SYDNEY, April 01, 2026 (GLOBE NEWSWIRE) -- Actinogen Medical Limited (ASX: ACW) announces that the first participant has been treated in the open-label extension (OLE) phase of the XanaMIA trial following completion of 36 weeks of treatment in the randomized phase.
Highlights of the OLE:
- Open-label phase of up to 25 months duration, during which all participants will receive active Xanamem® 10 mg once daily, with no placebo control group
- Open to all former and current participants who have completed the randomized phase of the XanaMIA pivotal Alzheimer’s disease trial in Australia and the United States
- Generates longer term safety data and observational data on key efficacy endpoints including CDR- SB1, cognition and activities of daily living
- Data will be analyzed and reported progressively and compared with relevant historical control cohorts of patients with mild to moderate Alzheimer’s disease, as well as with data from the randomized phase of the trial.
Dr Dana Hilt, the Company’s CMO said:
“The commencement of the open-label extension phase of the XanaMIA trial enables us to offer current and past participants access to active Xanamem therapy for up to 25 months. This is an important opportunity for our trial participants, particularly the half who initially received placebo, and allows all participants to contribute valuable data on the potential durability of Xanamem benefit and its long-term safety.”
“Xanamem has the potential to be a game-changer for patients with Alzheimer’s as a potentially safe and effective oral therapy to slow or halt disease progression. Unlike anti-amyloid antibody therapies, Xanamem does not require serial MRI monitoring for safety as there is no suggestion it causes brain swelling also known as ARIA.”
Upcoming milestones including XanaMIA topline final results in November
The randomized phase of the XanaMIA pivotal Alzheimer’s disease trial continues in parallel with the OLE, following a positive Independent Data Monitoring Committee safety and efficacy futility recommendation in January this year. Final topline safety and efficacy results for the full 36-week treatment period are expected in November 2026.
The XanaMIA trial is intended to serve as one of two pivotal trials supporting the earliest potential marketing approvals for Xanamem in Alzheimer’s disease. If the trial is positive, the Company will also explore accelerated approval pathways with relevant regulators, including discussions with the FDA regarding its recently announced policy supporting approval based on a single pivotal trial with adequate supporting evidence.
During the current quarter, the Company plans to meet with the European Medicines Agency to discuss opportunities to streamline the pathway to Xanamem approval in the European Union. This meeting will be similar in nature to the successful engagement held with the US FDA in 2025. In addition, publication of results from the XanaCIDD Phase 2a depression trial in a peer-reviewed journal is anticipated in the near term. Future depression trials will be considered once results from the pivotal XanaMIA trial are available.
Dr Steven Gourlay, the Company’s CEO said:
“Actinogen is proud to be advancing Xanamem toward pivotal trial results later this year while simultaneously offering active Xanamem to all eligible participants through the new open-label extension phase. A safe and effective oral therapy capable of slowing disease progression in Alzheimer’s disease is arguably the ‘holy grail’ in the field and we hope that Xanamem’s ability to control brain tissue cortisol levels will lead to clinically significant disease modulation.”
® Xanamem is a registered trademark of Actinogen Medical Limited
1 CDR-SB: Clinical Dementia Rating Scale – Sum of Boxes
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 500 people treated to date and has a promising safety and efficacy profile. ACW’s ongoing clinical trial, XanaMIA, is a phase 2b/3 pivotal trial of 247 participants with mild to moderate Alzheimer’s disease, treated for 36 weeks with Xanamem 10 mg or placebo, followed by an open-label extension phase with active Xanamem for completing participants. The trial is now closed for recruitment and will report topline final results from the randomized phase 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.
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
