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

项目摘要/摘要 对于大约530万美国人来说，只有暂时的、有症状的缓解 被认为已经患有阿尔茨海默病(AD)的人。在十年的失败之后， 仅有神经保护剂，神经元上升就发现并开发了一种治疗方法 不仅会阻止进一步的神经元丢失和功能障碍，而且会促进新的 存活到成熟的神经元，取代那些丢失的神经元。这种神经再生剂 从而阻止甚至逆转阿尔茨海默氏症患者的记忆损害。 Neuronascent的目标是向FDA提交治疗阿尔茨海默病的NNI-362的IND， 在完成IND申请之后。NIA支持FDA要求的GLP 安全性研究表明，该试剂也是安全的，在IND前与 美国食品和药物管理局。根据这一授予计划，Neuronascent将寻求监管部门的帮助 承包商提交IND申请，其中包括评估这一点的临床计划 NCE因其在健康老年人口中的安全性而获奖。在获得FDA批准后启动 Neuronascent首次对NNI-362进行人类阿尔茨海默病测试，旨在确定 50-72岁健康志愿者的安全性、耐受性和PK。男性和女性 志愿者将在三个提升阶段中的一个阶段口服单剂NNI-362 剂量(SAD)，有食物和没有食物。这之后将是14天的每日上升 剂量(MAD)试验。将对NNI-362的血浆水平进行评估，以确定 药代动力学和是否达到潜在有效剂量。如果NNI-362 被认为是安全和耐受性良好的，然后可以测试该试剂的促进能力 海马区新神经元(VMRI)与痴呆的停止或逆转和受损 阿尔茨海默氏症患者的功能，在扩大的安全性和有效性试验中。神经细胞上升 新的神经元再生治疗靶点衰老是高血压的最大危险因素 阿尔茨海默氏症和海马神经退行性变导致体积损失，这是直接的 与阿尔茨海默病的痴呆症相关。

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 alone, 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 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 a 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.