Neuronascent, a privately-held neuron regeneration therapeutics company, announced that the U.S. FDA has cleared the Company’s Investigational New Drug (IND) application for its proprietary oral therapeutic, NNI-362, to proceed into Phase 1 clinical testing in a healthy aged population.
The IND package demonstrates that NNI-362 administration reverses age- and disease-related cognitive deficits. This reversal of cognitive deficits by NNI-362 is associated directly with the stimulation of new neuron formation (that is, a reversal of the reduction of new neurons) in aged and Down syndrome-modeled animals, with brain neuron levels returning to levels seen in young and normal animals. Preclinical data for NNI-362 supports a reversal of disease progression and improved cognitive function, effectively returning the brain to a younger state.
The phase 1a trial is a placebo-controlled design and is planned to enroll 56 healthy subjects between 50 and 72 years of age, in single and multiple dose, dose-ascending cohorts. An Early Phase Clinical Unit (EPCU) will be the single clinical site. Safety, tolerability and pharmacokinetic measures will be assessed.
This IND and first-in-human Phase 1a study of NNI-362 is funded by a $2.25 million RO1 grant awarded to Neuronascent by National Institute on Aging (NIA), National Institutes of Health.
About Alzheimer’s disease
Alzheimer’s disease is a progressive disorder that causes brain cells to degenerate and die. It is believed to be caused by a combination of genetic, lifestyle and environmental factors that affect the brain over time. Two proteins are implicated in the progression of Alzheimer’s disease, Beta-amyloid and tau proteins. Beta-amyloid is a leftover fragment of a larger protein. When these fragments cluster together, they appear to have a toxic effect on neurons and to disrupt cell-to-cell communication. These clusters form larger deposits called amyloid plaques, which also include other cellular debris. Tau proteins play a part in a neuron’s internal support and transport system to carry nutrients and other essential materials. In Alzheimer’s disease, tau proteins change shape and organize themselves into structures called neurofibrillary tangles. The tangles disrupt the transport system and are toxic to cells.
NNI-362 is a patented oral therapeutic aimed at reversing age-related disorders by producing new neurons to replace those lost in chronic neurodegenerative disorders and aging. NNI-362 has a unique mechanism of action (MOA) that provides safety and selectivity specifically for neuron regeneration and was discovered through Neuronascent’s proprietary phenotypic screening program using human neural progenitors. This MOA takes advantage of the neural progenitors already in the aged brain to promote new neurons and protect them from degeneration in chronic diseases of the aged, specifically Alzheimer’s disease. New neuron regeneration from neural progenitors is a physiologic process in the brain, yet with aging and neurodegenerative disease there begins a pathological insufficiency in generating new neurons that survive to functionality. Thus, NNI-362 is aimed at reversing age-related neuron loss and restoring cognitive function in AD patients and should also be useful for other disorders of the elderly, including Parkinson’s disease and age-related hearing loss.