Actinogen receives positive EMA scientific advice aligned with prior FDA guidance for its Alzheimer’s disease program

XanaMIA randomized, pivotal trial of oral Xanamem 10 mg fully enrolled and remains on track for topline final results in November 2026

SYDNEY, May 28, 2026 (GLOBE NEWSWIRE) -- Actinogen Medical Limited (ASX: ACW) today announces the successful outcome of its scheduled scientific advice meeting (written response) on Alzheimer’s disease (AD) with the European Medicines Agency (EMA).

Actinogen and the EMA reached a common understanding of the pathway to marketing approval for AD in the European Union (EU). This includes agreements related to regulatory starting materials used in drug substance synthesis, design considerations for one additional pivotal clinical trial and the limited number of ancillary clinical pharmacology trials and nonclinical studies required.

Key understandings include:

1. Agreement on the suitability of the ‘Regulatory starting materials’ for the commercial manufacturing of Xanamem^® (emestedastat) drug substance
2. Design of one additional, well-controlled, pivotal (phase 3) trial to follow a positive XanaMIA pivotal trial – both use a 10 mg dose vs. placebo
3. Standard total number of people to be treated with Xanamem to be described in the Marketing Authorization Application – that is, the proposed makeup of the planned safety database consistent with EMA and FDA guidelines
4. Small number of ancillary clinical pharmacology trials to be conducted parallel with the next pivotal trial
5. Small number of nonclinical studies required to further characterize safety, metabolism and excretion pathways to be conducted parallel with the next pivotal trial
   
   

The outcome reached at this meeting aligns closely with the guidance received at an earlier meeting with the FDA’s Neurology-I Division in September 2025. With the new EMA advice, Actinogen now has a clear pathway and guidance towards marketing approvals in two key pharmaceutical markets. Approvals in the US and EU also contribute to approvals in many other countries and regions. The advice from both the FDA and EMA provides regulatory clarity for ongoing discussions with potential development and marketing partners.

Dr Steven Gourlay, the Company’s CEO said:

“We are pleased with the clear guidance from the EMA that closely conforms with our plans for streamlined development of Xanamem in Alzheimer’s disease, as agreed previously with the FDA. Importantly, the EMA agreed with our approach for only one additional, pivotal trial using a single 10 mg Xanamem dose vs. placebo to support a marketing application for Alzheimer’s in the EU.”

Topline results from the randomized phase of the XanaMIA pivotal Alzheimer’s disease trial of Xanamem 10 mg vs. placebo are expected in November this year. The trial enrolled 247 participants in the US and Australia with mild to moderate Alzheimer’s disease and elevated levels of plasma pTau181. Earlier this year the Independent Data Monitoring Committee (DMC) recommended the trial continue unchanged after a confidential assessment of unblinded safety and efficacy futility data.

Dr Dana Hilt, the Company’s CMO said:

“With positive feedback from both the FDA and EMA on our Xanamem program and a positive DMC interim analysis, we look forward to potentially groundbreaking XanaMIA trial results in November. Such results would provide important evidence that control of brain cortisol can safely slow the progression of Alzheimer’s disease. Our second pivotal trial in patients with mild to moderate Alzheimer’s disease would then commence mid next year in multiple regions.”

All XanaMIA participants are now potentially eligible to extend their treatment by participation in the Open Label Extension (OLE) phase. The OLE will provide valuable data on longer-term safety and efficacy trajectories.

The XanaMIA trial is intended to serve as one of two pivotal trials supporting the earliest possible marketing approvals for Xanamem in multiple regions. If the trial results are strongly positive in November, the Company will also explore expedited approval pathways with relevant regulators while commencing the new pivotal phase 3 trial. The FDA recently announced a new policy supporting approval based on a single pivotal trial with adequate supporting evidence.

^® 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 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 10 mg for completing participants. In January 2026 an Independent DMC conducted an interim analysis of safety and efficacy futility and recommended the trial proceed to completion. The trial is now fully enrolled 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 

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