A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of MGTA-145 in Combination With Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients With Sickle Cell Disease

This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.

开始日期2022-06-24

申办/合作机构

Ensoma, Inc.初创企业

[+1]

NCT04762875 / Terminated临床2期

A Phase II Study Evaluating the Safety and Efficacy of MGTA-145 in Combination With Plerixafor for the Mobilization and Transplantation of HLA-Matched Donor Hematopoietic Stem Cells in Recipients With Hematological Malignancies

This research study tests a new medicine for mobilizing stem cells so they can be collected and used for allogeneic stem cell transplant for treatment of hematological malignancies. MGTA-145, the new medicine, will be given with plerixafor.

开始日期2021-06-16

申办/合作机构

Ensoma, Inc.初创企业

[+1]

NCT04552743 / Completed临床2期IIT

Phase II Study of MGTA-145 in Combination With Plerixafor in the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Patients With Multiple Myeloma

This study evaluates a new drug MGTA-145 in combination with plerixafor (Mozobil) to mobilize stem cells into the peripheral blood for collection by apheresis. The stem cells will be used for autologous stem cell transplant for treatment of multiple myeloma.

开始日期2020-10-05

申办/合作机构

Stanford University

100 项与 GRO beta 相关的临床结果

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100 项与 GRO beta 相关的转化医学

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100 项与 GRO beta 相关的专利（医药）

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项与 GRO beta 相关的文献（医药）

2024-03-01·Archives of Biochemistry and Biophysics

A multifactorial study of in situ antioxidant activity of modified GrO in myocardial reperfusion injury using the Langerdorff model

Article

作者: Mys, Lidia A ; Sagach, Vadym F ; Sementsov, Yury I ; Goshovska, Yulia V ; Zhuravskyi, Sergey V ; Kolev, Hristo ; Sencha-Hlevatska, Kateryna V ; Korkach, Yulia P

Carbon nanomaterials possess antioxidant properties that can be applied in biomedicine and clinics for the development of new highly effective treatments against oxidative stress-induced diseases like ischemic heart disease. We previously reported the usage of graphene oxide (GrO) as a precursor for the elaboration of such prototypes. The promising findings led to the development of two new modifications of GrO: nitrogen-doped (N-GrO) and l-cysteine functionalized (S-GrO) derivatives as possible antioxidant agents in ischemia-reperfusion (I/R) conditions. In this study, the cardioprotective and antioxidant potential of modified GrO as a pre-treatment in rats was evaluated for the first time. In Langendorff isolated rat heart I/R model, the left ventricle developed pressure (LVDP), the end-diastolic pressure (EDP), the maximal (dP/dt_max) and minimal (dP/dt_min) value of the first derivative of LVDP, and heart rate (HR) were measured. The oxidative-nitrosative markers, in particular, the rate of O₂*^- and H₂O₂ generation, the content of malonic dialdehyde, diene conjugates, and leukotriene as well as cNOS and iNOS activity were estimated. Obtained results show a significant restoration of cadiodynamic parameters at the reperfusion period. Simultaneously, all samples significantly reduced the rate of reactive oxygen species (ROS) and lipid peroxidation markers in cardiac homogenates and preserved cNOS activity at the preischemic level. This evidence makes GrO derivatives promising candidates for the correction of reperfusion disorders affecting myocardial function.

2023-04-11·ACS omega

Tailoring Deep Eutectic Solvents to Provoke Solubility and Bioavailability of Naringin: Implications of a Computational Approach.

Article

作者: Korekar, Pawan P ; Borikar, Sachin P ; Pethe, Anil M ; Dangre, Pankaj V ; Chaturvedi, Kaushalendra K ; Borkar, Maheshkumar R

Recently, the applications of deep eutectic solvents (DESs) as green and sustainable solvents for the solubilization of functional foods and phytophenols have dramatically risen concerning global issues on the utilization of organic solvents. Nevertheless, developing a suitable DES system for phytocomponents to enhance its solubility and bioavailability is complex and requires a sound experimental setup. Herein, we have attempted to develop DES encompassing the choline chloride (ChCl) along with oxalic acid (OA), l-glutamine (l-Glu), urea (U), and glycerol (Gro) at different ratios to elicit the solubility and bioavailability of naringin (NAR). Several DES systems were designed and tested for solubility, kinematic viscosity, and pH. Among these, DES-NAR encompassing ChCl/Gro in a 1:3 ratio exhibited the maximum solubility of NAR (232.56 ± 7.1 mg/mL) and neutral characteristic and thus considered suitable for NAR. Further, the conductor-like screening model for real solvents (COSMO-RS) has been employed to estimate the molecular and electrostatic interactions. DES-NAR was evaluated by polarized optical microscopy, Fourier-transform infrared (FTIR), differential scanning calorimetry (DSC), and ¹H NMR to investigate the molecular transition and interaction. Further, diffusion and permeability studies were performed, which suggest significant improvements in DES-NAR. Likewise, the pharmacokinetic studies revealed a two times increase in the oral bioavailability of NAR in a designed DES system. Thus, the work represents a systematic and efficient development of the DES system for a potential phytocomponent considering the biosafety impact, which may widen the interest in pharmaceutical and food sciences.

2022-04-03·Free Radical Research

Graphene oxide nanoflakes prevent reperfusion injury of Langendorff isolated rat heart providing antioxidative activity in situ

Article

作者: Voitko, Kateryna V. ; Korkach, Yulia P. ; Mys, Lida A. ; Kolev, Hristo ; Demianenko, Eugeniy M. ; Zhuravskyi, Sergey V. ; Sementsov, Yury I. ; Goshovska, Yulia V. ; Sagach, Vadym F.

Carbon materials possess powerful antioxidant activity that might be promising for the development of new generation treatment of cardiovascular diseases, ischemic conditions, and reperfusion injury. The present study aimed to characterize the structure of nanosized graphene oxide (GrO) sample and evaluate the antioxidant efficacy of GrO in situ models of oxidative stress widely used in pre-clinical studies. The structure and surface chemistry of the initial samples were analyzed via LDS, RAMAN, LDI, TPD-MS, and FTIR methods. The GrO showed a strong ability to scavenge DPPH, hydroxyl, and superoxide anion free radicals and have a total antioxidant capacity. The DFT quantum-chemical calculation demonstrated the radical scavenging effect of GrO proceeding due to the physical adsorption of the free radical on the surface. For evaluation of the antioxidant effect of GrO in situ, we used the model of ischemia-reperfusion (I/R) of Langendorff isolated rat heart. We revealed that intravenous pretreatment of Wistar male rats with GrO significantly increased resistance of myocardium to I/R, improved restoration of heart function, prevented non-effective oxygen utilization, and I/R-induced reactive oxygen species production in cardiac tissue. Thus, our data demonstrate the perspective of further use of GrO for the development of antiischemic therapy.

项与 GRO beta 相关的新闻（医药）

2023-06-29

·药时代

5月生物医药上市盘点：两年来最大规模的Biotech IPO诞生

药时代：拥抱创新！拥抱未来！2023年5月，在生物医药领域，有2家公司通过IPO上市，但以其他方式上市的医药公司数量依旧维持在高位：其中4家公司宣布与SPAC合并上市，另有4家公司进行反向并购上市。一、IPO上市5月，有2家医药公司采用IPO形式上市，数量上比4月多1家，但仍处于历史低位，生物医药IPO市场依然不景气。1、Acelyrin(NASDAQ:SLRN)5月5日，Acelyrin在纳斯达克交易所上市，发行价定在区间顶格，为每股18美元，同时将发行规模从895万股扩大至3000万股，募集金额达5.4亿美元，是自2021年2月上市的Sana (NASDAQ:SANA)（5.88亿美元）之后，又一家募集金额超过5亿美元的Biotech公司，二者相隔达26个月之久。Acelyrin成立于2020年12月，由Horizon Therapeutics（被安进以260亿美元收购）的前研发负责人Shao-Lee Lin和前首席商务官Robert F. Carey共同创立。Acelyrin采取了对外买入研发管线的策略，寻找并购买具有市场前景的潜在颠覆性药物，试图复制Horizon的爆款药物Tepezza的成功故事。公司目前的在研管线全部都是从外部买入：Izokibep：一款白细胞介素 17A (IL-17A) 抑制剂，可以特异性结合IL-17A的两个亚基以及血清中的白蛋白，对靶点的亲和力高，分子量仅为单抗的十分之一，可以通过皮下注射，达到传统单抗静脉给药的吸收水平，同时还拥有不输于单抗的半衰期，在皮肤、关节等组织器官领域有着独特优势。目前处于治疗银屑病关节炎 (PsA) 、化脓性汗腺炎（HS）和葡萄膜炎的2b/3期临床试验阶段，治疗中轴型脊柱关节炎 (AxSpA)的试验也进入了临床2期。Izokibep此前发布了治疗银屑病关节炎的积极二期数据，有成为IL-17靶点的BIC产品的潜力。该药物最初是由Affibody公司研发，Acelyrin于2021年11月以2500万美元首付款和3亿美元里程碑付款的价格，引进了Izokibep的部分权益。而此前的2020年5月，国内的创响生物也引进Izokibep在大中华区、韩国的独家开发和商业化权利，以及日本外亚太地区的临床开发权利。Lonigutamab ：一款皮下给药的抗IGF-1R单抗药物，通过靶向IGF-1R的一个独特表位，可以增加疗效的持久性和耐受性，目前正在开展甲状腺眼病的临床1期试验。该药物由Acelyrin于2023年，以收购ValenzaBio公司的形式获得。SLRN-517：一种靶向 c-KIT 的全人源IgG1单克隆抗体，目前处于IND申报阶段。该药物由Acelyrin于2023年，以收购ValenzaBio公司的形式获得。Acelyrin是近些年为数不多的在一级市场屡创融资记录的Biotech公司。自成立以来，Acelyrin共完成了3轮融资，总融资金额达到5.58亿美元。上市后，公司股价首日暴涨30.56%。无论是IPO的定价、发行规模，还是首日涨幅，均显示出市场对公司的高度热情。Acelyrin从成立到上市，仅有短短25个月，累计获得10.98亿美元的融资，且完成在纳斯达克上市，不仅融资规模上，而且在上市速度上，都是Biotech行业所罕见，尤其是在过去一年多时间的Biotech行业下行阶段，更加难能可贵。Acelyrin获得资本市场追捧的原因主要是：热门靶点的晚期管线。白细胞介素17(IL-17)是由活化的T细胞分泌的促炎细胞因子，在免疫系统中发挥多种调节功能，与银屑病、强直性脊柱炎、牙周炎等自身免疫性疾病的发生相关。在IL-17靶点的药物中，已有Taltz、Cosentyx、Bimekizumab等多款药物获批，其中礼来的Taltz和诺华的Cosentyx分别在2022年实现了24.82亿美元和47.88亿美元的销售收入，是妥妥的blockbuster的存在。在Taltz和Cosentyx的后继追赶者中，资本市场也给与了高度期待。6月20日，礼来宣布以24亿美元的价格收购了DICE Therapeutics，DICE的主打药物便是处于临床二期的口服IL-17抑制剂DC-806。2022年4月，以合并SPAC上市的MoonLake(NASDAQ:MLTX)，其主打药物Sonelokimab是靶向IL-17的三抗药物，具有挑战Cosentyx的潜力，吸引了BVF、Cormorant、RTW等知名Biotech投资机构的投资，近一年涨幅达156.29%，是2022年以来表现最好的De-SPAC医药公司。优秀的创始人组合。Acelyrin的CEO Shao-Lee Lin、首席医学官Paul Peloso 、总裁Robert F. Carey等均来自于Horizon Therapeutics。其中Shao-Lee Lin曾担任Horizon的研发负责人，主导了甲状腺眼药Tepezza研发；Robert Carey曾在摩根大通担任生命健康领域的负责人，主导的融资金额超过100亿美元。Acelyrin的创始投资机构Westlake Village BioPartners由前安进公司研发主管Sean Harper和Beth Seidenberg 博士创立，与学术界、资本界的关系密切。凭借创始人和投资人的行业经验和资源，Acelyrin有能力去把握Biotech行业的趋势，有机会去实现潜力药物的收购，以及推进药物的实验和商业化。二、反向并购上市（Reverse Merger）5月，共有4家生物医药公司宣布或完成了反向并购上市。1、Magenta Therapeutics(NASDAQ:MGTA)5月3日，Magenta Therapeutics宣布与Dianthus Therapeucs以全股票交易的形式完成合并,预计将于2023年第三季度完成，合并后的公司预计将在纳斯达克交易，股票代码为“DNTH”。合并的同时，Dianthus Therapeucs进行一笔7000万美元的融资，投资方包括富达、Venrock Healthcare Capital、5AM Ventures、Wedbush、Avidity Partners等机构。Dianthus Therapeutics是一家新一代补体疗法研发公司，采用半衰期延长技术进行修饰，对活性补体蛋白具有高选择性，有潜力实现以更低的剂量水平和更低频率给药，为严重自身免疫性疾病的患者研发变革性药物。Dianthus目前主要的管线DNTH103是一款靶向C1s补体的抑制剂，采取皮下注射且低频率的给药方式，具有改善严重自身免疫性疾病患者生活的潜力。DNTH103后续研发的催化剂较多，今年底计划发布临床一期数据，2024年一季度启动重症肌无力的临床2期试验，以及两项针对其他神经系统疾病临床2期试验，2024年下半年发布计划发布治疗Cold Agglutinin疾病的数据。2022年4月，Dianthus Therapeucs完成了1亿美元的A轮融资，由5AM Ventures, Avidity Partners, 富达领投，Venrock Healthcare Capital、 Fairmount、Tellus BioVentures跟投。Dianthus的CEO Garcia拥有25年的Biotech从业经历，此前曾担任Zealand Pharma的高级副总裁；首席医疗官Simrat Randhawa博士拥有超过20年的临床实践经验，曾担任Aurinia Pharmaceuticals临床和医疗事务高级副总裁。选择卖壳的Magenta Therapeutics是一家旨在开发干细胞移植疗法用于血癌、遗传病和自身免疫性疾病患者的公司，曾经在一级市场也是备受追捧的明星项目，累计完成了1.52亿美元的融资，并于2018年6月IPO上市，融资规模达1亿美元。上市之后，得益于早期不错的数据，Magenta又完成了多次增发融资：2019年5月，以每股13.25美元的价格完成了5630万美元的融资；2020年6月，以每股8美元完成了6000万美元的融资；2021年5月，以每股9美元的价格完成了8640万美元的融资；参与增发的投资者中，不乏Deep Track Capital, TCG X, Great Point, OrbiMed, Janus Henderson等知名机构。在合作方面，也得到了诺华、Beam Therapeutics、Bluebird Bio等公司认可，然而2021年以来，Magenta的研发难言顺利：2021年7月，MGTA-117在急性髓系白血病（AML）和骨髓增生异常综合征（MDS）患者中的1/2期临床试验的IND申请遭到FDA的临床暂停，虽然后来暂停解除，但股价却再也没有回到之前的位置；2022年4月，Magenta宣布裁员，并暂停MGTA-145 (CXCR2激动剂) 的临床试验，用以将其现金储备延长至2024年第二季度；2022年12月，公司发布了MGTA-117治疗AML的I/II期剂量递增试验数据，出现多例安全性事件，公司股价当日暴跌52%；今年1月，Magenta宣布MGTA-117在治疗AML和MDS的I/II期剂量递增试验中，出现了一例患者死亡，暂停了相关临床研究。2月2日，Magenta宣布寻求出售公司/管线等战略选择，计划裁员84%，受到此消息影响，公司股价当日暴涨51.66%，侧面反应出股东们认为公司应该暂停意义不大的管线研发，选择其他可以实现股东价值最大化的途径。三、SPAC上市5月，有4家生物医药公司宣布与SPAC合并上市，不仅数量上比4月少了近50%，而且4家拟合并的生物医药公司质地均为一般，尚未凸显较大的投资价值。欢迎加入美股滚雪球的知识星球，查看我们的行业分享以及每周更新的多个投资机会：一、我们专注的领域1、美股生物医药股投资（核心领域，优势明显）；2、中概股、美股云计算板块、美股互联网板块投资（重点领域，擅长中长线）；3、美股IPO、次新股、SPAC、ETF投资（关注领域，水平中等）；二、我们提供的服务1、每日：医药板块速评（包括板块走势、持仓标的动态、新列入观察标的情况）、每天交易机会提示（不限于医药股）；2、每周：至少推荐一只值得交易的标的分析、建仓价格和仓位建议等；3、每周：白马股、成长股、短期交易股、烟蒂股、次新股等五大类，七小类关注标的名单定期更新；4、每季：核心标的和重点观察标的季报更新；5、不定期投资逻辑分享，如逆向并购概念股、SPAC公司、中概股打新等；封面图来源：123rf精彩推荐专利悬崖之后，有哪些商机？打败了降糖这个BOSS后，礼来Tirzepatide的「中国人群」数据还有哪些隐藏彩蛋？乘风破浪，聚力同行！——「博腾药物开发者论坛」成功举办！GLP-1赛道爆火！2023年都发生了哪些大事？面临的挑战有哪些？点击阅读原文，与药时代一起快乐学习！

并购IPO临床2期临床3期

2023-02-06

·药时代

患者死亡，在研管线全凉，股价却大涨50%……？

前言风干两年的咸鱼，下了油锅居然还蹦跶一下，这种奇事还真就发生了。2023年1月25日，Magenta Therapeutics宣布，由于一名患者在服用药物后死亡，研究暂停，并重新评估药物安全性。2月2日，Magenta宣布要对战略替代方案进行全面审查。隔天，Magenta股票居然大涨51.66%。即便如此，现在它的市值也不到巅峰时的一个零头。01撞到头破血流才停下的临床试验Magenta这款可能导致了患者死亡的MGTA-117，是一款ADC药物，靶点为CD117，主要作用于骨髓中的造血干细胞和癌细胞。该药物正在急性髓系白血病和髓系综合性复发患者中进行剂量递增试验。MGTA-117的I/II期临床试验一波三折，过强的毒性已经早早为悲剧埋下伏笔。一开始，MGTA-117的IND申请就被FDA打回。FDA要求Magenta开发额外的生物测定方法，用于安全检测和剂量递增试验设计。2021年9月，临床暂停解除，但随着试验阶段进行，剂量不断提升，又出事了。2022年12月，MGTA-117连续出现两起4级呼吸系统严重不良反应事件。这一消息导致MGTA股价大跌50%。Magenta决定停止该队列（队列4）用药，但依旧根据队列3为新患者用药。尽管队列3的药物剂量少于队列4，但2022年1月25日，又有一名患者出现了呼吸衰竭与心脏骤停的5级严重不良反应，最终导致患者死亡。Magenta表示，该事件可能与MGTA-117有关，试验暂停。图片来源：雪球02墙外开花的亮眼产品Magenta这家公司诞生伊始，本和ADC没啥关系，他们的核心领域一度是干细胞疗法。Magenta推出的第一款干细胞治疗产品MGTA-456 ，是一种经过扩增的 cd34+ 细胞治疗产品，可用于快速诱导并维持造血干细胞的植入。根据Magenta在2020年公布的一项2期临床数据，该产品在神经和认知功能方面具有临床意义和持久的益处，并可解决脑肾上腺脑营养不良症(CALD)患儿的脑炎症。Magenta借助MGTA-456画了个大饼，让不少投资机构吃撑了。Magenta2018年上市时可谓是众星捧月，Atlas venture、Third Rock等知名投资公司领投。不幸的是，2020年6月，MGTA-456由于患者入组困难、新冠影响与临床试验设计等原因研究终止。另一款与MGTA-456相似的干细胞疗法MGTA-145接过MGTA-456的接力棒，成为了Magenta的主要管线。Magenta目前的管线布局如果只有这两款产品，Magenta恐怕只是一家平平无奇的小biotech，在医药行业群星中，只能当一个小小的背景板。但有野心的Magenta没有止步于此，“罪魁祸首”MGTA-117诞生了。干细胞移植需要对患者进行清髓，清除患者体内原有的干细胞。传统清髓手段主要依靠化疗，而Magenta另辟蹊径，希望通过ADC药物MGTA-117耗尽血液或骨髓中表达CD117的靶细胞，让患者无需化疗即可完成清髓。就这样，一家研究干细胞疗法的公司也搞起了ADC。在2019年12月9日的美国血液学会年会（ASH）上，美国国立卫生研究院分子和临床血液学部主任John Tisdale博士的口头报告专门强调了MGTA-117的研究进展，表明“这是一次非常成功的试验，人类首次在不使用放、化疗的情况下，通过ADC的手段，成功将基因修饰细胞移植到非人灵长类动物中。”03Magenta为什么这么头铁2021年3月，Magenta公布了2020年财报，一年亏损七千多万美元，现金流只能维持到2023年。这一消息让投资者望风而逃，一年时间，股价从最高值14.2美元跌到4.4美元左右。Magenta也成为了2022医药行业裁员大潮中的一朵小浪花。2022年4月，Magenta宣布裁员14%，并暂停MGTA-145计划在健康受试者中进行的临床试验。自此Magenta的核心管线停摆，ADC产品MGTA-117成为了Magenta最后的希望。MGTA-117的成败直接关系到整个公司的命运。也许这就是在出现了严重不良反应之后，MGTA-117的临床研究仍没有停止的原因。最终，患者死亡事件为这家公司的结局标上句点。最后的赌博全盘皆输，资本不相信眼泪，留给Magenta的时间不多了。Magenta的结局，很可能是被收购，或者与其他公司合并。即便是乐观估计，恐怕也得卖掉自己的管线。也可能是由于卖身传闻，投资者才纷纷涌入，致使股价大涨。投资者们希望会出现溢价收购的情况，此刻购入或许只是为了抄底，赌一把可以在Magenta后续战略调整中抢到点残羹冷炙罢了，并非真的看好这家公司。Magenta这次股价大涨，不过是从0.56美元的低价涨到0.85美元，勉强追回2022年12月患者发生4级严重不良反应事件后的股价而已。咸鱼没有翻身，只是被油锅煎得翘了起来。03小结有破釜沉舟般的勇气，不撞南墙不回头，是科学界成功的要素之一。近些年杀出重围的传奇biotech们，多少都有一些这样的特质。但他们是坚信自己的产品能成功，而不是拒绝接受已经失败的事实。像Magenta这样不能“力拔山兮”却“气盖世”，连壮烈自刎留个体面的机会都没有了。临死前最后的回光返照，只是投机客的引渡冥灯，悲兮悲兮。参考资料https://endpts.com/magenta-halts-stem-cell-work-and-may-sell-itself-following-patient-death-clinical-hold/https://endpts.com/patient-death-spurs-trial-halt-for-magenta-therapeutics/magenta官网消息其他网络公开资料封面图来源：pixabay推荐阅读正月十五猜灯谜！全是新药专题，您能猜中几道？～~ 附：十五家药企、机构之精美元宵节海报！~~专栏 | 知名投资专家柳达：化学药的开发有无范式？卖了8年的PD-1，还能保持20%的增长率，它是怎么做到的？这款药被2022版上海市新冠病毒感染诊疗规范推荐，临床价值在哪里？罗氏2022，营收近700亿美元！新增长抵消新冠影响诺和诺德2022财报公布：全年销售额大涨25%，GLP-1产品拉动整架马车点击这里，报名创新药BD高阶研讨会【上海站】！

抗体药物偶联物临床2期临床申请临床1期细胞疗法

2023-02-03

·BioSpace

Magenta Searching for Options after Patient Death Halts Trial

Courtesy of Getty Images Magenta Therapeutics has suspended the development of its clinical programs and initiated a strategic review of business alternatives after a patient's death halted a Phase I/II trial. The Massachusetts biotech paused a dose-escalation study of its lead asset, MGTA-117, in acute myeloid leukemia on Jan. 26th after a patient suffered respiratory failure and cardiac arrest. The death was deemed possibly related to the study drug. The trial hold was voluntary but by the recommendations of a safety Cohort Review Committee. Magenta reported the incident to the FDA as a Suspected Unexpected Serious Adverse Reaction. As of Sept. 30th, 2022, Magenta still had cash, cash equivalents and marketable securities amounting to $128.3 million. The company revealed in its third-quarter financial results, posted November 2022. At the time, Magenta expected its cash runway to extend through the middle of 2024. As part of its business review, the company will assess the feasibility of an acquisition, merger, business combination or other similar transactions. Though Magenta made no assurances that the review would lead to an attractive business option, the company’s shares rose 47% post-market trading Thursday. Suspended Stem Cell Programs Magenta's pipeline is focused on stem cell transplantation. The company was developing novel antibody-drug conjugates to enable either mobilization and collection, wherein stem cells are extracted from a patient’s or donor’s bone marrow, or conditioning, which involves the removal of stem cells from the recipient’s bone marrow. While ADCs have long been known to be effective against specific cancers, Magenta has pioneered its use in stem cell transplantation. MGTA-117 was tested as a conditioning agent. The candidate targeted the CD117 receptor and was designed to selectively deplete cells bearing this protein, thereby reducing or eliminating the need for chemotherapy. CD117 is a cell surface-bound protein enriched in hematopoietic stem cells and leukemia cells, making it a good conditioning target for many blood disorders. Magenta was also developing a second investigational conditioning agent, a CD45-targeted ADC. This candidate was in preclinical studies and had recently completed dose-ranging toxicology assessments with no unexpected findings. MGTA-145, an agonist of the CXCR2 protein, was designed to be used with plerixafor, a CXCR4 antagonist, to target complementary pathways to improve the process of stem cells being released into the bloodstream. The candidate was in two Phase II trials.

临床2期财报细胞疗法抗体药物偶联物临床1期

100 项与 GRO beta 相关的药物交易

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研发状态

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
多发性骨髓瘤	临床2期	美国	Stanford University	2020-10-05
干细胞动员	临床2期	美国	Magenta Therapeutics, Inc.	2020-09-14
造血干细胞移植	临床1期	-	Magenta Therapeutics, Inc.	-

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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


NCT04762875 (CTgov)	临床2期	急性髓性白血病 \| 急性淋巴细胞白血病 \| 骨髓增生异常综合征	7	MGTA-145+Plerixafor	繭膚觸構構壓餘願遞夢(鹽壓餘窪觸選淵鬱獵餘) = 鑰衊選鏇遞網觸夢範襯鏇獵壓窪憲範鏇夢夢襯 (襯鹹襯憲願觸鏇衊網鑰, 網蓋艱簾築糧蓋觸衊顧 ~ 積壓糧構憲鏇廠獵糧築) 更多	-	2024-08-29
NCT04552743 (MGTA-145 + Plerixafor, CTgov)	临床2期	多发性骨髓瘤	25	MGTA-145+plerixafor	選襯構鹽齋獵積壓衊餘(範衊鑰鏇餘衊鑰築憲醖) = 積襯襯範觸窪鬱鑰鹹齋鑰網廠衊製築鬱鏇淵蓋 (鹽鏇窪壓壓築夢憲構餘, 窪齋餘鬱鑰願顧鑰觸餘 ~ 膚網積膚積夢顧遞憲醖) 更多	-	2022-09-10
NCT04552743 (MGTA-145 + Plerixafor, Tandem Meetings ASTCT and CIBMTR2022)	临床2期	多发性骨髓瘤	25	MGTA-145 + Plerixafor	簾膚製鬱壓鏇遞範廠鏇(窪窪築淵願蓋糧繭膚繭) = At least 1 treatment emergent AE (TEAE) with MGTA-145 was seen in 60% of patients (grade 1, n= 14, grade 2, n=1). Pain (all grade 1) was most common, seen in 44% (11), with 38% (9) patients experiencing acute onset transient bone pain with MGTA-145 (duration: 7 minutes, range: 3-28) 鏇襯窪夢襯構糧製壓鹽 (憲醖鏇鹽糧廠鑰襯築膚 )	积极	2022-03-01
NCT04552743 (ASCO2021)	临床2期	多发性骨髓瘤	25	MGTA-145 + plerixafor	(積夢願網淵糧夢選鏇夢) = At least 1 adverse event (AE) was seen in 90% of patients, 20% had grade 2 AEs (anemia, hypokalemia) and 20% had grade 3 AEs (worsening of baseline grade 3 anemia; hypocalcemia); all resolved. Acute & transient bone pain was seen in 40% of patients (back-2, hip-1, sternum-1), all grade 1, all on day 1, & resolved without intervention after 6 minutes (3-10) 廠憲窪鑰糧夢選願鏇鑰 (艱積夢遞淵製廠襯簾鬱 )	积极	2021-05-28
Tandem Meetings ASTCT and CIBMTR2021	N/A	-	-	MGTA-145 + plerixafor	襯淵衊憲製壓選夢醖憲(艱構顧襯鑰獵範壓遞鹹) = 顧選膚積願蓋積網糧醖廠繭衊構醖鹹糧膚糧糧 (獵鑰顧廠顧鹹膚膚網顧 )	-	2021-03-01
Phase 1 Clinical Study of MGTA-145 (ACR2020)	临床1期	CXCR2 \| MMP-9 \| TIMP-1	-	MGTA-145	(膚鹽獵鬱蓋顧醖鹽積齋) = MGTA-145 monotherapy was well tolerated with no significant adverse events (AEs). Grade 1, transient lower back pain that dissipated within minutes was reported. The combination of MGTA-145 with plerixafor was well tolerated, with some subjects experiencing grade 1/2 gastrointestinal AEs commonly observed with plerixafor and one grade 2 back pain with MGTA-145 at 0.075 mg/kg that resolved within minutes. 齋糧選膚鑰簾獵膚簾膚 (壓鑰繭憲窪簾廠醖獵蓋 ) 更多	积极	2020-11-06
NCT03932864 (P665, EBMT2020)	临床1期	一线	-	MGTA-145	(構膚網築餘夢餘鬱遞憲) = MGTA-145 monotherapy was well tolerated with no significant adverse events (AEs). Grade 1, transient lower back pain that dissipated within minutes was reported. The combination of MGTA-145 with plerixafor was well tolerated, with some subjects experiencing grade 1/2 gastrointestinal AEs commonly observed with plerixafor and one grade 2 back pain with MGTA-145 at 0.075 mg/kg that resolved within minutes. 夢蓋鏇糧艱壓糧積簾餘 (鏇膚廠觸憲衊築範範艱 )	积极	2020-08-29
EULAR2020	临床1期	一线	-	MGTA-145	(鑰衊願觸糧觸襯簾鑰築) = some subjects experiencing grade 1/2 gastrointestinal AEs commonly observed with plerixafor and one grade 2 back pain with MGTA-145 at 0.075 mg/kg that resolved within minutes 糧選醖構衊廠觸範繭夢 (鏇夢壓範廠選製觸憲齋 ) 更多	-	2020-06-03
Tandem Meetings ASTCT and CIBMTR2020	N/A	-	-	MGTA-145	觸壓鬱憲鬱構醖構鬱鑰(餘齋鏇製觸憲遞齋鹹獵) = MGTA-145 monotherapy was well tolerated with no significant adverse events (AEs). Grade 1, transient lower back pain that dissipated within minutes was reported. The combination of MGTA-145 with plerixafor was well tolerated, with some subjects experiencing grade 1/2 gastrointestinal AEs commonly observed with plerixafor and one grade 2 back pain with MGTA-145 at 0.075 mg/kg that resolved within minutes. 積膚顧鬱襯遞網製遞獵 (醖選衊淵積範蓋餘鬱蓋 ) 更多	-	2020-03-01
Tandem Meetings ASTCT and CIBMTR2019	N/A	-	-	MGTA-145 plus plerixafor	糧顧積繭淵網簾繭顧顧(廠構廠膚鹽顧願築積獵) = 範衊壓遞簾觸憲築蓋淵餘壓觸鏇醖網選願構襯 (觸夢製鹹艱網窪餘齋蓋 )	-	2019-03-01