Early data for Crinetics's atumelnant in congenital adrenal hyperplasia released

Crinetics Pharmaceuticals announced promising initial findings from a Phase II study examining the effectiveness of atumelnant for treating congenital adrenal hyperplasia (CAH). The results indicated significant and consistent suppression of androstenedione and 17-hydroxyprogesterone (17-OHP) levels. These findings will be detailed at the Endocrine Society’s annual meeting (ENDO) in June.

The study involved 10 patients who have classical CAH and had been on a stable glucocorticoid replacement regimen for at least six months. Participants were administered 80 mg of oral atumelnant daily for a period of 12 weeks. The primary focus of the study was to evaluate the early morning levels of androstenedione and 17-OHP as measures of efficacy.

For the initial report, Crinetics Pharmaceuticals shared data from the first four participants, who had a median age of 34 years. Baseline androstenedione levels in these individuals ranged between 116 ng/dL and 604 ng/dL, while baseline 17-OHP levels ranged from 4740 ng/dL to 6905 ng/dL. According to the study, treatment with atumelnant led to rapid and substantial reductions in both androstenedione and 17-OHP within two weeks, effects that were sustained throughout the 12-week treatment period. Specifically, reductions in androstenedione ranged from 74% to 99%, and reductions in 17-OHP ranged from 68% to over 99%.

Additionally, the study reported that there were no serious adverse events related to the treatment, and atumelnant was generally well-tolerated by the participants. These encouraging results have led to plans to further assess the safety and efficacy of atumelnant at various doses in the ongoing study.

The announcement marks a significant milestone in the treatment of CAH, a genetic disorder affecting the adrenal glands, leading to an overproduction of androgen and insufficient production of cortisol. Current treatments primarily involve glucocorticoids to manage symptoms, but these can come with significant long-term side effects. The successful suppression of key biomarkers androstenedione and 17-OHP without severe adverse effects suggests that atumelnant could offer an effective new treatment option for CAH patients.

These initial findings could pave the way for larger clinical trials to confirm the efficacy and safety of atumelnant, potentially leading to a new therapeutic option for managing congenital adrenal hyperplasia. The medical community eagerly anticipates the detailed presentation of these results at the upcoming ENDO meeting, which could provide further insights into this promising new treatment.

In summary, the early results from Crinetics Pharmaceuticals' Phase II study on atumelnant for CAH treatment show rapid and sustained suppression of critical hormonal markers, with no serious adverse events reported. This study could herald a significant advancement in the management of congenital adrenal hyperplasia, offering hope for improved treatment protocols in the future.