Endogenous Cushing’s Syndrome
A devastating orphan disease of cortisol excess causing severe physical and mental complications.
What is endogenous Cushing’s syndrome?
Endogenous Cushing’s syndrome (Cushing’s), also referred to as hypercortisolism, is the result of exposure to excess cortisol over an extended period of time. Cushing’s can lead to a wide range of serious health complications, including weight gain, high blood sugar (diabetes), high blood pressure (hypertension), thinning bones (osteoporosis), muscle loss and weakness, fragile skin that bruises easily, stretch marks, depression, and cognitive changes.
Cushing’s is caused by a secreting tumor often found on the pituitary or adrenal gland. The tumor, usually non-cancerous, causes an overproduction of cortisol.
Cushing’s can be difficult to diagnose, as symptoms vary and many are common to other health conditions. As a result, some patients endure harmful levels of cortisol for many years.
Cushing’s affects approximately 10-15 people per million each year.¹
The need.
Treatment for Cushing’s may involve surgery, radiation, chemotherapy, or therapies designed to either lower the body’s production of cortisol or block its ability to bind to certain receptors. Approved treatments often have limited efficacy and multiple side effects – or are simply not easily accessible. As patients are constantly in search of better options, the average duration of treatment with any one agent is only about 12 months.
There is a clear opportunity to develop a safer, more effective, and accessible option for patients and prescribers.
Our product candidate.
Our proprietary HSD-1 inhibitor, SPI-62, utilizes a novel mechanism of action to target a source of cortisol causing health problems, and has the potential to safely and effectively alleviate its associated symptoms. Additionally, HSD-1 inhibition may not share the undesirable effects of completely blocking the glucocorticoid receptor, lowering ACTH, or inhibiting multiple steroidogenic enzymes, as currently approved therapies do.
Oral, once-a-day administration, no complex monitoring necessary.
SPI-62 has demonstrated the ability to lower intracellular cortisol levels in vital organs, with a favorable safety and tolerability profile in five clinical trials. These studies also showed improvement in glucose, HbA1c, cholesterol, and triglycerides.
¹ http://pituitary.ucla.edu/cushings-disease