Dignify Therapeutics LLC — Department of Health and Human Services SBIR Phase I: 400

Description

PROJECT SUMMARY ABSTRACT Aging and diabetes as well as many neurological conditions such as multiple sclerosis Parkinsonandapos s disease spina bifida stroke and spinal cord injury SCI can result in loss of voluntary voiding of urine and require intermittent bladder catheterization for voiding Catheter use is associated with increased incidence of health problems predominately repeated urinary tract infections sepsis urethral trauma and hospitalization A short acting effective and safe product that induces on demand voiding would provide a paradigm shift in the management of neurogenic bladder and could reduce or eliminate the need for intermittent catheterization Dignify Therapeutics is developing a novel drug treatment DTI to provide an on demand rapid onset short duration drug induced voiding therapy for those who cannot void voluntarily When administered intravenously IV or subcutaneously SC to rats dogs and minipigs DTI rapidly induces bladder voiding However IV and SC routes of administration may be impractical for the multiple daily dosing that will be required by individuals with neurogenic bladder Therefore intranasal IN and orally disintegrating tablet ODT formulations of DTI are being developed Preliminary studies have shown that efficacious plasma concentrations and dose dependent bladder contractions can be achieved using both ODT and IN formulations Phase of this fast track SBIR proposal will use repeated dosing and measurements of voiding efficiency in conscious minipigs a species with subcutaneous nasal and oral tissues similar to humans to identify the preferred formulation and route of administration IN or ODT for use in future clinical studies In Phase this formulation will be optimized and manufactured according to FDA regulatory guidelines The optimized final formulation will then be used for preclinical in vivo testing in minipigs and rats to characterize the pharmacological mechanism of action as well as for mandatory general toxicity studies required for submission of an investigational new drug IND application and the initiation of studies in humans With proper support our product could be available as a therapeutic agent with the next five to seven years PROJECT NARRATIVE The ultimate aim of this project is to provide to individuals with neurological conditions a novel pharmaceutical alternative to multiple daily catheterizations of their bladder to void urine Catheter use can be embarrassing and uncomfortable and it is associated with urinary tract infections and various other complications This grant will enable the development of a convenient intranasal or sublingual dosage formulation for a novel drug to provide on demand voiding Success of our project will restore individual dignity improve daily quality of life and health and reduce associated health care costs for individuals with neurogenic voiding dysfunction