Positive phase 2a clinical data from an Alzheimer’s disease (AD) biomarker study, has been announced today (October 10). Actinogen Medical Limited shared the news that its study validates its planned program for Xanamem, a potential drug for the disease.
The biomarker study was conducted in 72 patients with available blood biomarker samples from the prior phase 2a placebo-controlled XanADu study of 185 patients.
Patients had a clinical diagnosis of mild AD, with a mini mental state examination (MMSE) score of 20 to 26 and were treated with Xanamem 10 mg or placebo once daily for 12 weeks. The trial was conducted in the U.S., U.K. and Australia. The average age of patients was 71 years, 57% were female and baseline mean CDR-SB score was 3.9.
Clinical dementia rating scale – sum of boxes effect (CDR-SB – a clinical endpoint used as a primary measure) of 0.6 to 0.8 points was observed in patients with elevated pTau biomarker with once daily, 10 mg, oral therapy.
Eminent authority on dementia, Michael Woodward, said: “The positive data for CDR-SB and other endpoints are encouraging and indicate a likely therapeutic effect of Xanamem in patients with the early stages of AD.
“The use of pTau blood levels to confirm the diagnosis of AD in future trials represents a practical and efficient method to select patients at risk of disease progression and in whom a treatment effect is more likely to be observed.”
The goals of the study were to measure Xanamem effects in patients with biomarker-positive AD by excluding the ‘noise’ from patients with other types of dementia who were unlikely to progress during the trial, and establish if there was any short-term effect of Xanamem on the levels of blood biomarkers themselves.
A short-term effect of Xanamem on protein biomarkers was considered unlikely because its mechanism is not via direct action on amyloid and tau proteins in the brain.
Twice as many patients (56%) in the Xanamem group were stable or improved compared with placebo and CDR-SB will be a primary endpoint in the upcoming six-month phase 2b trial.
It was found that the regulatory path to approval in AD is clear and uncontroversial using CDR-SB and that findings significantly de-risk and improve AD program efficiency.
Paul Rolan, Actinogen’s chief medical officer, said: “These clinical results provide further validation of our Alzheimer’s Disease program and are a significant step forward in the development of Xanamem as a new treatment for Alzheimer’s Disease with a novel, amyloid-independent mechanism of action.
“We are very pleased to see such positive clinical data for patients with biomarker-positive, mild Alzheimer’s Disease. The results extend findings of therapeutic effects on cognition in two prior trials of cognitively normal, older volunteers to patients with early Alzheimer’s Disease. The data also validate the dose range planned for our upcoming trials in Alzheimer’s Disease and Depression.”
The analysis was double-blind and pre-specified to avoid bias in the results.