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

项目总结/摘要 大约530万美国人认为， 已经患有阿尔茨海默病（AD）。十年来神经保护的失败 神经再生组织发现并开发了一种治疗方法，不仅可以阻止进一步的神经元 但这将促进新的神经元存活成熟，取代 那些失去的。这种神经元再生剂（NNI-362）然后应该停止甚至逆转 老年痴呆症患者的记忆障碍Neuronascent的目标是向FDA提交IND， NNI-362治疗阿尔茨海默病，完成IND申请后。NIA支持 FDA要求的GLP安全性研究表明，在IND前申请后， 与FDA会面。根据这项资助计划，Neuronascent将寻求监管机构的帮助， 承包商提交IND申请，其中包括评估NCE的临床计划， 在健康老年人群中的安全性。在获得FDA批准启动首次人体 测试NNI-362的阿尔茨海默氏病，Neuronascent旨在确定安全性，耐受性， 50-72岁健康志愿者中的PK。男性和女性志愿者将接受 NNI-362单次口服给药，三种递增剂量之一（SAD），伴或不伴食物。这 随后将进行为期14天的每日剂量递增（MAD）试验。NNI-362的血浆水平将 评估以确定药代动力学以及是否已经 达到了如果NNI-362被认为是安全的和耐受性良好的，那么该代理商可以测试能力 促进海马体中的新神经元（vMRI），并停止或逆转痴呆和受损 在阿尔茨海默病患者中的功能，在扩大的安全性和有效性试验中。Neuronascent的小说 神经元再生疗法的目标是衰老，这是阿尔茨海默氏症的最大风险因素， 和海马神经变性导致体积损失，这是直接相关的， 老年痴呆症 [原R 01补助金申请不变。]

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.]