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JAMA Neurology Publishes Positive Pivotal Clinical Trial with Vamorolone in Duchenne Muscular Dystrophy

Santhera Pharmaceuticals Holding AG
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Santhera Pharmaceuticals Holding AG
Santhera Pharmaceuticals Holding AG

  • Results from the VISION-DMD study showing efficacy and safety of vamorolone compared to placebo and to standard of care, prednisone, has been published in JAMA Neurology

  • Safety analysis on bone biomarkers and growth showed no negative effects of vamorolone

  • Regulatory filings for vamorolone with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) are planned to be completed in Q3-2022 and Q4-2022, respectively

Pratteln, Switzerland, and Rockville, MD, USA, September 1, 2022 –Santhera Pharmaceuticals (SIX: SANN) and ReveraGen (US: Private) announce that JAMA Neurology has published positive results of the 24-week primary efficacy and safety analysis from the VISION-DMD study evaluating vamorolone, an investigational drug for the treatment Duchenne muscular dystrophy (DMD). Vamorolone met its primary endpoint by demonstrating statistically significant and clinically relevant improvement in time to stand from floor compared to placebo, the first functional milestone to deteriorate in young children with DMD. Consistent results across multiple secondary endpoints support the results of the primary endpoint. The relative efficacy of vamorolone 6 mg/kg/day was comparable to that seen with prednisone 0.75 mg/kg/day across primary and secondary efficacy endpoints. Over the 24-week treatment period, no negative impact on biomarkers of bone health and no loss of linear growth were observed with vamorolone. Vamorolone was generally safe and well tolerated. The most commonly reported adverse events versus placebo study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

“Data from the VISION-DMD study continues to validate our ambition of developing a steroidal like treatment where we can retain the efficacy of traditional corticosteroids and reduce some of the toxicities that all too often lead to the premature discontinuation of treatment in children with DMD,” said Eric Hoffman, PhD, Professor of Pharmaceutical Sciences, Binghamton University - SUNY, and CEO of ReveraGen.

“Corticosteroids remain a cornerstone of treatment for many children with Duchenne muscular dystrophy and data published on the VISION-DMD study are an important advancement in the development of additional treatment options for these patients,” said Michela Guglieri, MD, Senior Lecturer and Consultant Neurologist, Newcastle University.

“Vamorolone has been developed as a potential first-in-class dissociative steroidal therapy,” said Shabir Hasham, MD, Chief Medical Officer of Santhera. “This pivotal data as well as other data presented at scientific congresses during 2022 continue to define vamorolone’s differentiated safety profile.”

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