CETP

NAARDEN, The Netherlands and MIAMI, Oct. 28, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced that it will present additional safety and efficacy data from the pivotal Phase 3 BROOKLYN study evaluating obicetrapib in patients with Heterozygous Familial Hypercholesterolemia in a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, taking place November 16 – 18, 2024 in Chicago, Illinois. Additionally, NewAmsterdam announced that company management will participate in the Jefferies London Healthcare Conference, taking place November 19 – 21, 2024 in London. Details are as follows: 2024 AHA Scientific Sessions Presentation Title: Safety and Efficacy of Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia (BROOKLYN)Session Title: Late-Breaking Science 8: New Targets and New Treatments: Advances in Lipid TherapeuticsPresentation Date and Time: Monday, November 18, 2024, 2:14 p.m. – 2:26 p.m. CST (3:14 p.m. – 3:26 p.m. ET)Presenter: Stephen Nicholls, M.B.B.S., Ph.D., Director, Monash Victorian Heart Institute and Professor of Cardiology, Monash University Jefferies London Healthcare Conference Fireside Chat Date and Time: Thursday, November 21, 2024, at 1:00pm GMTPresenters: Michael Davidson, M.D., Chief Executive Officer, NewAmsterdam Pharma, John Kastelein, M.D., Ph.D., FESC, Chief Scientific Officer, Ian Somaiya, Chief Financial Officer A live webcast of the Jefferies fireside chat will be available through the investor relations section of the NewAmsterdam Pharma website at ir.newamsterdampharma.com. Following the live webcast, an archived replay will be available on the Company’s website. About Obicetrapib Obicetrapib is a novel, oral, low-dose CETP inhibitor that NewAmsterdam is developing to overcome the limitations of current LDL-lowering treatments. In each of the Company’s Phase 2 trials, ROSE2, TULIP, ROSE, and OCEAN, as well as the Company’s Phase 3 BROOKLYN trial, evaluating obicetrapib as monotherapy or combination therapy, the Company observed statistically significant LDL-lowering combined with a side effect profile similar to that of placebo. The Company is conducting an additional Phase 3 pivotal trial BROADWAY, to evaluate obicetrapib as a monotherapy used as an adjunct to maximally tolerated lipid-lowering therapies to provide additional LDL-lowering for CVD patients, and TANDEM, to evaluate obicetrapib and ezetimibe as a fixed-dose combination. The Company began enrolling patients in BROADWAY in January 2022 and in TANDEM in March 2024; completing enrollment of BROADWAY in July 2023, and TANDEM in July 2024. The Company also commenced the Phase 3 PREVAIL cardiovascular outcomes trial in March 2022, which is designed to assess the potential of obicetrapib to reduce occurrences of major adverse cardiovascular events, including cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and non-elective coronary revascularization. NewAmsterdam completed enrollment of PREVAIL in April 2024 and randomized over 9,500 patients. Commercialization rights of obicetrapib in Europe, either as a monotherapy or as part of a fixed dose combination with ezetimibe, for cardiovascular diseases have been exclusively granted to the Menarini Group, an Italy-based, leading international pharmaceutical and diagnostics company. About NewAmsterdam NewAmsterdam Pharma (Nasdaq: NAMS) is a late-stage biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. We seek to fill a significant unmet need for a safe, well-tolerated and convenient LDL-lowering therapy. In multiple phase 3 studies, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated. Company Contact Matthew PhilippeP: 1-917-882-7512matthew.philippe@newamsterdampharma.com Media Contact Spectrum Science on behalf of NewAmsterdamBryan BlatsteinP: 1-917-714-2609bblatstein@spectrumscience.com Investor Contact Precision AQ on behalf of NewAmsterdamAustin MurtaghP: 1-212-698-8696austin.murtagh@precisionaq.com

▎药明康德内容团队编辑 今年年初，行业媒体Evaluate针对2024年生物医药产业趋势发布了一项分析报告，对2024年可能获批上市的潜在重磅疗法进行了预测。时光匆匆，转眼已经来到了2024年的第四季度，年初这份榜单上的10款疗法中已有8款获得了FDA的批准，其中包括FDA批准的首款非酒精性脂肪性肝炎（NASH）疗法Rezdiffra，数十年来首个治疗精神分裂症的新机制药物KarXT，全球首款端粒酶抑制剂Rytelo，20多年来具有新作用机制、用于慢性阻塞性肺病（COPD）维持治疗的首个吸入式疗法Ohtuvayre等等。这些疗法的获批标志着生物医药领域的重大进步，更重要的是，它们满足了很多此前没有适合的治疗方法患者的迫切需求，也为广大患者带来了疗效更好或使用更便捷的治疗选择。 图片来源：药明康德内容团队根据公开资料制表 除了上述提到的几款疗法，我们同样期待着未来有更多的新药、好药及早被开发出来，尽快送到需要的患者手中，给他们带去新的希望。在今天的文章中，药明康德内容团队将根据多家行业媒体公布的行业报告，盘点近期还有哪些潜在重磅疗法有望获批，以及还有哪些可能对整个行业产生显著影响的研发管线值得关注。 近期有望获批的潜在重磅疗法 据公开信息，2024年第四季度至明年1月，有4款潜在重磅疗法有望获得FDA的监管决定，包括1款抗体偶联药物（ADC）和3款小分子疗法，涉及的适应症包括非小细胞肺癌（NSCLC）、伴有心肌病的转甲状腺素蛋白介导的淀粉样变性（ATTR-CM）、囊性纤维化和急性疼痛。 图片来源：药明康德内容团队根据公开资料制表 疗法名称：datopotamab deruxtecan（Dato-DXd） 研发机构：阿斯利康（AstraZeneca）和第一三共（Daiichi Sankyo） 治疗疾病：局部晚期或转移性非鳞状NSCLC Datopotamab deruxtecan是一种由人源化、靶向Trop2的单克隆抗体与创新DNA拓扑异构酶I抑制剂（DXd）连接的在研ADC。DXd具有独特的作用机制，与常见化疗药物伊立替康（irinotecan）相比，活性提高10倍。而且此药物具有很强渗透细胞膜的能力，让它们在杀伤吞入ADC的癌细胞之后，能够杀死附近的癌细胞，产生“旁观者效应”（bystander effect）。 该疗法的上市申请主要基于关键TROPION-Lung01临床3期试验的结果。与目前的标准治疗多西他赛（docetaxel）相比，接受datopotamab deruxtecan治疗的局部晚期或转移性NSCLC患者在双重主要终点之一的无进展生存期（PFS）上显示出统计学上的显著改善，这些患者之前至少接受过一种先前疗法。根据新闻稿，如果获得批准，该疗法将会是首个获批用于治疗肺癌患者的Trop2靶向ADC疗法。除了非小细胞肺癌，datopotamab deruxtecan的另一项针对此前已接受过前期全身治疗的无法切除/转移性HR+、HER2-乳腺癌成人患者的生物制品许可申请（BLA）也在正接受美国FDA的审评。 疗法名称：acoramidis 研发机构：BridgeBio Pharma 治疗疾病：伴有心肌病的转甲状腺素蛋白介导的淀粉样变性 Acoramidis是一种新一代口服、强效的转甲状腺素蛋白（TTR）小分子稳定剂。它旨在模拟具有保护作用的TTR T119M突变的功能，维持TTR蛋白的正常四聚体构象，防止具有毒性的淀粉样蛋白的产生，开发用以治疗ATTR-CM患者。BridgeBio此前公布的结果显示，acoramidis治疗使血清TTR水平在治疗第28天显著增加且维持稳定，并与ATTR-CM参与者在第30个月时降低的总死亡率（ACM）、心血管死亡率（CVM）和心血管住院率（CVH）风险相关。该疗法被行业媒体Evaluate评估为2024年可能获批上市的关键疗法。BridgeBio公司此前的新闻稿也表示，acoramidis有潜力成为重磅疗法。 ▲Acoramidis作用机制（图片来源：参考资料[2]） 疗法名称：vanzacaftor 研发机构：Vertex Pharmaceuticals 治疗疾病：囊性纤维化 Vanzacaftor是一种小分子囊性纤维化跨膜传导调节因子（CFTR）调节剂。Vanzacaftor/tezacaftor/deutivacaft正作为治疗囊性纤维化的下一代三联疗法被开发。Vanzacaftor和tezacaftor旨在通过靶向CFTR蛋白的加工和运输缺陷，增加细胞表面成熟蛋白的数量。Deutivacaftor旨在使细胞表面的CFTR蛋白保持更长时间的开放，以改善穿过细胞膜的盐和水的流动，有助于水合和清除气道中的黏液。此前公布的2期临床试验结果表明，该三联组合疗法有望恢复CFTR蛋白功能，为患者带来临床益处。近期，行业媒体Evaluate发布的盘点文章中将这款疗法列为潜在重磅疗法。目前，vanzacaftor/tezacaftor/deutivacaftor的上市申请已被FDA和欧洲药品管理局接受。 ▲Vanzacaftor分子结构（图片来源：PubChem） 疗法名称：Suzetrigine（VX-548） 研发机构：Vertex Pharmaceuticals 治疗疾病：中度至重度急性疼痛 Suzetrigine是一种口服选择性NaV1.8抑制剂，与其它NaV离子通道相比，它对NaV1.8具有高度选择性。NaV1.8是在外周神经系统的痛觉信号传导中起到关键性作用的电压门控钠通道。它是经过遗传学验证的疼痛治疗靶点。Vertex的策略是通过选择性抑制NaV1.8，开发新的药物类型。与阿片类药物相比，此类药物可能在提供更好镇痛效果的同时，避免上瘾等副作用。此前，FDA已授予suzetrigine优先审评资格，预计在2025年1月30日之前完成审评。新闻稿指出，suzetrigine有望成为二十多年来首个用于治疗急性疼痛的新机制药物。 值得关注的研发管线 除了以上近期有望获批的潜在重磅疗法，根据行业媒体Evaluate和BioPharma Dive的报告，还有一些在研疗法有望对整个行业产生显著影响，并有潜力成为重磅。按针对的适应症进行分类，这些疗法多数集中在代谢疾病、自身免疫疾病、癌症以及精神疾病上。值得一提的是，GLP-1相关疗法在入选的研发项目中占据4席，显示了这一领域的研发热度和潜在潜力。 图片来源：药明康德内容团队根据公开资料制表 CagriSema为GLP-1受体激动剂司美格鲁肽与长效胰淀素类似物cagrilintide的固定剂量组合。去年8月公布的2期临床试验结果显示，CagriSema组患者经过32周的治疗后体重下降了15.6%，糖化血红蛋白（HbA1c）水平下降了2.18个百分点。Orforglipron是礼来公司开发的一种每日一次的口服、非肽类GLP-1受体小分子激动剂。该药物的一个显著优势是它是一种非肽分子，这使得它更容易被制造和包装成药片。与同类作用机制的疗法相比，这一特性还可能有助于提高其在患者中的可负担性和可及性。Retatrutide是礼来的在研靶向葡萄糖依赖性促胰岛素多肽、GLP-1和胰高血糖素受体的三重激动剂。在一项2期临床试验中，83%接受12 mg剂量retatrutide治疗的肥胖或超重成人患者在经过24周的治疗后，达成至少15%的体重下降。MariTide是一款潜在“first-in-class”的在研抗体多肽偶联药物，在靶向胃抑制肽受体（GIPR）的单克隆抗体的特定位点上偶联了两个胰高血糖素样肽1类似物，可在激活GLP-1受体的同时抑制GIPR。此前发表的一项1期临床试验结果显示，肥胖患者只需每月一针，接受高剂量MariTide治疗85天后，体重可降低14.5%（约26斤）。值得一提的是，这些患者在停药后150天，仍可维持体重降低11.2%。 行业报告显示，过去12个月间，全球的GLP-1临床候选药物（小分子和肽类）的数量从51个增长到了79个，增速为55%。值得一提的是，药明康德在行业风口到来之前已准确预判需求并落地布局、将产能迅速放大。过去12个月里，药明康德化学业务平台赋能的临床阶段GLP-1项目从10个增长到18个，增长率达到80%，超越行业增速。能够做到这一切的前提是药明康德管理团队富有远见的发展战略，以及与客户的深度合作。 除了GLP-1相关疗法，这些入选的潜在重磅疗法中有一些在近期更新了令人鼓舞的积极临床试验结果。例如，用于治疗杂合体家族性高胆固醇血症的口服、选择性CETP抑制剂obicetrapib达到了3期关键试验的主要终点，显著降低了患者的低密度脂蛋白胆固醇（LDL-C）水平，且疗效可维持达一年。靶向PD-1和VEGF的双特异性抗体ivonescimab一线治疗PD-L1阳性晚期NSCLC的3期临床试验结果显示，与获批重磅免疫检查点抑制剂相比，ivonescimab显著降低了患者的疾病进展或死亡的风险。行业媒体Fierce Pharma发表的文章表示，这一结果具有改变NSCLC一线标准治疗的潜力。CD40L靶向聚乙二醇化抗原结合片段dapirolizumab pegol联用标准疗法治疗中度至重度系统性红斑狼疮（SLE）的3期临床试验也达到了主要终点，患者的疾病活动程度获得了显著改善。期待研发机构和监管机构能够更密切地合作，进一步加快这些创新疗法的监管审评进程，为广大的患者群体带来新的、有效的治疗选择。 近年来，FDA批准的创新疗法数量显著增加，这是初创公司、大型药企共同推动的成果，也反映了全球医药创新加速。在这个过程中，药明康德很荣幸能与全球志同道合的生物医药合作伙伴携手，为一款又一款新药的问世提供从研究（R）、开发（D）、到商业化生产（M）的一体化全流程服务。 在近日举办的2024药明康德投资者开放日上，药明康德联席首席执行官陈民章博士指出，自2017年至今，药明康德已帮助合作伙伴商业化了50个化合物，并在今年上半年也保持这一强劲势头——2024年上半年美国FDA批准的14款小分子药物中，有3款由药明康德化学业务平台支持。而2023年全球销售额前十的小分子药物中，有4款由药明康德化学业务平台服务生产。不仅如此，由行业知名网站“猎药人”从上万个分子中评选出的2023年度10大明星小分子中，有7款由药明康德化学业务平台赋能。 这些以往的成果证明了药明康德作为创新赋能者的能力，也印证了药明康德独特CRDMO模式对公司自身发展以及对行业、客户和患者的价值。对于坚持CRDMO模式的未来，陈民章博士同样充满信心：“相信在未来获批的新疗法中，一定还会有能为患者提供巨大帮助的重磅疗法出现在药明康德赋能的管线里。” 成立二十余年，药明康德在行业发展中始终坚守初心。展望未来，药明康德也希望协助更多的合作伙伴将研究成果一步步转化为临床实际应用，共同实现“让天下没有难做的药，难治的病”这一伟大梦想！ ▲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] BridgeBio Shares Data on Serum TTR Increase When Switching Participants from Placebo and Tafamidis to Acoramidis in ATTRibute-CM and its Open-Label Extension. Retrieved August 30, 2024, from https://www.globenewswire.com/news-release/2024/08/30/2938591/0/en/BridgeBio-Shares-Data-on-Serum-TTR-Increase-When-Switching-Participants-from-Placebo-and-Tafamidis-to-Acoramidis-in-ATTRibute-CM-and-its-Open-Label-Extension.html [2] BBIO - ATTRibute-CM Detailed Results From ESC Congress 2023. Retrieved August 28, 2023 from https://investor.bridgebio.com/static-files/f3645003-5c8f-4ec2-b943-03bbad0c156e [3] Vertex Announces FDA Acceptance of New Drug Application for Vanzacaftor/Tezacaftor/Deutivacaftor, a Next-In-Class Triple Combination Treatment for Cystic Fibrosis. Retrieved August 28, 2023 from https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-fda-acceptance-new-drug-application#:~:text=Investigational%20vanzacaftor/tezacaftor/deutivacaftor%20was%20granted%20Fast%20Track%20and%20Orphan%20Drug 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

NAARDEN, the Netherlands and MIAMI, Sept. 06, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced that the Compensation Committee of NewAmsterdam’s Board of Directors approved the grant of inducement share options covering an aggregate of 111,000 of NewAmsterdam’s ordinary shares to three non-executive new hires. The share options were granted as an inducement material to the employees acceptance of employment with NewAmsterdam pursuant to the NewAmsterdam Pharma Company N.V. 2024 Inducement Plan (the “2024 Inducement Plan”) and in accordance with Nasdaq Listing Rule 5635(c)(4). The share options have an exercise price per share equal to $15.91, which represents the closing market price on the Nasdaq Stock Market of the Company’s ordinary shares on, September 3, 2024 the grant date. The shares subject to the options will vest over four years, with 25% of the shares vesting on the one-year anniversary of the applicable vesting commencement date and the balance of the shares vesting in a series of 36 equal monthly installments thereafter, subject to each employee’s continued service with NewAmsterdam on such vesting dates. The options are subject to the terms and conditions of the 2024 Inducement Plan and the terms and conditions of an option award agreement covering the grant. About NewAmsterdamNewAmsterdam Pharma (Nasdaq: NAMS) is a late-stage biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. We seek to fill a significant unmet need for a safe, well-tolerated and convenient LDL-lowering therapy. In multiple phase 3 studies, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated. Company ContactMatthew PhilippeP: 1-917-882-7512matthew.philippe@newamsterdampharma.com