ORPHAGEN PHARMACEUTICALS, INC. — Department of Health and Human Services SBIR Phase I: ? DESCRIPTION (provided by applicant): Endogenous Cushing's syndrome is a classic

ORPHAGEN PHARMACEUTICALS, INC. — SBIR Phase I award from Department of Health and Human Services.

Amount
$223,229
Agency
Department of Health and Human Services · National Institutes of Health
Program / Phase
SBIR · Phase I
Solicitation
PA14-071
NAICS
Place of performance
CA
Period

Description

? DESCRIPTION (provided by applicant): Endogenous Cushing's syndrome is a classic dysfunction of the endocrine system caused by prolonged exposure to inappropriately high levels of cortisol. The condition is tumor-driven and leads to obesity, diabetes,hypertension and psychiatric dysfunction, symptoms which improve with a reduction in cortisol. Cushing's syndrome disproportionately affects females, who make up 75% of the 3,000 new cases diagnosed each year in the United States. Two drugs recently approved for Cushing's, mifepristone and pasireotide, underscore the importance of developing new therapies. Pasireotide, indicated for patients with an adrenocorticotropic hormone (ACTH)-secreting pituitary tumor (Cushing's Disease), is effective in just 25% of cases and can cause hyperglycemia and diabetes. Mifepristone blocks glucocorticoid and progesterone receptors, leading to elevated cortisol and endometrial bleeding in women. Significantly, medical therapy of hypercortisolism in Cushing's is still predom