MLL1

「  本文共：16条资讯，阅读时长约：3分钟 」今日头条安斯泰来ADC报sBLA。安斯泰来靶向Nectin-4的ADC药物Padcev（enfortumab vedotin）的上市申请获CDE受理，联合PD-1抑制剂Keytruda一线治疗局部晚期或转移性尿路上皮癌（la/mUC）。在Ⅲ期EV-302临床中，与化疗相比，Padcev/Keytruda组合显著延长了患者的PFS和OS，使疾病进展或死亡风险降低55%，死亡风险降低53%。目前，该药用于治疗既往接受PD-1/L1和含铂化疗的la/mUC患者的上市申请也正接受CDE审查。国内药讯1.荣昌眼科新药完成Ⅲ期临床入组。荣昌生物宣布其VEGF/FGF双靶点融合蛋白RC28-E治疗糖尿病性黄斑水肿（DME）的Ⅲ期临床已完成全部患者入组。目前，荣昌生物正在开展RC28-E与阿柏西普头对头的两项Ⅲ期临床试验，分别用于糖尿病性黄斑水肿（DME）和湿性年龄相关性黄斑变性（wAMD）。RC28-E同时拮抗VEGF和成纤维细胞生长因子（FGF），有望基于FGF与VEGF的协同作用，发挥更强的治疗作用同时延长给药间隔。2.友芝友双抗上恶性腹水III期临床。武汉友芝友生物自主研发的EpCAM/CD3双抗M701在用于肿瘤所致恶性腹水的支持性治疗的III期临床完成了首例患者入组。M701可通过CD3招募患者自身免疫T细胞，继而通过EpCAM引导免疫细胞靶向肿瘤细胞，最后启动T细胞杀伤肿瘤细胞，从而达到治疗腹水的效果。在Ⅰ期临床中，M701总体客观反应率达到62.5%，疾病控制率为100%，总生存时间中位数（mOS）为152天。3.O药联合治疗结直肠癌优先审评。百时美施贵宝PD-1抑制剂Opdivo（nivolumab）与CTLA-4抗体Yervoy（ipilimumab）组合方案获CDE拟纳入优先审批，用于一线治疗不可切除或转移性微卫星高度不稳定性（MSI-H）或错配修复缺陷型（dMMR）结直肠癌（CRC）患者。在III期CheckMate-8HW研究中，与化疗相比，这一组合方案使这类患者的疾病进展或死亡风险降低了79%（p<0.0001）。本月初，这两款新药用于治疗上述适应症已被纳入突破性治疗品种。4.上海甫康TKI获胃癌孤儿药资格。甫康药业自主研发高脑渗透性HER2 TKI创新药CVL009获FDA授予孤儿药资格，用于治疗胃癌（包括胃食管交界处癌）。CVL009属于不可逆的作用于泛HER家族的药物，不仅可以抗HER2，也可以抑制HER1和HER 4，从而阻止HER家族之间形成二聚体后的下游传导通道，作用机制更强效。在国内，CVL009治疗胃食管癌脑转移患者的II期临床即将在河南省肿瘤医院等中心开展。5.国产十五价HPV疫苗启动I期临床。康乐卫士与成大生物合作开发的重组十五价人乳头瘤病毒疫苗（大肠埃希菌）已登记启动一项I期临床，拟评价用于9-45周岁健康女性受试者接种该种疫苗的安全性、耐受性和免疫原性。据悉，这是全球价型最高的在研HPV疫苗，覆盖WHO指出的全部13个HPV高危型，以及与尖锐湿疣高相关的HPV6/11型。该项试验主要研究者为江苏省疾控中心李靖欣主任医师。国际药讯1.肾性贫血创新药获FDA批准上市。Akebia公司口服缺氧诱导因子脯氨酰羟化酶抑制剂（HIF-PHI） Vafseo（vadadustat）片剂获FDA批准上市，用于治疗接受透析至少三个月的慢性肾病（CKD）相关贫血患者。HIF会调控铁元素的动员和促红细胞生成素（EPO）的产生，以此刺激血红细胞的生成，从而改善氧气运输。在两项INNO2VATE研究中，与darbepoetin α（一种血红细胞刺激剂）相比，vadadustat治疗使患者的血红蛋白（Hb）水平较基线变化达到非劣效性标准。2.Syndax公司白血病新药获优先审评。Syndax 公司潜在“first-in-class”menin抑制剂revumenib的上市申请获FDA受理，用于治疗携带KMT2A重排（KMT2Ar）的复发/难治性急性白血病患者，同时FDA也授予其优先审评资格。在关键AUGMENT-101研究中，revumenib在携带KMT2Ar的急性白血病人群中达到23%（p=0.0036）的完全缓解（CR）率或带部分血液学恢复的完全缓解（CRh）率。此前，该新药这一适应症已被FDA授予突破性疗法认定。3.Novocure公司肿瘤电场疗法肺癌Ⅲ期临床积极。Novocure公司肿瘤治疗电场（TTFields）治疗非小细胞肺癌（NSCLC）的Ⅲ期临床METIS达到了主要终点。TTFields旨在通过多种机制施加物理外力，以抑制肿瘤细胞有丝分裂，进而抑制其生长。数据显示，与支持性护理相比，TTFields联合标准治疗显著延长了患者无颅内进展的时间（21.9个月vs11.3个月，P=0.016）；关键性次要终点的初步分析未显示出统计学意义。此外，再鼎医药拥有TTFields的中国权益。4.迷幻色胺抑郁症Ⅱ期临床积极。Beckley Psytech公司基于短效迷幻色胺5-MeO-DMT开发的创新配方BPL-003，在治疗耐药性抑郁症（TRD）的Ⅱa期研究中获积极结果。BPL-003（10mg）单剂量鼻腔给药第二天后，有55%的患者的MADRS评分降低超过50%；第4周时，实现MADRS评分小于10分的临床缓解率达到55%，且疗效维持至第12周。BPL-003总体耐受性良好，药物的急性效应平均在不到两小时内消退。5.罕见遗传性眼病基因疗法早期临床积极。Opus Genetics公司基因疗法OPGx-LCA5治疗LCA5型Leber先天性黑朦（LCA）患者的Ⅰ/Ⅱ期临床试验结果积极。OPGx-LCA5旨在使用腺相关病毒8（AAV8）载体将功能性LCA5基因精确递送到外层视网膜。首批患者给药90天期间，患者视网膜已显示出明显的生物活性迹象；且OPGx-LCA5耐受性良好。该公司计划将OPGx-LCA5推进到下一个最高剂量，并扩大研究人群，纳入13岁或以上的受试者。6.Synthekine公司新型CAR-T获批IND。Synthekine公司CD19 CAR-T产品 SYNCAR-001获FDA批准开展新药研究，联合正交的IL-2配体STK-009，评估联合用药治疗非肾性系统性红斑狼疮（SLE）和狼疮性肾炎（LN）患者的疗效。SYNCAR-001+STK-009是一种基于Synthekine专有正交配体—受体信号平台的细胞因子诱导细胞治疗方案，也是一种双组分疗法。通过利用Synthekine的正交IL-2技术，STK-009有望驱动SYNCAR-001细胞的植入和受控的细胞增殖，而无需进行淋巴清除。7.艾伯维购进FIC自免病小分子。艾伯维与自免病药物公司Landos Biopharma签订最终协议，将以超过2亿美元的总交易金额收购Landos，并获得该公司拟开发用于治疗溃疡性结肠炎（UC）的临床期主打项目NX-13。NX-13是一款潜在“first-in-class”口服NLRX1激动剂，旨在减少活性氧和氧化应激，并同时减少促炎信号，具有抗炎作用并促进上皮修复的潜力。此项交易预计将于2024年第二季度完成。医药热点1. 翁建平任安徽医科大学校长。日前，安徽省委决定，任命翁建平同志为安徽医科大学校长；郑兰荣接替翁建平，任蚌埠医科大学党委副书记、校长。翁建平是中国著名的内分泌专家。翁建平于2020年担任蚌埠医学院院长，2023年12月，蚌埠医学院更名蚌埠医科大学，翁建平出任校长。目前，翁建平还担任中国科学技术大学临床医学院执行院长。2.国家规范助产服务资源调整。3月27日，国家卫健委发布关于加强助产服务管理的通知，强化助产服务规划布局。通知强调，各级妇幼保健机构、二级以上公立综合医院、承担危重孕产妇救治中心任务的公立医疗机构、地广人稀地区的公立基层医疗卫生机构原则上应保障产科持续服务。其他公立医疗机构拟关停产科要广泛征求建档孕产妇意见，书面征求当地街道办事处(乡镇政府)和县级卫生健康行政部门意见，切实保障群众看病就医权益。3.北京3月30日起执行国采政策。据北京市医保局官网3月27日消息，自3月30日起，北京市全面执行第九批国家组织药品集中带量采购中选结果及医保支付标准等相关政策，采购周期至2027年12月31日。第九批国家组织药品集中带量采购涵盖感染、肿瘤、心脑血管疾病、胃肠道疾病、精神疾病等常见病和慢性病用药，以及急抢救药、短缺药等重点药品。本次集采共计41种药品，中选药品价格平均降幅58%。评审动态  1. CDE新药受理情况（03月28日) 2. FDA新药获批情况（北美03月27日）股市资讯上个交易日 A 股医药板块 +0.52%涨幅前三     跌幅前三明德生物+10.02%  新 产 业 -9.71%莱茵生物 +9.25%  绿康生化-8.97%凯因科技 +6.65%  *ST太安-5.11%【泰恩康】全资子公司山东华铂凯盛生物科技有限公司于近日收到国家药品监督管理局签发的富马酸卢帕他定片境内生产药品注册上市许可申请《受理通知书》。【莱美药业】公司参股公司四川康德赛医疗科技有限公司的一款自主研发用于治疗失代偿期肝硬化的巨噬细胞CUD005注射液获得药物临床试验默示许可并取得由国家药品监督管理局核准签发的《药物临床试验批准通知书》。 【百奥泰】2023年营业收入7.05亿元，同比增长54.86%，归母净利润-3.95亿元，同比下降17.87%。 - The End -戳“阅读原文”，了解更多医药研发及股市资讯。

▎药明康德内容团队编辑Syndax Pharmaceuticals日前宣布，美国FDA接受其为在研疗法revumenib递交的新药申请（NDA）并授予其优先审评资格，用于治疗携带KMT2A重排（KMT2Ar）的复发/难治性急性白血病患者。Revumenib是一款潜在“first-in-class”的menin抑制剂。该NDA的提交得到了来自关键性临床试验AUGMENT-101的积极数据的支持，该试验研究了revumenib对携带KMT2Ar的急性髓系白血病（AML）和急性淋巴细胞白血病（ALL）成人和儿科患者的疗效。如先前报告，该试验在中期分析中达到了主要终点，在携带KMT2Ar的急性白血病人群中，57名可评估患者中有23%（13/57；95% CI：12.7，35.8，单边p=0.0036）达到完全缓解（CR）或带部分血液学恢复的完全缓解（CRh）。在达到CR/CRh且进行了最小残留病（MRD）评估的患者中，70%的患者MRD为阴性。此外，患者中的总缓解率（ORR）达到63%（36/57）。Revumenib是一种针对menin-KMT2A相互作用的强效、选择性小分子抑制剂。Revumenib已被FDA和欧洲委员会授予孤儿药资格，用于治疗AML患者，并被FDA授予快速通道资格，用于治疗携带KMT2A重排或NPM1突变的成人和儿科复发/难治性急性白血病患者。Revumenib还被FDA授予突破性疗法指定，用于治疗携带KMT2A重排的成人和儿科复发/难治性急性白血病患者。 大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲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：[1] Syndax Announces FDA Priority Review of NDA for Revumenib for the Treatment of Relapsed/Refractory KMT2Ar Acute Leukemia. Retrieved March 27, 2024, from https://www.prnewswire.com/news-releases/syndax-announces-fda-priority-review-of-nda-for-revumenib-for-the-treatment-of-relapsedrefractory-kmt2ar-acute-leukemia-302100145.html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

– Topline data expected in 4Q24 could support sNDA filing in 1H25 – – Revumenib has the potential to address $2 billion mNPM1 and KMT2Ar R/R acute leukemia U.S. market opportunity – WALTHAM, Mass., March 28, 2024 /PRNewswire/ -- Syndax Pharmaceuticals Inc.(Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with relapsed/refractory (R/R) mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth quarter of 2024 and could support a supplemental New Drug Application (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025. "We are thrilled to announce that we are one step closer to potentially expanding the therapeutic reach of revumenib in genetically defined acute leukemias," said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax. "We look forward to reporting topline data for this pivotal cohort in the fourth quarter of this year, which will follow closely behind a potential first approval of KMT2A acute leukemia in the third quarter." Michael A. Metzger, Chief Executive Officer, added, "With revumenib and axatilimab, two first-and best-in-class drugs, expected to launch in 2024 and the potential to expand beyond first approvals and into additional indications, Syndax is well positioned to deliver on its mission to improve the lives of cancer patients and create meaningful long-term value for shareholders." Sixty-four (64) adult and up to 20 pediatric patients with mNPM1 AML have been enrolled into the pivotal portion of AUGMENT-101, a pivotal trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered revumenib. The primary endpoint for the trial is efficacy as measured by complete remission (CR) or a CR with partial hematological recovery (CRh) rate (CR + CRh) per protocol, with secondary endpoints including duration of response (DOR) and overall survival (OS). A new drug application (NDA) is under review by the U.S. Food and Drug Administration (FDA) for revumenib in R/R KMT2Ar acute leukemia with a Prescription Drug User Fee Act (PDUFA) action date of September 26, 2024. The NDA submission is supported by positive data from the AUGMENT-101 pivotal trial cohort of revumenib in adult and pediatric patients with KMT2Ar AML and acute lymphoblastic leukemia (ALL). About Revumenib Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and NPM1-mutant AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology Annual Meeting and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. About NPM1-Mutant Acute Myeloid Leukemia NPM1-mutant AML, which is distinguished by mutations in the NPM1 gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult AML and represents approximately 30% of all adult AML cases. This subtype of AML has a five-year overall survival rate of approximately 50%. Similar to KMT2A-rearranged acute leukemia, NPM1-mutant AML is highly dependent on the expression of specific developmental genes shown to be negatively impacted by inhibitors of the menin-KMT2A interaction. NPM1-mutant AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies indicated for NPM1-mutant AML. About Syndax Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include revumenib, a highly selective inhibitor of the menin–KMT2A binding interaction, and axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. For more information, please visit. For more information, please visit or follow the Company on X (formerly Twitter) and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Syndax Contact Sharon Klahre Syndax Pharmaceuticals, Inc. sklahre@syndax.com Tel 781.684.9827 SNDX-G Company Codes: NASDAQ-NMS:SNDX