Development of a novel, regenerative therapy to reverse synapse loss in Alzheimer's Disease

Project Summary/Abstract The mission of the SBIR program is to “encourage domestic small businesses to engage in Federal Research/Research and Development (R/R&D) that has the potential for commercialization.” A mission of the NIA is to “support research on Alzheimer’s disease, and promote research on the etiology, treatment, and diagnosis of AD”. The proposed development program is necessary to enable the filing and acceptance of an IND with the FDA, an early prelude to commercialization. The three specific aims of this development grant are: 1. GMP Manufacture of an oral dosage form of SPG302, a novel synaptogenic molecule. 2. Conduct GLP safety pharmacology studies and conduct GLP IND-enabling toxicology studies in rats and Beagle dogs for 4 weeks with SPG302 3. Prepare and submit a successful IND request to the FDA. The proposed studies are designed to optimize time and resources while moving step wise through safety tests, resulting in an IND submission (for Phase 1 single ascending dose, multiple ascending dose and food interaction studies) within 24 months of the grant being funded. Spinogenix will work with a CRO to assemble the CMC, pharmacology and toxicology information and to prepare and submit an electronic IND to the FDA.