DuoPhos — Department of Health and Human Services SBIR Phase II: 400

Description

DESCRIPTION provided by applicant CKD chronic kidney disease is a progressive disease which is reported to affect of the worldandapos s population End stage renal disease ESRD is the fifth and final stage of CKD ESRD will affect million people worldwide in and more than Americans at a cost of more than $ billion in the U S alone Elevated serum phosphorus Pi is the major factor stimulating CKD progression and poor patient outcomes The NKF National Kidney Foundation recommended a target range for serum Pi of between mg dL and mg dL Less than of ESRD patients attain this goal under current treatments To address this modern epidemic DuoPhos developed an inhibitor of the intestinal protein responsible for to of dietary phosphorus absorption the intestinal Na phosphate cotransporter NaPi b andapos Fluorophosphophloretin FP is a small molecule inhibitor of NaPi b which has been demonstrated to reduce serum Pi to in animal models at daily doses of g kg body wt over week studies Preliminary toxicology studies indicate that FP shows no toxicity for up to weeks at concentrations X the efficacious dose The long term goal of this proposal is to deliver FP in a pill form to CKD stage and stage patients The goal of this proposal is to complete the toxicology studies required to complete the IND and begin human trials The specific aims are Synthesize kilogram quantities of FP that is GMP good manufacturing procedures quality suitable for clinical trials Assay the safety pharmacology of FP Determine the chronic toxicity of FP in rabbits Examine the chronic toxicity of FP in rats Completion of specific aim will accelerate human trials by providing a source and formulation pill form of FP for human trials GMP certified FP is required by the FDA for clinical trials Specific aims will complete the toxicology studies required by the FDA and complete the IND packet Completion of the IND will allow DuoPhos to either start Phase human trials or partner with a large pharmaceutical company to collaborate on the human trials and upon their completion take FP to market