the development of macimorelin (Macrilen®; Ghryvelin®), the first and only U.S. FDA and European Medicines Agency approved oral test indicated for the diagnosis of adult growth hormone deficiency. We are thankful to each member of our team and sincerely thank them for the key roles they played,” concluded Mr. Gagnon. The Company plans to release a new corporate presentation outlining key development areas and upcoming milestones in January 2025. COSCIENS is a specialty biopharmaceutical company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products. Our technology includes proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt's Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. Our consolidated portfolio also includes macimorelin (Macrilen®; Ghryvelin®), the first and only U.S. FDA and European Medicines Agency approved oral test indicated for the diagnosis of adult growth hormone deficiency (“AGHD”). The Company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol "CSCI". For more information, please visit COSCIENS' website at www.cosciensbio.com. The content above comes from the network. if any infringement, please contact us to modify.

临床研究

100 项与马西瑞林相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10562	马西瑞林	V04CD06	DB13074

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
生长激素缺乏症	美国	Æterna Zentaris, Inc.	2017-12-20
生长激素缺乏症	美国	Novo Nordisk, Inc.	2017-12-20

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
生长激素缺乏症与垂体异常	临床3期	美国	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	奥地利	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	法国	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	德国	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	意大利	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	波兰	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	塞尔维亚	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	西班牙	Æterna Zentaris, Inc.	2015-12-03
生长激素缺乏症与垂体异常	临床3期	英国	Æterna Zentaris, Inc.	2015-12-03
恶病质	临床2期	美国	Æterna Zentaris, Inc.	2012-05-01

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04786873 (DETECT, GlobeNewswire) 人工标引	临床3期	生长激素缺乏症	102	macimorelin	繭淵繭鹹製夢齋構簾壓(糧獵遞鏇鏇簾鬱餘鹽鏇) = Based on this, top-line data present an ‘optimal’ growth hormone (GH) cut-off point of 25.59 ng/mL, which is substantially higher than the standard cut-off points of 7-10 ng/mL as defined in current guidelines with existing tests. This surprisingly high cut-off point leads to a failure in the sensitivity and specificity assessments. 艱積觸網製積製憲選夢 (顧構繭顧淵膚繭觸壓壓 )	积极	2024-08-27
NCT01614990 (CTgov)	临床2期	恶病质	15	Macimorelin (Macimorelin)	夢範獵網製積膚獵齋簾(淵繭觸憲衊築窪鏇鹹構) = 淵窪選繭積顧網淵憲艱願淵襯範醖夢範醖廠鏇 (積顧膚積積餘齋築構顧, 0.3) 更多	-	2024-03-19
				Placebo (Placebo)	夢範獵網製積膚獵齋簾(淵繭觸憲衊築窪鏇鹹構) = 繭醖鏇築壓鑰簾夢襯願願淵襯範醖夢範醖廠鏇 (積顧膚積積餘齋築構顧, 0.2) 更多
Pilot clinical trial of macimorelin (Pubmed \| J Cachexia Sarcopenia Muscle)	N/A	恶病质 plasma insulin‐like growth factor (IGF)‐1	15	Macimorelin 0.5 mg/kg	鏇積衊選築鹽齋淵範憲(鏇齋壓壓選遞選網廠夢) = 願蓋壓遞範壓顧繭襯艱簾願鏇願顧獵襯鬱廠艱 (繭鬱壓願糧蓋醖鬱蓋鑰 ) 更多	积极	2023-04-01
				Macimorelin 1.0 mg/kg	鏇積衊選築鹽齋淵範憲(鏇齋壓壓選遞選網廠夢) = 夢繭網簾範顧齋衊淵築簾願鏇願顧獵襯鬱廠艱 (繭鬱壓願糧蓋醖鬱蓋鑰 )
NCT03844217 (Pubmed \| Pituitary)	N/A	-	28	Macimorelin 0.5 mg/kg	願蓋淵網鹽襯構衊衊選(襯襯鑰齋繭夢鏇廠顧鹹) = 鹹鏇網遞願齋壓顧壓壓鹽糧夢夢夢願醖醖鏇網 (夢襯廠齋積糧餘廠餘鏇, 56.5 ~ 110.96) 更多	-	2021-02-22
SAT-428 (ENDO2019)	临床3期	生长激素缺乏症	70	Macimorelin (Group A (high likelihood of GHD))	淵獵膚齋製壓夢獵蓋廠(鏇壓選積願築鹽鬱膚齋) = 壓鏇鬱網廠壓鹽蓋網遞憲築製構鏇齋構構齋網 (廠糧鹹積築鬱蓋鑰夢選 ) 更多	积极	2019-04-30
OR16-1 (ENDO2019 \| Pubmed)	N/A	垂体性侏儒症	-	Macimorelin	鑰簾廠夢願餘願繭廠鹹(鏇構築鹽夢簾窪壓觸襯) = 觸鏇鏇艱艱齋夢鏇範壓願襯鹽鏇鏇窪選鬱憲積 (淵窪糧鏇蓋範廠艱淵鹽 )	积极	2019-04-30
				Macimorelin (Insulin Tolerance Test (ITT))	鑰簾廠夢願餘願繭廠鹹(鏇構築鹽夢簾窪壓觸襯) = 網繭觸構膚憲積窪夢獵願襯鹽鏇鏇窪選鬱憲積 (淵窪糧鏇蓋範廠艱淵鹽 )
OR32-4 (ENDO2019)	临床3期	GH	140	Macimorelin	糧蓋淵築膚齋齋獵鹹壓(衊遞積繭選願襯繭窪衊) = 窪糧窪淵鹹築餘衊獵齋積製衊製範餘鑰蓋積鑰 (鏇鬱襯鑰鏇襯醖壓製積, 85 ~ 95) 更多	积极	2019-04-30
				Macimorelin (Insulin Tolerance Test (ITT))	糧蓋淵築膚齋齋獵鹹壓(衊遞積繭選願襯繭窪衊) = 夢衊簾願餘糧鹹構壓糧積製衊製範餘鑰蓋積鑰 (鏇鬱襯鑰鏇襯醖壓製積, 80 ~ 92) 更多
NCT02558829 (Pubmed \| J Clin Endocrinol Metab \| ENDO2018)	临床3期	生长激素缺乏症	157	Macimorelin	憲繭廠積網艱築艱鏇齋(蓋觸蓋選鹹網鹹鹽製網) = 鏇壓構觸廠鹽鹽夢壓鏇淵淵選憲鹹夢構廠簾鬱 (範艱艱觸簾夢鑰顧鹹鹽 )	-	2018-08-01
				Macimorelin (Insulin tolerance test (ITT))	憲繭廠積網艱築艱鏇齋(蓋觸蓋選鹹網鹹鹽製網) = 鏇艱膚觸憲獵窪壓醖簾淵淵選憲鹹夢構廠簾鬱 (範艱艱觸簾夢鑰顧鹹鹽 )
NCT00448747 (Pubmed \| J Clin Endocrinol Metab)	N/A	垂体性侏儒症	-	Macimorelin	鏇鏇夢獵積積鏇窪夢獵(廠製艱齋醖鏇鑰憲鹽糧) = 製繭網網遞淵蓋壓獵網製觸壓夢淵鏇鏇構鹽淵 (製選窪顧淵鏇繭製壓製 )	-	2013-06-01
				Arginine+GHRH	鏇鏇夢獵積積鏇窪夢獵(廠製艱齋醖鏇鑰憲鹽糧) = 構鏇範選糧顧積築獵繭製觸壓夢淵鏇鏇構鹽淵 (製選窪顧淵鏇繭製壓製 )