CRD-740

Dive Brief:Biotechnology startup Cardurion Pharmaceuticals on Tuesday raised a $260 million Series B round that will help it build its portfolio of heart disease drugs.Cardurion will use the funds to advance a pair of medicines into mid- and late-stage testing, run studies in newer indications and potentially acquire other assets for cardiovascular diseases. The round comes two months after the startup detailed datafrom a Phase 2 trial in heart failure.Ascenta Capital led the financing, which involved NEA, GV, Bain Capital Life Sciences and a handful of other investors. Bain has already invested heavily in Cardurion, having poured up to $300 million into the startup in 2021.Dive Insight:After years of being overlooked by investors, heart and metabolic disease startups are now getting closer attention,fueled in part by the spectacular success of obesity drugs as well as deals for companies working on cardiovascular research.Bristol Myers Squibb paid $13 billion in 2020 to buy MyoKardia and a drug the biotech had developed for a genetic heart condition, for example.Novartis spent $10 billion on a cholesterol drug from The Medicines Co. a year earlier. More recently, drugs like Wegovy and Zepbound have proven able to not only help people lose weight, but protect heart health as well, driving more interest in companies working in the field.Against that backdrop, Cardurion has been using its sizable bankroll to build a growing portfolio. The company was launched in 2017 with an alliance with Takeda, and has since advanced a group of medicines for heart failure and heart muscle dysfunctions. Its turned to deals to grow its pipeline too, in 2022 acquiring a medicine from now-defunct biotech Imara.In heart failure, Cardurion aims to improve upon standard care. Currently, about half of patients with the condition die within five years of their diagnosis. Though Novartis blockbuster medicine Entresto can reduce the risk of death or complications, there remains room for improvement.Cardurions drugs CRD-740 and CRD-750 block an enzyme called PDE9, a biological target thats been studied in other conditions like Alzheimers and sickle cell disease. In heart failure, PDE9 could prove beneficial because of its role regulating a molecule involved in the relaxation of heart muscles. Doing so could improve the hearts ability to pump blood, which is compromised in people with heart failure.Phase 2 data presented in May showed CRD-740 increased levels of that molecule, known as cGMP, after four weeks of treatment, meeting the studys primary goal.In a statement, the company said it is testing CRD-750 in two Phase 2 studies of people with heart failure. It will also use the funding to advance a second experimental drug targeting a protein linked to hyperactive muscle tissue. That drug will initially be tested in people with a rare condition called catecholaminergic polymorphic ventricular tachycardia. '

▎药明康德内容团队编辑潜在重磅心肌病疗法3期临床结果发布，今年递交监管申请Cytokinetics公司今日宣布，在研疗法aficamten在治疗有症状的梗阻性肥厚型心肌病（HCM）患者的关键性3期临床试验SEQUOIA-HCM中达到主要终点和所有次要终点，快速和持久改善患者症状和功能。详细数据发表在《新英格兰医学杂志》（NEJM）上，并在由欧洲心脏病学会举办的2024年心力衰竭（Heart Failure 2024）国际会议上报告。这款创新疗法被行业媒体Evaluate列为值得关注的十大潜在重磅疗法之一。SEQUOIA-HCM的结果显示，与安慰剂相比，aficamten治疗24周显著改善了患者的运动能力，通过心肺运动测试（CPET）测量的最高氧气摄取量（pVO2）增加了1.74 mL/kg/min（95% CI：1.04-2.44，p=0.000002）。值得注意的是，在不同患者亚组中均一致观察到aficamten的积极影响。▲Aficamten达到SEQUOIA-HCM的主要终点（图片来源：参考资料[1]）Aficamten是一款选择性小分子心肌肌球蛋白抑制剂，通过减少每个心动周期中活性肌球蛋白产力横桥的数量，抑制与肥厚型心肌病相关的心肌过度收缩。Cytokinetics计划在今年晚些时候向美国和欧盟的监管机构递交监管申请。只需每周或每月一次，血友病A长效疗法2期临床结果积极诺和诺德（Novo Nordisk）公司今天宣布了长效血友病A疗法Mim8的2期临床试验FRONTIER 2的主要结果，该试验评估了每周一次或每月一次皮下注射Mim8与未接受预防治疗以及与之前的凝血因子预防治疗相比，在12岁及以上带有或不带有凝血因子抑制物的血友病A患者中的疗效和安全性。数据显示，与不进行预防治疗及之前的凝血因子预防治疗相比，每周一次和每月一次Mim8均能显著减少需要治疗的出血事件，达到试验的共同主要终点。在未接受预防治疗的人群中，每周一次和每月一次Mim8分别将需要治疗的出血事件减少97%和99%。在接受过凝血因子预防治疗的患者中，与之前的凝血因子预防治疗相比，每周一次和每月一次Mim8分别将需要治疗的出血事件减少48%和43%。Mim8是一种模拟凝血因子VIIIa的双特异性抗体，通过皮下注射给药，它通过连接凝血因子IXa和X，替代缺失的因子VIII，有效恢复身体的凝血酶生成能力，帮助血液凝固。基于与监管机构的沟通结果，诺和诺德计划在2024年年底递交Mim8的首个监管申请。创新心力衰竭疗法获概念验证Cardurion Pharmaceuticals公司今日宣布，在研磷酸二酯酶-9（PDE9）抑制剂CRD-740在概念验证2期临床试验CARDINAL-HF中获得积极结果。数据显示，CRD-740在射血分数降低型心力衰竭（HFrEF）患者中达到了主要终点，治疗四周后，中位血浆环状鸟苷酸（cGMP）水平显著提高。血浆cGMP是细胞内cGMP的生物标志物，其水平反映了保护性心肌利钠肽（NP）信号通路的活性，该通路在心力衰竭中已证明具有临床益处。基于这一2a期临床试验的结果，Cardurion公司已启动两项总计包含640名患者的2期临床试验，其中一项在HFrEF中进行剂量探索，另一项将评估CRD-740治疗射血分数保留型心力衰竭患者的效果。▲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] Cytokinetics Announces Primary Results From SEQUOIA-HCM Presented in Late Breaking Clinical Trial Session at the European Society of Cardiology Heart Failure 2024 Congress and Published in the New England Journal of Medicine. Retrieved May 13, 2024, from https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-primary-results-sequoia-hcm-presented[2] Novo Nordisk A/S: Once-weekly and once-monthly Mim8 demonstrate superior reduction of treated bleeding episodes compared to on-demand and prior prophylaxis treatment in people with haemophilia A in the Frontier 2 trial. Retrieved May 13, 2024, from https://www.globenewswire.com/news-release/2024/05/13/2880715/0/en/Novo-Nordisk-A-S-Once-weekly-and-once-monthly-Mim8-demonstrate-superior-reduction-of-treated-bleeding-episodes-compared-to-on-demand-and-prior-prophylaxis-treatment-in-people-with-.html[3] Cardurion Pharmaceuticals Presents Positive Clinical Results from CARDINAL‑HF Phase 2a Clinical Trial of PDE9 Inhibitor in Patients With Heart Failure. Retrieved May 13, 2024, from https://www.businesswire.com/news/home/20240511287537/en/Cardurion-Pharmaceuticals-Presents-Positive-Clinical-Results-from-CARDINAL%E2%80%91HF-Phase-2a-Clinical-Trial-of-PDE9-Inhibitor-in-Patients-With-Heart-Failure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