Danish drugmaker H. Lundbeck announced Sunday that its experimental treatment asedebart demonstrated promising results and was well tolerated in a small mid-stage clinical study targeting Cushing’s disease, a rare disorder affecting the body’s hormonal balance.
The findings were unveiled at a medical conference held in Chicago, representing a significant step for Lundbeck as the company broadens its reach into rare diseases beyond its longstanding specialty in brain-related conditions like depression and migraine.
Data gathered from 12 trial participants revealed that following dose adjustments, urinary free cortisol — a measure of how much of the stress hormone cortisol the body produces in a single day — returned to normal levels in seven out of eight patients whose results could be evaluated. This normalization is considered a positive indicator in treating Cushing’s disease.
The drug, which is delivered intravenously, was reported to be generally well tolerated, with no unexpected harmful effects and no new safety concerns identified by researchers.
Johan Luthman, the company’s executive vice president and head of research and development, told Reuters: “All the adverse events we see are very, very consistent with the mode of action of the drug.”
Cushing’s disease is caused by a non-cancerous tumor on the pituitary gland that triggers the body to chronically overproduce cortisol. Asedebart targets this problem by reducing abnormal spikes in a hormone called ACTH, which in turn helps lower cortisol production.
Lundbeck said it intends to move to a new group of trial participants to study the drug when delivered via subcutaneous injections rather than intravenously, according to Luthman. A late-stage trial is expected to launch in the first half of 2027.
Asedebart has already been granted orphan drug designation — a special regulatory status reserved for treatments targeting rare diseases — for congenital adrenal hyperplasia in both the European Union and Japan.
