The European Medicines Agency (EMA) has granted approval to Iceland-based Arctic Therapeutics to commence a Phase IIa trial of small-molecule oral therapy, AT-001, targeting patients with Alzheimer’s disease.
The company’s lead investigational therapy, AT-001 is being developed for use in both rare and prevalent forms of dementia.
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The randomised, double-blind, dose-escalation, multi-centre, placebo-controlled trial will investigate the therapy in individuals aged between 50 and 85 years diagnosed with mild cognitive impairment (MCI) or mild Alzheimer’s disease.
It will be carried out at four sites across Iceland and Denmark, in partnership with contract research organisation (CRO) Sanos Group.
Subjects will be enrolled for a 12-month treatment time and receive increasing oral doses of the therapy.
Monthly safety laboratory checks will be conducted along with biomarker evaluations every three months.
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By GlobalDataMagnetic resonance imaging (MRI) scans will be performed at baseline, midway through the trial, and at completion, while positron emission tomography (PET) scans will take place at both the start and end of the trial period.
The study is centred on evaluating the safety of AT-001 and its efficacy based on biomarkers.
Among the biomarkers being assessed are plasma pTau217 and total Tau, which will be measured using Quanterix Simoa technology to monitor early tau pathology and neuronal injury.
The trial will also analyse neurofilament light chain (NfL) as an indicator of axonal damage and neurodegeneration, as well as toxic amyloid-β oligomers, which will be detected with the SOBA assay to identify synaptotoxic species associated with memory loss.
The imaging techniques that will be employed are MRI scans for assessing brain atrophy and white matter integrity, while PET scans will visualise amyloid plaque burden and help determine whether AT-001 can reduce these deposits.
At the conclusion of the study, Arctic Therapeutics plans to analyse biomarker data and brain amyloid outcomes alongside any clinical improvements noted among participants, especially in comparison with outcomes from recent monoclonal antibody therapies aimed at reducing amyloid accumulation.
This latest authorisation follows the EMA’s previous approval for the company to conduct a Phase IIb/III trial of AT-001 for hereditary cystatin C amyloid angiopathy (HCCAA).
Arctic Therapeutics founder Dr Hakon Hakonarson said: “Through our clinical trials targeting both rare and common forms of dementia, we are pushing the science forward with the goal of not only delaying progression, but ultimately preventing these diseases altogether.”
