Emestedastat

（识别二维码，解锁更多澳洲牛肉烹饪教程） （识别二维码，选购奔富马年新年限定款） 整理：星尚澳洲编辑部 本文参考来源：ntnews/abc/heraldsun/网络 注：本文综合整理媒体资讯，无任何立场！ 一种每天只需一次的口服药物，正在让全球医学界重新审视阿尔茨海默病的干预方式。 这项研究从澳洲起步，历经十余年探索，如今已进入关键验证阶段。 在谨慎、系统、透明的科学流程中，它正在接受最重要的一次检验。 从一个分子发现开始的长期研究 故事的起点，并不在临床病房。 十多年前，英国爱丁堡大学的科研人员首次识别出一种具有潜在调节作用的分子结构。 这一发现随后被澳洲悉尼的生物技术公司Actinogen Medical持续推进，并逐步发展为系统性药物研发项目。 最终，这款候选药物被命名为Emestedastat，商品名为Xanamem。 它并非沿用传统阿尔茨海默病治疗中常见的蛋白靶点思路，而是选择了一条不同的研究路径。 聚焦被忽视的生理信号 在长期研究中，科学家将注意力集中在一种与压力反应相关的激素上。 这种激素名为皮质醇。 皮质醇在短期内对人体具有重要调节意义，但当其在大脑中长期处于较高水平时，可能会对神经系统的稳定状态产生影响。 Xanamem的设计目标，正是调节大脑中特定区域的皮质醇生成水平。 该药物具备穿越血脑屏障的能力，并以每日一次口服的形式进行给药，便于长期使用与观察。 一项进入关键节点的临床试验 目前，这款药物正通过一项名为XanaMIA的关键性临床试验接受验证。 XanaMIA是一项IIb/III期、为期36周的随机、安慰剂对照研究。 试验共招募了220名轻度至中度阿尔茨海默病患者。 所有入组患者均被筛选出血液中pTau181水平升高，这是一种与疾病进程相关的生物标志物。 该试验的核心目标，是评估Xanamem是否能够在全球通用的临床痴呆评定量表中，展现出对疾病进展速度的调节作用。 独立评估机制的中期审查 在这项试验中，一个由美国主导的独立数据监测委员会，承担着阶段性评估任务。 这一委员会的职责，是在不解盲数据的前提下，判断试验是否具备继续推进的科学合理性。 这种评估在医学研究中被称为“可行性或非无效性分析”。 简单来说，它并不是为了提前给出结论，而是判断研究是否值得继续投入时间与资源。 在去年11月，该委员会已确认试验在安全性方面符合继续标准。 而最新一轮评估结果，正被视为XanaMIA试验的重要节点之一。 试验的完整顶线数据，仍计划于今年11月对外公布，并提交同行评审期刊。 来自研究团队的谨慎期待 Actinogen Medical首席执行官史蒂文·古尔雷博士（Dr Steven Gourlay）表示，研究团队对试验方向保持信心。 他同时强调，独立委员会的存在本身，就是为了确保研究过程的客观性与科学严谨性。 目前，所有临床数据仍处于盲态管理之中，任何结论都需等待最终分析完成。 真实世界中的观察样本 在严格的科研框架之外，部分参与者的变化也为研究提供了现实参考。 在澳洲与美国，已有约400名患者在不同阶段接受过Xanamem相关治疗。 其中，一些参与者在试验期间，于交流意愿、情绪稳定度及部分认知测试中呈现出积极变化。 研究人员指出，这些观察结果本身并不构成结论，但有助于理解药物在真实使用环境中的表现。 从专科治疗走向更早期管理的可能性 随着血液检测技术的发展，阿尔茨海默病的识别时间点正在逐步前移。 史蒂文·古尔雷博士（Dr Steven Gourlay）曾表示，若未来顺利完成审批，这类安全性与可及性较高的口服药物，或具备由全科医生开具处方的潜力。 这意味着疾病管理，有望从更早阶段开始介入，并以长期稳定方式进行。 持续推进的科研基础 在资金层面，Actinogen Medical近期获得了约430万澳元的研发税收激励预付款。 这笔资金来自其预期联邦研发返税计划的一部分，为当前研究阶段提供了稳定支持。 研究团队表示，这类非稀释性资金，有助于确保试验按既定节奏推进。 写在关键结果之前 从分子发现，到多国临床试验。 从实验室推演，到独立委员会的审慎评估。 Xanamem正在经历一条完整而严格的科学验证路径。 今年11月，XanaMIA试验的最终结果即将公布。 这项从澳洲出发的研究，是否能为阿尔茨海默病的长期管理提供新的可能，时间将给出答案。 而此刻，医学界正在耐心等待。 今日话题讨论  对此，你有什么看法？ 欢迎留言评论～ - END -  欢迎将文章分享到朋友圈  本公众号所发布的内容，部分文字、图片、视频等素材来源于网络，仅用于学习、交流与非商业性用途。我们始终尊重原创、尊重版权，尽最大努力注明来源和作者，绝无恶意侵犯他人合法权益之意。 若本号发布的内容无意中侵犯了您的版权、著作权、名誉权、隐私权、肖像权等合法权益，请您通过后台留言或邮件及时与我们联系，我们将第一时间核实处理，立即修改或删除相关内容，避免不必要的争议和损失。 请勿直接投诉或举报，可先友好沟通解决！

XanaMIA trial fully enrolled with final results expected in November 2026 SYDNEY, Feb. 03, 2026 (GLOBE NEWSWIRE) -- Actinogen Medical Limited (ASX: ACW) announces that the XanaMIA pivotal Alzheimer’s disease (AD) trial’s independent Data Monitoring Committee (DMC) has recommended that the trial continue without amendment after its interim analysis. In doing so, the DMC determined that the unblinded safety and efficacy data it reviewed support continuing the trial to its expected completion in November this year. Highlights: In a confidential process designed to preserve the statistical power of the trial, the external DMC considered unblinded safety and efficacy data from approximately 37% of the expected final dataset, including 136 participants with one or more efficacy datapoints and 52 participants who had finished the full 36 weeks of treatment. All ACW staff and XanaMIA trial personnel remain blinded to participant treatment assignment Following the positive DMC recommendation, the XanaMIA trial will continue to treat ongoing participants with either Xanamem ® 10 mg or placebo for a total of 36 weeks – enrolment of the final 247 th participant occurred in December last year and the last participant’s final evaluation visit is expected in September this year Topline final results are due in November of this year, with subsequent presentation at a key Alzheimer’s disease scientific meeting and publication in a peer-reviewed journal All XanaMIA participants are potentially eligible to participate in the open-label extension (OLE) phase of the trial and will receive active Xanamem 10 mg A second and final global pivotal trial, similar to XanaMIA but larger, will commence in 2027 along with open-label and clinical pharmacology trials. This relatively streamlined path to approval in the US, including the design of the new trials, was agreed with the FDA in a Type C meeting last year. Actinogen CEO and MD, Dr Steven Gourlay said: “The positive recommendation from our independent Data Monitoring Committee is an important milestone in the successful conduct of our XanaMIA pivotal trial in patients with Alzheimer’s disease. We look forward to November when we can report final results for the full dataset of 247 participants. We anticipate that many XanaMIA participants will take up the opportunity for a longer period of active Xanamem therapy in the new open-label extension phase of the trial which will run for approximately two years. ” Dr Dana Hilt, CMO, commented: “ We are highly confident in the expert review conducted by the Data Monitoring Committee which was chaired by Dr Hans Moebius, who is a highly experienced Alzheimer’s leader and trials expert. Xanamem has the potential to be a game-changer for Alzheimer’s patients as a safe and effective oral therapy to slow or halt disease progression. ” An exciting year ahead The 2026-year heralds further key late-stage milestones for Actinogen in its Xanamem development program. In addition to this announcement regarding the positive outcome of the interim analysis for the XanaMIA trial, the company expects to announce the publication of the results of its XanaCIDD phase 2a major depressive disorder trial in a peer-reviewed journal during the coming months. Preparations continue for an open-label extension trial to allow all participants in the XanaMIA trial to access longer-term active Xanamem therapy. All participants in the OLE will receive active Xanamem 10 mg once daily for up to 25 months’ treatment. The OLE will provide longer term safety data for at least 14 months for all participants and measure key efficacy endpoints such as the CDR-SB, cognition and activities of daily living. The Company expects to receive scientific advice from the European Medicines Agency on its Xanamem Alzheimer’s development program in the second quarter and to attend and present at several industry and partnering conferences throughout the year. Topline final safety and efficacy results for the full 36 weeks of treatment from all participants in the XanaMIA AD trial will be available in November 2026. Positive XanaMIA results will position Xanamem as a unique and differentiated oral therapy – the first to convincingly slow and potentially stabilize Alzheimer’s disease progression. The company announced today that it is fully funded beyond the XanaMIA results in November by way of a strongly supported placement to existing and new institutional shareholders, and a share purchase plan which provides all Actinogen shareholders the opportunity to participate. ® Xanamem is a registered trademark of Actinogen Medical Limited About Actinogen Medical Actinogen Medical (ACW) is an ASX-listed, biotechnology company in the late clinical stages of development for Xanamem ® (emestedastat), its novel oral therapy for Alzheimer’s disease and depression. The Company is based in Sydney, Australia with operations and clinical trials in Australia and the US. Xanamem, has been studied in eight clinical trials with more than approximately 500 people treated to date and has a promising safety and efficacy profile. ACW’s ongoing clinical trial, XanaMIA, is a phase 2b/3 trial of 247 participants with mild to moderate Alzheimer’s disease (AD), treated for 36 weeks, followed by an open-label extension phase. The trial is now closed for recruitment and will report topline final results in November this year. About Xanamem (emestedastat) Xanamem’s novel mechanism of action is to control the level of cortisol in the important areas of the brain through the inhibition of the cortisol synthesis enzyme, 11β-HSD1, without blocking normal production of cortisol by the adrenal glands. Xanamem is a first-in-class, once-a-day pill designed to deliver high levels of brain cortisol control in regions where 11β-HSD1 is highly expressed such as the hippocampus. Chronically elevated cortisol is associated with progression in Alzheimer’s Disease and excess cortisol is known to be toxic to brain cells. Elevated cortisol is also associated with depressive symptoms. Xanamem has demonstrated excellent brain target engagement and in human trials has shown potential to slow progression of Alzheimer’s disease and improve depressive symptoms in patients with moderately severe depression. To view Xanamem’s two-minute Mechanism of Action animation, click here . Xanamem is an investigational product and is not approved for use outside of a clinical trial by the FDA or by any global regulatory authority. Xanamem ® is a trademark of Actinogen Medical. Disclaimer This announcement and attachments may contain certain "forward-looking statements" that are not historical facts; are based on subjective estimates, assumptions and qualifications; and relate to circumstances and events that have not taken place and may not take place. Such forward looking statements should be considered “at-risk statements” - not to be relied upon as they are subject to known and unknown risks, uncertainties and other factors (such as significant business, economic and competitive uncertainties / contingencies and regulatory and clinical development risks, future outcomes and uncertainties) that may lead to actual results being materially different from any forward looking statement or the performance expressed or implied by such forward looking statements. You are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Actinogen Medical does not undertake any obligation to revise such statements to reflect events or any change in circumstances arising after the date hereof, or to reflect the occurrence of or non-occurrence of any future events. Past performance is not a reliable indicator of future performance. Actinogen Medical does not make any guarantee, representation or warranty as to the likelihood of achievement or reasonableness of any forward-looking statements and there can be no assurance or guarantee that any forward-looking statements will be realized. CONTACT: Dr. Steven Gourlay CEO & Managing Director E. steven.gourlay@actinogen.com.au Investors Michael Roberts Investor Relations E. michael.roberts@actinogen.com.au Media George Hazim Media & Public Affairs Australia E: georgehazim@mediaaffairs.com.au