Opsidio LLC is commercializing a monoclonal antibody (MAb) that will prevent and treat chronic kidney disease (CKD) that can lead to end stage renal disease (ESRD) and dialysis or kidney transplantation. At the present time, there is no known therapy for CKD that can alter the progression to end stage kidney failure. The CDC estimates that there are 20 million adult patients with CKD in the US and the cost of disease treatment is over $40 billion per year. The quality of life for patients with CKD or ESRD is compromised with patients feeling sick and tired, frequent doctor visits and tests, numerous medications, and dietary restrictions. Opsidio has licensed the patent for a MAb specific for a disease-associated form of Stem Cell Factor (SCF) from the University of Michigan. SCF stimulates two kinds of immune cells, mast cells and eosinophils, to produce hormones that drive chronic tissue remodeling and fibrosis, a process in which normal tissue is replaced by scar tissue. This process is especially relevant in CKD, where the amount of SCF present has been correlated to severity of kidney disease, and the degree of fibrosis and mast cell accumulation in the kidney. The design of the antibody allows it to bind only to the disease associated SCF248, and not the other forms of SCF that maintain normal functions. Opsidio has generated preliminary data that supports the hypothesis that SCF contributes to human CKD. The focus for this phase 1 STTR will be to validate in animal models of CKD that the monoclonal antibody that is specific for the SCF248 form can arrest and possibly reverse the fibrosis that is the cause of renal failure. In addition, we will use a large collection of blood, urine and kidney biopsy samples from human patients with various types of kidney disease to show that levels of SCF248 are correlated with the severity of human CKD. An animal model that resembles human disease will be performed with a subcontract to kidney researchers at the University of Michigan. They will demonstrate that the MAb can prevent or treat the decline in kidney function. The second major goal of this phase 1 STTR will be the analyses of data from a large clinical cohort (NEPTUNE) that is already established at the University of Michigan with ~600 human CKD patients to further demonstrate that SCF levels correlate to disease severity and progression of human CKD. The demonstration that 1) blocking the abnormal form of SCF prevents or reverses CKD in an animal model, and 2) that the same mechanism of abnormal SCF driving CKD is operative in humans with known CKD will provide strong evidence for Opsidio, LLC to continue the development of these MAbs directed against SCF248, and progress to a phase 2 STTR with a strong commercialization plan. The ultimate goal of Opsidio, LLC is to commercialize the anti-SCF248 monoclonal antibody as a drug that can stabilize or reverse renal fibrosis in human CKD patients.
Public Health Relevance Statement: Narrative Opsidio, LLC is developing a monoclonal antibody therapy that will treat or reverse chronic kidney disease that could otherwise progress to dialysis or transplantation. At the present time there is no known therapy for chronic kidney disease and the estimated 20 million US patients have few options. Thus, this therapeutic would be a first in class drug to address the ongoing needs of a large patient population.
Project Terms: Address; Adult; Adverse effects; Albumins; American; Animal Model; Animals; Antibodies; Automobile Driving; Binding; Biological; Biopsy; Biopsy Specimen; Blood; Cause of Death; Cell Count; Cells; Centers for Disease Control and Prevention (U.S.); Chronic; Chronic Kidney Failure; Cicatrix; Clinical; cohort; Collection; commercial application; commercialization; cost; Cost of Illness; Creatinine; Data; Data Analyses; Dependence; design; Development; Diabetes Mellitus; Dialysis procedure; dietary restriction; Disease; Disease Progression; Economics; End stage renal failure; eosinophil; experimental study; Feeling; Fibrosis; Future; Glomerular Filtration Rate; Goals; Growth; Hemodialysis; Hormones; Human; human disease; human monoclonal antibodies; Hypertension; Immune; improved; Inflammation; Injury; Kidney; Kidney Diseases; Kidney Failure; Kidney Transplantation; Knowledge; Lead; Legal patent; Licensing; Lung; mast cell; Measures; Mediator of activation protein; Methods; Michigan; Monoclonal Antibodies; Monoclonal Antibody Therapy; mouse model; Normal tissue morphology; Outcome; patient population; Patients; Pharmaceutical Preparations; Pharmacology; Phase; prevent; Process; Protein Isoforms; Proto-Oncogene Protein c-kit; Quality of life; Reaction; receptor; Recruitment Activity; Renal function; Research Personnel; Sampling; Serum; Severities; Severity of illness; Small Business Technology Transfer Research; Specificity; Stem Cell Factor; technological innovation; Technology; Testing; Therapeutic; Therapeutic Monoclonal Antibodies; Time; Tissues; Transplantation; Treatment Cost; Universities; Up-Regulation; urinary; Urine; Visit
