Emestedastat

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

2025年12月18日，全球阿尔茨海默病（AD）治疗领域迎来关键进展！澳大利亚生物科技公司Actinogen Medical Limited (ASX: ACW) 宣布，其口服创新疗法Xanamem® (emestedastat)治疗阿尔茨海默病的关键性IIb/III期临床试验XanaMIA已成功完成所有患者的随机分组和治疗启动。这项包含247名患者的研究，计划于2026年1月下旬进行中期分析，并有望在2026年11月公布最终顶线结果。作为一款旨在控制脑部皮质醇水平的口服药物，Xanamem因其潜在的安全性、便捷性及独特作用机制，被寄予厚望成为AD治疗的“游戏规则改变者”。 一、里程碑时刻：XanaMIA试验，全面冲刺 Actinogen Medical宣布，由于近期加速的筛选和入组，XanaMIA试验已成功完成所有参与者的随机分组和治疗。原计划招募220名患者，最终实际入组人数达到247名，体现了研究的强大吸引力与急迫需求。 Actinogen首席医疗官Dana Hilt博士表示：“尽管近期出现了新的单克隆抗体疗法，但阿尔茨海默病患者在所有阶段仍然面临巨大的未满足医疗需求。口服Xanamem因其安全性、易用性和潜在的疗效优势，有望成为阿尔茨海默病患者的‘游戏规则改变者’。” 关键时间点一览： 完成入组及治疗启动：2025年12月 中期分析（安全性与无效性）：2026年1月下旬（由独立数据监察委员会DMC进行） 最终顶线结果公布：2026年11月 开放标签扩展阶段（Open-label extension）：2026年第一季度启动，所有已完成主试验的参与者均可接受Xanamem治疗。 二、揭秘XanaMIA：严谨设计，聚焦核心 XanaMIA是一项双盲、36周治疗期、安慰剂对照、平行组设计的临床试验，旨在评估Xanamem对轻中度阿尔茨海默病（AD）患者的疗效和安全性。 患者选择：入组患者不仅根据临床标准判断为轻中度AD并伴有疾病进展，还通过血液pTau181生物标志物水平升高进行了确认。这一生物标志物被认为是AD的早期且可靠指标。 给药方案：参与者每日口服10毫克Xanamem或安慰剂。 主要终点：国际公认的临床痴呆评定量表-总和盒（CDR-SB），这是一个衡量AD进展和认知功能下降的核心指标。支持数据：在XanaMIA试验设计之前，Actinogen已有多项独立的安慰剂对照试验和人体PET研究数据，支持Xanamem的成功可能性。 在轻度AD且pTau181升高的患者中，Xanamem每日10毫克治疗12周后，CDR-SB终点显示出显著获益（n=34），其他如ADCOMs、MMSE和执行功能也有改善趋势。 在认知正常的老年志愿者中，5毫克、10毫克和20毫克剂量的Xanamem均显示出改善注意力和工作记忆的能力。 三、创新机制：Xanamem如何“解压”大脑？ Xanamem（emestedastat）的作用机制独特且引人注目，它并非直接清除淀粉样斑块或Tau蛋白缠结，而是着眼于控制大脑关键区域的皮质醇水平。 靶点：Xanamem通过抑制11β-HSD1酶来发挥作用。这种酶负责将非活性皮质醇转化为活性皮质醇，尤其在大脑海马体等与记忆和认知密切相关的区域高表达。 作用原理：通过抑制11β-HSD1，Xanamem能够降低大脑中过高的皮质醇水平。 皮质醇，也被称为“压力荷尔蒙”，慢性升高已被证实与阿尔茨海默病的进展密切相关，并对脑细胞具有毒性。 过高的皮质醇水平不仅损害认知功能，还与抑郁症状有关。 优势：作为一款首创的、每日一次的口服药物，Xanamem在临床试验中已显示出良好的脑部靶点结合能力，并有望减缓AD进展并改善抑郁症状。重要的是，它不会阻碍肾上腺正常产生皮质醇，从而避免了全身性副作用。四、全球布局与未来展望 Actinogen Medical总部位于澳大利亚悉尼，并在澳大利亚和美国开展临床试验。除了XanaMIA试验，公司还计划在2026年与欧洲药品管理局（EMA）进行互动，探讨Xanamem在欧盟的批准途径，并规划未来的临床和生产项目。 公司首席执行官兼董事总经理Steven Gourlay博士表示：“我们很高兴能进入XanaMIA随机对照试验的最后阶段，并对2026年11月公布最终顶线结果的计划充满信心。” 随着AD新药研发的竞争日益激烈，像Xanamem这样具有独特作用机制和口服便捷性的疗法，无疑为患者带来了新的希望。它不仅可能提供一种有别于现有单抗疗法的选择，也可能与现有疗法形成互补，为AD患者提供更全面、更个性化的治疗方案。 免责声明： 本文旨在传递医药前沿信息，不构成任何投资或医疗建议。文中提及的药物尚处于临床试验或审批阶段，其安全性及有效性尚未获得最终审批。股票市场有风险，投资需谨慎。请在专业医生指导下进行疾病诊断与治疗。