SYDNEY, Dec. 18, 2025 (GLOBE NEWSWIRE) -- Actinogen Medical Limited (ASX: ACW) announces that due to the recent accelerated screening and enrolment in the XanaMIA phase 2b/3 Alzheimer’s disease (AD) trial, the company has randomized and commenced treatment of all participants. This means that topline, final results will be available in November next year
Dr Dana Hilt, CMO, commented:
“There remains an enormous unmet medical need for patients at all stages of Alzheimer’s despite the recent availability of new monoclonal antibody therapies. Oral Xanamem has the potential to be a game-changer for Alzheimer’s patients because of its safety, ease-of-use and potential efficacy advantages. We are very grateful to trial staff, patients and their caregivers for their enthusiastic partnership in the Xanamem program.”
Key points:
- XanaMIA is a randomized trial of 36 weeks treatment with Xanamem® or placebo in patients with mild-moderate Alzheimer’s disease followed by an open-label extension phase
- Final and total enrolment is 247 participants (originally targeted 220)
- Topline final results are confirmed for November of next year (previously mid Q4) with full analysis to be completed in the months following
- A webinar to discuss this announcement and related information was held during Sydney business hours, December 18, 2025 – details on how to access a recording of the webinar are outlined below.
The robust recent enrolment in the XanaMIA trial validates the attractiveness of Xanamem as an easy-to-use once-daily oral therapy for AD with a novel mechanism designed to control elevated brain cortisol (aka the “stress hormone”). The trial was designed using data from the analysis of 34 AD patients from the previous XanADu phase 2 trial with a diagnosis newly confirmed by elevated blood pTau181 levels in a prospectively designed, double-blind, “in silico” trial. Similar patients have been selected for the current XanaMIA trial based on these data.
Actinogen has four independent, placebo-controlled trials supporting the likelihood of success in its pivotal trial program in addition to high levels of target engagement shown in a human brain PET study:
- Large Xanamem benefit on the CDR-SB endpoint seen after 12 weeks of treatment with 10 mg daily in patients with mild AD and elevated blood pTau181, with trends towards benefit on other endpoints such as ADCOMs, MMSE1 and executive function (n = 34)
- Durable (4 weeks post treatment) trend towards benefit on depressive symptoms in a moderately-severe population, validating the clinical activity of a 10 mg daily dose in the brain (n =165)
- Improvement in attention and working memory in two trials of cognition in cognitively normal, older volunteers using doses of 5 mg, 10 mg and 20 mg daily (n = 30-40 per group in both trials).
The XanaMIA trial is a double-blind, 36-week treatment, placebo-controlled, parallel group design trial in participants with mild to moderate AD and progressive disease, determined by clinical criteria and confirmed by an elevated level of the pTau181 blood biomarker. Participants receive Xanamem 10 mg or placebo, once daily, and its ability to slow progression of AD over 36 weeks of treatment is assessed with a variety of endpoints. The primary endpoint of the trial is the internationally-recognized CDR-SB (Clinical Dementia Rating scale – Sum of Boxes). The trial is being conducted in Australia and the US. All past and present participants in the trial are eligible to receive active Xanamem 10 mg treatment in the open-label extension phase commencing in Q1 next year, even if they finished the main part of the trial in 2025.
A formal interim analysis of safety and efficacy futility from the partially completed trial will be conducted by an independent Data Monitoring Committee (DMC) in late January 2026. The DMC comprises independent clinical and statistical experts who are not connected to the day-to-day conduct or analysis of the trial. The committee will review, in a highly confidential process, unblinded data for safety and efficacy futility from all available participant visits including many who will have completed the 36-week treatment period.
Topline final safety and efficacy results for the full 36 weeks of treatment from all participants will be available in November 2026. The final analysis will assess the difference in efficacy and safety between Xanamem and placebo-treated participants after the “double-blind” code is opened by trial statisticians.
Dr Steven Gourlay, the company’s CEO and MD, commented:
“We are excited to be entering the final stage of the XanaMIA randomized controlled trial with confidence in our November 2026 timeline for topline final results. As the trial continues, the team will be busy commencing the open-label extension phase so that all participants can receive active Xanamem treatment for a longer period.”
“In 2026 we will be conducting the formal interim analysis of the trial, interacting with the European Medicines Agency on the path to approval in the EU in Q2, and planning for the upcoming clinical and manufacturing programs.”
Actinogen is grateful to the XanaMIA trial staff, participants with Alzheimer’s disease and their caregivers for their significant contributions and to those who have participated in previous Xanamem trials.
® Xanamem is a registered trademark of Actinogen Medical Limited
1 ADCOMs: Alzheimer’s Disease Composite Score. MMSE: Mini Mental State Examination
Webinar recording available
Following the announcement to the Australian Securities Exchange Thursday morning Sydney time, the company held a webinar to discuss this announcement and related information for the Xanamem program.
A recording of the webinar is available on the company’s InvestorHub:
https://investors.actinogen.com.au/webinars/VyEXvP-december-webinar
View the ASX announcement (including the webinar presentation slides) on the Actinogen InvestorHub: https://investors.actinogen.com.au/link/eNm4Jy
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.
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.
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
