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Athira Pharma Inc.

Leen Kawas, PhD | Washington, United States

Athira Pharma Inc.

Leen Kawas, PhD | Washington, United States

Phase I clinical trials of a novel neurotrophic activator drug to treat Alzheimer's disease

Increasing life expectancy has increased the incidence of Alzheimer's disease (AD) and dementia in industrialized nations. In the course of AD development, a patient experience systematic brain tissue loss before the onset of cognitive impairment. Current treatment options only help manage symptoms rather than treat the underlying cause of AD. There is no treatment capable of changing the course of cognitive decline nor restoring lost brain tissue. Without an effective therapy, the incidence of AD and dementia is expected to triple by 2050, posing an urgent public health challenge. One method to restore the lost cognitive function is to restore the neurons that are lost during AD progression. This can be achieved by selectively activating neurotrophic growth factors, a naturally occurring substance capable of stimulating brain cell survival and healing. One such growth factor with perhaps the greatest potential is hepatocyte growth factor (HGF). When activated, HGF can fight against massive brain tissue loss prior to the onset of cognitive decline in AD. Activation of HGF has been shown to be effective in numerous animal models of neurodegenerative disorders and age related cognitive decline, including AD. To leverage the neuro-protective capacity of HGF, M3 Biotechnology (now called Athira Pharma) has developed a small molecule oral drug, NDX-1017, to treat AD. NDX-1017 is a subcutaneous injection, rapidly converts to its parent active compound NDX -1001 capable of activating HGF in the brain at low concentrations. It has been demonstrated that the parent compound NDX-1001 can restore brain functions in aged dementia animal studies. Our project aims to move NDX-1017 into Phase 1a human clinical trials, to evaluate the safety profile, determine a safe dosage range, and identify side effects in humans. NDX-1017 is being advanced as a first-in-class disease-modifying treatment for AD.