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Santhera Pharma has announced the results of its LIONHEART study, which evaluated vamorolone – a corticosteroid – and confirmed its action as a mineralcorticoid receptor antagonist (MRA).

The LIONHEART study was an open-label, randomised, placeboand eplerenone-controlled study involving 30 healthy adult males. The endpoint was met, with data showing a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo following a fludrocortisone challenge.

These findings further differentiate the drug’s pharmacological profile, coupled with its known properties as a dissociative glucocorticoid receptor agonist, which sets it apart from other corticosteroids. Vamorolone is indicated for use in patients with Duchenne muscular dystrophy (DMD) where cardiac complications like cardiomyopathy are a leading cause of morbidity and mortality.

“MRAs are strongly recommended but late when cardiac function is already reduced, and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi PhD MD, cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid.”

Shabir Hasham MD, chief medical officer of Santhera, commented: “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone. We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterise long-term outcomes including any beneficial impact on cardiac complications in DMD.”

DMD is the most common type of MD. In the UK, about 100 boys are born with DMD each year, and there are about 2,500 people living with the condition in the UK at any one time. In the UK, around 70,000 people have muscular dystrophy or a related condition.