Crinetics Announces Positive Initial Findings at ENDO 2024 for Atumelnant in Two Ongoing, Open-Label Studies for the Treatment of Congenital Adrenal Hyperplasia (CAH) and ACTH-Dependent Cushing's Syndrome (ADCS)

Core Insights - Crinetics Pharmaceuticals announced promising initial findings from its clinical product candidate, atumelnant (CRN04894), a novel oral ACTH receptor antagonist, during the Endocrine Society's annual meeting (ENDO2024) [1][2] Group 1: Clinical Trial Results - In the Phase 2 TouCAHn study, participants with congenital adrenal hyperplasia (CAH) showed over 90% reduction in androstenedione (A4) and 97% reduction in 17-hydroxyprogesterone (17-OHP) after 12 weeks of treatment with atumelnant (80 mg) [1][3] - 100% of CAH participants maintained A4 levels below the upper limit of normal at all time points during the treatment [1][3] - The Phase 1b/2a study for ACTH-dependent Cushing's syndrome demonstrated that 100% of participants achieved serum cortisol levels below 5 mcg/dL within 10 days of atumelnant administration [6][9] Group 2: Safety and Tolerability - No severe or serious treatment-emergent adverse events were reported in the ongoing trials, with all adverse events being mild to moderate [4][6] - The most common treatment-emergent adverse events included fatigue, headache, and upper respiratory tract infection [4][6] Group 3: Mechanism and Significance - Atumelnant is the first once-daily oral ACTH receptor antagonist, which directly inhibits ACTH at its receptor, offering a novel approach compared to traditional glucocorticoid treatments [2][5] - The drug has shown strong binding affinity for the melanocortin type 2 receptor (MC2R) and effectively suppresses adrenally derived glucocorticoids and androgens [11] Group 4: Future Developments - The TouCAHn study aims to enroll up to 30 patients and is ongoing, with topline results expected in the second half of 2024 [12] - Crinetics will host an investor conference call to discuss these findings further [7]