David Katz, PhD, Chief Scientific Officer and Founder of Sparrow Pharmaceuticals, discusses how clofutriben addresses the challenges of hypercortisolism in Cushing’s syndrome.
Cushing’s syndrome is a rare endocrine disorder caused by prolonged exposure of the body’s tissues to high levels of cortisol. Signs and symptoms of Cushing’s syndrome include:
- Upper body obesity
- Fatigue
- Muscle weakness
- High blood pressure
- Backache
- High blood sugar
- Easy bruising and bluish-red stretch marks on the skin
Affected women may also experience irregular menstrual periods and increased growth of body and facial hair. This condition may be caused by a variety of factors including long-term use of corticosteroid medications, tumors in the pituitary gland or adrenal adenomas.
The first line treatment for Cushing’s syndrome typically involves surgical removal of the tumor. While this may cure the disease for some patients, for those who are ineligible for surgery or who do not respond, medical treatments are necessary. These therapies target and reduce adrenocorticotropic hormone (ACTH) secretion and cortisol production. However, most of the available treatments have a significant side effect of adrenal insufficiency.
Clofutriben is a small molecule HSD-1 inhibitor that targets intracellular cortisol. The treatment aims to address hypercortisolism in Cushing’s syndrome while avoiding adrenal insufficiency.
An interim analysis of clinical trials evaluating clofutriben was recently presented at the ENDO 2024 conference. Of the outcomes observed, was a normalized urine free cortisol (UFC) in over 60% of patients with endogenous Cushing’s syndrome. This was without suppressing serum cortisol to levels associated with adrenal insufficiency. These results may indicate a differentiation with clofutriben in the detailed dose titration required by most treatments for Cushing’s syndrome in order to mitigate side effects.
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To learn more about Cushing’s syndrome and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/
