Actinogen Secures FDA Alignment on Xanamem’s Path to Alzheimer’s Approval.
- Noel Ong
- Sep 24
- 5 min read
Announcement
Actinogen Medical Limited (ASX: ACW) has announced a significant milestone in the development of Xanamem®, its lead therapy for Alzheimer’s disease (AD). Following a successful Type C meeting with the US Food and Drug Administration (FDA), the Company now has clear guidance on the remaining clinical, manufacturing, and regulatory requirements for a New Drug Application (NDA) submission in the US. This outcome brings Actinogen a step closer to offering a novel, brain-targeted therapy aimed at slowing the progression of AD — a condition with few effective treatment options.
Key Highlights
FDA Agreement on Development Pathway: Actinogen and the FDA agreed on the key regulatory starting materials for drug substance synthesis, clinical trial design, and the limited number of remaining pharmacology and nonclinical studies required for NDA submission.
Streamlined Clinical Development: The FDA confirmed support for a single additional pivotal Phase 3 trial using a 10 mg once-daily Xanamem dose versus placebo, alongside interim analysis from the ongoing XanaMIA trial.
Global Regulatory Strategy: Similar engagement is planned with the European Medicines Agency (EMA) in 2026, followed by the UK MHRA and other global regulators, aligning the development program across major markets.
Safety and Efficacy Profile: Xanamem has now been tested in around 400 volunteers and patients across eight clinical trials, showing a promising safety profile and clinically meaningful effects on cognition and depression.
Upcoming Milestones: Interim analysis results from XanaMIA are expected in January 2026, with final results due by Q4 2026 — key catalysts for investors tracking ACW’s progress.
Summary of Xanamem’s Alzheimer’s disease program timeline:
• H2 2025 – FDA meeting to review planned program for US regulatory approval
• Q4 2025 or January 2026 – Full enrolment of 220 XanaMIA phase 2b/3 trial participants
• January 2026 – Independent DMC review of safety and efficacy data for XanaMIA
• Q1 2026 – Open-label extension trial enrols the first patient following on from XanaMIA
• Q4 2026 – Final XanaMIA results announced
• Q1 2027 – Commencement of second pivotal, phase 3 trial.
Clinical Development Program
XanaMIA Phase 2b/3 Alzheimer’s Disease Trial.
Design: Double-blind, placebo-controlled, parallel group trial with 220 patients (mild to moderate AD with elevated pTau181 biomarker).
Treatment: 10 mg Xanamem daily for 36 weeks.
Primary Endpoint: Clinical Dementia Rating – Sum of Boxes (CDR-SB).
Geography: Conducted across Australia and the US.
Results: Interim readout due January 2026, final results Q4 2026.
XanaMIA-DUR Extension Study.
Design: Open-label extension (up to 24 months) for all XanaMIA participants.
Focus: Safety and long-term efficacy endpoints such as CDR-SB progression.
Start Date: Q1 2026.
XanaCIDD Depression Trial.
Completed Phase 2a in 2024 with statistically significant improvements in depressive symptoms measured by MADRS and PGI-S. Cognitive benefits were seen in both arms, supporting Xanamem’s mechanism of action in mood regulation.
Dr Steven Gourlay, the Company’s CEO and MD, commented:
“We are pleased with the clear guidance from the FDA that confirms our plans for streamlined development of Xanamem in Alzheimer’s disease. Importantly, the Agency agreed with our approach for only one additional, pivotal trial using a single 10 mg Xanamem dose design vs. placebo to support a marketing application for Alzheimer’s in the US.”
About Xanamem® (Emestedastat)
Xanamem is a first-in-class oral therapy that selectively inhibits the 11β-HSD1 enzyme to reduce cortisol inside brain cells, aiming to protect neurons and slow disease progression. Chronic elevation of cortisol is strongly associated with AD progression and cognitive decline. High brain target engagement has been shown even at low doses (5 mg), with a well-tolerated safety profile.
Samso Concluding Comments
Actinogen’s FDA alignment marks a critical de-risking moment for the Xanamem program. Regulatory clarity often becomes the difference between hesitation and conviction for investors following clinical-stage biotech companies. With a single pivotal trial now endorsed by the FDA, the development path is shorter and more focused than many would have expected.
For those following the ASX biotech sector, this is a prime example of how regulatory progress can materially change the risk-reward profile of a company. The January 2026 interim readout now takes on heightened importance, as a positive signal could bring forward partnering discussions and accelerate value recognition.
It’s also worth noting that Actinogen continues to explore additional indications, including depression and potentially Fragile X Syndrome, which broadens the commercial potential of Xanamem. These parallel development tracks could make ACW a more attractive partner for larger pharmaceutical companies seeking differentiated CNS assets.
As always, investors should watch for the next catalysts: the interim data, EMA and MHRA interactions, and the initiation of the pivotal Phase 3 trial. Each milestone represents another validation step in bringing this novel therapy closer to market.
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