Thetis Pharmaceuticals LLC — Department of Health and Human Services SBIR Phase I: 300

Description

ABSTRACTUlcerative colitisUCis a chronicrelapsing form of Inflammatory Bowel DiseaseIBDcharacterized by inflammation and ulceration of the colon and rectumUncontrolled UC can lead to serious complicationsincluding toxic megacolon and colorectal cancerThe disease affects approximatelypeople in the USwith up toof patients diagnosed before ageYoung UC patients typically present with more severe symptomsare subject to heavy psycho social burdenand suffer from growth and development issuesMoreoverthe side effect profiles of approved therapies are frequently more severe in childrenlimiting the therapies available for this vulnerable populationIn particularthere is a need for novelsafe therapeutic options suitable for chronic administration in pediatric and adolescent UC patientsTo answer this unmet medical needThetis proposes to develop magnesium lysicosapentateor TPan innovative new molecular entityNMEas a safeoral therapy to maintain remission of symptoms in children and adolescents with UCTPdelivers eicosapentaenoic acidEPAan Omegapolyunsaturated fatty acidPUFAshown to have clinical efficacy for treatment of UCTPovercomes the physico chemical and commercial deficits inherent in EPA itself to enable development and registration as a prescription drug with an indication for maintenance of remission in pediatric UC patientsImportantlyin a previous preclinical study conducted by ThetisTPwas shown toideliver therapeutic levels of EPA to colonic tissue andiifavorably change colon tissue levels of key eicosanoid mediators that regulate inflammationBased on these biological findingsalong with technicalpharmaceutical and IP advantages of TPThetis plans to undertake the development of TPto maintain remission in pediatric UC patientsIn this SBIR Fast Track projectThetis will complete the preclinical activities required for IND submission and initiation of clinical studiesThis goal will be achieved through three Specific AimsPhase I will include an efficacy study performed using a dextran sulfate sodiumDSSmouse model of UCSpecific AimThe efficacy of TPwill be evaluated aloneSub aimaand in combination with current treatmentSub aimbIn Phase IIwe will determine the juvenile safety profile of TPin support of treatment of pediatric UC patientsSpecific AimOnce the efficacy and acceptable toxicity profile of TPare demonstratedThetis will conduct the manufacture and testing of GMP Drug SubstanceSpecific Aimwhich itself involves two sub aims including prototype drug product developmentSub aimafollowed by manufacturing scale uptestingpackaging and release of drug product for clinical suppliesSub aimbUpon completion of this projectThetis will be able to prepare and submit an IND filing as a prerequisite to initiate Phase I clinical studiesThis SBIR project is critical to enabling development of the TPprogram to address the unmet medical need in this vulnerable pediatric population NARRATIVE Ulcerative colitisUCis a chronicrelapsing form of Inflammatory Bowel DiseaseIBDcharacterized by inflammation and ulceration of the colon and rectumUncontrolled UC can lead to serious complicationsincluding toxic megacolon and colorectal cancerThe disease affects approximatelypeople in the USwith up toof patients diagnosed before ageYoung UC patients typically present with more severe symptomsare subject to heavy psycho social burdenand suffer from growth and development issuesMoreoverthe side effect profiles of approved therapies are frequently more severe in childrenlimiting the therapies available for this vulnerable populationIn particularthere is a need for novelsafe therapeutic options suitable for chronic administration in pediatric and adolescent UC patientsThetis Pharmaceuticals proposes to develop TPas a safeoral therapy to maintain remission of symptoms in children and adolescents with UC