Actinogen Medical Ltd (ASX:ACW, OTC:ATGGF) has outlined the scientific rationale supporting its lead drug candidate Xanamem® (emestedastat) as a potential treatment for Alzheimer’s disease, with results from the company’s first pivotal clinical trial expected in November 2026.
The company presented an overview of Xanamem and its development program in a 12-minute podium presentation titled Advancing Oral Xanamem in Alzheimer’s Disease at the annual Bioshares Biotech Summit in Queenstown, New Zealand.
Xanamem is an oral small-molecule inhibitor designed to control elevated levels of cortisol, commonly known as the stress hormone, in the brain.
The drug targets the enzyme 11β-hydroxysteroid dehydrogenase type 1, or 11β-HSD1, which converts inactive cortisone into active cortisol within brain tissue.
Cortisol linked to cognitive impairment
Actinogen said there was a substantial body of human evidence supporting the role of excessive cortisol in cognitive decline and Alzheimer’s disease.
Exposure to therapeutic corticosteroids and conditions associated with excess cortisol, such as Cushing’s syndrome, have been linked to impaired memory, learning and problem-solving abilities, as well as psychiatric symptoms and reductions in brain tissue volume.
Elevated cortisol levels in cerebrospinal fluid have also been associated with Alzheimer’s disease and with the ApoE4 gene variant, the largest known genetic risk factor for developing the condition.
The company also highlighted genetic evidence involving 11β-HSD1. Increased expression of the enzyme has been linked to cognitive decline, while the absence of 11β-HSD1 expression appears to have a protective effect.
By inhibiting the enzyme responsible for producing cortisol in the brain, Xanamem is intended to reduce the adverse effects of excessive cortisol on cognitive function.
Previous studies show preliminary activity
Xanamem has previously demonstrated preliminary clinical activity in patients with mild Alzheimer’s disease.
The drug has also produced positive effects on measures of attention and working memory in studies involving cognitively healthy older volunteers.
Actinogen is now evaluating Xanamem in a pivotal Alzheimer’s disease trial involving 247 participants.
The company expects to report final topline results from the study in November 2026.
The trial represents a key milestone for Actinogen and is designed to assess whether controlling cortisol production in the brain can slow cognitive decline and improve outcomes for people with Alzheimer’s disease.