Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care

Classic congenital adrenal hyperplasia, or CAH, is a potentially life-threatening rare genetic disorder characterized by an inability to produce the stress hormone cortisol while causing excess production of androgens, or male sex hormones. The condition has long-been treated with steroids, but about 70 percent of patients with the condition have poorly controlled disease. The problem is that it is difficult to give someone with CAH enough steroids to control the androgens without causing problems such as weight gain, increased blood sugar, and high cholesterol. As a result, doctors often underdose patients. Spruce Biosciences is developing an experimental therapy called tildacerfont, a non-steroidal therapy that binds to a receptor on the pituitary glands to limit the production of adrenal androgens and address that aspect of the disease. We spoke to Richard King, CEO of Spruce Biosciences, about CAH, how tildacerfont works, and what the company is doing to build a pipeline of other rare endocrine therapies behind it.