A novel drug may offer new hope for Cushing's disease patients. Asedebart, a first-in-class monoclonal antibody targeting ACTH, normalized urinary free cortisol in 7 of 8 patients in early phase 2 data presented at ENDO 2026. A phase 3 trial could begin as early as 2027 if further results hold up.
A new treatment approach for Cushing's disease is showing early promise. Asedebart, a humanized monoclonal antibody developed by H. Lundbeck, works by binding to adrenocorticotropic hormone (ACTH) — the hormone overproduced by pituitary adenomas in Cushing's disease — effectively reducing excess cortisol production at the adrenal gland. Interim phase 2 data from the BalanCeD trial, presented at ENDO 2026, showed that 7 of 8 patients who completed IV dose titration achieved normalization of urinary free cortisol (UFC ≤170 nmol/24h).
The drug was generally well tolerated, with adverse events largely consistent with its mechanism of action — including glucocorticoid deficiency and peripheral edema. Three of 12 patients experienced serious adverse events, and one patient died from dilated cardiomyopathy deemed unrelated to the study drug. The next phase will evaluate subcutaneous administration, with a potential phase 3 pivotal trial slated for 2027.
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Why it matters: Cushing's disease is a rare but debilitating condition, and over 30% of patients are ineligible for surgery — the current first-line treatment. Asedebart's novel ACTH-targeting mechanism could fill a critical gap, and its potential dual use in congenital adrenal hyperplasia makes it an even more compelling pipeline candidate.
