A Multi-Center, Single-Dose and Repeat-Dose Over Eight Weeks, Sequential Cohort Study to Evaluate Safety and Tolerability As Well As Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% Administered Subcutaneously in Subjects with Alpha1-Antitrypsin Deficiency

The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).

开始日期2021-08-13

申办/合作机构

Grifols Therapeutics, Inc.

NCT04547140

/ Terminated临床2期

A Multicenter, Randomized, Double-blind, Placebo-Controlled, Parallel Group Study to Evaluate the Safety and Efficacy of Liquid Alpha1-Proteinase Inhibitor (Human) Plus Standard Medical Treatment (SMT) Versus Placebo Plus SMT in Hospitalized Subjects With COVID-19

The purpose of the study is to determine if Liquid Alpha1-Proteinase Inhibitor (Human) (Liquid Alpha1-PI) plus SMT can reduce the proportion of participants dying or requiring intensive care unit (ICU) admission on or before Day 29 or who are dependent on high flow oxygen devices or invasive mechanical ventilation on Day 29 versus placebo plus SMT in hospitalized participants with COVID-19.

开始日期2021-01-29

申办/合作机构

Grifols Therapeutics, Inc.

NCT04495101

/ Completed临床2期

A Multicenter, Randomized, Open-label, Parallel Group Pilot Study to Evaluate the Safety and Efficacy of Prolastin Plus Standard Medical Treatment (SMT) Versus SMT Alone in Hospitalized Subjects With COVID-19

The primary objective of the study is to determine if Prolastin plus SMT can reduce the proportion of subjects dying or requiring intensive care unit (ICU) admission on or before Day 15 or who are dependent on invasive mechanical ventilation on Day 15 versus SMT alone in hospitalized subjects with Coronavirus disease 2019 (COVID-19).

开始日期2020-07-29

申办/合作机构

Instituto Grifols SA

100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的临床结果

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100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的转化医学

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100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的专利（医药）

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项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的文献（医药）

2025-04-01·Phytomedicine

Apigenin alleviates inflammation as a natural IRAK4 inhibitor

Article

作者: Ji, Kailong ; Gongpan, Pianchou ; Zhang, Yufei ; Wang, Xue ; Xu, Tingting ; Li, Jia ; Sun, Yili ; Kong, Lingmei ; Chen, Hongman ; Geng, Chang-An ; Song, Qishi

BACKGROUNDUncontrolled inflammation is a key factor in the development of many diseases, and targeting pivotal kinases involved in the inflammatory response, such as interleukin-1 receptor-associated kinase 4 (IRAK4), holds promise for the treatment of inflammatory conditions. Apigenin (Api) is a popular element in numerous plants, possessing anti-inflammatory properties. Many studies have shown that Api modulates NF-κB signaling and MAPK cascade to reduce inflammation, but the exact mechanisms by which Api regulates these pathways remain unclear.PURPOSEThe aim of this study was to investigate the role of Api on acute inflammation and its specific mechanism in mediating inflammation.METHODSThe dextran sulfate sodium (DSS) induced ulcerative colitis (UC) model and LPS induced acute inflammation mouse model were established to investigate the anti-inflammatory effects of Api. Subsequently, the anti-inflammatory activity of Api was validated in vitro and vivo by RNA-seq, qPCR, Western blot, cytokine ELISA, immunofluorescence and histological analysis. A series of experiments were performed to study the effects of Api on IRAK4, including ADP-Glo™ kinase assay, surface plasmon resonance (SPR), cellular thermal shift assay (CETSA), and molecular docking simulation. The targeting of Api to IRAK4 was verified by IRAK4 inhibitor and siRNA.RESULTSOral administration of Api significantly ameliorated inflammatory conditions in the LPS induced acute inflammation and DSS induced UC mouse models. Furthermore, Api inhibited the expression of interleukin and chemotactic factor genes and downregulated the immune response of macrophages at the transcriptome level. Mechanistically, Api acted as a novel IRAK4 inhibitor, inhibiting kinase activity by direct binding to IRAK4 (Kd = 4.78 μM) with an IC₅₀ of 1.74 μM, interfering with extracellular signaling to the NF-κB and MAPK pathways, and reducing the expression of pro-inflammatory factors in an IRAK4 dependent manner.CONCLUSIONIn this study, Api was identified for the first time as a natural IRAK4 inhibitor that suppresses cytokine signaling pathways and modulates the immune response at the level of the transcriptome. The results provided valuable insights into the specific mechanism of Api inhibition of inflammatory activation and shed light on opportunities for the development of novel IRAK4 inhibitors based on Api, which is found in various plants.

2025-02-13·Journal of Medicinal Chemistry

Development of a Novel Human Serum Albumin-Based Tool for Effective Drug Discovery: The Investigation of Protein Quality and Immobilization

Article

作者: Molnár, Zsófia ; Koplányi, Gábor ; Kenéz, Balázs ; Katona, Gábor ; Balogh, György T. ; Decsi, Balázs ; Horváth, Péter ; Balogh-Weiser, Diána

The binding ability of human serum albumin (HSA) on active pharmaceutical ingredients (APIs) is one of the most important parameters in the early stages of drug discovery. In this study, an immobilized HSA-based tool was developed for the rapid and easy in vitro screening of API binding. The work explored the serious incompleteness in the identification of HSA used for in vitro screening published in the last five years. To mitigate this problem, a comprehensive analysis and immobilization studies were performed on the most used HSA types. Serious differences in the colloidal stability of HSAs and their API binding ability on a selected set of APIs were observed. HSAs were immobilized on magnetic nanoparticles with glutardialdehyde (GDA) or cyclohexyl-diglycidyl ether (CDGE) linkers, which have never been used for HSA immobilization before. The HSA-MNP-CDGE complexes achieved a higher immobilization yield and preserved API binding ability; however, the esterase-like enzymatic activity of HSA reduced significantly.

2025-01-02·Drug Development and Industrial Pharmacy

Solid dispersion of alectinib HCl: preclinical evaluation for improving bioavailability and establishing an IVIVC model

Article

作者: Jadhav, Ajinkya ; Saha, Sumit Kumar ; Singh, Romi ; Dubey, Kiran ; Panigrahy, Bala Krishna ; Jhanana Kailash, Some ; Arya, Vipin ; Joshi, Amita

OBJECTIVEAlectinib HCl (ALB-HCl) is a BCS class IV molecule with low solubility and low oral bioavailability. Owing to its low bioavailability, a high dose of ALB-HCl is recommended with food to meet clinical efficacy. Thus, there is a need for a delivery system to overcome the bioavailability concerns.METHODSThree solid dispersion (SD) formulations (I, II, and III) were evaluated for in-vitro dissolution and in-vivo pharmacokinetics (PK) study in Wistar rats. An in-vitro and in-vivo correlation (IVIVC) model was developed to establish a relationship between in-vitro dissolution data and in-vivo PK data. The formulations were subjected to stability studies.RESULTSAll formulations showed enhanced dissolution in all the media except Formulation I in FaSSIF media. In-vivo PK studies displayed that Formulation I was inferior to API alone. Formulations II and III (amorphous SD [ASD]) exhibited two-fold higher C_max and AUC_0-last than API alone. Level A IVIVC model was established for C_max and AUC_0-last with an acceptable % prediction error (PE). When evaluated for external predictability, the model was found validated for C_max (% PE <10%), however, it was inconclusive for AUC_0-last (%PE -14.03). Stability studies showed ASD formulations were stable during storage.CONCLUSIONA stable ASD formulation of ALB-HCl was successfully developed with improved bioavailability. Developing an IVIVC model can act as a surrogate to predict in-vivo performance. The selection of formulation components in ASD shall be rationalized for bioavailability and stability before clinical evaluation.

项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的新闻（医药）

2024-09-12

·Healthcare Digital

Grifols & Orange Business Launch Egypt Plasma Project

Grifols' Cairo project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines. Grifols & Orange Business Complete landmark phase of Data Centre for Plasma Derivatives Facility, designed to boost healthcare in Egypt Global healthcare company Grifols is working with French telecoms giant Orange to create a sustainable supply of essential therapeutics for life-threatening diseases in Egypt. Orange Business – the enterprise division of telecoms company, Orange Group – has completed the initial stage of a new data centre and communications infrastructure in Cairo, for Grifols Egypt for Plasma Derivatives (GEPD). GEPD is a joint venture between Grifols and Egypt's National Service Projects Organisation (NSPO), which manufactures military and civilian products and provides contracting services. The project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines -- vital treatments extracted from blood plasma for various serious medical conditions. Partnership to boost Egyptian healthcare Grifols, a specialist in plasma-derived medicines, is seeking to interconnect critical healthcare facilities, including a plasma laboratory, co-location data centre, plasma warehouse, 20 plasma donation centres, and a state-of-the-art manufacturing facility. Orange Business is providing co-location services, which allows GEPD to house its servers in Orange's data centre. The company is also supplying communication services and support with equipment procurement. The networked healthcare infrastructure is designed to ensure efficient operations and “the timely delivery of plasma-derived medicines to patients in need”, said Dr Ahmed Serag, Chief Strategy and Projects Officer at GEPD. Kristof Symons, CEO International at Orange Business, said: "Our technical skills, experienced resources, and global knowledge were pivotal in enabling Grifols to replicate its global business model in Egypt. This project shows how Orange Business can support its customers in creating a positive impact in today's society." About Grifols Multinational healthcare company Grifols was founded in 1940 in Barcelona, Spain employs 24,000 people worldwide, and specialises in the production of plasma-derived medicines and the development of diagnostic solutions. Its core activities include plasma collection, the manufacture of protein therapies derived from plasma, and fractionation, a process that separates a mixture into two or more fractions with different chemical and physical properties. Grifols is conducting clinical trials for its Alzheimer's treatment , a plasma-based therapy aimed at slowing cognitive decline in patients with mild to moderate Alzheimer's disease. The company is also researching treatments for liver diseases and immunodeficiencies. Among Grifols' products are Gamunex, an immune globulin therapy used to treat primary immunodeficiency, and Prolastin, used in the treatment of alpha-1 antitrypsin deficiency. The firm also produces albumin solutions for fluid replacement and coagulation factors for haemophilia treatment. In the diagnostic sector, Grifols offers blood typing systems and infectious disease screening tools. The company operates a network of plasma donation centres, primarily in the US, which form a crucial part of its supply chain for plasma-derived therapies.

2024-08-04

·药明康德

使90%阿尔茨海默病患者认知功能稳定或改善的NK细胞疗法；旨在功能性治愈乙肝的创新疗法进入临床…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 治疗阿尔茨海默病（AD）的自然杀伤（NK）细胞疗法SNK01在一项1期临床试验中使90%患者的认知功能稳定或有改善。 2. FDA批准了能在体内制造嵌合抗原受体（CAR）-T细胞的创新疗法UB-VV111的IND申请，预计将于今年年底前启动1期临床试验。 3. 有望功能性治愈乙型肝炎病毒（HBV）感染的重组多克隆抗体疗法GIGA-2339获许可进入临床。药明康德内容团队整理 SNK01：公布1期临床试验的新数据 NKGen Biotech公布了其在研NK细胞疗法SNK01治疗阿尔茨海默病患者的临床试验数据。SNK01是一款自体、非基因工程改造的NK细胞产品，它具有增强的细胞毒性，活化受体表达率超过90%，可从任何供体中稳定生产。该候选疗法能够有效降低AD患者脑脊液（CSF）中的α-突触核蛋白（α-syn）水平，该蛋白的升高被认为与认知能力下降有关。目前，已批准的AD疗法均未将α-syn作为靶点。此前公布的1期试验结果显示，SNK01除了先前披露的对淀粉样蛋白β（Aβ）和神经炎症生物标志物的积极影响外，对晚期AD患者的认知功能也具有临床疗效。在10名可评估的AD患者中，90%患者的阿尔茨海默病综合评分（ADCOMS，一种反映患者认知功能的评分）较基线时有改善或保持稳定。此次公布的结果显示，尽管70%的受试者接受的是相对低剂量的SNK01治疗，仍有60%的受试者（6/10）的CSF α-syn水平较基线时有所下降。第11周时，6名患者中有5名受试者的α-syn下降与ADCOMS的稳定/下降相对应。安全性方面，没有观察到与治疗相关的不良事件。AD受试者对SNK01的耐受性良好，在所有测试剂量下均未观察到剂量限制性毒性。该公司表示，这些数据值得在更大剂量、更长疗程、更大规模的2期试验中进一步研究。 UB-VV111：IND申请获得FDA许可 Umoja Biopharma宣布，美国FDA已批准其在研CD19靶向原位生成（in-situ generated）CAR-T细胞疗法UB-VV111的IND申请，用以治疗血液恶性肿瘤。Umoja预计将在2024年底前启动1期试验并进行首位患者给药。根据新闻稿，UB-VV111可能是血液学人体试验中首个原位生成CD19靶向CAR-T细胞疗法。 UB-VV111是一款通过VivoVec平台开发的体内CAR-T细胞疗法。UB-VV111包含一个表面经过工程化的病毒包膜，以及编码CD19靶向CAR和雷帕霉素激活细胞因子受体（RACRTM）的转基因，旨在于患者体内扩增UB-VV111工程化CAR-T细胞。该疗法是VivoVec基因递送平台进入临床的首个管线。 GIGA-2339：IND申请获得FDA许可 GigaGen公司宣布，美国FDA已批准其IND申请，将于2024年第四季度启动一项1期试验，以评估该公司首款用于治疗HBV感染的重组多克隆抗体疗法GIGA-2339。GIGA-2339采用GigaGen的下一代平台开发，含有1000多种全人重组抗HBV抗体，旨在复制人体的自然免疫反应。GIGA-2339的效力是血浆衍生HBV药物的2000多倍，涵盖了广泛的循环HBV变异。在小鼠模型中，GIGA-2339中和并清除了HBV的病毒DNA及其抗原。通过这种独特的作用机制，GIGA-2339有可能清除病毒颗粒并激活免疫反应，从而提供HBV感染患者的功能性治愈。 Sabirnetug（ACU193）：公布1期临床试验的新数据 Acumen Pharmaceuticals公司公布了sabirnetug（ACU193）用于治疗AD的1期临床试验的新结果。Sabirnetug是一种选择性靶向毒性可溶性β淀粉样蛋白寡聚体（AβOs）的人源化单克隆抗体。Sabirnetug能够结合AβOs，由于其独特的结合特性，sabirnetug有可能在产生淀粉样蛋白相关成像异常（ARIA，一种抗体疗法产生的常见主要副作用）风险较低的情况下提供治疗益处，因为sabirnetug可以在不直接靶向淀粉样斑块的情况下阻断AβOs的毒性作用。此次公布的结果显示，接受sabirnetug给药3次可显著降低患者脑脊液中突触前和突触后蛋白水平，这与sabirnetug抑制AβOs突触结合的作用机制一致。VAMP2是一种与突触损伤相关的生物标志物，在所有多剂量递增组中均显著降低，似乎是该研究中发现的对sabirnetug最敏感的生物标志物。Acumen公司计划在正在进行的为期18个月的2期临床试验中评估生物标志物的长期变化及其与临床结果的关系，以进一步证实sabirnetug的作用机制。此外，Acumen公司还宣布了sabirnetug皮下制剂治疗早期AD的1期临床试验已完成首例受试者给药。 Pepinemab：公布1b/2a期临床试验数据 Vaccinex公司公布了pepinemab治疗AD的1b/2期试验数据的良好初步结果。Pepinemab是一种人源化IgG4单克隆抗体，旨在阻断SEMA4D与plexin-B1受体结合，从而避免引发细胞内肌动蛋白细胞骨架的崩溃，以及进一步导致的大脑中星形胶质细胞和其他胶质细胞以及免疫组织中树突状细胞的稳态功能丧失。此次公布的结果证实了以激活星形胶质细胞为目标的新型作用机制有望延缓AD从早期轻度认知障碍发展为老年痴呆症的进程。该研究结果与此前pepinemab治疗亨廷顿病的2期临床试验结果类似，因为AD与亨廷顿病在神经免疫病理学方面具有相似性。 RGX-202：公布1/2期临床试验的新数据 REGENXBIO公司宣布，其在研杜氏肌营养不良症（DMD）基因疗法RGX-202在治疗1至11岁DMD患者的1/2期临床试验中获得了积极的中期安全性和疗效数据。在接受关键性临床试验推荐剂量（剂量水平2）的RGX-202治疗的5.8岁和8.5岁患者中，接受治疗后三个月时微抗肌营养不良蛋白表达水平分别为健康对照组的77.2%和46.5%。该试验中按剂量和年龄分类的微抗肌营养不良蛋白表达结果如下表所示： ▲AFFINITY DUCHENNE试验中接受不同剂量RGX-202治疗患者的微抗肌营养不良蛋白表达水平（图片来源：参考资料[1]） RGX-202是一种一次性基于腺相关病毒载体（AAV）的基因疗法，它编码包含天然抗肌营养不良蛋白C端（CT）结构域的微抗肌营养不良蛋白，以更好地模拟天然蛋白的功能。临床前研究中，CT结构域已被证明可以保护肌肉免受收缩引起的应力，并提高其自我修复的能力。 XPro：公布1期临床试验数据 INmune Bio公司公布了其下一代肿瘤F坏死因子（TNF）抑制剂XPro用于治疗AD的1b期临床试验结果。XPro的作用与现有的TNF抑制剂不同，它能中和可溶性TNF（sTNF），而不影响跨膜TNF（tmTNF）或TNF受体。XPro可在不抑制免疫的情况下减轻神经炎症，从而对神经系统疾病患者产生潜在的实质性益处。此次公布的结果显示，接受XPro治疗12周后，患者的突触蛋白发生了显著变化，而突触蛋白对神经元之间的交流至关重要。 NTLA-3001：获批在英国启动1/2期临床试验 Intellia Therapeutics公司宣布其临床试验申请（CTA）获得英国药品与保健品管理局（MHRA）的授权，可启动一项1/2期研究以评估NTLA-3001治疗α1抗胰蛋白酶缺乏症（AATD）相关的肺部疾病的效果。AATD是一种罕见的遗传性疾病，最常见的表现是由于α1抗胰蛋白酶（AAT）蛋白水平不足导致的肺功能障碍。NTLA-3001是一种全身给药的体内一次性基因编辑疗法，它旨在通过CRISPR/Cas9技术精确插入编码AAT蛋白的SERPINA1基因的健康拷贝，单次给药就有可能使功能性AAT蛋白永久表达并恢复到治疗水平。这种方法旨在改善患者的治疗效果，包括消除每周静脉注射AAT的疗法或严重病例对肺移植的需求。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202. Retrieved August 1, 2024, from https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-new-positive-data-affinity-duchenner-trial [2] Acumen Pharmaceuticals Presents Patient Experience and Biomarker Data from Phase 1 INTERCEPT-AD Study at the Alzheimer’s Association International Conference (AAIC®) 2024. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/28/2919969/0/en/Acumen-Pharmaceuticals-Presents-Patient-Experience-and-Biomarker-Data-from-Phase-1-INTERCEPT-AD-Study-at-the-Alzheimer-s-Association-International-Conference-AAIC-2024.html [3] Acumen Pharmaceuticals Announces First Subject Dosed in Phase 1 Study of Subcutaneous Sabirnetug (ACU193) for Early Alzheimer’s Disease. Retrieved August 2, 2024, from https://investors.acumenpharm.com/news-releases/news-release-details/acumen-pharmaceuticals-announces-first-subject-dosed-phase-1 [4] NKGen Biotech Presents New Positive SNK01 Biomarker Data at the 2024 Alzheimer’s Association International Conference. Retrieved August 2, 2024, from https://nkgenbiotech.com/nkgen-biotech-presents-new-positive-snk01-biomarker-data-at-the-2024-alzheimers-association-international-conference/ [5] Umoja Biopharma Announces FDA Clearance of IND Application for UB-VV111, a CD19 Directed in situ CAR T for Hematologic Malignancies. Retrieved July 31, 2024 from https://www.umoja-biopharma.com/news/umoja-biopharma-announces-fda-clearance-of-ind-application-for-ub-vv111-a-cd19-directed-in-situ-car-t-for-hematologic-malignancies/ [6] Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2920893/0/en/Intellia-Therapeutics-Receives-Authorization-to-Initiate-Phase-1-2-Clinical-Trial-of-NTLA-3001-for-the-Treatment-of-Alpha-1-Antitrypsin-Deficiency.html [7] Vaccinex Reports Positive Data for SIGNAL-AD Phase 1b/2 trial of Pepinemab in Alzheimer’s Disease. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921918/34858/en/Vaccinex-Reports-Positive-Data-for-SIGNAL-AD-Phase-1b-2-trial-of-Pepinemab-in-Alzheimer-s-Disease.html [8] Citryll Completes Patient Dosing In The Repeat Dose Stage Of The Phase 1 Trial. Retrieved August 2, 2024, from https://citryll.com/newsitems/citryll-completes-patient-dosing-in-the-repeat-dose-stage/ [9] GigaGen Receives FDA Clearance of IND Application for Phase 1 Trial of Recombinant Polyclonal for HBV Treatment, GIGA-2339. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921826/0/en/GigaGen-Receives-FDA-Clearance-of-IND-Application-for-Phase-1-Trial-of-Recombinant-Polyclonal-for-HBV-Treatment-GIGA-2339.html [10] Compugen Announces FDA Clearance of IND for COM503 for the Treatment of Solid Tumors. Retrieved August 2, 2024, from https://www.prnewswire.com/news-releases/compugen-announces-fda-clearance-of-ind-for-com503-for-the-treatment-of-solid-tumors-302208442.html [11] Georgiamune Announces First Patient Dosed in A First-In-Class Phase 1 Cancer Treatment Trial of GIM-531. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2921055/0/en/Georgiamune-Announces-First-Patient-Dosed-in-A-First-In-Class-Phase-1-Cancer-Treatment-Trial-of-GIM-531.html [12] INmune Bio Inc. Announces New Phase 1 Study Data Demonstrating Dose-Dependent Effect of XPro™ on Proteins that Regulation Synapses in Alzheimer’s Patients. Retrieved August 2, 2024, from https://www.inmunebio.com/index.php/newsroom/2024-news/muneioncnnouncesewhase1tudyataemonstrati20240729060507 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床申请临床1期免疫疗法临床2期

2023-11-15

·GlobeNewswire-Health Care

Grifols Completes Cohort 1 in Clinical Study of Alpha-1 15%, Evaluating First-in-Human Subcutaneous Dosing Option for Patients with Alpha1-Antitrypsin Deficiency

Study designed to demonstrate impact of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), an alpha1 antitrypsin treatment, compared with Liquid Alpha1-Proteinase Inhibitor (Human) intravenousThis first in-human subcutaneous approach to treating alpha1-antitrypsin deficiency, if proven successful in clinical trials, could give patients the convenience and flexibility to administer their medication from home BARCELONA, Spain, Nov. 15, 2023 (GLOBE NEWSWIRE) -- Grifols (MCE: GRF, MCE: GRF.P NASDAQ: GRFS), one of the world’s leading producers of plasma-derived medicines, today announced that it has completed Cohort 1 of its Phase 1/2 study (NCT04722887) evaluating Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), a subcutaneous (SC) alpha1 antitrypsin (AAT) treatment being compared to Liquid Alpha1-Proteinase Inhibitor (Human) intravenous (IV). Alpha1-antitrypsin deficiency (also known as alpha-1) is an underdiagnosed1 genetic disorder that occurs when a patient has low levels of AAT, a protective protein that safeguards the lungs. Augmentation therapy with IV AAT is the standard medical treatment option for patients with severe AAT deficiency and emphysema. If proven successful in clinical trials, a SC option could provide alpha-1 patients the ability to independently administer AAT therapy from home, allowing for greater convenience and flexibility. In this multi-center, single-dose and repeat-dose study over eight weeks, Cohort 1 has been completed and demonstrated no safety issues with Alpha-1 15% that would prevent the study from moving forward into Cohort 2. “A subcutaneous option would be a first for alpha-1 patients, providing more freedom when it comes to managing their AAT deficiency by allowing patients to administer their medication from the comfort of their own home. We are pleased to announce this milestone during Alpha-1 Awareness Month, contributing to raising further visibility about this rare disease,” said Jörg Schüttrumpf, Chief Scientific Innovation Officer, Grifols. “We look forward to moving this study into Cohort 2. Grifols has a strong commitment to the alpha-1 community and continues to innovate to find additional treatment options for patients living with this disease, in addition to more convenient alpha-1 testing alternatives such as AlphaID™ At Home, our recently launched direct-to-consumer genetic health risk service.” “During Alpha-1 Awareness month, we are excited to share this exciting news to the Alpha-1 community of a new clinical trial advancement in Alpha-1. We are thrilled that Grifols has completed Cohort 1 in clinical study of Alpha-1 15% and moving forward into Cohort 2. This study evaluating first-in-human subcutaneous dosing option for Alpha-1 patients could provide a new option that could easily be administered from their own homes,” said Scott Santarella, Alpha-1 Foundation President and CEO. About Grifols Grifols is a global healthcare company founded in Barcelona in 1909 committed to improving the health and well-being of people around the world. A leader in essential plasma-derived medicines and transfusion medicine, the company develops, produces and provides innovative healthcare services and solutions in more than 110 countries. Patient needs and Grifols’ ever-growing knowledge of many chronic, rare and prevalent conditions, at times life-threatening, drive the company’s innovation in both plasma and other biopharmaceuticals to enhance quality of life. Grifols is focused on treating conditions across a broad range of therapeutic areas: immunology, hepatology and intensive care, pulmonology, hematology, neurology and infectious diseases. A pioneer in the plasma industry, Grifols continues to grow its network of donation centers, the world’s largest with over 390 across North America, Europe, Africa and the Middle East and China. As a recognized leader in transfusion medicine, Grifols offers a comprehensive portfolio of solutions designed to enhance safety from donation to transfusion, in addition to clinical diagnostic technologies. It provides high-quality biological supplies for life-science research, clinical trials, and for manufacturing pharmaceutical and diagnostic products. The company also supplies tools, information and services that enable hospitals, pharmacies and healthcare professionals to efficiently deliver expert medical care. Grifols, with more than 24,000 employees in more than 30 countries and regions, is committed to a sustainable business model that sets the standard for continuous innovation, quality, safety and ethical leadership. In 2022, Grifols’ economic impact in its core countries of operation was EUR 9.6 billion. The company also generated 193,000 jobs, including indirect and induced. The company’s class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Grifols non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ through ADRs (NASDAQ:GRFS). For more information, please visit www.grifols.com MEDIA CONTACT:Media Press Officemedia@grifols.com Tel. +34 93 571 00 02 INVESTORS: Investors Relations Department & Sustainabilityinversores@grifols.com - investors@grifols.com Tel. +34 93 571 02 21 LEGAL DISCLAIMER The facts and figures contained in this report that do not refer to historical data are “future projections and assumptions”. Words and expressions such as “believe”, “hope”, “anticipate”, “predict”, “expect”, “intend”, “should”, “will seek to achieve”, “it is estimated”, “future” and similar expressions, insofar as they relate to the Grifols group, are used to identify future projections and assumptions. These expressions reflect the assumptions, hypotheses, expectations and predictions of the management team at the time of writing this report, and these are subject to a number of factors that mean that the actual results may be materially different. The future results of the Grifols group could be affected by events relating to its own activities, such as a shortage of supplies of raw materials for the manufacture of its products, the appearance of competitor products on the market, or changes to the regulatory framework of the markets in which it operates, among others. At the date of compiling this report, the Grifols group has adopted the necessary measures to mitigate the potential impact of these events. Grifols, S.A. does not accept any obligation to publicly report, revise or update future projections or assumptions to adapt them to events or circumstances subsequent to the date of writing this report, except where expressly required by the applicable legislation. This document does not constitute an offer or invitation to buy or subscribe shares in accordance with the provisions of the following Spanish legislation: Royal Legislative Decree 4/2015, of 23 October, approving recast text of Securities Market Law; Royal Decree Law 5/2005, of 11 March and/or Royal Decree 1310/2005, of 4 November, and any regulations developing this legislation. In addition, this document does not constitute an offer of purchase, sale or exchange, or a request for an offer of purchase, sale or exchange of securities, or a request for any vote or approval in any other jurisdiction. The information included in this document has not been verified nor reviewed by the external auditors of the Grifols group 1 American Thoracic Society; European Respiratory Society. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am J Respir Crit Care Med. 2003;168(7):818-900. doi:10.1164/rccm.168.7.818

100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Alpha1-proteinase inhibitor(Grifols SA)	B02AB02	DB00058

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
α1-抗胰蛋白酶缺乏症	日本	OrphanPacific, Inc.	2021-01-22
肺气肿	美国	Grifols Therapeutics, Inc.	1987-12-02

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
新型冠状病毒感染	临床2期	西班牙	Instituto Grifols SA	2020-07-29
1型糖尿病	临床2期	美国	Grifols Therapeutics, Inc.	2014-03-01
HIV感染	临床2期	美国	Grifols Biologicals, Inc.	2012-04-01
新型冠状病毒感染	药物发现	西班牙	Instituto Grifols SA	2020-07-29
α1-抗胰蛋白酶缺乏症	药物发现	英国	Grifols Therapeutics, Inc.	2006-06-01
α1-抗胰蛋白酶缺乏症	药物发现	美国	Grifols Therapeutics, Inc.	2006-06-01
囊性纤维化	药物发现	-	Grifols Therapeutics, Inc.	2003-12-01
气肿	药物发现	英国	Grifols Therapeutics, Inc.	2003-12-01
气肿	药物发现	丹麦	Grifols Therapeutics, Inc.	2003-12-01
气肿	药物发现	瑞典	Grifols Therapeutics, Inc.	2003-12-01

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临床结果

适应症

分期

评价

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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


NCT02947087 (CTgov)	临床1/2期	慢性胰腺炎	48	Prolastin-C (Prolastin-C)	選鏇選網夢鹽襯鏇淵憲(襯廠選顧築構繭簾簾選) = 憲憲願廠夢衊鬱襯鏇鹽鹽築觸蓋願憲範顧壓廠 (鏇憲選夢繭網膚窪憲憲, 構遞蓋積廠製願願獵繭 ~ 構夢遞顧衊淵築窪衊獵) 更多	-	2023-05-09
				Placebo (Placebo)	選鏇選網夢鹽襯鏇淵憲(襯廠選顧築構繭簾簾選) = 鹽醖選鏇構構餘築遞觸鹽築觸蓋願憲範顧壓廠 (鏇憲選夢繭網膚窪憲憲, 築選鹹襯鏇齋鏇齋鑰鬱 ~ 醖鹹艱構蓋鑰繭積觸簾) 更多
NCT04547140 (CTgov)	临床2期	新型冠状病毒感染	57	Standard Medical Treatment	膚憲鏇鏇遞廠壓醖鏇遞(繭積膚糧觸顧鏇觸餘襯) = 鹽鹽廠鑰窪膚簾壓鑰簾願蓋餘膚範製選廠膚鑰 (構簾簾膚鏇製膚鏇製網, 醖艱廠醖衊製鏇鏇繭構 ~ 製糧憲鹽鑰襯獵醖網構) 更多	-	2023-03-22
NCT02870348 (GTI1401-OLE, CTgov)	临床1/2期	α1-抗胰蛋白酶缺乏症	4	Alpha-1 MP	廠衊衊艱選餘餘鏇願選(鏇夢鹹淵夢觸壓鬱膚淵) = 夢壓鹽繭壓齋繭構窪遞獵襯襯網憲繭網鏇夢觸 (繭蓋簾獵鹹齋築壓鑰繭, 衊糧鬱願製獵繭憲遞製 ~ 廠構鏇選壓壓襯簾鬱廠) 更多	-	2022-03-21
NCT02093221 (CTgov)	临床2期	1型糖尿病	76	180 mg/kg Alpha1-PI (Alpha1-PI 180 mg/kg/wk, 26 Weeks)	鬱顧鹹觸餘衊衊繭糧鹹(築簾網蓋鏇艱艱觸簾構) = 構夢獵糧觸艱鑰製鏇積繭蓋襯築憲繭積鏇鹹鏇 (憲積製築積範鹽觸鬱築, 憲鑰觸醖簾鏇願廠鹹選 ~ 齋製糧範夢襯膚憲製遞) 更多	-	2018-04-19
				180 mg/kg Alpha1-PI (180 mg/kg/wk Alpha1-PI, 13 Weeks)	鬱顧鹹觸餘衊衊繭糧鹹(築簾網蓋鏇艱艱觸簾構) = 鹹衊構艱壓簾願壓鏇願繭蓋襯築憲繭積鏇鹹鏇 (憲積製築積範鹽觸鬱築, 憲築構選壓糧鹽夢簾築 ~ 糧襯醖繭積膚齋繭餘獵) 更多
NCT02282527 (CTgov)	临床2/3期	α1-抗胰蛋白酶缺乏症	32	Alpha1-PI (Liquid Alpha₁-PI)	艱醖鏇膚願觸顧築廠鹽(顧衊蓋製網願鹹窪夢艱) = 網廠鬱積艱獵醖積鬱醖蓋艱醖積壓鹹獵鬱醖遞 (鏇夢鏇獵鏇鹽蓋齋夢網, 艱選鹹糧壓醖醖範蓋願 ~ 蓋齋襯觸簾糧製憲糧鬱) 更多	-	2017-03-13
				Prolastin-C (Prolastin-C)	艱醖鏇膚願觸顧築廠鹽(顧衊蓋製網願鹹窪夢艱) = 艱鹹齋衊壓鬱夢範鑰艱蓋艱醖積壓鹹獵鬱醖遞 (鏇夢鏇獵鏇鹽蓋齋夢網, 獵範鏇選構衊醖構簾衊 ~ 淵鏇簾衊選築製齋鹹願) 更多
NCT01936896 (VCU-α1RT \| VCU-Alpha1RT, CTgov)	临床1/2期	ST段抬高心肌梗死 \| 急性心肌梗塞	10	Alpha 1-Antitrypsin	(淵選願鏇壓繭餘衊衊鏇) = 夢夢齋廠壓蓋齋鹹襯窪艱鬱糧遞鬱鬱襯範顧觸 (蓋遞觸簾簾鏇鏇網簾鑰, 糧齋願願壓鏇積構製觸 ~ 鑰糧積廠窪獵廠積遞簾) 更多	-	2016-02-12
NCT00295061 (Pubmed)	临床3期	α1-抗胰蛋白酶缺乏症	24	Prolastin-C	鹹顧範鹽夢範艱衊窪鏇(鹹壓選選窪淵窪選築築) = 簾壓構願觸膚廠襯願淵醖製衊艱憲蓋鏇願製鬱 (鏇壓獵鑰鏇鹽窪艱鹹廠 )	-	2010-09-30
				Prolastin	鹹顧範鹽夢範艱衊窪鏇(鹹壓選選窪淵窪選築築) = 遞廠觸艱鑰襯構範醖鹽醖製衊艱憲蓋鏇願製鬱 (鏇壓獵鑰鏇鹽窪艱鹹廠 )