IND-Enabling Pre-clinical Development of Modified P8 for the Treatment of Alzheimer's Disease

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Summary The parent Commercialization Readiness Program grant was to conduct studies necessary to support regulatory submissions relating to pre-clinical development of Cenna’s peptide drug candidate mP8. mP8 is being developed as a new, first-in-class peptide drug for the treatment of Alzheimer’s disease. The funded grant builds on the substantial progress made under the previously funded SBIR Phase 2 and 2B grants to carry out activities to support a future first in human clinical study of mP8. A necessary component of the studies that is required by the FDA is to conduct IND-enabling drug safety toxicity and GLP safety pharmacology studies in two species of animals. For peptides, small molecules and other drug modalities, studies using a rodent plus a non-rodent species are required by the FDA to support clinical development and licensing. When the parent grant was submitted, we had proposed to use the cynomolgus monkey as the non-rodent animal model for these studies as it is an accepted species for preclinical toxicity testing and is more closely related, both phylogenetically and physiologically, to humans than dogs. Monkeys are also much smaller than dogs and so would require much less peptide. This is important since our studies to date have indicated that our original peptide P8 is not toxic even at the highest dose. We therefore anticipate having to use 50X-100X the efficacious dose if we cannot produce toxicity, making the cost of the peptide very high if we were to test it in dogs. Since the funding of the grant, because of the pandemic and recent trade-wars with China, where monkeys are sourced, there is a dire shortage of non-human primates. As a result, the price per monkey has more than doubled. This Administrative Supplement application is to help offset the increased cost of the monkey studies that are within the scope of the approved award but were unforeseen when the application was submitted and reviewed.