OPSIDIO LLC — Department of Health and Human Services STTR Phase I: 400

Description

Abstract 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 million adult patients with CKD in the US and the cost of disease treatment is over $ 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 SCF 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 STTR will be to validate in animal models of CKD that the monoclonal antibody that is specific for the SCF 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 SCF 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 STTR will be the analyses of data from a large clinical cohort NEPTUNE that is already established at the University of Michigan with human CKD patients to further demonstrate that SCF levels correlate to disease severity and progression of human CKD The demonstration that blocking the abnormal form of SCF prevents or reverses CKD in an animal model and 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 SCF and progress to a phase STTR with a strong commercialization plan The ultimate goal of Opsidio LLC is to commercialize the anti SCF monoclonal antibody as a drug that can stabilize or reverse renal fibrosis in human CKD patients 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 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