Santhera declares positive results from LIONHEART study with Agamree demonstrating unique mineralocorticoid receptor antagonism

Santhera Pharmaceuticals has announced positive findings from the LIONHEART study, confirming vamorolone’s unique role as a mineralocorticoid receptor antagonist (MRA), distinguishing it from other corticosteroids.

The study, involving 30 healthy male subjects, an open-label, placebo- and eplerenone-controlled trial, met its primary goal by demonstrating a significant increase in the urinary sodium/potassium ratio (p<0.0001) in the vamorolone group following a fludrocortisone challenge. This supports vamorolone's dual role as both a dissociative glucocorticoid receptor agonist and an MRA.

Cardiac complications, like cardiomyopathy, are a leading cause of mortality in boys with Duchenne muscular dystrophy (DMD). Corticosteroids and ACE inhibitors have delayed the onset of cardiomyopathy, and MRAs like eplerenone have shown improvements in heart function. The LIONHEART study provides proof of concept for vamorolone’s potential as a cardioprotective treatment, particularly for DMD patients, making it a promising alternative to conventional corticosteroids.