Actinogen Medical Limited (ASX: ACW) has announced that the World Health Organization (WHO) has granted the nonproprietary name ‘emestedastat’ to its once-daily small molecule Xanamem® (UE2343). The designation follows WHO’s Procedure for the Selection of Recommended International Nonproprietary Names (INN) for Pharmaceutical Substances.

An INN is a globally recognized, unique name for pharmaceutical drugs and active ingredients, ensuring clear identification, safe prescription, and regulatory compliance. The newly designated ‘-stedastat’ suffix classifies emestedastat as part of a new drug class of 11β-HSD1 enzyme inhibitors, making it the first in its category as a tissue cortisol synthesis inhibitor. By targeting and blocking the 11β-HSD1 enzyme in the brain, Xanamem aims to regulate cortisol levels, a key factor in neurodegenerative and mood disorders.

Dr. Steven Gourlay, CEO and Managing Director of Actinogen, emphasized the significance of this recognition, stating, "The award of an INN with a unique suffix underscores Actinogen’s leadership in 11β-HSD1 enzyme inhibition. With promising results from phase 2 trials in both Alzheimer’s disease (AD) and major depressive disorder (MDD), we are confident in the potential success of current and future trials.”

Actinogen's XanaMIA Phase 2b/3 trial for Alzheimer’s disease is progressing, with all 25 trial sites in the USA and Australia actively pre-screening participants. Over 300 individuals have undergone a blood pTau test, and approximately 40 patients have entered the 36-week treatment phase. The study is enrolling 220 participants with mild to moderate AD and elevated levels of pTau181, a biomarker linked to disease progression. The primary endpoint is the Clinical Dementia Rating – Sum of Boxes (CDR-SB) scale, alongside assessments of cognition and functional ability. Interim results are anticipated in Q4 2025, with final outcomes expected in late 2026.

As Xanamem moves closer to potential commercialization, Actinogen is actively preparing for market readiness. The company has appointed Andrew Udell as Chief Commercial Officer, is engaging with the FDA for regulatory approvals, and is in discussions with potential co-development partners. Additionally, Actinogen is expanding its patent portfolio, securing data exclusivity protections to safeguard Xanamem’s market position into the 2040s.

With manufacturing scale-up, regulatory meetings, and further clinical validation underway, Actinogen continues to position Xanamem as a first-in-class treatment for neurodegenerative and psychiatric conditions.