Dignify Therapeutics LLC — Department of Health and Human Services SBIR Phase II: NICHD

Description

DESCRIPTION provided by applicant Loss of voluntary control over bladder and bowel function as a result of spinal cord injury SCI has profound impact on the mental and physical health status and quality of life of individuals It is estimated that people in the USA have SCI https www nscisc uab edu PublicDocuments fact figures docs pdf Urinary retention as a result of SCI is irreversible and can be life threatening The only available pharmacotherapy for urinary retention consists of cholinergic agonists which have minimal efficacy and severe side effects Consequently many people with SCI catheterize multiple times daily to empty their bladder Bladder catheter use is associated with increased incidence of health problems predominately repeated urinary tract infections sepsis isolation depression and hospitalization An andquot on demandandquot safe and effective pharmaceutical alternative to catheterization would be a life changing improvement in the daily routine of bladder management for SCI individuals and a significant reduction in individual and community health care costs Dignify Therapeutics is a drug development company focused on advancing novel drug therapies for patients with SCI multiple sclerosis and similar diseases that result in voidin dysfunction By combining novel pharmaceuticals with novel drug delivery technology Dignify Therapeutics hopes to redefine treatment of voiding disorders and restore the dignity of voluntary excretory function in a way that mimics normal micturition Dignify Therapeuticsandapos lead compound DTI is being developed as the first andquot on demandandquot pharmaceutical to induce bladder voiding without catheterization Phase II studies are proposed in a translational rat model of SCI to establish the efficacy of DTI Chronic SCI often results in detrusor sphincter dyssynergia DSD in which the external urethral sphincter fails to properly relax during micturition resulting in impaired voiding efficiency Proposed studies will examine the effects of DTI on bladder function in a rat SCI model with and without urethral smooth and or striated muscle activity These studies are essential to confirm the utility of DTI in treating chronic SCI induced voiding dysfunction in humans To enable submission of an Investigational New Drug IND application and subsequent use of DTI in humans FDA regulated drug safety and toxicology studies are required Phase II studies are proposed to establish a toxicity and safety profile for DTI that is acceptable for IND submission Studies including repeated dose toxicology cardiovascular and pulmonary safety studies in two animal species will be performed to identify an acceptable dose range for human administration Manufacture of DTI drug product for use in clinical studies will also be completed Successful completion of these studies will enable a clinical study of the safety tolerability and pharmacodynamics of DTI in humans and suggest therapeutic approaches to reduce urethral resistance in patients with high resistance e g DSD PUBLIC HEALTH RELEVANCE The ultimate aim of this project is to provide spinal cord injured individuals a novel pharmaceutical alternative to multiple daily catheterization of their bladder to void urine Passage of a catheter into the urethra and bladder can be discomforting stigmatizing and is associated with urinary tract infections and various other complications Success of our project will restore individual dignity improve daily quality of life and health ad reduce associated health care costs for people with bladder dysfunction due to spinal cord injury This grant will provide animal efficacy toxicity and safety data to allow clinical studie of a novel drug that may produce efficient voiding and hence provide an alternative to catheterization in individuals with spinal cord injury