FASN

Phase 2b study evaluating diagnostic performance of 18F-RAD101 for suspected recurrent brain metastases from solid tumors of different origins Underscores Radiopharm’s commitment to developing transformative oncology radiopharmaceuticals April 28, 2025 -- Radiopharm Theranostics (ASX:RAD, Nasdaq: RADX, “Radiopharm” or the “Company”), a clinical-stage biopharmaceutical company focused on developing innovative oncology radiopharmaceuticals for areas of high unmet medical need, today announced the dosing of the first patient in its U.S. Phase 2b imaging study of 18F-RAD101 in suspected recurrent brain metastasis. The U.S. multicenter, open-label, single arm Phase 2b clinical trial1 is evaluating the diagnostic performance of 18F-RAD101 in 30 individuals with confirmed recurrent brain metastases from solid tumors of different origins. The primary objective of the study is concordance between 18F-RAD101 positive lesions and those seen in conventional imaging (MRI with gadolinium) in participants with suspected recurrent brain metastases. RAD101 is a novel imaging small molecule that targets fatty acid synthase (FASN), a multi-enzyme protein that catalyses fatty acid synthesis and is overexpressed in many solid tumors, including cerebral metastasis. Disruption of FASN activity allows for the accurate detection of cancer cells, representing a strongly viable target for the imaging of brain metastasis. Positive data from the Imperial College of London’s Phase 2a imaging trial of 18F-RAD101 in patients with brain metastases showed significant tumor uptake that was consistent with and independent from the tumor of origin.2 “We are proud to pioneer the first U.S. clinical trial of RAD101,” said Harshad R. Kulkarni, MD, Chief Medical Advisor at BAMF Health and Principal Investigator of this Phase 2b study. “This marks an important step toward improving diagnostic precision and enabling more evidence-based, individualized treatment decisions for patients with brain metastases following stereotactic radiosurgery.” “This trial is an excellent illustration of BAMF Health’s clinical trials platform in action,” added BAMF Health’s Director of Clinical Trials. “Our Radiopharmacy is producing the imaging agent on-site, our clinic team is caring for the patient and providing the best image in the world, and our clinical trials team expertly coordinates it all. BAMF’s facility and team were built to do trials just like this.” “Current standard of care imaging is less sensitive in discriminating between tumor recurrence and radiation necrosis in patients with brain metastasis who have received anticancer treatments, including radiation,” said Riccardo Canevari, CEO and Managing Director of Radiopharm Theranostics Ltd. “18F-RAD101 has the strong potential to improve diagnostic accuracy of brain metastases, and holds promise for discriminating between treatment effect and true progression in the more than 300,000 patients diagnosed with brain metastasis each year in the U.S. alone. We look forward to advancing this clinical trial and to reporting topline data in the second half of 2025.” Radiopharm Theranostics is a clinical stage radiotherapeutics company developing a world-class platform of innovative radiopharmaceutical products for diagnostic and therapeutic applications in areas of high unmet medical need. Radiopharm is listed on ASX (RAD) and on NASDAQ (RADX). The company has a pipeline of distinct and highly differentiated platform technologies spanning peptides, small molecules and monoclonal antibodies for use in cancer. The clinical program includes one Phase 2 and three Phase 1 trials in a variety of solid tumor cancers including lung, breast, and brain. Learn more at radiopharmtheranostics.com. BAMF Health is the world’s first vertically integrated platform for intelligence-based precision medicine. Headquartered in Grand Rapids, Michigan, BAMF Health employs the most advanced theranostic imaging technology to detect and treat cancer and other diseases and conduct advanced clinical trials. Our overriding mission is to empower patients to become people again. With a team of data scientists, researchers, software engineers, and clinicians —all working in lockstep—we’re making good on it. To learn more about BAMF Health, visit www.bamfhealth.com._______________________ 1 https://www.clinicaltrials.gov/study/NCT06777433 2 S. Islam et. Al., EJNMMI; 07 February 2025. https://doi.org/10.1007/s00259-025-07118-0 The content above comes from the network. if any infringement, please contact us to modify.

点击“蓝字”关注我们代谢相关脂肪性肝病（MAFLD）是全球肝病的主要病因之一，影响着全球超1/4的成年人，在生活方式改变与代谢疾病流行的背景下，其发病率逐年攀升。刚刚召开的世界糖尿病联盟2025世界糖尿病大会（IDF 2025）上，组委会特别设立了“The liver and diabetes: diabetic liver and hepatogenous diabetes”专题，以探讨糖肝共管策略。本刊特邀温州医科大学附属第一医院郑明华教授接受采访，分享MAFLD的诊疗策略与最新进展郑明华 教授温州医科大学附属第一医院温州医科大学附属第一医院感染内科肝病中心主任医师、教授、博士生导师亚太肝病学会代谢相关脂肪性肝病联盟委员CHESS-MAFLD协作组组长中国研究型医院学会肝病学分会委员中国研究型医院学会肝病学分会脂肪肝学组委员中国医师协会循证医学专业委员会委员中国中西医结合学会循证医学专业委员会委员浙江省医学会肝病学分会委员浙江省数理医学会肝病学分会副主任委员Global NASH Council memberGOASIA member长期致力于脂肪肝的流行病学与发病机制研究，平台发展态势良好，积淀深厚，与国内外相关团队深度合作且有较高知名度。在Nature、Cell、Science、Science Translational Medicine、Nature Review Gastroenterology Hepatology、Nature Review Nephrology、Nature Review Endocrinology、Nature Communications、Journal of Clinical Investigation、Journal of Hepatology、Gut、Hepatology、Gastroenterology、Lancet Gastroenterology & hepatology等刊物发表或合作发表相关工作，H指数62。牵头编写脂肪肝国际多学科专家共识4项，参编脂肪肝国际专家共识/指南2项（全球及亚太指南），及中国2024版“代谢相关（非酒精性）脂肪性肝病防治指南”。问题1：您带领团队深耕于肝病领域，针对MAFLD的筛查、诊断、评估以及治疗等在权威期刊上发表了一系列研究论文。基于这些研究成果，请您介绍当前全球及我国MAFLD的疾病现状？郑明华教授：既往，脂肪肝在临床上并不受重视，无论是在肝病科还是糖尿病科。而随着对脂肪肝认识的不断深入，临床医生和患者对脂肪肝的重视程度都在不断增加。目前，脂肪肝在全球范围内的疾病负担相对较重，最新的流行病学调查数据显示全球范围内脂肪肝的患病率约为30%，中国的患病率约为29%，而在糖尿病人群中，脂肪肝的患病率更高，为55%~65%。但是，由于脂肪肝没有明显的外在表现（如疼痛），且疾病进展缓慢，出现肝病相关事件（如肝硬化失代偿期、肝硬化腹水、肝癌、死亡等）需要10年以上时间，因此既往临床医生和患者很容易忽视这一疾病。此外，脂肪肝的管理较为困难，涉及到多学科合作，因为脂肪肝往往不是单独出现，而是合并其他系统疾病共同发生，比如糖脂代谢异常、心血管疾病、肾脏病、骨关节炎、骨质疏松等。但是这一情况目前只有部分医生有相关认知，因此仍需要时间来加深临床医生对脂肪肝的认识，以提高临床医生对脂肪肝的重视程度。而在糖尿病的管理中，脂肪肝也是非常重要的一环，因此在本次IDF 2025大会中，组委会特别设置了“糖肝”专题，会议现场的参与人员非常多，表明现在越来越多人逐渐认识到脂肪肝管理的重要性。问题2：目前临床上MAFLD的治疗策略是什么？还存在哪些短板？郑明华教授：疾病的治疗永远是患者和医生最关心的问题。但是，一直以来，脂肪肝的治疗是依靠生活方式管理进行的，因为既往临床上无针对脂肪肝的特效药。直至去年，美国食品药品监督管理局（FDA）批准了第一款用于治疗代谢功能障碍相关脂肪性肝炎的药物瑞美替罗，但是这款药物目前只在美国上市，因此国内仍然没有相应的治疗药物。而且瑞美替罗的价格非常高，连续使用一年的价格将近33万，但针对肝纤维化的有效率仅三分之一，这意味着多数患者是无法从中获益的。因此，目前临床上管理脂肪肝的主要策略和基石仍是生活方式管理。此外，还有一些药物可以用于脂肪肝的管理，如维生素E、保肝药等，但是需要注意保肝药的应用需要适当，因为其无法根治脂肪肝。运动和饮食管理才是根治脂肪肝的策略。然而，生活方式管理这一医嘱的执行率偏低，很多患者很难坚持下去，这就导致只有部分患者可以从中获益。目前，除了生活方式管理，胰高血糖素样肽-1（GLP-1）受体激动剂（如司美格鲁肽）也可以用于脂肪肝的管理，但是在临床上处方司美格鲁肽时需要患者合并糖尿病或者肥胖症，单纯脂肪肝患者无法处方司美格鲁肽。而且目前糖尿病患者使用司美格鲁肽可以进行医保报销，而肥胖症患者使用司美格鲁肽是自费的。除了此类药物，维生素E是临床上常用的药物之一，早在2010年，新英格兰医学杂志上就发表过相关研究。在我国，2016年由施军平院长牵头开展了维生素E用于脂肪肝治疗的相关研究，研究结果于去年年底正式发表。这一研究提示，中低剂量维生素E对中国人群的脂肪肝具有保护作用。而且维生素E的价格较低，对患者的经济负担较小，因此也是目前临床上常用的常规处方。问题3：请问MAFLD新型药物的研发进展有哪些？这些新型药物展现出哪些令人期待的潜力或前景？郑明华教授：脂肪肝这一赛道的竞争非常激烈，目前约有200种药物正在研发中，而已经进入临床前期阶段的仅30%~35%，也就是六七十种；进入Ⅲ期临床试验的仅6种，其他药物大多都集中在Ⅱ期临床试验。而且这些药物的种类很多，比如GLP-1受体激动剂、成纤维细胞生长因子21（FGF21）类似物等具有强代谢调节作用的药物，但目前的研究显示这些药物对肝脏的保护不太强。目前，司美格鲁肽用于脂肪肝的Ⅲ期试验已经完成1.5年的结果，最新释放的证据显示其对脂肪性肝炎和纤维化的改善达到了预期目标，而且不良反应是可以接受的。而对于FGF21类药物，具有调节代谢的作用，主要在抗纤维化方面展现出显著效果，这也是非常令人期待的。针对纤维化4期，即早期肝硬化的患者，FGF21类似物是一个可行方案。除了上述具有代谢调节作用的药物以外，另一大类是具有肝脏保护作用的药物，如瑞美替罗，一种甲状腺激素受体-β（THR-β）选择性激动剂。此外还有乙酰辅酶A羧化酶（ACC）抑制剂、脂肪酸合成酶（FASN）抑制剂等。这些药物具有肝脏保护作用，但是调节代谢的作用较弱。未来，有可能使用代谢调节药物+肝脏保护药物的双联方案来治疗MAFLD，当然，这一设想仍需相应的研究进行验证。此外，还有一种可行方案是基因靶向治疗药物，如针对PNPLA3基因以及针对HSD17B13基因的靶向药物，这两款药物都是长效制剂，注射一针疗效持续1~3个月。但是这两款药物仍处于Ⅱ期临床试验阶段，是否可以真正上市仍未可知。综上所述，目前脂肪肝这一赛道有很多靶点药物正在研发，所以未来攻克脂肪肝不是一个缥缈的梦想，是可以期待的。问题4：2025年2月，MAFLD被正式写入《健康中国战略之慢病综合管理蓝皮书》，提到其与糖尿病、心脑血管疾病等代谢相关慢病的互筛互查非常重要，这具有怎样的现实意义和战略价值？它又将如何影响我国MAFLD的防治与管理工作？郑明华教授：作为医生，我们能做的就是治疗疾病。在国家层面，则通过政策引导患者科学就医、规范治疗，因此将脂肪肝纳入慢病管理体系是一个正确决策。今年提出的三年体重管理年这一方针也表明国家已经意识到慢病给社会、民众和经济带来的重大负担，因此必须通过政策来引导和防治。如果只依靠医生进行疾病的治疗，那能获益的患者只是一部分，而且会给医疗系统带来巨大的压力。因此，全社会共同努力进行慢病的管控和防治是必须要做的事情。通过科普宣教，在全社会形成慢病管理和生活方式管理的风潮，才能够真正降低慢病对全社会的负担。我也非常庆幸能够看到我国将脂肪肝纳入蓝皮书，这意味着脂肪肝正在受到更多的重视，也期待通过政策的引导，可以提高公众对脂肪肝这一疾病的重视程度，促进脂肪肝的早筛早治，将脂肪肝诊疗和预防关口提前，构筑疾病管理的长城，使更多患者真正获益。声明：本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。最新《国际糖尿病》读者专属微信交流群建好了，快快加入吧！扫描左边《国际糖尿病》小助手二维码（微信号：guojitnb），回复“国际糖尿病读者”，ta会尽快拉您入群滴！（来源：《心肾代谢时讯》编辑部）

April 23, 2025 -- Bluejay Therapeutics, a clinical-stage biopharmaceutical company dedicated to developing potentially life-changing therapeutics for serious viral and liver diseases, today announced it will present new preclinical data on the characterization of BJT-188, a liver-targeted fatty acid synthase (FASN) inhibitor being investigated for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), at the European Association for the Study of the Liver (EASL) Congress 2025, taking place May 7-10, 2025, in Amsterdam. The presentation will also be available on Bluejay’s website following the meeting. BJT-188 targets FASN, a key enzyme in the de novo lipogenesis (DNL) pathway responsible for converting dietary sugar metabolites into saturated fatty acids, which serve as precursor for lipotoxic lipids such as triglycerides. Overactivation of FASN has been implicated in the pathogenesis of MASH, primarily through excessive lipid accumulation in the liver, leading to inflammation and fibrosis. “The presentation of BJT-188 preclinical data represents an important milestone for Bluejay as we leverage our Liver-Targeting Advanced Platform (L-TAP) to expand our strategic focus beyond viral hepatitis into MASH, a serious liver disease with limited treatment options affecting millions globally,” said Keting Chu, M.D., Ph.D., Founder and Chief Executive Officer of Bluejay Therapeutics. Previous studies of other FASN inhibitors have demonstrated therapeutic potential in MASH; however, their clinical utility has been limited due to systemic side effects. BJT-188 addresses this challenge by leveraging Bluejay’s proprietary Liver-Targeting Advanced Platform (L-TAP), designed to enhance drug delivery to the liver. This targeted approach potentially reduces systemic exposure, thereby minimizing side effects and improving the overall therapeutic profile. Poster presentation details are as follows: Title: Preclinical characterization of BJT-188, a liver-targeted fatty acid synthase inhibitor for the treatment of MASH Abstract number: 2721 Session: Poster - MASLD: Experimental and pathophysiology Date: Friday, May 9 Time: 8:30 a.m. CET Presenter: Jerome Deval, Ph.D., Bluejay Therapeutics Metabolic dysfunction-associated steatohepatitis (MASH) is a severe liver disease that affects an estimated 5% of the global adult population. In the United States, it is estimated that MASH currently impacts approximately 13 million adults and is expected to impact more than 19 million by 2039. MASH is characterized by fat accumulation, inflammation and fibrosis in the liver. Increasing fibrosis is associated with a greater risk for progression to liver-related complications, including cirrhosis, liver cancer and liver-related death. MASH is also a leading cause of liver transplant. There are limited treatment options for MASH worldwide, with only one treatment currently approved in the United States. BJT-188 is a preclinical, liver-targeted fatty acid synthase (FASN) inhibitor currently under investigation by Bluejay Therapeutics for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). BJT-188 targets the FASN enzyme, which is involved in converting dietary sugar metabolites into lipotoxic lipids. BJT-188 was developed utilizing Bluejay Therapeutics’ proprietary Liver-Targeting Advanced Platform (L-TAP) to enhance its liver-specific delivery. Bluejay Therapeutics’ Liver-Targeting Advanced Platform (L-TAP) integrates molecular modeling and drug design to create novel therapeutics with optimized liver targeting. By enhancing liver specificity, L-TAP aims to reduce drug distribution to peripheral tissues, thereby improving therapeutic efficacy and minimizing side effects. L-TAP has been applied to the development of BJT-628, a hepatitis B virus (HBV) transcript inhibitor currently under evaluation as part of potential combination regimens aimed at achieving a functional cure for chronic hepatitis B (CHB), and BJT-188, a fatty acid synthase (FASN) inhibitor being investigated for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). Both BJT-628 and BJT-188 have demonstrated promising results in preclinical studies. Bluejay Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing potentially life-changing therapeutics for serious viral and liver diseases. The company is currently investigating brelovitug for the treatment of chronic hepatitis D (CHD) and chronic hepatitis B (CHB) viral infections. Additionally, Bluejay is advancing several innovative programs with the goal of developing a combination regimen to achieve a functional cure for chronic hepatitis B, including a proprietary TLR9 agonist (cavrotolimod) and a liver-targeted HBV transcript inhibitor (BJT-628). The company is also investigating BJT-188, a preclinical liver-targeted fatty acid synthase (FASN) inhibitor, for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The content above comes from the network. if any infringement, please contact us to modify.