Core Viewpoint - Actinogen Medical Limited's XanaMIA trial for Alzheimer's disease has been fully enrolled, with positive interim results leading to the continuation of the trial, and final results expected in November 2026 [1][3][9]. Company Developments - The independent Data Monitoring Committee (DMC) has recommended that the XanaMIA trial continue without amendments, indicating confidence in the safety and efficacy data reviewed [1][4]. - The trial involves 247 participants and aims to report topline final results in November 2026, with the last participant's evaluation visit expected in September 2026 [8][10]. - An open-label extension phase will allow participants to receive active Xanamem therapy for up to 25 months, providing additional long-term safety data [6][10]. Product Information - Xanamem (emestedastat) is a novel oral therapy designed to control cortisol levels in the brain, potentially slowing the progression of Alzheimer's disease and improving depressive symptoms [11]. - The drug has shown promising safety and efficacy in previous trials, with over 500 individuals treated to date [10]. Future Expectations - The company anticipates further key milestones in 2026, including the publication of results from the XanaCIDD phase 2a trial for major depressive disorder [5][7]. - Actinogen expects to receive scientific advice from the European Medicines Agency regarding its Alzheimer's development program in the second quarter of 2026 [7].

Core Insights - Actinogen Medical Limited is progressing with its pivotal phase 2/3 trial for Xanamem, targeting Alzheimer's disease, with interim analysis expected in January 2026 and final results anticipated in late 2026 [1][2] - The company has reached a significant agreement with the FDA regarding the pathway to marketing approval for Xanamem, which includes the design of an additional pivotal clinical trial and a limited number of ancillary studies [2][3] Group 1: Regulatory Developments - The FDA meeting confirmed the regulatory starting materials for drug substance synthesis and the design of one additional pivotal clinical trial [2][6] - Actinogen plans to submit a New Drug Application (NDA) in the US and to other global regulators, following the FDA's guidance [2][3] - A similar meeting with the European Medicines Agency is scheduled for 2026, indicating a global regulatory strategy [3] Group 2: Clinical Trial Details - The ongoing XanaMIA trial involves 220 participants with mild to moderate Alzheimer's disease and is currently enrolling in Australia and the US [7] - The pivotal trial will utilize a single 10 mg dose of Xanamem compared to a placebo, as agreed upon with the FDA [4][6] - The trial design includes a small number of ancillary clinical pharmacology trials and nonclinical studies to further characterize Xanamem [6] Group 3: Product Information - Xanamem (emestedastat) is a novel oral therapy designed to control cortisol levels in the brain, which is linked to Alzheimer's disease progression and depressive symptoms [8] - The drug has shown promising safety and efficacy in previous clinical trials, with over 400 participants treated [7][8] - Xanamem's mechanism of action involves inhibiting the cortisol synthesis enzyme 11β-HSD1, targeting areas of the brain where cortisol is known to be toxic [8]

Xanamem Clinical Trial & Results - Xanamem demonstrated clinically and statistically significant benefits in depression symptoms in the XanaCIDD phase 2a trial, with a 2.7-point difference compared to placebo at the post-treatment follow-up (p < 0.05)[5] - Phase 2a data suggests a greater benefit on CDR-SB in high pTau181 patients, with a mean change from baseline of 0.6 units compared to placebo in a study of 34 participants (p = 0.09)[8] - Human PET study shows extensive binding of Xanamem to the 11β-HSD1 enzyme throughout the brain after 7 days of daily dosing at 5mg, 10mg and 20mg doses[10] XanaMIA Trial Design & Expectations - The XanaMIA trial is designed with 220 participants and includes an interim analysis at 24 weeks with N=100, with initial interim results expected in January 2026 and final results in Q4 2026[16] - The primary endpoint for the XanaMIA trial is CDR-SB (functional and cognitive measure) at 36 weeks, with key secondary endpoints including a cognitive test battery and the Amsterdam Activity of Daily Living[16] - The company hopes Xanamem will be better than anti-amyloid drugs on the primary endpoint of CDR-SB and other endpoints[21] Commercialization & Market Potential - The anti-depressant market is currently approximately $20 billion, with significant opportunities for novel mechanisms and better-tolerated drugs[34] - The Alzheimer's market is projected to reach $20 billion by 2030, presenting a major opportunity for a safe and effective oral agent like Xanamem[34] - US neurologists indicate that uptake of a safe and effective oral drug for Alzheimer's would be rapid, potentially positioning Xanamem as a first-line therapy[29]

Core Insights - Actinogen Medical Limited is progressing with its Phase 2b/3 trial for Alzheimer's disease, expecting to enroll 100 participants by the end of the current quarter, which will trigger an interim analysis in approximately six months [1][2][3] Group 1: Trial Progress and Design - The XanaMIA trial is designed to enroll 220 participants with biomarker-positive, mild to moderate Alzheimer's disease, specifically those with elevated blood biomarker pTau181 [2][13] - Currently, 60 participants have entered the treatment phase, with an additional 35 expected to enroll soon as recruitment accelerates with new clinical sites in the US, bringing the total to 35 sites [3][13] - The primary endpoint of the trial is the Clinical Dementia Rating – Sum of Boxes scale, with additional measures assessing cognition and functional ability [2][13] Group 2: FDA Interactions and Future Plans - Actinogen had a successful meeting with the FDA regarding its Major Depressive Disorder (MDD) program, which provided guidance for additional clinical trials needed for marketing approval of Xanamem [4][5] - A similar Type C meeting for Alzheimer's disease is scheduled with the FDA's Neurology Division later in 2025 to outline the path to marketing approval [5] Group 3: Mechanism and Future Applications - Xanamem, the lead compound, aims to control cortisol levels in the brain, which is linked to cognitive decline and depressive symptoms [15][16] - The company plans to explore Xanamem's potential in treating other neurological and psychiatric conditions, including Fragile X Syndrome [12][15] Group 4: Upcoming Events and Engagement - Actinogen will host a Clinical Trials Science Forum webinar on May 14, 2025, to discuss current and potential treatments for Alzheimer's disease and commercialization planning [7][8]