The multicentre, randomized, double-blind, placebo-controlled, dose-escalation study will assess the safety profile of AT-001, as well as its impact on novel blood-based biomarkers and brain amyloid deposition.

The study authorisation in AD follows the EMA’s authorisation last year to initiate a Phase IIb/III study of AT-001 in HCCAA, an ultra-rare condition marked by amyloid buildup in the brain’s blood vessels that can cause cerebral haemorrhage, stroke and progressive neurological decline, including dementia. In addition, the EMA has granted AT-001 ODD for the treatment of HCCAA, a form of hereditary cerebral amyloid angiopathy (CAA).

“This is a significant milestone and underscores the urgent unmet medical need in HCCAA and provides ATx with an important regulatory milestone to advance AT-001 in this rare and devastating condition,” says CEO and co-founder Ivar Hakonarson.

About the Phase IIa Study

The study will evaluate the safety, tolerability, and biomarker-based efficacy of AT-001, a small molecule oral therapy, in patients aged 50–85 with mild cognitive impairment (MCI) or mild Alzheimer’s disease. The study will be a multicentre, randomized, double-blind, placebo-controlled trial conducted at four sites across Denmark and Iceland in collaboration with Sanos Group, a global multi-niche CRO.

The trial will enrol patients for 12 months of treatment with escalating oral doses of AT-001. Safety labs will be monitored monthly, together with biomarker assessments every three months, MRI at baseline, mid- and end of study, and PET scans at baseline and study completion.