Administrative Supplement to Promote Diversity in Research and Development Small Businesses

Abstract Neuroene Therapeutics is a biotech company established to develop and commercialize safe effective therapies for epilepsyinpeople develop epilepsy in their lifetimehowever the currently available anti epileptic drugsAEDshave several issues and liabilities that leave unmet market needssuch asnot controlling seizures forof patients with epilepsycurrent AEDs only treat the symptoms of epilepsy and do not modify underlying diseaseadverse effectsWe have discovered new anti epilepsy compounds that protect mitochondrial and neuronal health and have successfully completed the Phase I STTRwe have optimized the lead compound through careful design and synthesiswhich now has excellent serum half lifehrcompared to previous tofhrrententionand oral bioavailabilityin vivo in miceas well as increased antiseizure activity compared to our prior leadOur goal now is to complete IND enabling studies for our lead compoundThe ultimate goal of Neuroene Therapeutics is to develop a new generation of AED for patients with medication resistant epilepsy and those with severe side effects from their current medicationThere are currently no AEDs that target mitochondrial dysfunctiondespite being a major contributing factor for epilepsyTargeting an alternative mechanism of actionand having a low therapeutic dose compared to current AEDs on the market means this novel therapy is likely to be more effective with fewer potential side effectsEstablishing the optimal oral formulation and pharmacokineticPKand pharmacodynamicPDrelationship in rodents will reveal the best dosing strategies and the best epilepsy subset for the lead compoundThese results will enable future studies of this potential oral based AED in higher mammals and humansAimSynthesisADMETand formulationOur ultimate goal is to produce a safe AED that can be orally delivered in humansAn optimized oral formulation with a minimum oforal bioavailability will be deliveredAimOral dosing and brain bioavailabilityWe will determine the oral maximum tolerated doseMTDoptimum doseand dosing regimen of the lead compound in rodentsThe lead compound should have no observable genoor chronic toxicityand will maintain a significant CMAX in brain tissue with excellent target selectivityAimAnti seizure efficacyThe lead compound will be evaluated for anti seizure efficacy in multiple rodent seizure modelsThe lead compound will be considered efficacious against rodent models of medication resistant epilepsyby reducing incidence of seizures at doses below MTDand PDduration of acute anti epileptic effectwill be correlated with PKAt the end of this Phase II studythe lead compound will be deemed feasible as a new generation of AED based on achieving in vivo efficacy and PK PD milestonesallowing for studies in higher mammals required for pre IND filing for human studiesPartnerships with Pharma and Biotech will be sought to take the therapeutic agent into human clinical trials and to complete commercialization of the product!Narrativeinpeople develop epilepsy in their lifetimehowever the currently available anti epileptic drugs have several issues and liabilities and are not effective forof the population with active epilepsyTraining the next generation of scientists with the appropriate research and business skills is essential for the development of pharmacotherapies for this difficult to treat neurological disease!