Actinogen Medical’s pivotal XanaMIA trial for Alzheimer’s disease has received a green light from its independent Data Monitoring Committee to continue, reinforcing confidence in the drug’s safety and efficacy profile.
- Independent Data Monitoring Committee recommends continuation of XanaMIA trial
- Interim analysis covers 37% of final dataset with positive safety and efficacy signals
- Final topline results expected November 2026 after full enrolment of 247 participants
- Open-label extension phase to begin March 2026 offering active treatment to participants
- Second larger pivotal trial planned for 2027 following FDA consultation
Positive Interim Analysis Boosts Confidence
Actinogen Medical Limited (ASX, ACW) has announced a significant milestone in its development of Xanamem, a novel oral therapy targeting Alzheimer’s disease. The company’s independent Data Monitoring Committee (DMC) has recommended that the XanaMIA pivotal trial continue without changes following an interim analysis. This endorsement comes after the DMC reviewed unblinded safety and efficacy data from approximately 37% of the trial’s participants, including 136 patients with efficacy data and 52 who completed the full 36-week treatment period.
Such a recommendation is crucial in clinical development, as it suggests that the drug’s safety profile remains acceptable and that early efficacy signals justify completing the trial as planned. Importantly, the trial remains blinded to Actinogen staff and investigators, preserving the integrity of the data.
Trial Progress and Next Steps
The XanaMIA trial enrolled its final participant in December 2025, reaching a total of 247 patients with mild to moderate Alzheimer’s disease confirmed by biomarker criteria. Participants are receiving either 10 mg of Xanamem or placebo daily for 36 weeks. The trial is being conducted across Australia and the United States, with topline results anticipated in November 2026.
Following the positive interim review, Actinogen is set to launch an open-label extension phase in March 2026. This phase will allow all XanaMIA participants to receive active Xanamem treatment for up to 25 months, providing further data on long-term safety and efficacy.
Looking Ahead, Regulatory and Development Plans
Actinogen’s leadership expressed optimism about the trial’s progress. CEO Dr Steven Gourlay highlighted the importance of the DMC’s recommendation as a validation of the trial’s design and execution. Chief Medical Officer Dr Dana Hilt emphasised Xanamem’s potential as a game-changing therapy that targets elevated brain cortisol, a factor implicated in Alzheimer’s progression.
In parallel, Actinogen is preparing for a second, larger pivotal trial scheduled to commence in 2027 across multiple countries, including Australia. This next step follows a Type C meeting with the US Food and Drug Administration (FDA), which agreed on a streamlined path to potential approval. The company also plans additional open-label and clinical pharmacology studies to support regulatory submissions.
Contextualising Xanamem’s Promise
Xanamem works by inhibiting an enzyme involved in cortisol synthesis within the brain, aiming to reduce the toxic effects of elevated cortisol on brain cells. This mechanism is distinct from other Alzheimer’s treatments and addresses a significant unmet need in slowing disease progression. Previous trials, including a phase 2 study in depression, have demonstrated Xanamem’s safety and some clinical benefits, bolstering confidence in its therapeutic potential.
While the interim data is encouraging, the final results later this year will be critical in determining Xanamem’s future. The Alzheimer’s treatment landscape is highly competitive and challenging, but Actinogen’s approach offers a fresh avenue that could complement or improve upon existing therapies.
Bottom Line?
As Actinogen advances toward final data and broader trials, all eyes will be on November’s topline results to gauge Xanamem’s true potential in Alzheimer’s care.
Questions in the middle?
- Will the final trial data confirm the early safety and efficacy signals observed?
- How will the FDA respond to the upcoming larger pivotal trial design and data?
- What impact might Xanamem have on the competitive Alzheimer’s treatment market if approved?