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iStock, Sundry Photography Despite comments made by a Novo Nordisk official this week, the company confirmed to BioSpace that it has no additional clinical trials of its GLP-1 drugs in addiction beyond a Phase II trial testing semaglutide and two other drugs with alcohol use as a secondary endpoint. Thursday, Reuters reported that Martin Holst Lange, head of development at Novo, had told an online conference on Thursday that his company plans to investigate how its GLP-1 medicines can help people with addiction. However, in response to questions from Biospace , a Novo spokesperson said that Lange was referring to already ongoing work studying semaglutide on liver damage and alcohol use, and that the company has no plans to study the drug in broader addiction indications. “There is nothing else in terms of clinical trials we have ongoing in this space at the moment,” the spokesperson said in an email. Novo currently markets its GLP-1 treatments, which both contain the active ingredient semaglutide, as Wegovy for weight loss and Ozempic for type II diabetes. However, there is an early but growing interest in the potential of these molecules to treat substance abuse disorders, their related effects and a bevy of other conditions . The Phase II trial, launched in May 2024 and currently recruiting globally, is studying the effectiveness of semaglutide and two other drugs—cagrilintide, an amylin analogue, and NNC0194-0499, an FGF21 analog—used alone or in combination, in patients with alcoholic liver disease. Treatment of liver damage is that trial’s primary endpoint and alcohol consumption is a secondary endpoint. Three months ago, Novo’s biggest competitor Eli Lilly shared its plans to study its own GLP-1 medicines in alcohol and drug abuse. While Lilly has not officially announced any trials, CEO David Ricks said at an event in December that the company would begin large studies of its obesity drugs in alcohol and drug abuse next year, according to  Endpoints News . Currently, pharmaceutical treatment for addiction and substance-use disorders is limited, with most treatments aimed at avoiding overdoses or reducing withdrawal symptoms. But the industry is attempting inroads, with psychedelic therapies like LSD and ketamine drawing attention from researchers and biopharma companies. Another area of interest is in vaccination. In 2021 the NIH awarded a $25 million grant to scientists at Boston Children’s Hospital to study a vaccine against opioids. The hope is that the vaccine would generate antibodies to prevent the molecules from crossing into the brain. Novo is making progress with GLP-1 on another neurological front. In October 2024, semaglutide was linked with a significant reduction—40% to 70%—in the risk of Alzheimer’s disease in a real-world study of patients with type 2 diabetes. Novo is running two Phase III trials, EVOKE and EVOKE Plus, formally testing semaglutide in patients with Alzheimer’s. Those trials are expected to read out in September of this year.

Elisa Cascade, CPO at Advarra explains why addressing the challenges faced by clinical research sites is vital for improving patient trust and the overall viability of clinical trials. Lori Ellis, head of insights at Biospace , and Elisa Cascade , CPO of Advarra discuss the challenges faced by clinical research sites at SCOPE 2025. Cascade highlights that many sites are reporting significant staff shortages of over 50%. This issue is expected to worsen. Since patient experience and trust are influenced by the sites, she suggests a “patient-centric” approach incorporates a “site-centric” approach. Key Takeaways Staff shortages put sites in a disadvantaged position, making it harder for them to be patient-centric and build trust with patients. Improving “site-centricity” - focusing on the needs and challenges of clinical research sites - is crucial, as sites are the primary point of contact with patients in clinical trials. Two key gaps that could be addressed to help sites include streamlining the login process and improving the clinical trial startup process. There is a need for more collaboration and alignment across the industry to support clinical research sites and ensure the sustainability of the clinical research ecosystem. ALL COVERAGE Drug Development SCOPE Summit 2025 Key Takeaways: A Look Inside, Outside and Beyond the Cybersecurity Wall Jay Ferro, EVP, CIO, CPO & CTO at Clario discusses safety, trust and what keeps him up at night. February 13, 2025 · 1 min read · Lori Ellis Drug Development Healthcare at a Crossroads: Maintaining Good Science in Uncertain Times Continuing our SCOPE 2025 coverage, Rohit Nambisan, CEO at Lokavant addresses not only current challenges, but the life sciences industry’s responsibility to maintain scientific integrity. February 17, 2025 · 1 min read · Lori Ellis Drug Development Site Survival: Why Clinical Research Sites Need a Lifeline Elisa Cascade, CPO at Advarra explains why addressing the challenges faced by clinical research sites is vital for improving patient trust and the overall viability of clinical trials. February 19, 2025 · 1 min read · Lori Ellis Drug Development Diversity at the Forefront: Life Sciences Industry Champions Inclusive Representation The industry remains unwavering in the commitment to increased clinical trial accessibility and representation. February 25, 2025 · 1 min read · Lori Ellis Drug Development Beyond Dr. Google: The Roles of Industry and AI in Fighting Misinformation and Fueling Patient Empowerment As communication gaps in the US healthcare market widen, the emphasis on the need for credible information and patient empowerment is paramount. February 27, 2025 · 1 min read · Lori Ellis Drug Development Building Patient Trust Amid an Environment of Distrust While at SCOPE 2025, Sam Srivastava, CEO at WCG Clinical discusses the challenges and responsibilities of the life sciences industry in building public trust amidst growing anger towards healthcare. February 28, 2025 · 1 min read · Lori Ellis Drug Development The Chilling Effect: State Actions, Patient Data Vulnerability, and DEI Uncertainty In the current legal and political landscape, it is all about survival for DEI initiatives. March 3, 2025 · 1 min read Drug Development Trust, Tech, and Tomorrow: A Pharmaceutical Executive’s WEF Insights Konstantina Katcheves, Senior VP of Innovative Global Business Development at Teva Pharmaceuticals brings insights from the World Economic Forum to SCOPE 2025. March 5, 2025 · 1 min read · Lori Ellis

（限时福利直播）如何借助AI使用PASS和R软件计算样本量 2015年至今，FDA共批准新药465款，其中2018年批准数量最高，达59款；2016年批准数量最低，达22款。 2024年，FDA药品评价与研究中心（CDER）共批准了50款新药，其中包括34款新分子实体和16款生物制品。 从疾病领域角度分析，肿瘤（28%）依然是获批数量最多的领域，其次是罕见病（20%），之后依次为心血管及代谢、感染、自身免疫性疾病、皮肤病、精神和神经类等疾病领域。 其次从技术角度分析，首款mRNA疫苗、首款TCR-T细胞疗法（Adaptimmune的Tecelra）、首款TIL疗法（Iovance的Lifileucel）等多款里程碑进展于2024年获批。 2025年，FDA继续加大审批力度，预计第一季度审批新药共计44款，表格如下。 值得一提的是，知名行业媒体Biospace统计了2025年第一季度最值得期待的五项监管决定。 Biogen/卫材的Leqembi 适应症：早期阿兹海默症的静脉维持治疗 PDUFA日期：2025年1月25日 Leqembi（仑卡奈单抗）是人源化免疫球蛋白γ 1（IgG1）单克隆抗体，靶向聚集性可溶性（原纤维）和不溶性淀粉样蛋白β（aβ），由Biogen与卫材合作开发的阿尔茨海默症早期药物。 目前，两家公司正在推动Leqembi每月一次静脉维持治疗方案的审批，预计结果将于2025年1月25日公布。现有用药方案显示，Leqembi为两周一次静脉输注给药，每次大约需要一个小时。而最新用药方案，则是将Leqembi的给药间隔延长至一个月（初步的双周启动阶段后）。 Biogen与卫材表示，新的每月维持治疗方案旨在保持体内Leqembi的有效浓度，以抑制有毒原纤维，减少神经损伤和脑损伤的风险。此方案基于IIb期研究201及其开放标签扩展研究的数据模型，以及III期Clarity AD研究和其扩展阶段的数据。 此外，两家公司还在开发Leqembi的皮下注射剂型，并已开始滚动提交生物制品许可申请，预计于2026年3月获得批准。目前，约有6,000名患者正在等待接受这种疗法的初始治疗。一旦解决了基础设施和行政挑战，这可能转化为未来的增长动力。 阿斯利康和第一三共的Dato-DXd 适应症：转移性、HR阳性、HER2阴性乳腺癌 PDUFA日期：2025年1月29日 Dato-DXd是一款TROP2 ADC，由阿斯利康与第一三共共同开发。2024年初，两家公司提交了该药物的BLA，适用于可手术或转移性、HR阳性且HER2阴性或低表达的乳腺癌，PDUFA日期为今年1月29日。 根据III期TROPION-Breast01研究的数据，Dato-DXd相较于化疗，在降低死亡或疾病进展风险方面展现了37%的显著优势，这一效果被认为具有统计学意义和临床重要性。然而，后续数据显示，尽管Dato-DXd对无进展生存率有明显改善，但在总生存率上未能显著提升。 安全性方面，TROPION-Breast01研究表明Dato-DXd总体耐受良好，其不良事件概况优于传统化疗。最常见的3级或以上副作用包括中性粒细胞减少症、疲劳和贫血。 目前，除了乳腺癌外，阿斯利康和第一三共还在探索Dato-DXd用于非小细胞肺癌的效果，但在2024年9月，该ADC同样未能在该领域显示显著的整体生存改善。 Vertex的Suzetrigine 适应症：中度至重度急性疼痛 PDUFA日期：2025年1月30日 Suzetrigine是一种在研的选择性NaV1.8疼痛信号抑制剂，旨在通过特异性阻断周围神经中的钠通道来阻止疼痛信号传达到大脑。由于其作用机制仅限于外周神经系统，该药物不具备阿片类药物的成瘾风险。 目前，Vertex正在推动这款非阿片类止痛药用于治疗中度至重度急性疼痛，PDUFA日期为2025年1月30日。 2024年药时代报道了该药物用于治疗坐骨神经痛（LSR）的临床试验结果（推荐阅读：安慰剂疗效显著？Vertex一夜蒸发130亿美元）。为了推动此次Suzetrigine的审评，Vertex提供了两项II期研究和三项III期研究的数据，其中包括NAVIGATE 1和NAVIGATE 2试验。这些试验分别评估了该药物在进行拇外翻手术和腹部整形术患者中的疗效。 结果显示，Suzetrigine单药治疗显著降低了患者的疼痛评分，在腹部整形术后降低了48.4分，而在接受脚趾手术的患者中则降低了29.3分。与安慰剂相比，这两种治疗效果均具有统计学意义。 如果获得批准，suzetrigine将成为二十多年来首个用于急性疼痛的新药类别，为临床医生提供一种新的工具来管理中度至重度急性疼痛，同时也为患者带来了无需依赖阿片类药物的治疗选项。 Alnylam的Amvuttra 适应症：转甲状腺素蛋白淀粉样变心肌病 PDUFA日期：2025年3月23日 2024年11月22日，FDA批准了BridgeBio的Attruby，用于治疗野生型或变异型转甲状腺素介导的淀粉样变性心肌病（ATTR-CM）成人以降低心血管死亡和心血管相关的住院。Attruby成为全球首款转甲状腺素近完全稳定的治疗剂。 Alnylam紧跟其后，希望旗下Amvuttra拓展ATTR-CM适应症。Amvuttra是一款皮下给药的RNAi疗法，此前已批准用于治疗遗传性转甲状腺素蛋白淀粉样变多发性神经病。 为了支持其ATTR-CM申请，Alnylam引用了III期HELIOS-B研究的数据。该研究表明，Amvuttra对ATTR-CM患者的生存率、心血管事件、功能能力和生活质量产生了“有利影响”。特别是，在2024年美国心脏协会科学会议上展示的数据指出，Amvuttra治疗使得所有原因死亡率和复发性心血管事件的综合结果降低了28%。这一积极成果促使FDA接受了Alnylam的补充新药申请，预计评审结果将于2025年3月23日公布。 如果获得批准，Amvuttra将区别于现有TTR稳定剂（BridgeBio的Attruby；辉瑞的Vyndaqel、Vyndamax），成为首款用于ATTR-CM的RNA干扰疗法。 赛诺菲的Fitusiran 适应症：A型或B型血友病 PDUFA日期：2025年3月28日 Fitusiran是一款由赛诺菲和Alnylam公司联合开发的靶向抗凝血酶的siRNA，通过与表达抗凝血酶的RNA相结合，降低抗凝血酶（AT）的产生，从而恢复凝血因子和抗凝血因子之间的平衡，达到降低血友病患者出血事件的作用。该创新疗法只需每个月皮下注射一次，与常规预防性凝血因子注射相比，为患者控制出血风险提供了很多便利。 目前，该药物正在等待FDA批准，用于在没有抑制剂的情况下，降低 A 型和 B型血友病患者的AT水平并增加凝血酶的产生，PDUFA日期为2025年3月28日。 在一项名为ATLAS-INH的研究中，66%接受Fitusiran治疗的参与者达到了每月零出血发作，而给予按需旁路剂的对照组中仅有5%达到此效果。另一项试验ATLAS A/B则招募了没有抑制物的患者，其中51%在接受Fitusiran治疗后实现了每月零出血，相比之下，接受按需凝血因子治疗的对照组这一比例为5%。综合来看，这两项研究表明，与各自的对照组相比，预防性使用Fitusiran可将年化出血率降低90%。 随着越来越多血友病基因疗法（BioMarin的Roctavian；辉瑞的Beqvez）的获批，Fitusiran将有望成为血友病治疗领域的一个重要补充。 参考资料： 1.5 FDA Decisions to Watch in Q1（Biospace） 2.FDA 2025年第一季度审批全览（CPHI制药在线） 3.其他公开资料 封面图来源：网络 版权声明/免责声明 本文为编译文章。 本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。 文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。 如有任何问题，请与我们联系。 衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 点击查看更多精彩！