Phase 2 Trial Planned for Xanamem, Oral Treatment for Fragile X

Actinogen Medical announced plans to open a Phase 2 trial of its experimental oral therapy Xanamem in boys with fragile X syndrome early next year.

The study, to be called XanaFX and take place in Australia, will be the first to evaluate whether targeting the stress hormone cortisol alleviates anxiety, sleep, and behavioral symptoms in people with fragile X.

“We are delighted to embark on this significant new trial program and particularly excited to announce Fragile X syndrome as a new indication for Xanamem,” Bill Ketelbey, Actinogen’s CEO and managing director, said in a press release.

XanaFX will be a double-blind and placebo-controlled study in about 40 boys with fragile X, ages 12-18, and assess Xanamem’s efficacy and safety at two different doses. Treatment effectiveness will be measured by changes in levels of anxiety, sleep patterns, and behavioral issues.

The study will be conducted at two sites in Melbourne — the Murdoch Children’s Research Institute and Royal Children’s Hospital. XanaFX is expected to open by June, and results to be available within one year of its start. 

Xanamem acts by blocking the cortisol-activating enzyme 11beta-HSD1, which is highly expressed in brain regions associated with cognition and memory. Elevated cortisol can cause profound alterations to the brain that affect cognition and behavior. By blocking 11beta-HSD1, Xanamem should inhibit cortisol production.

A Phase 1 study called XanaHES (NCT03830762), completed earlier this year, tested the safety and tolerability of Xanamem in 42 healthy, older adults (ages 50 to 75). A once-daily dose of Xanamem at 20 mg was found to have no serious side effects and to significantly benefit cognition compared with a placebo, the company reported.

These data and other findings, Actinogen stated, support the use of Xanamem in treating cognitive impairment associated with elevated cortisol.

Actinogen also announced plans for a Phase 2 safety and efficacy trial, named XanaMIA, of Xanamem treating mild cognitive impairments in people with very early stage Alzheimer’s disease. 

“We look forward to initiating the planned studies next year and building on the significant progress made to date with the development of Xanamem,” Ketelbey added.

Actinogen, which is based in Australia, is undertaking a number of initiatives to raise capital to support continued development of Xanamem, its lead treatment compound.