Alzheimer's disease (AD) is characterized by progressively worsening dementia eventually leading to death. It affects over five million people in the United States and costs the healthcare system over $200 billion per year. Currently there is no available therapy for slowing, reversing or preventing the disease. There is strong evidence suggesting that AD starts with the accumulation of the 4.5 kDa peptide, amyloid beta (A?), in the brain leading to concentration-dependent A? self-assembly into neurotoxic oligomers. A? accumulation in sporadic AD is related to a decreased net clearance rate of A? from the brain rather than A? overproduction. Despite the strong correlation between A? accumulation and AD progression, most amyloid-targeted therapeutic approaches have failed to demonstrate significant clinical benefits. However, most A?-targeted therapies have focused on removing specific forms of A? (e.g. plaques or oligomers), inhibiting A? production, or shifting the equilibrium distribution of A? between the brain and the periphery. Few, if any, attempts have been made to enhance A? brain clearance rates by sustainably increasing the irreversible systemic clearance and degradation of A?. Akston proposes to address this critical need by developing therapeutic candidates that specifically bind and remove A? from the body at a high enough rate to increase the net clearance of A? from the brain into the periphery and reduce the overall steady state amyloid burden throughout the body. The company will leverage its key scientists'extensive experience in chemical modification of biomolecules for targeted biodistribution. Therapeutic candidates that are chemically-engineered with various targeting moieties will be infused intravenously in aged beagle dogs to select a candidate with the highest potential to reduce peripheral A? levels. The main goal of this one-year Phase 1 feasibility study is to demonstrate that this candidate, when infused intravenously for two weeks in aged beagle dogs, can significantly reduce brain and CSF A? levels. This candidate could then be evaluated in large-animal and human clinical testing to determine if increased systemic A clearance can reverse or stall the degradation in cognitive function associated with AD.

Public Health Relevance

Alzheimer's disease (AD), which is characterized by progressively worsening dementia eventually leading to death and a buildup of amyloid beta in the brain, currently affects over five million people in the United States and costs the healthcare system over $200 billion per year. If left untreated, by 2050 it is estimated that 15 million peopl in the US will be afflicted with the disease leading to over $1 trillion in annual costs to the national healthcare system. To address this critical need, Akston Biosciences Corporation is developing a technology platform capable of rapidly and irreversibly clearing A from the periphery, resulting in lower amyloid burden in the brain, and thereby potentially halting or reversing the progression of AD.

National Institute of Health (NIH)
National Institute on Aging (NIA)
Small Business Innovation Research Grants (SBIR) - Phase I (R43)
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Special Emphasis Panel (ZRG1-ETTN-M (11))
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Refolo, Lorenzo
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Akston Biosciences Corporation
United States
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