Key pipeline drug for Lundbeck delivers in Cushing syndrome

Lundbeck has reported positive mid-stage results with Cushing's disease therapy asedebart, one of four experimental drugs the Danish pharma group is banking on to deliver growth as it prepares for a patent cliff before the end of the decade.

Anti-adrenocorticotropic hormone (ACTH) asedebart (formerly Lu AG13909) is designed to reduce the production of the stress hormone cortisol in Cushing's disease, a condition caused by a non-cancerous tumour on the pituitary gland.

The tumour causes over-production of ACTH, which stimulates the adrenal glands to release dangerously high levels of cortisol and leads to a wide range of symptoms and can raise the risk of serious complications like cardiovascular disease, diabetes, and osteoporosis.

In preliminary data from a phase 2a trial reported at the ENDO 2026 conference, asedebart was shown to normalise levels of urinary free cortisol (UFC) – a biomarker for Cushing's disease – in seven of eight evaluable patients.

So far, 12 patients have been enrolled into the phase 2a stage of the study, which is evaluating intravenous and subcutaneous formulations of asedebart. A second stage, looking at subcutaneous dosing only, is already underway.

"The data from the ongoing phase 2 study highlight[s] the potential of direct ACTH neutralisation as a novel therapeutic approach in Cushing's disease, a neuroendocrine condition where major unmet medical needs remain," said Johan Luthman, Lundbeck's head of R&D.

"The UFC normalisation observed in most evaluable patients is very encouraging and strengthens our confidence in asedebart's continued development," he added.

At the moment, the main treatment for Cushing's disease is surgery to remove the tumour, but that isn't suitable for all patients, and sometimes surgery alone is not enough to keep patients in remission.

The potential for drug treatment of Cushing's disease is apparent from Recordati's oral cortisol synthesis inhibitor Isturisa (osilodrostat), which has been growing strongly since launch and is tipped by some analysts to be capable of clearing $1 billion in annual sales.

Lundbeck is facing a hit to its revenues in the coming years as it loses patent protection for antidepressant Trintellix/Brintellix (vortioxetine) and current growth driver Rexulti (brexpiprazole), used to treat depression, schizophrenia, and agitation associated with Alzheimer's dementia. The two drugs alone accounted for around 43% of the company's total revenues last year.

The company sees asedebart coming in to plug the gap when generics of the two brands come to market, along with anti-alpha-synuclein antibody amlenetug for multiple system atrophy (MSA), anti-PACAP drug bocunebart for migraine prevention, and 5-HT 2C receptor super-agonist bexicaserin for seizures associated with rare forms of epilepsy.

Along with Cushing's disease, asedebart is also being tested as a potential treatment for congenital adrenal hyperplasia.