Vamorolone, a new anti-inflammatory, improves motor function in DMD without the side effects of corticosteroids

Vamorolone (VBP15) is a steroid analogue developed by ReveraGen Biopharma, a subsidiary of Santhera Pharmaceuticals. This anti-inflammatory acts like glucocorticoids without having side effects. The intermediate outcomes of the evaluation of vamorolone in a phase II clinical trial lasting 6 months followed by its open label extension which lasted 2 years have just been published in the journal PlosOne.

Medium-term motor improvement with few side effects

In these 2 successive trials, 46 participants, boys aged 4 to 7 years, with Duchenne muscular dystrophy were treated with oral vamorolone. The published results focus on two groups of children who received for 18 months (12 months of the open-label extension and 6 months of the preliminary test) 2 or 6 mg / Kg / day of vamorolone. The results indicate an improvement in the motor performance of the boys. The speed to stand up from the ground, that to climb 4 steps, walk / run for 10 meters, the distance covered during 6 minutes of walking, as well as the overall measurements of motor skills with the NSAA motor scale have significantly higher values. These measurements, apart from those concerning the speed to get up from the ground, were significantly better in boys treated with vamorolone compared to an external control group of 19 boys of the same age with DMD and without treatment (neither vamorolone nor glucocorticoids ), from the CINRG natural history study. The classic side effects of glucocorticoids (growth, weight gain, behavioral problems, water retention) are minimized in this trial.

 

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension studyEdward C Smith , Laurie S Conklin, Eric P Hoffman, Paula R Clemens, Jean K Mah, Richard S Finkel, Michela Guglieri, Mar Tulinius, Yoram Nevo, Monique M Ryan, Richard Webster, Diana Castro, Nancy L Kuntz, Laurie Kerchner, Lauren P Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Phil Shale, Heather Gordish-Dressman, Laura Hagerty, Utkarsh J Dang, Jesse M Damsker, Benjamin D Schwartz, Laurel J Mengle-Gaw, Craig M McDonald, CINRG VBP15 and DNHS Investigators. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. PMID: 32956407; PMCID: PMC7505441.