Neuron regenerative therapeutic, NNI-362, for first-in-human safety and PK for AD

项目摘要/摘要

Project Summary / Abstract There is only temporary, symptomatic relief for the approximately 5.3 million Americans thought to already suffer from Alzheimer's disease (AD). With a decade of failures for neuroprotective agents, Neuronascent discovered and developed a therapy that would not just stop further neuron loss and dysfunction, but that would promote new neurons that survive to maturation, replacing those lost. This neuron regenerative agent (NNI-362) should then halt or even reverse the memory impairment in Alzheimer's patients. Neuronascent aims to submit an IND to the FDA for NNI-362 for Alzheimer's disease, following completion of the IND application. The NIA supported the FDA-required GLP safety studies showing the agent to also be safe, following a pre-IND meeting with the FDA. Neuronascent, under this granting program, will enlist the help of regulatory contractors to submit an IND application that includes a Clinical plan that assesses this NCE for its safety in a healthy aged population. On obtaining FDA approval to initiate the first-in-human testing of NNI-362 for Alzheimer's disease, Neuronascent aims to determine the safety, tolerability and PK in healthy volunteers aged 50-72. Male and female volunteers will be administered a single dose of NNI-362 orally at one of three ascending doses (SAD) , with and without food. This will be followed by a 14-day daily ascending dose (MAD) test. Plasma levels of NNI-362 will be assessed to determine pharmacokinetics and whether a potential efficacious dose has been reached. If NNI-362 is deemed safe and well-tolerated, the agent could then be tested for ability to promote new neurons in the hippocampus (vMRI) and halt or reverse dementia and impaired function in Alzheimer's patients, in expanded safety and efficacy trials. Neuronascent's novel neuron regenerative therapeutic targets aging, which is the greatest risk-factor for Alzheimer's, and hippocampal neurodegeneration causing volume loss, that is directly correlated with dementia in Alzheimer's disease. [Unchanged from original R01 grant application.]