June 14 (Reuters) – Danish drugmaker H. Lundbeck said on Sunday its investigational drug asedebart showed encouraging tolerability and results in a small mid-stage study for the treatment of Cushing’s disease, a rare hormonal disorder.
The results, presented at a medical conference in Chicago, mark a step forward for Lundbeck as it expands into rare conditions beyond its traditional focus on brain diseases such as depression and migraine.
Trial data from 12 patients showed that, after adjusting the drug dose, urinary free cortisol, or the amount of hormone cortisol released by the body in a day, returned to normal in seven out of eight assessable patients, a sign of improvement in Cushing’s disease.
• The drug, given intravenously, was generally well tolerated with no unexpected adverse events, and researchers did not observe any new safety signals
• “All the adverse events we see are very, very consistent with the mode of action of the drug,” Johan Luthman, executive vice president and head of R&D, told Reuters
• Cushing’s disease is a rare hormonal disorder caused by a benign tumour on the pituitary gland that leads to chronic overproduction of cortisol
• Asedebart works by reducing abnormal increases in a hormone called ACTH, which helps bring down the cortisol overproduction
• Lundbeck plans to shift to a new trial cohort examining the drug when administered in subcutaneous injections, Luthman said
• It expects to begin a late-stage trial in the first half of 2027, he added
• Asedebart has already received an orphan drug designation, a status given by regulators for the development of rare disease treatments, for congenital adrenal hyperplasia in the European Union and Japan
(Reporting by Vera Dvorakova in Gdansk and Bhanvi Satija in London; Editing by Milla Nissi-Prussak)
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