Studies in Cells and Mice

The development of vamorolone (originally called VBP15) included testing in cells to determine if it performed well in terms of inhibiting NFkB danger signals (transrepression), while losing some of the activities of corticosteroids that seem to drive side effects (transactivation). Once vamorolone passed testing in cells, it was then tested in mouse models of Duchenne muscular dystrophy (mdx mouse), where it showed improvements of strength of mice but without the side effects of corticosteroids.

Corticosteroids are prescribed for many conditions, and vamorolone (VBP15) was then tested in a series of mouse models of human disease where corticosteroids are used, including:

• asthma
• multiple sclerosis
• inflammatory bowel disease
• dysferlin-deficient muscular dystrophy (LGMD2B)
• brain tumors
• critical illness muscle disease
• arthritis

In each of the mouse models of human disease, vamorolone appeared to perform as well or better than corticosteroids, while showing less evidence of the side effects seen with corticosteroids.

Vamorolone was then tested for toxicity, pharmacology, and a formulation developed for use in humans.