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New data for better treatment of Duchenne muscular dystrophy

The first part of a pivotal clinical study trialling a new steroid-like drug called vamorolone for treating Duchenne muscular dystrophy (DMD) is now over. Data points to improved motor function and fewer side effects than other steroids.

Health

DMD is a genetic disorder that weakens and damages the muscles in the body over time. Almost always affecting boys, DMD typically begins with muscle loss around the age of four and progresses rapidly. By 12 years of age, most children with DMD are no longer able to walk. As the disease also attacks the heart and lung muscles, patients also experience heart and respiratory complications, with most patients dying by 30 years of age. While there’s no known cure for DMD, medications such as corticosteroids help to slow muscle degeneration and delay disease progression. However, they come with a wide range of side effects that lower patients’ quality of life. A clinical trial conducted with support from the EU-funded VISION DMD project is now testing the efficacy, safety and tolerability of an innovative steroid-like drug called vamorolone as an alternative to current corticosteroids. The first half of the study has showed that vamorolone – discovered by US-based project partner ReveraGen Biopharma, Inc. – improved motor function and had significantly fewer corticosteroid-associated side effects than other steroids. “The use of glucocorticoids, despite having proven benefits in the treatment of DMD, is severely limited due to side effects and poor tolerability. Our expectation is that vamorolone will have the benefits but avoids many of the tolerability issues that limit the use of this standard of care,” noted ReveraGen Biopharma CEO Eric Hoffman in a press release posted on the VISION DMD website. In the first period of the 48-week randomised, double-blind clinical trial, researchers compared the efficacy, safety and tolerability of vamorolone with the corticosteroid prednisone and a placebo. Over a period of 24 weeks, DMD patients aged 4 to less than 7 years and able to walk were randomly assigned either vamorolone at 2.0 mg/kg or 6.0 mg/kg a day, a placebo or prednisone at 0.75 mg/kg per day. The 2 different doses of vamorolone were given to a total of 48 patients, 41 of whom have been treated and evaluated over a 2.5-year period. In the second half of the trial, all boys will receive either the low or high dose of vamorolone.

Potential of vamorolone confirmed

The results so far showed that treatment with vamorolone leads to clinical improvement equivalent to an approximately 2-year delay before the speed with which a patient rises from supine to standing declines. Both daily doses of vamorolone were also found to be safe and well tolerated in the 2.5-year treatment period. Additionally, fewer side effects such as behavioural changes and abnormal hair growth were reported for the steroid-like drug, which – unlike corticosteroids prednisone and deflazacort – also didn’t stunt growth. “These findings indicate the long-term maintenance of treatment effect and disease modifying potential with vamorolone,” stated Dario Eklund, CEO of Swiss company Santhera Pharmaceuticals that is developing the drug in collaboration with ReveraGen Biopharma. “We are expecting the 6-month results from the pivotal Phase 2b VISION-DMD study in Q2-2021 and are confident that they will provide further evidence to establish vamorolone as an effective treatment and valuable alternative to corticosteroids for the long-term treatment for DMD,” he went on to say in a press release posted on ‘PharmiWeb.com’. The 5-year VISION DMD (VISION-DMD – Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy) project ends in December 2021. For more information, please see: VISION DMD project website

Keywords

VISION DMD, Duchenne muscular dystrophy, DMD, vamorolone, corticosteroid