The search for an effective treatment for neurological conditions like Alzheimer’s disease remains one of the most urgent challenges in global healthcare. In 2025, Australian biotech company Actinogen Medical Ltd (ASX:ACW) is making significant strides in tackling this issue with its groundbreaking therapy, Xanamem.

Actinogen has entered the new year with a major milestone: The World Health Organisation (WHO) granted Xanamem a medical, non-proprietary name – “emestedastat” – cementing its status as a novel treatment candidate.

This key recognition not only validates the drug’s potential but also marks it as the first in a new class of medications designed to regulate brain cortisol levels; a critical factor in Alzheimer’s progression.

The WHO’s designation is a pivotal step toward the commercialisation of Xanamem/emestedastat. International Non-proprietary Names (INNs) are essential for the global identification and safe prescription of pharmaceuticals, allowing widespread adoption in scientific literature, labelling, and regulatory approvals. 

With the suffix ‘stedastat,’ the WHO acknowledges emestedastat as part of a new class of drugs – enzyme inhibitors of 11β-HSD1 – that target elevated cortisol levels in the brain. It’s this distinction that sets Xanamem apart as a first-in-class ‘tissue cortisol synthesis inhibitor,’ a groundbreaking approach to Alzheimer’s treatment.

“This recognition highlights Actinogen’s leadership in 11β-HSD1 enzyme inhibition, a mechanism designed to control brain cortisol and deliver clinically meaningful benefits for patients with Alzheimer’s disease and major depressive disorder,” Actinogen’s CEO and managing director Dr. Steven Gourlay, said.

“Based on positive outcomes in Phase 2 trials with the 10mg daily dose of Xanamem in both Alzheimer’s and major depression, we have strong confidence in the drug’s potential to drive meaningful progress in future trials.”

Extensive research has linked elevated cortisol levels in the brain to an increased risk of Alzheimer’s and early memory loss. The enzyme 11β-HSD1, responsible for producing cortisol in brain tissue, is found at high levels in key regions associated with cognitive impairment, including the hippocampus, frontal cortex, and cerebellum. 

By inhibiting this enzyme, Xanamem directly targets these brain regions, potentially slowing disease progression. The drug has been evaluated for safety and efficacy in eight clinical trials, involving approximately 400 volunteers and patients, showing promising effects in biomarker-positive Alzheimer’s patients and those with moderate depression. 

Actinogen’s XanaMIA Phase 2b/3 trial is now underway, enrolling 220 participants in Australia and the US. These patients, diagnosed with mild to moderate Alzheimer’s and elevated levels of the pTau181 biomarker (a predictor of disease progression), will receive either a 10mg daily dose of Xanamem or a placebo. 

The trial’s primary endpoint is the Clinical Dementia Rating Scale – Sum of Boxes (CDR-SB), an internationally recognized standard for assessing Alzheimer’s progression. Initial results from an interim analysis of the first 100 participants after 24 weeks of treatment are expected in Q4 2025, with final results anticipated in the second half of 2026. 

So far, 25 trial sites have begun pre-screening potential participants, with approximately 400 individuals tested for the pTau biomarker.

More than 40 patients have already entered the 36-week treatment phase, and an additional 20 are in the final stages of screening.

Adding to this momentum, a recently published peer-reviewed paper in Clinical Pharmacology in Drug Development confirmed a 10mg daily dose of Xanamem is optimal for current and future trials. The study validated the use of advanced pharmacodynamic measures – including PET imaging and cognitive testing – to determine the ideal dosage for maximum clinical efficacy.

Beyond clinical trials, Actinogen is preparing for the commercialisation of Xanamem. In a strategic move, the company appointed U.S.-based Andrew Udell as chief commercial officer, reinforcing its market expansion efforts.

Key regulatory discussions are also on the horizon. Actinogen is engaging with the Food and Drug Administration (FDA) to define marketing approval criteria for Alzheimer’s and major depressive disorder. And, the company is in talks with potential co-development partners to accelerate the drug’s path to market.

To safeguard its innovation, Actinogen is strengthening its intellectual property portfolio. Data exclusivity laws in all major markets provide robust protection against generic competition, while newly granted patents ensure long-term market positioning for Xanamem. 

Meanwhile, the company is ramping up drug substance manufacturing and preparing additional peer-reviewed publications to further validate its scientific findings. In parallel with the XanaMIA trial, Actinogen is conducting ancillary studies to support late-stage clinical development. 

With its first-in-class status, strong clinical trial momentum, and strategic commercialisation efforts, Actinogen is positioned for a pivotal year in 2025. If successful, Xanamem/emestedastat could reshape the treatment landscape for Alzheimer’s and major depressive disorder, offering hope to millions worldwide.

Actinogen shares opened at 3.2 cents each through Wednesday morning.

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