Biotherapeutics, Inc. — Department of Health and Human Services SBIR Phase I: NIAID

Description

BTA First in class Oral Therapeutic for Inflammatory Bowel Disease Biotherapeutics IncBTIdevelops oral first in class therapeutics for autoimmune diseasesBTa lead compound for treating inflammatory bowel diseaseIBDbelongs to a new chemotype and targets the novel Lanthionine Synthetase C LikeLANCLpathwayBTI will file an IND for BTinmonthsSignificanceIBD afflicts overmillion Americans andmillion people worldwideAn unmet clinical need for safermore effective drugs remainsas current therapies have limited efficacy and adverse side effectsThe Technology andampProductBTI validated LANCLas a therapeutic target for IBD and patented a novel chemotypewhich encompassesprivileged scaffolds and andgtbillion new chemical entitiesNCEsincluding BTan orally active small molecule that binds to LANCLin the gastrointestinalGItractBThas efficacy inmouse models of IBDmg kg p ohas no toxicity in Ames and hERG screeningand has a benign safety profile in rats at doses up tomg kg p oThe Specific Aims for the SBIR Fast Track Phase I application are toAssess the therapeutic and prophylactic efficacy of oralrectal versus intravenousI VBTinthe MDRmouse model of IBDUsing dose response studies in the MDRmouse model of IBDwewill assess the efficacy of BTin dose response studies using three routes of administrationCompare efficacy of BTversus current drugs and INDs in the MDRmouse model of IBDWewill compare BTs efficacy with standard of care drugsASAsprednisoneazathioprineand antiTNFand INDsantianti ILandamp ILtofacitiniband GEDTransition Milestones areOral BToutperforms current treatments by andgtin effects on inflammatory biomarkers and histopathological grading in the MDRmouse model of IBDWe provide preliminary data in MDRmice in support of being able to meet the transition milestones with BTand do not anticipate any unforeseen challenges that prevent meeting the transition milestonesThe Specific Aims for the SBIR Fast Track Phase II application are toCharacterize the potential for human translation of BTvia cytokine profiling in ex vivoorganotypic intestinal systemsWe will use a novel organotypic human intestinal systemileum andcolonto assess the effect of BTon cytokine profiles in the gut from IBD patientsEvaluate the distribution and toxicological profile of BTWe will perform dose range findingDRFstudies and in vitro genetic toxicology testing before moving toweek repeated dose toxicity GLPstudies of BTin rats as required for IND statusWe will also identify potential off target effectsLong Term GoalThe goal of BTI s LANCLbased technology is to provide a first in class oral therapeutic for IBD that addresses an unmet clinical need for a market exceeding $billion and growingannually Public Health Relevance Inflammatory bowel diseaseIBDafflicts overpeople in the United States andpeople worldwideThis SBIR Fast Track application builds on the successful development and preclinical testing of a novel chemotype of anti inflammatories that target lanthionine synthetase C likeLANCLfor treating IBDThis project will advance BTa first in class oral IBD therapeutictoward investigational new drugINDapproval and Phasehuman clinical testing inmonths