clofutriben
A highly potent and selective, small molecule HSD-1 inhibitor.
What is clofutriben?
A new way to control cortisol levels and mitigate their negative effects.
clofutriben is our proprietary, oral small molecule, new chemical entity, HSD-1 inhibitor, designed to reduce intracellular cortisol levels and mitigate its numerous adverse effects. We are currently developing clofutriben for conditions directly caused by an overproduction of cortisol in the body.
Clofutriben may represent the first new mechanism of action in decades approved for patients suffering from endogenous Cushing’s syndrome (CS) and Autonomous Cortisol Secretion (ACS).
How it Works
Targeting intracellular cortisol.
HSD-1 is a major source of intracellular cortisol in key tissues where excess cortisol causes morbidity, such as the liver, adipose (fat), bone, and brain.
By inhibiting HSD-1, clofutriben targets a source of cortisol that is available to act within cells, and thereby potentially mitigates its negative effects.
Potential Benefits
High Efficacy
Previous clinical trials of clofutriben showed potent targeting of HSD-1 in the brain and liver, and a corresponding lowering of intracellular cortisol levels in the liver. Furthermore, patients with Cushing’s and constitutionally low HSD-1 activity showed no symptoms of their severe hypercortisolism.¹ ²
Strong Safety
In five clinical trials, clofutriben has demonstrated a favorable safety and tolerability profile with few adverse effects.
High Adherence
As a small molecule with predictable pharmacokinetics, clofutriben is expected to be administered orally, once-a-day.
No Titration
The risk of adrenal insufficiency from HSD-1 inhibition is expected to be low, alleviating the need for careful dose titration and complex monitoring.
Future Plans
Phase 2 clinical trials of clofutriben for patients with Cushing’s and ACS initiated in 2022.
¹ J Clin Endocrinol Metab (2002) 87:57-62.
² Endocrine J (2008) 55:709-715.