Скачать или смотреть Atumelnant for the Treatment of Congenital Adrenal Hyperplasia

Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses atumelnant for the treatment of patients with congenital adrenal hyperplasia (CAH).

CAH is a genetic condition characterized by an enzyme deficiency in the adrenal glands. This causes underproduction of cortisol and the overproduction of androgens. Symptoms may include ambiguous genitalia at birth, dehydration, poor feeding, diarrhea, vomiting and other health problems. People with milder forms may not be diagnosed with the condition until adolescence or adulthood when they experience early signs of puberty or fertility problems.

Treatment of these patients includes the lifelong use of glucocorticoids to help replace the missing cortisol. However, the doses of this treatment are often higher than the natural levels of cortisol found in the body. This can result in many complications such as obesity, metabolic issues, diabetes, Cushing’s syndrome, and diminished growth potential in children. Treatment of this condition can be difficult due to the need to balance glucocorticoid doses and androgen production.

Atumelnant, an ACTH receptor antagonist, is currently entering phase 3 studies for the treatment of CAH. The compound works to block receptors in the adrenal gland, lowering steroid production. Results from an ongoing phase 2 study of atumelnant for treatment of patients with CAH were presented at the ENDO 2025 meeting.

With three months of treatment of atumelnant, reductions in biomarkers in CAH activity and reduction in size of overgrown adrenal glands were observed. These results indicate that the treatment is engaging the target, reducing not only the biochemical excess output of the adrenal glands but physically reducing the size. Additionally, the atumelnant has been well-tolerated.

Chapters:
Introduction 00:00
CAH Overview 00:14
Treatment 2:59
Poster Overview 4:10