Actinogen Medical (ASX: ACW) has received a positive recommendation from its independent Data Monitoring Committee (DMC) to continue the XanaMIA pivotal Alzheimer’s disease trial without amendment following an interim analysis. The DMC reviewed unblinded safety and efficacy data and determined the trial should proceed to completion, preserving its statistical power and validating the trial’s integrity ahead of topline Actinogen XanaMIA trial results expected in November 2026.
The committee assessed approximately 37% of the expected final dataset, including data from 136 participants with one or more efficacy datapoints and 52 participants who completed the full 36 weeks of treatment. Enrolment of the final 247th participant occurred in December last year. This positive interim recommendation represents a significant de-risking event for investors, as the DMC reviewed actual efficacy data and found no reason to halt or modify the trial design.
The recommendation to continue without amendment signals that no safety concerns or futility signals were detected in the unblinded data. All Actinogen staff and XanaMIA trial personnel remain blinded to participant treatment assignments, maintaining the integrity of the double-blind study design.
What is a Data Monitoring Committee and why does this matter?
A Data Monitoring Committee is an independent, external group of clinical trial experts who have access to unblinded data that trial staff and company personnel cannot see. This separation ensures objective oversight of trial safety and efficacy throughout the study period. The DMC’s role is to protect participant welfare and assess whether a trial should continue as planned, be modified, or be stopped early due to safety concerns or futility.
For the XanaMIA trial, the DMC is chaired by Dr Hans Moebius, a highly experienced Alzheimer’s disease leader and trials expert. When a DMC recommends continuing “without amendment,” it means the committee found no safety issues requiring intervention and no futility signal suggesting the trial is unlikely to meet its endpoints. This represents a critical gatekeeping function in clinical development.
The practical significance for retail investors is substantial. DMCs can halt trials for safety reasons or recommend stopping for futility if early data suggests the treatment is unlikely to work. A positive continuation recommendation after reviewing real efficacy data provides meaningful validation that the trial remains on track. While not a guarantee of ultimate success, it reduces binary risk ahead of the final readout.
Key trial milestones and timeline to November results
The XanaMIA trial is progressing through its final stages, with several important milestones scheduled before topline Actinogen XanaMIA trial results are announced. The last participant’s final evaluation visit is expected in September this year, with topline data release following in November. All trial participants are potentially eligible to enrol in an open-label extension phase opening in March, where everyone will receive active Xanamem 10 mg once daily.
| Milestone | Expected Timing |
|---|---|
| Open-label extension enrolment opens | March 2026 |
| Last participant final evaluation | September 2026 |
| Topline results announcement | November 2026 |
| Scientific meeting presentation | Post-November 2026 |
| Peer-reviewed publication | Post-November 2026 |
The open-label extension provides an opportunity for long-term safety and efficacy data collection, with the phase expected to run for approximately two years. This extended data will be valuable for regulatory submissions and commercial positioning.
Xanamem’s novel approach to slowing Alzheimer’s progression
Xanamem represents a first-in-class therapeutic approach targeting elevated cortisol levels in the brain through inhibition of the 11β-HSD1 enzyme. Unlike existing Alzheimer’s treatments such as anti-amyloid antibodies that require regular infusions, Xanamem is an oral tablet taken once daily. The therapy works by controlling cortisol specifically in brain regions critical for memory and cognition, including the hippocampus and frontal cortex, without affecting the body’s normal cortisol production by the adrenal glands.
Chronically elevated cortisol is associated with progression in Alzheimer’s disease and is known to be toxic to brain cells. By targeting this mechanism, Xanamem aims to slow or halt disease progression through a pathway distinct from current approved therapies. The oral, once-daily dosing format offers potential convenience advantages for patients and healthcare systems compared to injectable treatments requiring clinic visits.
Actinogen has studied Xanamem in approximately 400 volunteers and patients across eight clinical trials, establishing a promising safety profile. The drug has demonstrated clinical activity in patients with biomarker-positive Alzheimer’s disease, patients with depression, and cognitively normal volunteers. Key features of Xanamem’s profile include:
- First-in-class mechanism targeting brain cortisol rather than amyloid plaques
- Oral tablet administered once daily, eliminating need for infusions
- Targeted delivery to hippocampus and frontal cortex where memory and cognition are processed
- Safety profile established across multiple clinical trials and patient populations
For investors, the oral, disease-modifying approach positions Xanamem as a potential competitor or complement to injectable anti-amyloid therapies. If successful, the convenience factor and different mechanism of action could support market differentiation and accessibility advantages.
Regulatory pathway agreed with FDA
Actinogen has secured regulatory alignment with the US Food and Drug Administration (FDA) through a Type C meeting. The agreed development plan establishes a clear pathway forward, including a second pivotal trial commencing in 2027 in multiple countries including Australia. This regulatory clarity reduces development uncertainty and provides visibility on the steps required for potential approval.
The agreed programme includes additional open-label studies and clinical pharmacology trials beyond the second pivotal study. According to company guidance, this represents a relatively streamlined path to approval in the US market. The multi-country approach for the second pivotal trial should accelerate recruitment and provide geographic diversity in the patient population.
FDA alignment at this stage is significant for investors, as it confirms the regulatory agency has reviewed the XanaMIA trial design and agreed on requirements for a New Drug Application. This removes a layer of regulatory risk that often affects biotech development programmes.
What’s next for Actinogen and XanaMIA investors
The positive DMC recommendation strengthens the investment thesis for Actinogen (ASX: ACW) ahead of the pivotal November 2026 catalyst. The committee’s access to real, unblinded efficacy data and subsequent recommendation to continue without modification provides meaningful validation that the trial is tracking appropriately. While not definitive proof of success, it represents a significant de-risking milestone in the binary nature of clinical development.
The open-label extension commencing in March offers an additional data stream, with all 247 participants eligible to receive active treatment for up to 25 months. This long-term exposure data will support regulatory submissions and provide evidence of sustained safety and efficacy beyond the initial 36-week treatment period.
CEO Commentary
“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 topline results for the full dataset of 247 participants.”
— Dr Steven Gourlay, CEO & Managing Director
Looking ahead, November 2026 remains the critical catalyst for Actinogen shareholders. The topline results will determine whether Xanamem demonstrates statistically significant slowing of Alzheimer’s disease progression on the primary endpoint, the Clinical Dementia Rating scale – Sum of Boxes (CDR-SB). With FDA alignment secured and the second pivotal trial planned for 2027, the company has established a clear regulatory pathway contingent on positive phase 2b/3 data.
The DMC’s positive interim assessment provides investors with increased confidence that the trial will reach its planned conclusion with data integrity intact, setting the stage for what could be a transformative readout in the Alzheimer’s disease treatment landscape.
Ready to Learn More About Actinogen’s Alzheimer’s Treatment Programme?
The positive DMC recommendation following their review of unblinded efficacy data represents a significant de-risking event ahead of the November 2026 topline results catalyst. With FDA alignment secured and a clear regulatory pathway established, Actinogen’s oral, once-daily Xanamem could potentially transform Alzheimer’s disease treatment if the pivotal trial demonstrates statistical significance.
To explore Actinogen’s clinical development strategy and regulatory milestones in detail, visit the Actinogen investor centre. Track the company’s progress through the final stages of the XanaMIA trial and understand the commercial opportunity for this first-in-class therapeutic approach targeting brain cortisol.