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Arctic Therapeutics Receives EMA Approval for Phase IIa Study of AT-001 in Alzheimer’s Disease

Arctic Therapeutics (ATx), a burgeoning biopharmaceutical firm based in Iceland, has made significant strides in the fight against dementia with its latest announcement regarding its lead investigational therapy, AT-001. Recently, the European Medicines Agency (EMA) authorized the initiation of a Phase IIa clinical trial aimed at evaluating the safety and biometric efficacy of AT-001 in patients suffering from mild cognitive impairment (MCI) or mild dementia attributed to Alzheimer’s disease (AD). This milestone comes at a crucial time as Alzheimer's disease represents one of the major health challenges worldwide.

In the Phase IIa study, ATx plans to conduct a rigorous, multicentre, randomized, double-blind, placebo-controlled trial at several sites across Denmark and Iceland, collaborating with Sanos Group, a renowned global multi-niche Contract Research Organization (CRO). This trial's design will allow the team to assess the treatment's safety profile as well as observe its effects on innovative blood-based biomarkers and brain amyloid deposition levels.

Dr. Hakon Hakonarson, the founder of ATx, highlighted the urgency of addressing Alzheimer’s disease by stating, "Alzheimer's disease is one of the world's most urgent healthcare challenges. Advances in early diagnosis, combined with treatments that enable earlier intervention, hold the potential to transform how we approach Alzheimer's, as well as other forms of dementia." He emphasized that through their clinical trials, ATx aims to not only delay the progression of dementia but to work towards its prevention altogether.

The recent EMA authorization is particularly important, as it follows last year’s endorsement from the agency for a Phase IIb/III study of AT-001 targeting hereditary cystatin C amyloid angiopathy (HCCAA), an ultrarare form of familial dementia. HCCAA is characterized by amyloid accumulation within blood vessels in the brain, leading to severe complications like cerebral hemorrhage, strokes, and the deterioration of neurological functions, including dementia. Furthermore, AT-001 has been granted Orphan Drug Designation (ODD) for treating HCCAA, underscoring its potential applicability in a range of dementia-related conditions.

"The ODD status confirms the groundbreaking nature of AT-001 as a potentially disease-modifying treatment for HCCAA and underpins our expansion into other forms of dementia, including Alzheimer's," remarked Ivar Hakonarson, the CEO and co-founder of ATx. This emphasizes both the innovative approach of the company and the pressing medical needs being addressed within the sphere of dementia.

About the Phase IIa Study

The Phase IIa study is set to enroll patients aged between 50 to 85 with confirmed mild cognitive impairment or mild Alzheimer's disease. Over a year, participants will receive escalating oral doses of AT-001, with their safety and health monitored regularly. Monthly safety labs and biomarker assessments will be executed every three months, alongside MRI scans at the start, halfway point, and end of the study, and PET scans at both baseline and completion.

Among the selected biomarkers that will be monitored are plasma pTau217 and total Tau, which will be measured using Quanterix Simoa® technology to track early indicators of tau-related neurodegeneration, as well as Neurofilament Light Chain (NfL), known for indicating axonal injury. Moreover, the levels of toxic amyloid-β oligomers will be assessed using the newly developed SOBA assay, intended to identify the synaptotoxic agents responsible for memory deterioration.

The study’s imaging endpoints will include MRI evaluations aimed at assessing the degree of brain atrophy and the health of white matter. PET scans will be utilized to visualize amyloid plaque accumulation, a critical aspect of Alzheimer's pathology, and to identify any potential reductions resulting from treatment with AT-001.

Upon completion of the trial, the analysis of biomarker outcomes within the context of clinical improvements observed will provide valuable insights into the efficacy of monoclonal antibody therapies that target amyloid reduction.

About Arctic Therapeutics

Founded in 2015, Arctic Therapeutics is a pioneering drug discovery and development company that has emerged from the Center for Applied Genomics in the United States. It leverages cutting-edge genomics to develop transformative treatments for some of the most challenging diseases, now holding operations in both Iceland and the US while collaborating across Europe. With AT-001 in the forefront, Arctic Therapeutics continues to push the boundaries of what’s possible in the realm of dementia treatment. For further details, visit their official website at www.arctictherapeutics.com or follow their updates on LinkedIn.