Vitae Pharmaceuticals LLC

HANGZHOU, China, Sept. 5, 2024 /PRNewswire/ -- Venus Medtech (Hangzhou) Inc. (Venus Medtech, 02500.HK), a leading provider of integrated solutions for transcatheter structural heart valvular therapies in China, announced that its in-house developed next-generation balloon-expandable dry-tissue transcatheter aortic valve replacement (TAVR) system, Venus-Vitae, successfully completed two inaugural implantations as part of its global multicenter pivotal trial, Venus-Vitae SMART-ALIGN, at Prince of Wales Hospital in Hong Kong, China. Continue Reading These successful implantations mark another leap forward for the company into the frontier of next-generation dry-tissue valves, representing a significant addition to our transcatheter heart valve solutions. Following the first two successful implantations, Venus-Vitae SMART-ALIGN pivotal clinical trial will be progressively launched at around 20 centers in Europe, North America and China, enrolling a total of 150 patients. The trial data will be used to support registration applications in the EU, Canada, China and other territories. The procedures were performed by Prof. So Chak Yu Kent and the team at Prince of Wales Hospital in collaboration with Prof. Scott Lim of University of Virginia Medical Center, supported by a multidisciplinary team from Ultrasound, Anesthesiology, and Imaging Departments. Both patients received 23mm Venus-Vitae implants. The procedures presented good immediate post-operative results with significant hemodynamic improvement. Venus-Vitae Smart-ALIGN, abbreviated from Multicenter Pivotal Study to Evaluate the TreatMent of Severe AoRTic VALve StenosIs UsinG the VeNus-Vitae Transcatheter Heart Valve System, is a prospective, multicenter, non-randomized interventional study to evaluate the safety and efficacy of the Venus-Vitae TAVR system in patients with severe aortic stenosis. "Recently, my colleague Dr. Michael Hainstock and I, at the University of Virginia Medical Center, completed the first patient implantation in the VenusP-Valve PROTEUS pivotal clinical trial in the United States, " said Prof. Scott Lim." Two months later, I am very pleased to have participated with Dr. Kent So in the implantation of the first two patients in the Venus-Vitae SMART-ALIGN international multicenter pivotal clinical trial at Prince of Wales Hospital in Hong Kong. These both represent important milestones in those studies. Additionally, both the VenusP Valve and the Vitae valve have been previously used in clinical applications, with excellent published results. Of note, the Venus-Vitae valve's stent frame is the shortest among current transcatheter aortic valves, and I am encouraged by its potential to treat aortic stenosis while maintaining coronary access. Furthermore, its dry valve leaflet technology facilitated rapid preoperative preparation time. We look forward to further international enrollment in the SMART-ALIGN clinical trial in the near future." "It was an honor to collaborate with Dr. Scott Lim to complete the first two confirmatory clinical implantations of this innovative dry valve," said Prof. So Chak Yu Kent after the procedures. "Venus-Vitae's active anti-PVL technology and short frame design effectively prevent leakage and minimize the risk of coronary artery obstruction. I'm eager to see expedited enrollment of patients for Venus-Vitae, so that more patients can benefit soon." "Venus-Vitae is our next generation balloon-expandable TAVR system, with global patent coverage," said Hou-Sen Lim, General Manager and CEO of Venus Medtech. "We have strong confidence in its international multi-center clinical trials and global strategy. The successful initial implantations provide a solid foundation for forthcoming trials in Europe, North America, and China. We are committed to advancing Vitae's global clinical application and regulatory registration, bringing our cutting-edge technology to physicians and patients worldwide." About Venus-Vitae Venus-Vitae utilizes the innovative Venus-Endura technology, which integrates multiple anti-calcification techniques, immunogenicity removal technology and 3D force-controlled dehydration technology, providing exceptional durability, biocompatibility, and excellent anti-calcification performance. In addition, this design allows for room temperature storage of the valve in a dry-tissue condition. Additionally, the product features a unique patented technology, Lockwire Technology, ensuring the stability and accuracy of the valve during delivery and deployment. The short stent frame of Venus-Vitae's valve along with its steerable NAVIMASTER delivery system, significantly optimizes the performance across the aortic arch and positioning within the native valve. Venus-Vitae also features a unique Coronary Alignment technology, addressing the fundamental challenge that current TAVR products have been unable to address—aiding in protection of the coronary arteries. By utilizing three gold markers at the base of the valve leaflets in conjunction with the delivery system's steering, telescoping, and balloon rotation capabilities, Venus-Vitae can effortlessly and precisely achieve coronary alignment during the deployment process. This aids in accomplishing coronary protection throughout the implantation procedure. Furthermore, Venus-Vitae features a self-adaptive anti-PVL technology, utilizing an adaptive polymer skirt. The proprietary polymer skirt material possesses a high compression ratio, excellent resilience, self-expanding properties, and adaptive sealing. It demonstrates adaptive deformation during valve crimping, without enlarging the profile of the delivery system. Upon deployment, it actively fills the gaps around the valve perimeter, achieving remarkable anti-PVL performance. About Venus Medtech Venus Medtech (Hangzhou) Inc. (02500.HK) is committed to structural heart innovation. We are developing and commercializing comprehensive solutions for structural heart disease. Our robust pipeline, encompassing all four heart valves from TAVR, TPVR, TMVR, and TTVR to hypertensive renal denervation (RDN) therapy, underscores our unwavering commitment. For more information, please visit *Provided for informational and academic purposes only, this content is not intended as professional medical or legal advice. Venus Medtech makes no representations, warranties or guarantees regarding the completeness, accuracy, or timeliness of this content. *Venus Medtech makes no representations, warranties or guarantees regarding the property or clinical performance of any medical devices mentioned. *VENUSMEDTECH, the stylized QI logo, Venus-Vitae, Venus-Endura etc. are trademarks of Venus Medtech (Hangzhou) Inc. *Venus-Vitae is currently restricted to research only in the EU, North America, and China. SOURCE Venus Medtech (Hangzhou) Inc.

BRISBANE, Calif.--( BUSINESS WIRE )--Gate Bioscience, a biotechnology company developing a new class of small molecule medicines to eliminate disease-causing proteins at their source, today announced the appointment of Jeff Hatfield to its Board of Directors. Mr. Hatfield brings more than three decades of successful industry leadership across biotech and pharmaceuticals. “We are thrilled that Jeff is joining Gate’s Board of Directors,” said Jordi Mata-Fink, Ph.D., Co-founder and Chief Executive Officer of Gate. “Jeff has built and led multiple chemistry platform companies based on novel cutting-edge approaches, leading to many first-in-class programs in critical disease areas and successful outcomes for patients and shareholders alike. We look forward to benefiting from his expertise as we develop our Molecular Gate medicines.” Mr. Hatfield’s significant platform company leadership experience includes serving as Chief Executive Officer of Vividion Therapeutics, a pioneer in the development of chemoproteomics. At Vividion, he led the development of a portfolio of first-in-class oncology and immunology programs before the company’s 2021 acquisition by Bayer Pharmaceuticals, which delivered $1.5 billion upfront and $0.5 billion in CVR earn-outs to shareholders. He also served as CEO of Vitae Pharmaceuticals, where he built an early innovator position in AI/ML computational drug discovery before that company’s 2016 acquisition by Allergan for $640 million. Mr. Hatfield currently serves as Chair of ROME Therapeutics and Vividion, as well as serving on the board of directors for VIR Biotechnology and the advisory boards of Lime Therapeutics and the Harvard Business School’s Blavatnik Fellowship in Life Science Entrepreneurship. Earlier in his career, Mr. Hatfield held senior leadership roles at Bristol Myers Squibb, including Senior Vice President, Immunology; President, BMS-Canada; and Head of U.S. Market Access. He earned an MBA from The Wharton School of the University of Pennsylvania, and a B.S. from the Purdue University College of Pharmacy. “Gate’s new therapeutic mechanism – selectively blocking the secretory translocon with small molecules to potently eliminate extracellular disease-causing proteins – is unique and powerful,” said Mr. Hatfield. “This science has the potential to quickly identify high-value, first-in-class therapeutics in the inflammatory, cancer and neurodegenerative fields. This is an exciting time to join the company, with the platform fully operational and multiple discovery programs rapidly taking shape. I look forward to working with Gate to bring new therapies to patients in need.” Mr. Hatfield joins Gate’s Board of Directors that includes Dr. Mata-Fink, Thomas Daniel, M.D., former President of Global Research and Early Development at Celgene, Clare Ozawa, Ph.D., Managing Director at Versant Ventures, and Vineeta Agarwala, M.D. Ph.D., General Partner at a16z Bio + Health. About Gate Bioscience Gate Bioscience is creating a new class of medicines called Molecular Gates, small molecules that selectively eliminate harmful extracellular proteins at their origin: inside the cell. Every extracellular protein—of which there are more than 4,000—must pass through a single channel in the cell before it can be secreted into the body. Molecular Gates recognize and block specific disease-causing proteins from exiting the channel, causing them to be degraded by the cell instead of being secreted. By eliminating the harmful protein at the source, Gate aims to eliminate the disease for patients. Founded in 2021, Gate is backed by Versant Ventures, a16z Bio + Health, ARCH Venture Partners, and GV. Learn more at www.gatebio.com .

▲8月15-16日 NDC2024生物医药创新者峰会 扫码立即报名  注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。 2024年3月26日，Syndax Pharmaceuticals宣布美国FDA接受其在研管线Revumenib递交的NDA并授予优先审评资格，用于治疗携带KMT2A重排（KMT2Ar）的复发/难治性急性白血病患者。如果进展顺利的话，Revumenib有望于2024年在美国获批。从药融云数据库获悉，Syndax Pharmaceuticals（以下简称Syndax）成立于2005年，是一家临床阶段的生物制药公司，致力于开发创新的癌症疗法管线。 药融云数据库：融资信息 2023年底，Syndax宣布完成此前宣布的12432431股普通股的承销公开发行，其中包括行使全部承销商购买1621621股额外股票的选择权。在扣除承销折扣和佣金以及预计发行费用之前，Syndax从此次发行中获得的总收益约为2.3亿美元。此次增发无疑又为公司的融资和业务合作提供了更多机会，公司目前合作伙伴有Incyte、Vitae Pharmaceuticals（艾伯维的子公司）、UCB和拜耳。截止2024年5月23日，Syndax总市值17.09亿美元（124亿人民币）。截止2024年6月13日，收盘，每股17.40美元。 公司市值 研发管线 Syndax目前与合作伙伴进行公司管线的研发，主要包括以下三条研发管线，各管线具体进展情况如下： 01  Revumenib Revumenib（SNDX-5613）是一种新型口服Menin-KMT2A抑制剂。KMT2A重排的白血病发生在婴儿、儿童和成人患者中，NPM1突变是急性髓系白血病中最常见的基因改变，KMT2A重排和NPM1突变合计占急性髓系白血病（AML）病例的40%。在KMT2A重排或NPM1突变的急性白血病中，menin蛋白与KMT2A的相互作用对于驱动白血病促进基因的表达至关重要。临床前研究表明，靶向menin和KMT2A之间的相互作用可能是治疗携带这些突变的癌症的有效策略。目前正在开发Revumenib用于治疗KMT2Ar（KMT2A重排，也称为混合谱系白血病重排或MLLR）急性白血病（包括急性淋巴细胞白血病和急性髓系白血病），以及NPM1突变（NPM1m）急性髓系白血病（AML）。 Revumenib目前正在多项临床试验中进行评估，包括用于治疗复发/难治性（R/R）急性白血病的关键性AUGMENT-101试验，各项临床试验具体进展情况如下：（1）AUGMENT-101-2A是Revumenib单药治疗复发/难治性KMT2A基因重排急性淋巴细胞白血病（ALL）的试验，这是一项临床试验1/2期开放标签、剂量递增和剂量扩展队列研究。（2）AUGMENT-101-2B是Revumenib单药治疗复发/难治性KMT2A基因突变急性髓系白血病的试验。（3）AUGMENT-101-2C是Revumenib单药治疗复发/难治性NPM1m（核磷蛋白1突变）的急性髓系白血病的试验。2024年3月，公司宣布完成针对R/RNPM1mAML患者的AUGMENT-101关键试验队列的入组，顶线数据预计将于2024年第四季度公布，并可能支持2025年上半年Revumenib在R/R NPM1m AML中的sNDA（补充新药申请）。 药融云数据库：药品信息 此外，Revumenib治疗NPM1m和KMT2Ar急性白血病的多项临床试验1期联合研究正在进行，这些试验目前正在扩大，以验证推荐的临床试验2期剂量，预计2024年下半年将获得更多数据。这些试验包括：（1）AUGMENT-102试验是Revumenib联合化疗治疗KMT2Ar或NPM1m急性白血病患者的临床试验1期研究。（2）BEAT-AML主要评估Revumenib与维奈克拉（VEN）和阿扎胞苷（AZA）联合治疗一线AML患者的情况，该试验是白血病和淋巴瘤协会的Beat AML临床试验的一部分。（VEN+AZA：VEN是一种口服的B细胞淋巴瘤2选择性抑制剂，目前多项研究及临床试验证实VEN联合去甲基化药物或低剂量阿糖胞苷在AML患者中可获得较高的治疗反应和无病生存率，2020年10月FDA批准VEN与AZA、地西他滨或LDAC联合用于≥75岁或因合并症不能耐受强化诱导化疗的初治AML患者，且VEN+AZA方案于2020年被写入AML NCCN指南）（3）Revumenib在不可切除的转移性微卫星稳定型结直肠癌患者中的临床试验1期概念验证正在招募中，预计在2024年第二季度提供试验的最新情况。（4）2024年第一季度启动了一项Revumenib联合7+3化疗治疗新诊断的NPM1m或KMT2Ar急性白血病患者的临床试验1期研究。（5）此外，Syndax计划在2024年底前启动一项关键性试验，将Revumenib联合维奈克拉和阿扎胞苷用于新诊断的NPM1m或KMT2Ar不适合接受强化化疗的急性白血病患者。 在2023年年底，Syndax Pharmaceuticals分享了AUGMENT-101试验关键部分的顶线数据，并且提前停止了这项临床试验，却使公司的股价下跌9%。2024年3月，公司宣布FDA已批准Revumenib的新药上市申请（NDA）优先审评，NDA正在根据FDA的实时肿瘤学审查计划（RTOR）进行审查，并已将PDUFA的目标行动日期定为2024年9月26日。 02  Axatilimab Axatilimab（SNDX-6352）是一种靶向CSF-1R（集落刺激因子-1受体）的单克隆抗体，与CSF-1R具有高亲和力结合，并阻断两种已知的CSF-1R配体CSF-1和IL-34的结合。CSF-1R是一种被认为可以控制单核细胞和巨噬细胞存活和功能的细胞表面蛋白。在临床前模型中，通过CSF-1受体抑制信号传导已被证明可以减少疾病介导巨噬细胞及其单核细胞前体的数量，这已被证明在慢性移植物抗宿主病（cGVHD）和特发性肺纤维化（IPF）等疾病的纤维化疾病过程中起关键作用。Axatilimab是根据Syndax和UCB于2016年签订的UCB全球独家许可开发的，2021年9月，Syndax和Incyte就Axatilimab签订了全球独家联合开发和联合商业化许可协议。 AGAVE-201试验是一项临床试验2期研究，旨在评估Axatilimab在复发或难治性活动性cGVHD患者中以三种不同剂量治疗的疗效、安全性和耐受性。2023年7月，Syndax与合作伙伴Incyte宣布，在AGAVE-201试验中，Axatilimab在cGVHD患者中进行了两次或两次以上的治疗，所有三个剂量组（每两周0.3 mg/kg、每两周1.0 mg/kg和每四周3.0 mg/kg）均达到主要终点。在0.3 mg/kg剂量治疗的前六个月内，总反应率为74%，其中60%的患者在一年内仍有反应。此外，Axatilimab总体耐受性良好，最常见的不良事件与目标效应和既往试验一致。2024年2月，FDA已接受Axatilimab（用于治疗至少两种先前的全身治疗失败后的cGVHD）的BLA（生物制品许可申请）的优先审查，并指定PDUFA行动日期为2024年8月28日。 药融云数据库：药品信息 2024年1月，Syndax宣布根据其与Incyte的2021年合作协议，它已行使其选择权，在美国共同商业化Axatilimab。此外还宣布一项临床试验2期研究（随机、双盲和安慰剂对照试验）现已公开招募，该试验旨在评估Axatilimab在IPF患者中的疗效、安全性和耐受性。其合作伙伴Incyte还计划在2024年年中启动两项Axatilimab治疗cGVHD的联合试验，包括与鲁索利替尼（JAK抑制剂，可以阻止免疫系统攻击黑色素细胞）联合的临床试验2期研究和与类固醇联合的临床试验3期研究。 03  Entinostat（恩替司他） 恩替司他是一种组蛋白去乙酰化酶（HDAC）抑制剂,可选择性抑制I类和IV类HDACs，抑制细胞增殖、促进终末分化和/或诱导凋亡，发挥抗肿瘤作用。最早由拜耳制药研发，2007年转让于Syndax，2013年Syndax将中国的商业权许可给了亿腾景昂。 图源：国家药品监督管理局 2024年4月24日，亿腾景昂宣布恩替司他片（商品名：景助达）新药上市申请获中国国家药品监督管理局（NMPA）正式批准，适应症为联合芳香化酶抑制剂用于治疗激素受体（HR）阳性、人类表皮生长因子受体-2（HER-2）阴性，经内分泌治疗复发或进展的局部晚期或转移性乳腺癌患者。 财务状况 Syndax 2023年度的研发费用从上年同期的1.185亿美元增加至1.630亿美元，研发费用的同比增长主要是由于员工相关费用和专业费用的增加以及临床和制造费用的增加；一般、销售和管理费用从上年同期的3330万美元增加到6690万美元，该项的同比增长主要是由于员工相关费用和专业费用的增加以及Revumenib和Axatilimab商业化活动的增加；年度归属于普通股股东的净亏损为2.09亿美元，或每股2.98美元。 截止2024年3月31日，Syndax尚未产生任何产品收入，因为其创造收入和盈利的能力取决于获得候选产品的上市批准并成功商业化的能力。2024年第一季度研发费用从上年同期的3410万美元增至5650万美元，研发费用的增加主要是由于临床开发和制造成本增加、员工相关费用和专业费用增加以及本期确认的开发里程碑费用；一般、销售和管理费用从上年同期的1200万美元增至2300万美元，该项的增加主要是由于员工相关费用和专业费用的增加，以及Revumenib和Axatilimab的商业化活动增加。该季度报告的归属于普通股股东的净亏损为7240万美元，或每股0.85 美元。 截至2024年3月31日，Syndax 拥有5.22亿美元的现金、现金等价物，预计可以支持公司的研究、临床开发和商业运作到2026年。对于第二季度，Syndax预计研发费用为5000~5500万美元，总运营费用为8000~8500万美元。对于2024年全年，Syndax预计研发费用为2.4~2.6亿美元，总运营费用为3.55~3.75亿美元，其中包括预计4300万美元的非现金股票补偿费用。 【关于药融圈】 药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。