Medosome Biotec, LLC — Department of Health and Human Services STTR Phase I: NICHD

Description

ABSTRACTUp toof low birth weight preterm infants manifest some form of periventricular white matter injuryPWMImaking it the most common form of brain injury affecting premature infantsPWMI is associated with significant morbidityas affected individuals may have profound intellectual impairment and cerebral palsyHighlighting the magnitude of PWMIeach year more thaninfants are born prematurely in the United StatesOf these infantsare born at risk for PWMIand aboutchildren per year will develop PWMIThusfinding a prevention and treatment for PWMI is of major public health importanceOligodendrocytesOLsare the myelinating cells of the central nervous system and play a critical role in white matter formationIt is believed that loss of Pre oligodendrocytesPreOLswhich are proliferative cells that develop into myelinating OLsplays a major role in PWMI causationPresentlythere are few pharmacological approaches that specifically target PreOLsresulting in increased proliferation of these cells and increased brain myelinationMedosome BiotecLLCMBTand its research partners at the University of Florida believe a significant market exists to develop and commercialize a therapeutic treatment for white matter injury in premature infants and other brain myelination disordersTo address the shortcomings of current therapeuticsthe teamled by DrScott RivkeesPIat University of Floridaused high throughput screening to identify compounds that to stimulate PreOL proliferationThese studies identifiedcompounds as having potential meritIn preliminary studieswe assessed myelination and toxicological properties of these compounds in brain slice and in vivo studiesThese studies identified the compound Kas the most potent stimulator of myelinationThis compound was subsequently tested in short term and long term toxicology studies in neonatal mice and found to be non toxicThis compound also stimulated myelination in neonatal miceBuilding on our promising datathe proposed STTR fast track application will first focus on testing this compound for efficacy in models of white matter injury in the PhasecomponentIf protectiveas anticipatedin the Phasecomponentwe will be to perform IND enabling studies that will lead to an IND and clinical studiesTo achieve these goalsour team will include DrsAvery and McCardywho direct the University of Florida Translational Drug Development CoreCharles River and ChemDiv Laboratorieswhich are CROs with extensive experience in the execution of pre IND testingand Pharmlex which has more thanyears of experience in assisting a pharmaceutical companies in the United States and around the world to bring new therapeutics to the marketplaceThis project presents an excellent opportunity to commercialize novel approaches for treating and preventing white matter injury in the tens of thousands of premature infants born and hospitalized each yearAs suchwe have the opportunity to develop new therapeutics for a huge unmet need PROJECT NARATIVE Loss of Pre oligodendrocytesPreOLswhich are proliferative cells that develop into myelinating OLsplay a major role in white matter injury causationPresentlythere are few pharmacological approaches that specifically target PreOLsresulting in increased proliferation and differentiation of these cells and increased brain myelinationThe goal of this work is to develop novel therapeutic agents for the treatment of white matter injury in premature infants and other disorders of myelination