Semaglutide (Novo Nordisk)

Welcome back to Endpoints Weekly! This week was another busy one in DC, with two Senate hearings for Trump’s picks to lead the NIH and FDA, more actions from the Department of Government Efficiency, talk of tariffs, and more. We’ll walk you through it. Plus, another big pharma is joining the obesity race — but not with a GLP-1. And a small Swiss biotech quietly disclosed that it would halt a trial for an oral Alzheimer’s drug after two of its top competitors Eli Lilly and Biogen also scrapped their own oral Alzheimer’s drugs. Endpoints’ Kyle LaHucik has the latest on those stories below. Stay tuned next week as we cover the Senate HELP Committee’s scheduled votes on FDA nominee Marty Makary and NIH pick Jay Bhattacharya. On the same day, the committee is set to hold a confirmation hearing for CDC nominee David Weldon. See you on Monday! — Nicole DeFeudis President Donald Trump’s picks to lead the NIH and FDA emerged from Senate confirmation hearings relatively unscathed this week. Here’s a run-down of where the nominees stand on key topics: 💉 Vaccines: FDA nominee Marty Makary said “vaccines save lives” during his hearing on Thursday. The statement is a departure from comments made by HHS Secretary Robert F. Kennedy Jr., who declined to refute his past claims about a link between vaccines and autism in a January Senate hearing. NIH pick Jay Bhattacharya said he “fully supports” measles vaccinations and does not believe there’s a link between autism and vaccines. However, he called for more study to combat falling vaccination rates in some parts of the country. “I’m convinced we have good data on MMR and autism, but if other people don’t agree with me and don’t vaccinate their children, the one lever I have is to give them good data,” he said. 💼 Staff cuts: Both nominees said they were not involved in recent staff cuts at the NIH and FDA. When pressed by Sen. Tim Kaine (D-VA) about his thoughts on “willy, nilly, random” layoffs at the FDA, Makary cited his surgical experience. “Look, I’m a surgeon, so you know, I’m going to give you a surgical answer,” he said. Bhattacharya said he would “look very carefully at the personnel decisions.” When repeatedly asked, he said, “the personnel decisions are hard to talk about unless I’m actually confirmed and I have more data.” 💵 NIH grants: Sens. Susan Collins (R-ME) and Patty Murray (D-WA) both raised concerns about the NIH’s planned 15% cap on indirect cost reimbursements for grant recipients. Bhattacharya didn’t contradict the Trump administration’s cuts or say he would reverse course. “I want to make sure that the money goes to the research,” he said. He told Collins he would “make sure that your concerns are addressed.” 💊 Medication abortion: Makary was asked numerous times about his stance on access to the abortion pill mifepristone . Some Republican senators asked if he would reinstate an in-person dispensing requirement for mifepristone, which was removed by the Biden administration in 2021. Makary said he has “no preconceived plans” on mifepristone, and will “take a solid, hard look at the data.” The Senate HELP committee is expected to vote on Bhattacharya and Makary’s nominations next Thursday. 🏭 President Donald Trump’s tariffs are causing the biopharma industry to rethink where it produces and manufactures its drug products, with several notable companies commenting on the topic this week. Pfizer CEO Albert Bourla, who has visibly tried to put himself in Trump’s good graces since the election, said his company could “quickly” onshore drug production if it needed to mitigate the effects of the tariffs. Pfizer is in a better position thanks to its US manufacturing network, Bourla said, potentially giving it an advantage over some rivals. But he cautioned the situation remains “volatile,” and he said last month that any tariffs in Europe would likely impact Pfizer in some form. Trump has yet to specifically impose tariffs against pharma goods produced in Europe, but has threatened a 25% tax import on all pharma goods into the US. Eli Lilly, meanwhile, is asking state governments to send pitches about why the company should invest in them as part of the company’s $27 billion US manufacturing promise . It’s an unusual move, but one that comes due to the “unprecedented nature” of the investment, according to a company spokesperson. Lilly is already in negotiations with an undisclosed number of states. At least one expert believes the company will likely select areas with universities and academic institutions to tap into their local talent pools. Merck KGaA also expects to be able to weather the tariffs thanks to its earlier efforts to move manufacturing sites to the US. CEO Belén Garijo believes the situation will be “manageable,” and in October the company announced plans to boost antibody-drug conjugate manufacturing in St. Louis. But one company that could see stronger headwinds is Bavarian Nordic , which has contracts with the US government to stockpile its drug in the event of smallpox and mpox outbreaks. CEO Paul Chaplin said the onshoring process would be doable, but he warned it could take five to 10 years to complete . One of the last Big Pharma holdouts in obesity is now in the game, as AbbVie partnered with the Danish biotech Gubra on an experimental medicine called GUBamy, also known as GUB014295. The program is not a GLP-1, but rather a long-acting amylin analog, which is a peptide-based drug that could be more complex to manufacture. It’s currently in the multiple ascending dose portion of Phase 1 testing, with interim results expected before July. There are multiple other companies in the amylin space, including Zealand, Metsera, Structure and AstraZeneca, among others. But recent attempts at amylin have not always succeeded. Novo Nordisk tested its GLP-1 drug Wegovy in combination with an amylin program and disappointed investors , while Zealand is still searching for a partner for its own amylin drug. 🥼Two days after announcing plans to shut down an FDA lab devoted to ensuring the US drug supply remains safe and potent, the Trump administration changed its plans . The St. Louis lab, known as the Office of Testing and Research, tests the quality of drugs and investigates high-profile drug safety issues. The lab was one of about 30 FDA facilities whose leases were marked for termination by DOGE. “They are the FDA’s premier drug analysis lab,” former FDA Acting Commissioner Janet Woodcock told Endpoints. “Congress has been constantly on FDA to make sure imported drugs are of adequate quality, and the St. Louis lab is the foundation of that.” A small Swiss biotech called Asceneuron quietly disclosed it would halt a trial for an oral Alzheimer’s drug this week, posting an update to the government’s clinical trials registry saying it was due to a “strategic decision.” The company’s CEO, Barbara Angehrn Pavik, declined to provide a reason for the trial termination. Asceneuron’s decision comes after two top competitors, Eli Lilly and Biogen, both scrapped their own oral Alzheimer’s drugs. DON’T MISS:

中国北京，2025年3月7日——近日，全球领先的生物制药公司诺和诺德举办媒体沟通会，全球管理层与国际媒体分享了多疾病领域的创新要点：直击慢性疾病之间的相互关联，以整体性方式打破壁垒，强化协同，全面拥抱心血管代谢疾病领域的创新变革；同时继续深耕罕见病领域，引领药物研发创新，探索整合解决方案，全力构筑更健康的未来。 诺和诺德全球总裁兼首席执行官周赋德（Lars Fruergaard Jørgensen）说道：“一个多世纪以来，诺和诺德专注于‘驱动改变，携手战胜严重慢性疾病’，为社会创造价值。着眼未来，心血管代谢疾病领域充满潜力，公司在这方面拥有领先的科研经验。我们有望与合作伙伴一道，共同解锁‘长期健康’的更多可能性。” 诺和诺德全球总裁兼首席执行官周赋德（Lars Fruergaard Jørgensen） 01 解锁GLP-1价值，应对更广泛的心血管代谢疾病 基于对慢性疾病的深入研究和科学理解，诺和诺德重点介绍了其在心血管代谢疾病方面的洞察。心血管疾病、慢性肾病和包括2型糖尿病、肥胖症及代谢功能障碍相关脂肪性肝炎（MASH）在内的代谢疾病，常常在同一患者身上交叠或并存。这类相互关联的疾病被称为心血管代谢疾病。 为此，诺和诺德持续开发创新药物和健康解决方案——应对这些相互关联的代谢和心血管疾病，提供相互补充的疗效。以司美格鲁肽2.4 mg为例，SELECT心血管结局试验的结果显示：司美格鲁肽2.4 mg可使成年超重或肥胖症患者主要不良心血管事件风险降低20%。 诺和诺德执行副总裁兼商务策略及企业事务负责人柯米拉（Camilla Sylvest）表示：“我们的胰高糖素样肽-1受体激动剂（GLP-1RA）类药物为糖尿病、肥胖症和相关疾病的患者带来了积极的健康获益，同时也改变了社会看待和治疗肥胖症与糖尿病的方式。2024年，全球超过4,500万糖尿病和肥胖症患者获益于诺和诺德的创新产品。未来，我们将在推动科学创新的同时，持续提升产能，服务更多患者。” 诺和诺德执行副总裁兼商务策略及企业事务负责人柯米拉（Camilla Sylvest） 谈及心血管代谢疾病领域的研发创新，诺和诺德全球开发执行副总裁Martin Holst Lange介绍道：“心血管代谢疾病是一类非常复杂的疾病，是多种因素相互作用的结果，可能带来严重的健康风险。这需要以患者为中心，采用全面、综合的管理方法。因此，我们在为该领域开发创新疗法时，会从作用机制、有效性与耐受性、合并症及便利性等多个维度进行综合考虑，提升产品管线的深度和广度。” 诺和诺德全球开发执行副总裁Martin Holst Lange 目前，诺和诺德正通过一系列丰富的临床研究（SELECT研究、FLOW研究、SOUL研究、STRIDE研究、ESSENCE研究、EVOKE研究），进一步挖掘司美格鲁肽在心血管代谢领域更广泛的治疗潜力，并根据从中获得的经验，打造心血管代谢领域差异化的未来产品组合。此前，司美格鲁肽获批用于2型糖尿病治疗已超过7年，获批用于肥胖症治疗近4年。 值得一提的是，诺和诺德已在GLP-1与胰淀素二者的联合作用机制方面取得重要进展：CagriSema的3期临床试验REDEFINE 1结果显示，在伴有一种或多种合并症的超重或肥胖症患者中，接受CagriSema治疗68周后，平均体重降幅达22.7%，并展示出良好的安全性。此外，针对皮下注射Amycretin开展的1b/2a期试验已成功完成，对Amycretin的安全性、耐受性和药代动力学进行研究，并完成概念验证，证实了其减重潜力。 02 聚焦个性化需求，惠及更多罕见病患者 过去45年来，诺和诺德深耕罕见血液疾病、罕见内分泌疾病治疗领域，持续开发创新疗法，满足患者多样化的需求。本次活动上，诺和诺德罕见病业务部执行副总裁Ludovic Helfgott介绍了公司在罕见病治疗领域的三大方向： 进入仍存在巨大未满足需求的其他罕见病治疗领域 我们的目标是开发药物管线，更好地服务患者的个体化需求，并推动血友病管理和其他罕见血液疾病如镰状细胞病的重大变革 药物创新之外，我们致力于提供整合的解决方案，从而赋能医生和患者，提升治疗的依从性 诺和诺德罕见病业务部执行副总裁Ludovic Helfgott 全球有近800万人患有镰状细胞病1，而患者在治疗选择、医疗可及性和长期管理等方面存在重重挑战。诺和诺德的目标是改善全球镰状细胞病患者的治疗标准，通过开发包括新型疾病修饰、基因编辑技术，以及可穿戴设备在内的整合解决方案，帮助患者减少疼痛、提高生活质量。 在罕见出血性疾病治疗领域，诺和诺德从未停止创新探索，不断开发新治疗方案（口服制剂、单克隆抗体、下一代基因组编辑技术），并持续倡导尽早进行规范化预防治疗，以期改善治疗结局。 03 “中国同创”五周年，引领创新“加速度” 2020年，诺和诺德正式启动“中国同创”项目，推动中国全面纳入全球同步研发体系，旨在实现从临床试验到新药递交申请全球同步，让中国患者同步受益于全球创新药。经过五年多的发展，项目取得了里程碑式进展：2024年，全球首个胰岛素周制剂诺和期®（依柯胰岛素注射液）在中国获批，开启胰岛素治疗周制剂时代。这是“中国同创”项目的首个落地成果，实现国内外同步研发、同步获批，同步上市。 依托“中国同创”战略框架，2024年，诺和诺德中国在多个疾病领域达成了历史性突破：覆盖糖尿病、肥胖症、罕见病三个疾病领域的3款创新药物同月获批，包括全球首个用于长期体重管理的GLP-1RA周制剂诺和盈®、中国首个长效重组凝血因子Ⅷ诺易特®。 “中国同创”项目在为患者带来创新治疗方案的同时，也为中国医药创新生态系统建设贡献力量——与多家医疗机构建立“临床研究战略合作中心”，合力提升临床试验能力和诊疗水平。中国也成为诺和诺德全球首个正式使用“战略合作中心”模式来推动临床试验开展的分部。截至2024年12月，诺和诺德临床研究战略合作中心已经拓展至10余家医疗机构共40余个专业科室。 诺和诺德大中国区医药和质量部企业副总裁张克洲表示：“随着药品审评审批制度改革持续深化，创新药国内外获批‘时差’进一步缩短，中国患者能够更早获益。五年间，‘中国同创’项目取得的显著进展就是最好的例证。这也让我们更加充满信心，加速将全球创新成果带给中国患者。目前，在肥胖症、血友病、生长障碍以及其他严重慢性疾病如心血管疾病、慢性肾病、代谢功能障碍相关脂肪性肝炎、阿尔茨海默病等多个新治疗领域，我们在准备或正在开展多项临床试验。从临床试验来看，诺和诺德中国已全面进入诺和诺德所有疾病领域。” 诺和诺德大中国区医药和质量部企业副总裁张克洲 诺和诺德全球高级副总裁兼大中国区总裁周霞萍表示：“作为一家使命驱动的企业，诺和诺德积极应对全球健康挑战，加速布局多疾病领域。在中国，我们的创新步伐也越来越快。同时，诺和诺德携手合作伙伴，提升疾病认知，推动疾病的早诊早治。此外，我们积极践行社会、环境、财务三重责任，推动可持续发展。未来，诺和诺德也将继续履行对患者的长期承诺，为建设‘健康中国’做出贡献。” 诺和诺德全球高级副总裁兼大中国区总裁周霞萍 参考资料： 1.  Thomson, AM, et al. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease Study 2021. Lancet Haematol. 2023;10(8):e585-e599. 一审| 黄佳 二审| 李芳晨 三审| 李静芝