A Clinical Study of Latiglutenase as a Treatment for Type 1 Diabetics with Celiac Disease

Project Summary The goal of this work is to evaluate the efficacy of a therapeutic agent for protecting individuals with TypediabetesT Dand celiac diseaseCDfrom intestinal and symptomatic distress they suffer due to minute ingestion of gluten proteinBoth T D and CD are lifelong autoimmune diseases whose management is anchored upon very restrictive and distinct dietsThe co morbidity of these two diseases is highwhereas CD alone affectsof the general population in most countriesthe prevalence of CD among patients with T D has been estimated to beCurrently the only therapeutic option to avoid the symptoms and potentially long term health consequences of CD is the life long strict adherence to a gluten free dietGFDWhen superimposed on the need for tight blood sugar control in a T D patientthe burden of disease is enormous especially because many commercially available gluten free foods have high glycemic indicesThusthere is a considerable unmet need for a therapeutic solution to alleviating the burden of a GFD in patients with both T D and CDImmunogenX is a clinical stage biopharmaceutical company developing therapeutic and diagnostic solutions for CDThe company s lead productlatiglutenaseis an orally administereddual enzyme product that proteolyzes gluten and shows clinical evidence for histologic protection and symptomatic reduction in CD patientsIn particularevidence has been observed for symptom relief due to latiglutenase relative to placebo in a subpopulation of CD patients who remained seropositive despite adhering to a GFDAccordinglyin this SBIR Fast TrackPhase I IIproposalImmunogenX will team up with researchers at Stanford University who have extensive T D and CD expertise to demonstrate symptom relief in persistently seropositive patients with a diagnosis of both T D and CDThe Stanford team will conduct a double blindplacebo controlledrandomized withdrawal study on subjects with T D and CD who remain seropositive for CD specific antibodies notwithstanding attempts to maintain a GFD for at least one year following their CD diagnosisIn addition to supplying latiglutenase for this clinical trialImmunogenX will also provide the recently validated Celiac Disease Symptom Diary clinical outcome assessmentCDSDCOAinstrument for CD symptoms as well as logistical support for the Stanford trialA novel urine biomarker will be studied to estimate systemic exposure to dietary gluten over the duration of the trialOur patient enrollment target is based on adequate powering of the primary endpoint for symptom reductionWe anticipate the need to prescreenpatients with both T D and CD to enrollseropositive patients into the run in screening periodultimately achievingcompleted patientsPhase I will provide a refined trial designIRB approvals as well as trial preparation activities such as development of clinical suppliesThe actual trial conduct will occur in Phase II Project Narrative We propose to perform a clinical trial designed to test the efficacy of a promising investigational druglatiglutenasefor alleviating the extraordinarily high burden of a gluten free diet that is experienced by patients with a dual diagnosis of TypeDiabetesT Dand celiac diseaseCD