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.

项目摘要该请求要求进行管理补充的目的是支付GMP肽的成本增加制造业和体内和体外毒理学研究的成本增加，将在第二年进行。从最初提交U01提案，多肽，我们的肽以来的制造业。制造CRO，500克GMP批次PNA5的成本（$ 970/克加上稳定性测试，CMC 文档，最终填充和完成）现在为786,000美元，而我们的原始预算为525,000美元，150克（$ 2500/克加上稳定性测试，CMC文档）。我们计划的需要增加批处理大小延长21天的重复剂量正式毒性研究计划在YR 3中计划，也足以完成我们的计划在人类中进行的1A/B研究期，因此消除了生产第二个GMP批次的需求计划在人类研究领域。成本差将为$ 261,000。这最终将节省时间和金钱是不需要第二批次GMP批次在人类研究批处理和大量$/克储蓄之前。关于大鼠和狗和体外测定的7天MTD毒理学研究（HERG，染色体测定，艾姆斯），我们已经能够加速这些研究的时机，现在计划完成这些研究的时间第2年。在原始提案中，原始报价我们的原始供应商（MPI）总计$ 452,800。从那以后，那是供应商被查尔斯·河（Charles River）收购，这些研究的售价为691,456美元。成本差异是$ 238,656。我们包括了原始报价和新报价之间的逐项成本比较以下。因此，对于第二年，我们要求额外的499,656美元的直接费用来支付增加制造成本以及增加体内和体外毒理学成本。