权益分类	功能权益	普通用户	{{item.name}}会员
{{category.name}}	{{benefitItem.name}}

IND Enabling Studies for a Novel Mas Receptor Agonist for Treatment of Cognitive Impairment in Patients at Risk for Alzheimer's Disease Related Dementia

新型 Mas 受体激动剂的 IND 使研究能够治疗有阿尔茨海默病相关痴呆风险的患者的认知障碍

基本信息

批准号：
10437202
负责人：
Meredith Hay
金额：
$ 76.7万
依托单位：
UNIVERSITY OF ARIZONA
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-04-15 至 2024-03-31
项目状态：
已结题

项目摘要

PROJECT SUMMARY The purpose of this request for an administrative supplement is to cover the increased cost of GMP peptide manufacturing and the increased cost of the in-vivo and in-vitro toxicology studies that will occur in year 2. With regards to manufacturing, since the original submission of this U01 proposal, Polypeptide, our Peptide manufacturing CRO, the cost of a 500gram GMP batch of PNA5 ($970/gram plus stability testing, CMC documentation, and final fill and finish) is now $786,000 vs our original budget of $525,000 for 150 grams ($2500/gram plus stability testing, CMC documentation). This increased batch size is required for our planned extended 21-day repeated dose formal toxicity studies planned in Yr 3 and will also be enough to complete our planned Phase 1a/b studies in humans, thus eliminating the need to produce a second GMP batch for the planned first in human study. The cost difference will be $261,000. This will ultimately save both time and money by not requiring a 2nd GMP batch prior to first-in-human studies batch and a significant $/gram savings. Regarding the 7-day MTD toxicology studies in rat and dog and in vitro assays (hERG, chromosome assay, AMES), we have been able to accelerate the timing of these studies and now plan to have these completed in Year 2. In the original proposal, the original quotes our original vendor (MPI) totaled $452,800. Since then, that vendor was acquired by Charles River and those same studies are now costing $691,456. The cost difference is $238,656. We have included an itemized cost comparison between the original quote and the new quote below. Thus, for Year 2, we are requesting an additional $499,656 in direct costs to cover the increased manufacturing costs and for the increase in-vivo and in-vitro toxicology costs.

项目摘要本行政补充申请的目的是支付GMP肽增加的成本生产和体内和体外毒理学研究的成本增加，将在第2年进行。与关于生产，自最初提交本U 01提案以来，多肽，我们的肽生产CRO，500克GMP批次的PNA 5的成本（970美元/克加上稳定性测试，CMC 文件，以及最终填充和完成）现在是786，000美元，而我们最初的预算为150克525，000美元（2500美元/克加上稳定性测试、CMC文档）。这一增加的批量是我们计划的计划在第3年进行为期21天的延长重复给药正式毒性研究，这也足以完成我们的计划的人体1a/B期研究，从而无需生产第二批GMP批次在人类研究中首次提出。费用差额为261 000美元。这将最终节省时间，通过在首次人体研究批次之前不需要第二个GMP批次来节省资金，并显著节省$/g。关于大鼠和犬的7天MTD毒理学研究和体外试验（hERG，染色体试验，我们已能加快进行这些研究的时间，并计划在二零零零年完成这些研究。第二年。在原始提案中，我们的原始供应商（MPI）的原始报价总计为452，800美元。从那时起， Charles River收购了一家供应商，这些研究现在的成本为691，456美元。成本差异是238656美元我们已经包括了原始报价和新报价之间的逐项成本比较下面因此，对于第二年，我们要求增加499，656美元的直接费用，以支付增加的制造成本以及体内和体外毒理学成本的增加。