XanaMIA Trial Enters 25-Month Open-Label Extension with Active Xanamem
Actinogen Medical has initiated the open-label extension of its XanaMIA Alzheimer’s trial, offering all participants active Xanamem treatment for up to 25 months to gather long-term safety and efficacy data ahead of final trial results.
- Open-label extension phase started with first participant treated
- All former and current XanaMIA trial participants eligible for active Xanamem
- Up to 25 months of treatment to assess long-term safety and efficacy
- Randomised placebo-controlled phase continues with results due November 2026
- Xanamem targets brain cortisol to potentially slow Alzheimer’s progression
Open-Label Extension Begins
Actinogen Medical (ASX:ACW) has officially commenced the open-label extension (OLE) phase of its pivotal XanaMIA trial for Alzheimer’s disease, marking a significant milestone in the development of its lead compound, Xanamem. The first participant was treated on 31 March 2026, signalling the start of up to 25 months of active treatment for all eligible participants.
This extension phase invites both former and current participants who completed the randomized, placebo-controlled segment of the trial in Australia and the United States to receive Xanamem 10 mg daily. Unlike the earlier phase, the OLE has no placebo group, allowing all participants to benefit from the active therapy while generating valuable long-term safety and observational efficacy data.
Why the Open-Label Phase Matters
The open-label design enables Actinogen to monitor the durability of Xanamem’s effects on Alzheimer’s progression, focusing on key clinical endpoints such as the Clinical Dementia Rating Scale – Sum of Boxes (CDR-SB), cognition, and daily living activities. This data will be instrumental in supporting future regulatory submissions and marketing applications, providing a richer picture of the drug’s safety profile over an extended period.
Dr Dana Hilt, Actinogen’s Chief Medical Officer, emphasised the importance of this phase for participants and the company alike, noting that half of the trial’s initial cohort received placebo and now have the opportunity to access active treatment. She highlighted Xanamem’s potential as a game-changing oral therapy that could slow or halt Alzheimer’s disease progression without the safety concerns linked to anti-amyloid antibody treatments, such as brain swelling (ARIA).
Continuing the Randomised Trial and Broader Context
While the open-label phase unfolds, the original randomized, placebo-controlled trial continues in parallel, with topline results expected in November 2026. This dual approach allows Actinogen to maintain rigorous efficacy evaluation while expanding safety and observational data collection.
Xanamem’s novel mechanism targets elevated cortisol levels in the brain; a stress hormone implicated in cognitive decline and neurodegeneration; by inhibiting the enzyme 11β-HSD1. This approach differentiates it from other therapies and has shown promise in prior trials, including a phase 2a depression study where Xanamem demonstrated significant benefits on depressive symptoms and cognition.
Looking Ahead
With over 500 volunteers and patients having participated in Xanamem trials to date, Actinogen is building a comprehensive clinical profile for this first-in-class therapy. The open-label extension phase is a critical step in validating the long-term safety and potential sustained efficacy of Xanamem, which could address a substantial unmet need in Alzheimer’s treatment.
Investors and observers will be watching closely as data from this phase emerges progressively, alongside the awaited November topline results, to gauge whether Xanamem can fulfil its promise as a safe, effective oral treatment for a devastating disease.
Bottom Line?
The open-label extension sets the stage for deeper insights into Xanamem’s long-term impact, with pivotal trial results looming later this year.
Questions in the middle?
- How will the open-label data compare with placebo-controlled results in demonstrating efficacy?
- What regulatory hurdles remain before Xanamem could reach the market?
- Could Xanamem’s cortisol-targeting mechanism extend to other neuropsychiatric conditions?
