Vamorolone, a novel compound for the treatment of Duchenne Muscular Dystrophy, holds potential to better preserve muscle function and prolong ambulation for the patient, without some of the side effects associated with glucocorticoid therapy.
ALLSCHWIL, SWITZERLAND, and ROCKVILLE (MD), USA – 03 November 2016 –Actelion Ltd (SIX: ATLN) and ReveraGen BioPharma, Inc., a privately held company engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases, announced today that they have entered into an agreement. By this agreement, Actelion has obtained an exclusive option to in-license ReveraGen’s lead compound vamorolone for the treatment of Duchenne Muscular Dystrophy at two different stages in its development.

Enrollment has begun in the Phase 2a clinical trial of vamorolone (VBP15) in the treatment of boys with Duchenne muscular dystrophy (DMD). The Phase 2a trial sponsored by ReveraGen BioPharma, is an open-label, multiple ascending dose study that will be followed by a 2a extension trial. The Phase 2a trials are part of the VISION-DMD project: An Innovative Steroid-like Intervention on DMD.

PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and animal data which suggests it has none of the side effects of steroids, does not impact linear growth, and does not promote insulin resistance. NIH has awarded $3 million for Vamorolone to continue clinical studies in boys 4 -7 years of age. PPMD is proud to be an early funder of Vamorolone and are hopeful that this compound will one day prove to be an alternative option for steroids.