Andexanet alfa

SAN DIEGO, CA, USA I January 17, 2024 I Tr1X, Inc. today announced its emergence from stealth with a $75 million Series A financing to bring universal allogeneic regulatory T (Treg) and CAR-Treg cell therapies to the clinic to treat and potentially cure autoimmune and inflammatory diseases. The financing was led by Bay Area-based The Column Group, with participation from NEVA SGR and Alexandria Ventures. The Company appointed William Lis Chief Executive Officer, strengthened its leadership team with other biopharma industry veterans, and announced members of the Board of Directors and Scientific Advisory Board. "We are thrilled to partner with Bill Lis, Maria Grazia Roncarolo and Tr1X's world-class team to revolutionize the field through its innovative, breakthrough science," said Leon Chen, Ph.D., MBA, Partner at The Column Group and Tr1X Board Member. "With its experienced management team at the helm, we are confident in Tr1X's ability to bring curative cell therapies to patients who currently require life-long treatment and management." Science and Pipeline The Company's science is primarily based on the work of Tr1X's Scientific Founder Maria Grazia Roncarolo, M.D., the discoverer of Type 1 regulatory T (Tr1) cells, which have features that can benefit patients with autoimmune and inflammatory diseases. Tr1 cells, a differentiated subpopulation of regulatory T cells, are crucial for maintaining homeostasis and tolerance in healthy individuals and have several important functions. These include dampening of local inflammation and downregulation of the inflammasome, suppression of pathogenic effector T cell responses, and induction of long-term tolerance as observed in preclinical models and patients. Tr1X's proprietary technology enables the conversion of CD4 + T cells isolated from healthy donors into Treg-like cells that have a similar function and profile to naturally occurring Tr1 cells. These cells can be further engineered to target specific tissues or organs to enable local, targeted immunomodulation. With a proprietary, GMP-grade, closed-loop system that provides consistency, quality and reliability at scale, Tr1X can enable production of its products at commercial volume. Currently, Tr1X is working on its first investigational Tr1 cell therapy, TRX103, which it plans to evaluate in a Phase 1 trial to prevent Graft versus Host Disease (GvHD) in patients undergoing mismatched hematopoietic stem cell transplant. The Company is developing additional pipeline programs to treat inflammatory bowel disease, Type 1 diabetes and multiple B-cell mediated autoimmune diseases. "The ability to develop a pipeline of medicines based on our work on regulatory T cells represents the culmination of decades of discovery and research into the underpinnings of immunological tolerance and autoimmunity," said Dr. Roncarolo, Scientific Founder, President and Head of R&D at Tr1X. "Tr1 cells have unique properties and represent the ideal therapeutic platform from which to develop 'immune reset' products. Our two platforms have the required attributes to address a broad set of indications, including dual targeting of pathogenic T and B cells. These engineered cells have the potential to induce tolerance, which could transform the lives of people living with chronic autoimmune diseases by providing them with a cure instead of ongoing treatment." Proven Leadership Team, Board of Directors and Scientific Advisory Board Tr1X is led by industry veterans with cell therapy expertise who have a track record of discovery, development and commercialization of more than a dozen novel first- or best-in-class medicines. Chief Executive Officer William "Bill" Lis has more than 25 years of biotech executive leadership experience in building companies and product pipelines from R&D stage through commercialization. He most recently served as Chairman and interim CEO of Jasper Therapeutics, Inc., from 2019 to 2022, where he led the company's Series A and follow-on financings and successful brequilimab Phase 1 study readouts in hematopoietic stem cell transplantation. Before that, he served as Chief Executive Officer and a Director of Portola Pharmaceuticals, Inc. from 2008 to 2018 after serving as Chief Operating Officer, where he built the company from a private research-stage startup into a multi-billion dollar public company that launched Andexxa ® and Bevyxxa ® . Portola was acquired by Alexion Pharmaceuticals, Inc. in 2020. Previously, Mr. Lis held executive positions at Scios, Inc. (a Johnson & Johnson company) where he last served as Senior Vice President of New Product Development and Business Development, leading in-licensing efforts, development and commercial P&L operations for Xarelto ® and the company's therapeutics pipeline. Earlier in his career, he held positions at Millennium Pharmaceuticals, Inc., COR Therapeutics, Inc. and Rhone Poulenc Rorer. He is a director of Zai Laboratories, Inc. Scientific Founder, President and Head of R&D Dr. Maria Grazia Roncarolo is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and Medicine, Founder of the Center for Definitive and Curative Medicine, and former Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University. Dr. Roncarolo has spent her career translating discoveries in immune-mediated diseases and regenerative medicine into novel patient therapies. She and her team were the first to discover Type 1 regulatory T cells in patients and the first to develop curative treatments for ADA-SCID. A pediatric immunologist by training, she was instrumental in bringing novel cell and gene therapies to market, including Strimvelis ® and Libmeldy ® . She is a Co-Founder of the gene-editing companies Graphite Bio and Kamau Therapeutics. Co-Founder and Chief Operating Officer David de Vries has over a decade of experience founding and scaling companies at the intersection of healthcare, biology and technology. Prior to Tr1X, he was Co-Founder and Chief Operating Officer of Arine, a medication intelligence and therapeutic optimization company he helped start, launch and scale to serve millions of patients, working with some of the largest healthcare organizations in the U.S. Previously, he worked at Proteus Digital Health, an innovative drug-device company and the first in the field to obtain FDA approval for a digital medicine, Abilify MyCite ® . "Tr1X is committed to bringing cures to millions of patients suffering from autoimmune diseases through our scalable, universal allogeneic cell therapy process that has been demonstrated to yield billions of cells in a single run," said Mr. Lis, CEO of Tr1X. "In just 18 months, the Tr1X team has successfully closed a Series A financing and extension, assembled an experienced leadership team, board of directors and scientific advisory board, and established a scalable GMP-grade manufacturing process. We are well positioned to advance our pipeline of first-of-their kind allogeneic engineered Treg and CAR-Treg cell therapies that have the potential to address the significant limitations of current cell therapies for autoimmune diseases, which include, among others, poor trafficking, lack of stability, limited persistence and induction of cytokine-associated toxicities, at a fraction of the cost per dose." In addition to Mr. Lis, Dr. Roncarolo and Mr. de Vries, the Tr1X leadership team includes Xavier Paliard, Pharm.D., Ph.D., Chief Scientific Officer; James Adams, MBA, Chief Technical Officer; Jonathan Perrin, J.D., Co-Founder and General Counsel; and Erik Poulsen, M.S., Head of Regulatory Affairs. Joining Mr. Lis and Dr. Roncarolo on Tr1X's Board of Directors are: Tr1X has recruited experts in immunology, stem cell and gene therapy, T-cell biology and immune-mediated diseases to join its Scientific Advisory Board. They include: About Tr1X Tr1X is a privately held biotechnology company focused on engineering cures for immune and inflammatory diseases. Founded by industry veterans, including the scientists behind the discovery of Tr1 cells, the company's pipeline of universal allogeneic cell therapies is being developed for diseases with high unmet medical need. The company is headquartered in San Diego, has developed a robust intellectual property portfolio, and is backed by leading investors, including The Column Group, NEVA SGR and Alexandria Ventures. For more information visit: www.tr1x.bio . SOURCE: Tr1X

「  本文共：16条资讯，阅读时长约：3分钟 」今日头条诺诚健华TYK2抑制剂Ⅱ期临床积极。诺诚健华新型酪氨酸激酶2（TYK2）抑制剂ICP-332治疗中重度特应性皮炎（AD）的Ⅱ期临床达到主要终点。与安慰剂相比，ICP-332（80m、120mg）每日一次治疗组患者的EASI（湿疹面积和严重程度指数）评分较基线的平均百分比变化显著更高（78.2%和72.5%，vs16.7%），达成EASI 75应答的患者比例更高（64%和64%，vs8%）。此外，药物耐受性良好。国内药讯1.罗氏眼科双抗中国获批上市。罗氏眼科注射双特异性抗体法瑞西单抗（商品名：罗视佳®，Vabysmo®）获国家药监局批准上市，用于治疗糖尿病黄斑水肿（DME）。这是全球首个双通路眼底创新治疗药物，同时靶向抗血管生成素-2（Ang-2）和血管内皮生长因子A（VEGF-A）的双重作用机制。在美国，该产品目前已获批DME、湿性年龄相关性黄斑变性（wAMD）和视网膜静脉阻塞继发黄斑水等适应症。2.AZ创新止血疗法中国报产。阿斯利康与百特公司申报的3.1类生物制品注射用andexanet alfa（AndexXa）的上市申请获CDE受理。andexanet alfa是一款凝血因子Xa抑制剂逆转剂，能够快速逆转因子Xa抑制剂rivaroxaban和apixaban的抗凝血作用，控制严重和不可控制的出血。今年6月，阿斯利康宣布Andexxa在上市后IV期试验（ANNEXA-I）中期结果中达到止血优效性标准，并提前终止该项研究。3.信达HER3-ADC上实体瘤临床。信达生物靶向HER3的ADC新药IBI133在Clinicaltrials.gov网站上注册一项Ⅰ期临床，计划在澳洲评估IBI133用于治疗晚期实体瘤患者的安全性与有效性。该项试验预计2025年底初步完成。目前全球范围内尚未有靶向HER3的疗法获批上市。目前，信达生物已有5款ADC进入临床阶段，分别为CEACAM5 ADC（赛诺菲引进）、Claudin18.2 ADC、HER2 ADC、Trop2 ADC和HER3 ADC。4.九天生物SMA基因疗法上I期临床。九天生物AAV基因治疗药物SKG0201注射液获国家药监局批准开展I期临床，拟评估用于治疗I型脊髓性肌萎缩症(SMA)的安全性与有效性。SKG0201包含由独特启动子调控并全面优化的人源SMN1 cDNA。临床前数据表明，SKG0201单次静脉注射不仅可快速、持久地恢复功能性SMN蛋白表达，而且在低剂量水平下即可显著增加SMA疾病模型小鼠的体重并有效地延长其寿命。5.礼来IL-13抗体中国获批临床。礼来1类生物制品lebrikizumab注射液（Ebglyss）获国家药监局临床默示许可，拟开发用于治疗中重度特应性皮炎（AD）青少年和成人患者。lebrikizumab是Almirall公司与礼来联合开发的IL-13抑制剂，通过与IL-13结合，以阻止IL-13Rα1/IL-4Rα异二聚体复合物的形成以及后续信号传导。今年11月，欧盟委员会已批准Ebglyss上市，用于治疗12岁及以上中重度特应性皮炎患者。6.优诺DC细胞疗法获批胰腺癌临床。广东优诺生物UNODC0407注射液获国家药监局临床试验默示许可，拟开发用于治疗不可手术切除的局部进展期胰腺癌或转移性胰腺癌。UNODC0407是利用肿瘤抗原对患者自体树突状细胞（DC）进行特异性免疫刺激培养的一款肿瘤治疗性疫苗。它通过DC将肿瘤匿名抗原呈递给效应性淋巴细胞，从而诱导更有效的肿瘤免疫反应并杀伤肿瘤细胞。国际药讯1.DMD类固醇新药获欧盟批准上市。Santhera公司类固醇药物AGAMREE®（vamorolone）获欧盟（EU）批准上市，用于治疗4岁及以上患者的杜氏肌营养不良症（DMD）。在IIb期临床（VISION-DMD）中，vamorolone的疗效与强的松相比达到非劣效标准；而且并未发现vamorolone在骨骼健康生物标志与线性生长上有负面的影响。在中国，曙方医药拥有该产品在大中华区开发和商业化的独家权益。2.全球首款CRISPR基因疗法获欧盟支持上市。Vertex公司与CRISPR Therapeutics开发的CRISPR/Cas9基因编辑疗法Casgevy（exa-cel）获欧洲药品管理局（EMA）人用药品委员会（CHMP）推荐有条件批准上市。这是全球首款CRISPR基因编辑疗法，FDA已于12月8日批准Casgevy上市，用于治疗12岁及以上患有复发性血管闭塞危象（VOC）的镰刀型细胞贫血病（SCD）患者。据媒体报道，该产品每位患者的治疗费用高达220万美元。3.肿瘤mRNA疫苗联合K药3年随访数据积极。默沙东与Moderna开发的肿瘤新抗原mRNA疫苗V940（mRNA-4157）联合PD-1抑制剂Keytruda治疗完全切除的高危黑色素瘤（III/IV期）患者的IIb期KEYNOTE-942/mRNA-4157-P201研究最新结果积极。中位随访为3年时，V940组合辅助治疗较Keytruda持续改善患者的无复发生存期（RFS），将复发或死亡风险降低49%（HR=0.510 [95%CI：0.288-0.906]；P=0.0095）；将发生远处转移或死亡的风险降低62%（HR=0.384 [95%CI：0.172-0.858]; p=0.0077）。该研究中V940的安全性与之前报告一致。4.GLP-1基因疗法减重临床前研究积极。Fractyl Health公司开发的基因疗法GLP-1 PGTx在治疗肥胖症的临床前研究中获积极结果。与司美格鲁肽（10 nmol/kg/day）治疗相比，小鼠肥胖模型单剂输注GLP-1 PGTx后28天，小鼠体重降幅显著更多（27%vs21%）；在小鼠停用司美格鲁肽之后接受单剂GLP-1 PGTx或安慰剂治疗，转为接受GLP-1 PGTx治疗的小鼠体重降幅较基线达到26%，而转为接受安慰剂的小鼠体重增加19%。该公司计划明年进行IPO，以推进GLP-1 PGTx的临床开发。5.眼科补体C3疗法未获欧盟支持上市。Apellis公司靶向C3补体蛋白的聚乙二醇化（PEGylated）双环肽疗法pegcetacoplan（Syfovre）的上市许可申请(MAA)未获得欧洲药品管理局（EMA）人用药品委员会（CHMP）支持，以用于治疗年龄相关性黄斑变性(AMD)继发地理性萎缩(GA)。发表在《柳叶刀》上的Ⅲ期（OAKS和DERBY）研究数据显示，每隔一个月和每月一次的Syfovre治疗均能显著减少GA病变的生长。今年2月，FDA已批准Syfovre用于治疗由AMD引起的地图样萎缩（GA）。6.AI小分子药物公司Deep Apple完成A轮融资。人工智能（AI）小分子药物公司Deep Apple宣布完成5200万美元的A轮融资，以用于推进GPCR调节剂开发，应用于代谢紊乱、炎症、免疫学和内分泌疾病。Deep Apple利用其冷冻电镜技术、深度学习和超大型分子对接筛选文库，能够在12个月内完成从靶标识别到先导物优化。Deep Apple的发现引擎适用于广泛疾病领域，尤其适合针对完整膜蛋白的加速发现。医药热点1.广东省全面启动加速消除宫颈癌行动。12月13日，广东省卫健委公布《广东省加速消除宫颈癌行动计划（2023—2030年）》，明确了2030年前完成三大目标：持续推进适龄女孩HPV疫苗接种服务，15岁之前适龄女生HPV疫苗首针接种完成率达到90%；35—64岁妇女宫颈癌筛查率达到70%；确诊宫颈癌及癌前病变患者治疗率达到90%。2.瑞金医院卢湾分院帕金森病诊疗中心成立。12月15日，上海交通大学医学院附属瑞金医院卢湾分院“帕金森病卓越诊疗中心”正式揭牌。瑞金医院卢湾分院脑病中心主任陆勇表示，帕金森病卓越诊疗中心以创新技术群和医疗新模式为先导，为帕金森病患者提供高效、全面的一站式服务，旨在有效减轻患者的残疾症状、提高生活质量，通过数字化智能及互联网技术赋能居家医疗，实现全流程智能化服务。3.北大医院大兴院区正式启用。12月18日，北京大学第一医院大兴院区正式启用。北大医院大兴院区距离中心院区直线距离约22公里，建有门诊楼、医技楼、急诊楼、住院楼、科研楼、行政教学楼，占地面积21.6万平方米，编制床位数1200张，其中儿科床位400张、妇产科床位200张、其他综合床位600张。大兴院区开办将以“先门诊、后病房，先平台、后临床，先专科、后综合”的基本步骤实施，以“大专科、小综合”进行学科布局，与中心院区差异化发展。评审动态  1. CDE新药受理情况（12月16日) 2. FDA新药获批情况（北美12月15日）股市资讯上个交易日 A 股医药板块 +0.02%涨幅前三     跌幅前三科华生物+10.03%  英 诺 特 -5.90%大理药业 +9.99%  ST吉药 -5.90%赛托生物+6.90%  *ST太安-5.14%【安科生物】安徽安科生物工程（集团）股份有限公司收到国家药品监督管理局下发的AK1012 吸入用溶液《药物临床试验批准通知书》，批准公司“AK1012 吸入用溶液”开展临床试验【海思科】获得创新药 HSK39297 片《药物临床试验批准通知书》。【华东医药】全资子公司杭州中美华东制药有限公司美国合作方对外宣布，其用于治疗9岁及以上患者脂溢性皮炎的药物 ZORYVE®（罗氟司特）泡沫剂（0.3%）获得美国食品药品监督管理局（FDA）批准上市。- The End -戳“阅读原文”，了解更多医药研发及股市资讯。