Edgewise Therapeutics, Inc.

Novo Holdings CEO Kasim Kutay asked, \"who’s going to IPO an asset in this environment?\"\n The CEO of Novo Holdings has warned that his $160 billion fund will slow down investment activity until the dust settles after the Trump administration’s tariff blitz.Trump’s attack on global trade—and fast retreat from his initial tariffs—has left investors uncertain about where the economy is heading. That could make life hard for investors such as Novo Holdings. The fund, which is best known as the controlling shareholder of Novo Nordisk, deployed (PDF) 34 billion Danish kroner ($5.2 billion) in life sciences last year. Now, Novo Holdings CEO Kasim Kutay is pumping the brakes.“Until there is more clarity on the prospects for economic growth, we will slow down our investment activities,” Kutay told Bloomberg. “We’re in the midst of uncharted waters.”The shift in Novo Holdings’ approach could have implications for biotechs that are seeking to raise cash. Last year was one of the most active years in the 25-year history of Novo Holdings’ venture investments group, which has pumped money into biotechs including Alentis Therapeutics, Amolyt, Edgewise, Numab and Vaxcyte. Novo Holdings’ venture portfolio was valued at 16 billion Danish krone ($2.4 billion) at the end of 2024. Around two-thirds of the 60 companies in the venture portfolio are private. Novo Holdings had planned to exit some of the assets this year but those actions could now be pushed to 2026.“I mean, who’s going to IPO an asset in this environment? Unless you have to, it’s probably not advisable,” Kutay said. “The view on interest rates, which is a key barometer and a driver of how and when to invest, is all over the place.”Kutay’s comments come shortly after Roche’s CEO Thomas Schinecker said tariffs could make it “more difficult to make financial sense of any M&A deals.” Merck & Co. CEO Rob Davis also said that \"clearly what’s happening does make it more complex to get things done because [of] the uncertainty everyone is wrestling with.”

Dive Brief:Bristol Myers Squibb on Monday said its drug Camzyos failed a Phase 3 trial in people with a progressive heart condition, closing off an opportunity to expand use of a medicine it sees as a future blockbuster.According to Bristol Myers, Camzyos missed the dual main goals of a study focused on the non-obstructive form of HCM, or hypertrophic cardiomyopathy. It failed to meaningfully improve peak oxygen consumption as well as scores on an assessment of heart health. The company didnt provide study details, but said more information will be shared with the scientific community in the future.Camzyos was acquired through the $13 billion buyout of MyoKardia in 2020 and two years later became the first drug cleared for use in the obstructive and more common form of the disease. Biotechnology companies Cytokinetics and Edgewise Therapeutics are developing similar medicines that are both in the advanced stages of clinical testing. Cytokinetics drug, aficamten, could be approved in the U.S. later this year.Dive Insight:Camzyos setback carries implications for Bristol Myers as well as the companies trying to develop better medicines for HCM, a chronic condition characterized by a potentially deadly stiffening of the heart muscle.Bristol Myers is bracing for the losses of billions of dollars in yearly revenue when the patents expire for its cancer immunotherapy Opdivo and blood thinner Eliquis. The company already announced a cost-cutting plan under CEO Chris Boerner, and is looking to a handful of medicines, Camzyos among them, to grow sales in the years ahead.Camzyos generated $602 million in sales in 2024. But Bristol Myers has said that number could reach $4 billion in 2029, and one way to get there was a clearance in the non-obstructive setting, which is estimated to account for about a third of the people with the condition. The setback is a disappointment, wrote Leerink Partners analyst David Risinger, though Bristol Myers could rebound if a series of coming Phase 3 readouts are successful.The study failure also stirred debate among analysts as to whether drugs like Camzyos, known as cardiac myosin inhibitors, can help all people with HCM. Camzyos has proven beneficial for the obstructive form that impedes blood flow and can lead to heart failure as well as other potentially serious complications. The non-obstructive type doesnt, but can still put people at risk of poor health outcomes. The new study tested whether Camzyos might be similarly helpful for those people as well.The fact that Camzyos wasnt suggests obstructive and non-obstructive HCM are two unique diseases, said Milind Desai, the director of the Cleveland Clinics HCM Center, in a statement provided by Bristol Myers.Analysts were mixed on whether the cardiac myosin inhibitors Cytokinetics and Edgewise are developing might similarly struggle in non-obstructive HCM. Cytokinetics drug, aficamten, is under Food and Drug Administration review in obstructive HCM and in a Phase 3 trial in non-obstructive disease. Edgewises EDG-7500 could join it in late-stage testing next year.Matt Phipps, of the investment bank William Blair, wrote that Camzyos shaky performance in the latest study as well as Phase 2 testing in non-obstructive HCM patients may be more attributed to disease biology, which has negative implications for the whole drug class.That form of the disease is also more heterogeneous, making it tougher for a drug to succeed in testing, added RBC Capital Markets Leonid Timashev.Still, there are key differences between the drugs and their development plans that leave some analysts optimistic about Cytokinetics and Edgewises chances. Aficamten appeared to perform better in earlier testing in non-obstructive patients, and Cytokinetics Phase 3 study is designed differently, wrote Mizuho Securities Salim Syed. RBCs Timashev added that EDG-7500s unique mechanism could overcome the limitations of other medicines in non-obstructive HCM.We now see a 2-handed market split between Cytokinetics and Edgewise, Timashev wrote. '

▎药明康德内容团队编辑首款！诺华肾病疗法获FDA加速批准诺华（Novartis）今日宣布，美国FDA已加速批准Vanrafia（atrasentan）上市，用于降低具有快速疾病进展风险的原发性免疫球蛋白A肾病（IgAN）成人患者的蛋白尿水平。Vanrafia是一种高效且选择性强的内皮素A（ETA）受体拮抗剂，每日一次口服使用，可作为支持治疗的一部分，与肾素-血管紧张素系统（RAS）抑制剂联用，亦可联合钠-葡萄糖共转运蛋白2（SGLT2）抑制剂使用。新闻稿指出，Vanrafia是首款获批用于降低IgAN患者蛋白尿水平的ETA受体拮抗剂。Vanrafia的加速批准基于3期临床试验ALIGN预设的中期分析，该研究测量了与安慰剂相比，在36周时蛋白尿的降低情况。数据显示，接受Vanrafia联合RAS抑制剂治疗的患者与安慰剂组相比，实现了具有临床意义且统计学显著的蛋白尿降低，幅度达36.1%（P<0.0001）。该效果在第6周即可见，并持续至第36周。Vanrafia的影响在各亚组中均表现出一致性。在另一组同时接受RAS抑制剂和SGLT2抑制剂治疗的患者中，Vanrafia也显示出类似的治疗效果。穿越血脑屏障！Denali启动突破性疗法申请递交Denali Therapeutics日前宣布，公司已启动滚动提交tividenofusp alfa的生物制品许可申请（BLA），寻求用于治疗亨特综合征（MPS II）的加速批准。Denali持续与FDA保持沟通，并就BLA数据包内容达成一致，包括使用脑脊液中硫酸乙酰肝素（CSF HS）水平作为支持加速批准的替代终点。Denali预计将在2025年5月上旬完成BLA提交。Tividenofusp alfa是一款由艾杜糖醛酸-2-硫酸酯酶（IDS）与Denali的酶转运载体（ETV）结合的融合蛋白药物。Denali的转运载体平台含有可与天然转运受体结合的工程化Fc片段，通过与在血脑屏障上表达的天然转运受体结合，借着转胞吞作用（transcytosis）将大分子递送到大脑，从而缓解MPS II的中枢神经系统症状。它已经获得美国FDA授予的快速通道资格和突破性疗法认定，用于治疗MPS II。此前公布的1/2期临床试验结果显示，tividenofusp alfa将患者CSF HS水平降低到正常水平，与溶酶体功能和神经退行性病变相关的生物标志物指标也得到改善。经过24周治疗，患者的肝脏体积恢复正常，各测试频段的听力阈值均有所改善，同时大多数受试者在适应性行为和认知测评上均表现出改善。肥厚型心肌病疗法2期临床结果积极Edgewise Therapeutics日前宣布，在研疗法EDG-7500，在治疗梗阻性和非梗阻性肥厚型心肌病（HCM）患者的2期临床试验CIRRUS-HCM中获得了积极的顶线数据。EDG-7500是一款创新口服、选择性心肌节调节剂，专门设计用于减缓早期收缩速度并改善与HCM相关的心脏舒张功能障碍，同时不影响其收缩功能。在梗阻性HCM患者中，接受EDG-7500（100 mg）治疗的患者，其静息和诱发左室流出道压力梯度（LVOT-G）与基线相比，平均分别降低了71%和58%。值得注意的是，这些梯度降低是在未引起左心室射血分数（LVEF）明显变化的情况下实现的。EDG-7500治疗还使关键心衰生物标志物NT-proBNP与基线相比平均降低了62%。在非梗阻性HCM患者中，EDG-7500给药亦呈现剂量依赖性降低NT-proBNP的效果，100 mg剂量组与基线相比，NT-proBNP平均降低42%。针对难以成药靶点，赛诺菲获得靶向蛋白降解疗法开发权益Nurix Therapeutics公司日前宣布，赛诺菲（Sanofi）已获得Nurix一个未公开项目的独家研发许可，该项目针对先前被认为难以成药的转录因子，用于治疗自身免疫疾病。该靶点是炎症反应的中心调控因子。根据2019年合作协议，Nurix已从赛诺菲获得1500万美元的许可费用，并有资格就该项目获得额外4.65亿美元的研发、监管及商业里程碑付款。赛诺菲在2019年与Nurix达成研发合作，根据合作协议，Nurix利用其专有的药物发现平台，发现利用E3连接酶诱导降解特定靶点的创新候选分子。赛诺菲有权获得由这一合作产生的候选药物的研发许可。迄今为止，Nurix已从这一合作中获得1.05亿美元付款。▲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] Novartis receives FDA accelerated approval for Vanrafia® (atrasentan), the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN). Retrieved April 3, 2025, from https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-vanrafia-atrasentan-first-and-only-selective-endothelin-receptor-antagonist-proteinuria-reduction-primary-iga-nephropathy-igan[2] Nurix Licenses a Drug Discovery Program to Sanofi Targeting a Novel Transcription Factor for Autoimmune Diseases. Retrieved April 3, 2025, from https://www.globenewswire.com/news-release/2025/04/02/3054111/0/en/Nurix-Licenses-a-Drug-Discovery-Program-to-Sanofi-Targeting-a-Novel-Transcription-Factor-for-Autoimmune-Diseases.html[3] Edgewise Therapeutics Announces Positive Top-Line Results from Phase 2 CIRRUS-HCM Four-Week Trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM). Retrieved April 3, 2025, from https://www.prnewswire.com/news-releases/edgewise-therapeutics-announces-positive-top-line-results-from-phase-2-cirrus-hcm-four-week-trial-of-edg-7500-in-hypertrophic-cardiomyopathy-hcm-302417857.html[4] Denali Therapeutics Announces Initiation of BLA Filing for Accelerated Approval of Tividenofusp Alfa for the Treatment of Hunter Syndrome (MPS II) and Positive Ongoing Interactions with FDA on DNL126 Through START Program. Retrieved April 3, 2025, from https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-initiation-bla-filing-accelerated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