Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company’s clinical trial. This is a Phase 2 trial that is testing the company’s drug tildacerfont. This drug, which is classified as an orally available corticotropin-releasing factor type-1 receptor antagonist, is being tested in this trial as a treatment for congenital adrenal hyperplasia. Spruce Biosciences is focused on the development of therapies for rare endocrine system disorders.

About Congenital Adrenal Hyperplasia (CAH)

Congenital adrenal hyperplasia (CAH) is a group of several related genetic disorders that appear as a result of certain genetic mutations that affect the production of sex steroids, mineralocorticoids, and glucocorticoids. The different variants of congenital adrenal hyperplasia are distinguished by which gene is affected. Symptoms vary depending on the sex of the patient and the form of the disorder. These symptoms can include hypogonadism, infertility, vomiting, undervirilization (in males), ambiguous genitalia (in females), early puberty or delayed puberty, rapid growth, menstrual irregularities, and excessive facial hair. Management of congenital adrenal hyperplasia typically involves supplementation of the substance that is affected, such as replacement glucocorticoids, mineralocorticoids, or sex steroids. Other treatments may be used to control growth rate. There is no known cure for these disorders. To learn more about congenital adrenal hyperplasia, click here.

About The Clinical Trial

This clinical trial involved a total of 18 congenital adrenal hyperplasia patients. These patients were divided in to two separate cohorts, one consisting of ten patients and another of eight, in order to test escalating doses of tildacerfont. The first cohort was treated with 200, 600 and 1000 mg doses of the drug once each day over a period of treatment that lasted six weeks. The second cohort were treated with 200 mg of tildacerfont twice per day for two weeks.

The results from the trial indicate that tildacerfont was well tolerated by the participants and no serious adverse effects were reported. The majority of patients in the clinical trial saw meaningful reductions in their concentrations of adrenal androgens, which are commonly present in excessive amounts in patients with congenital adrenal hyperplasia and are being used in this study as an indicator of disease modifying activity.

These results are encouraging and suggest that tildacerfont could one day become the first disease modifying therapy for congenital adrenal hyperplasia.


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