Saga University

近100%有效！FDA批准新型单抗CSL日前宣布，美国FDA批准Andembry（garadacimab），用于预防成人和12岁及以上儿童患者遗传性血管性水肿（HAE）发作。值得一提的是，Andembry可通过自我使用的皮下自动注射器在15秒或更短时间内完成给药。此外，根据新闻稿，Andembry是用于治疗此类患者群体的首款因子XIIa靶向疗法。HAE是一种罕见、慢性且可能危及生命的遗传性疾病，其特征是反复且不可预测的血管性水肿发作。HAE发作通常很痛苦，可能影响身体的多个部位，包括腹部、喉部、面部和四肢。HAE发生率为约每5万人中1人，任何种族群体都可能患病。通过靶向因子XIIa（一种在HAE患者肿胀发作中起关键作用的血浆蛋白），Andembry抑制HAE级联反应的顶端以防止HAE发作。FDA的批准基于关键性安慰剂对照3期VANGUARD试验的数据支持。结果显示，62%接受Andembry治疗的患者在治疗期间未出现发作。与安慰剂相比，Andembry组患者的HAE发作次数中位减少超过99%，最小二乘均值减少89.2%；需使用按需治疗的发作中位数减少超过99%，均值减少88%；中度或重度发作的中位数同样减少超过99%，均值减少90%。试验中最常见的不良反应（发生率≥7%）为鼻咽炎和腹痛。“癌症之王”总生存率近100%！潜在“best-in-class”小分子亮眼数据公布Immuneering公司今日公布其在研口服、潜在“best-in-class”MEK抑制剂atebimetinib（IMM-1-104）与改良型吉西他滨/白蛋白紫杉醇方案（mGnP）联合用于胰腺癌一线治疗的2a期临床试验积极结果。在纳入的34例患者中，6个月时，联合治疗实现94%的总生存率和72%的无进展生存率，中位总生存期（OS）和无进展生存期（PFS）尚未达到。数据显示，治疗方案已展现出显著的肿瘤缩小效果，总缓解率达39%，疾病控制率达到81%。多位患者出现肿瘤持续缩小，甚至部分病灶消失。此外，该联合治疗方案也在安全性方面表现良好。基于当前的积极数据，公司计划于2026年启动atebimetinib联合mGnP，用于胰腺癌患者一线治疗的关键性注册试验。这一进展标志着Immuneering在胰腺癌精准治疗领域的重要进步。▲Atebimetinib药物试验的OS与PFS结果（图片来源：参考资料[2]）13亿美元！礼来收购Verve Therapeutics礼来（Eli Lilly and Company）与Verve Therapeutics今日联合宣布，双方已达成最终协议，礼来将以约13亿美元总额收购Verve。此次收购将进一步强化礼来在心血管疾病领域的创新布局，特别是在基因编辑疗法方面的战略扩展。Verve致力于开发一次给药即可能带来持久疗效的基因编辑药物，以应对动脉粥样硬化性心血管疾病（ASCVD）的根本病因。其核心项目VERVE-102为潜在“first-in-class”体内基因编辑疗法，靶向与胆固醇水平和心血管健康密切相关的PCSK9基因，适用于异合子型家族性高胆固醇血症（HeFH）及部分早发性冠心病（CAD）患者。VERVE-102目前正在开展1b期临床试验，并已获得美国FDA授予的快速通道资格。参考资料：[1] U.S. Food and Drug Administration Approves CSL's ANDEMBRY® (garadacimab-gxii), the Only Prophylactic Hereditary Angioedema (HAE) Treatment Targeting Factor XIIa with Once-Monthly Dosing for All Patients From the Start. Retrieved June 17, 2025 from https://www.prnewswire.com/news-releases/us-food-and-drug-administration-approves-csls-andembry-garadacimab-gxii-the-only-prophylactic-hereditary-angioedema-hae-treatment-targeting-factor-xiia-with-once-monthly-dosing-for-all-patients-from-the-start-302483058.html[2] Immuneering Reports Positive Overall Survival Data for Atebimetinib (IMM-1-104) from Ongoing Phase 2a Trial in First-Line Pancreatic Cancer Patients. Retrieved June 17, 2025 from https://ir.immuneering.com/news-releases/news-release-details/immuneering-reports-positive-overall-survival-data-atebimetinib[3] Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk. Retrieved June 17, 2025 from https://ir.vervetx.com/news-releases/news-release-details/lilly-acquire-verve-therapeutics-advance-one-time-treatments免责声明：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：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

The collaboration will conduct a study that will evaluate the tumour microenvironment (TME) using the BostonGene Tumour Portrait test, which uses tissue samples collected before EGFR-TKI treatment and before ICI administration BostonGene, Saga University collaborate on biomarkers. (Credit: Julia Koblitz on Unsplash) BostonGene has teamed up with Japan’s Saga University to discover biomarkers for immunotherapy (IO) treatment response and treatment-related toxicity in advanced non-small cell lung cancer (NSCLC) patients. Epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) are the preferred treatment options for advanced NSCLC patients with EGFR mutations. According to BostonGene, the acquired resistance often leads to recurrence within a couple of years, presenting a challenge for post-treatment optimisation. Immune checkpoint inhibitors (ICIs) are standard therapy for NSCLC, but they lack efficacy in EGFR mutation-positive cases. However, combination therapy with ICIs and anti-angiogenic agents has shown promise in EGFR-positive lung cancer. The collaboration will conduct a study that will evaluate the tumour microenvironment (TME) using the BostonGene Tumour Portrait test. The test will use tissue samples collected before EGFR-TKI treatment and before ICI administration in patients with EGFR-positive lung adenocarcinoma. The study will examine the relationship between TME characteristics and the effectiveness of ICIs in these patients and investigate TME changes resulting from EGFR-TKI therapy. Saga University professor Naoko Aragane said: “By partnering with BostonGene, we aim to uncover novel biomarkers to revolutionize how we approach immunotherapy treatment in EGFR-positive lung cancer, ultimately improving patient outcomes.” The researchers will review cases where small-cell transformation due to EGFR-TKI resistance, to elucidate the molecular mechanisms underlying the transformation. They will enhance the understanding of TME dynamics and its implications for treatment response, informing improved therapeutic strategies for EGFR-positive lung adenocarcinoma. In 2023, BostonGene, NEC Corporation and Japan Industrial Partners established a joint venture, dubbed BostonGene Japan. The JV leverages BostonGene’s advanced molecular technology and NEC’s biocomputational algorithms. It aims to deliver personalised tests to cancer patients in Japan and collaborate with academic and industry partners to advance the development of targeted therapies. BostonGene chief medical officer Nathan Fowler said: “Our collaboration with Saga University underscores our dedication to advancing the standard of care in Japan by bringing novel technologies to the region. “The study marks an opportunity to delve deep into the complexities of the tumour microenvironment in EGFR-positive lung cancer patients, equipping oncologists with more targeted and effective immunotherapy strategies for their patients.”

Study designed to explore novel markers for identifying responders with immune checkpoint inhibitors in patients resistant to targeted therapies WALTHAM, Mass.--(BUSINESS WIRE)-- BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, and Saga University located in Saga, Japan and known for its commitment to educating and training researchers and professionals in the medical and healthcare fields, announced today the launch of a collaboration aimed at discovering biomarkers for immunotherapy (IO) treatment response and treatment-related toxicity in advanced non-small cell lung cancer (NSCLC) patients. This press release features multimedia. View the full release here: EGFR-TKI treatment effectively targets EGFR mutation-positive NSCLC. Still, acquired resistance often leads to recurrence within 1 to 2 years, presenting a challenge for post-treatment optimization. Immune checkpoint inhibitors (ICIs) are standard therapy for NSCLC, but the efficacy in EGFR mutation-positive cases is limited. However, combination therapy with ICIs and anti-angiogenic agents has shown promise in EGFR-positive lung cancer. This study will evaluate the tumor microenvironment (TME) utilizing the BostonGene Tumor Portrait™ test in tissue samples collected before EGFR-TKI treatment and before ICI administration in patients with EGFR-positive lung adenocarcinoma. The study aims to examine the relationship between TME characteristics and the effectiveness of ICIs in these patients and investigate TME changes resulting from EGFR-TKI therapy. Furthermore, researchers will review cases where small-cell transformation occurred as a mechanism of EGFR-TKI resistance, aiming to elucidate the molecular mechanisms underlying this transformation within the TME context. By addressing these objectives, we enhance our understanding of TME dynamics and its implications for treatment response, ultimately informing improved therapeutic strategies for EGFR-positive lung adenocarcinoma. “We are dedicated to advancing medical research directly impacting patient care. By partnering with BostonGene, we aim to uncover novel biomarkers to revolutionize how we approach immunotherapy treatment in EGFR-positive lung cancer, ultimately improving patient outcomes,” said Dr. Naoko Aragane, Professor of Saga University. “Our collaboration with Saga University underscores our dedication to advancing the standard of care in Japan by bringing novel technologies to the region. The study marks an opportunity to delve deep into the complexities of the tumor microenvironment in EGFR-positive lung cancer patients, equipping oncologists with more targeted and effective immunotherapy strategies for their patients,” said Nathan Fowler, MD, Chief Medical Officer at BostonGene. BostonGene, NEC Corporation and Japan Industrial Partners established BostonGene Japan Inc., a joint venture leveraging BostonGene's advanced molecular technology and NEC's biocomputational algorithms in 2023. The company aims to deliver its industry-leading personalized tests to cancer patients in Japan and collaborate with academic and industry partners to accelerate the development of targeted therapies. About BostonGene Corporation BostonGene has a mission to provide transformative, AI-integrated molecular analytics and biomarker discovery for precision matching of therapies to improve the lives of patients living with cancer and other immune-related diseases. BostonGene’s concierge-service model provides customized client solutions using a multi-omic approach prioritized for real-world impact to optimize standard-of-care therapies, accelerate research and provide cost-effective, measurable data-driven results. BostonGene’s tests reveal key drivers of each patient’s unique disease profile, including an in-depth pro the immune microenvironment, actionable mutations, biomarkers of response to diverse therapies, and recommended therapies. Through these comprehensive analyses, BostonGene’s tests generate a personalized roadmap for therapeutic decision-making for each patient. For more information, visit BostonGene at .