Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes.

ABSTRACT:

Background

Glucocorticoid drugs are highly effective anti-inflammatory agents, but chronic use is associated with extensive pharmacodynamic safety concerns that have a considerable negative impact on patient quality of life.

Purpose

Vamorolone (VBP15) is a first-in-class steroidal multi-functional drug that shows potent inhibition of pro-inflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor, high affinity antagonism for the mineralocorticoid receptor, and membrane stabilization properties. Pre-clinical data in multiple mouse models of inflammation showed retention of anti-inflammatory efficacy, but loss of most or all side effects.

Experimental approach

We report first-in-human Phase 1 clinical trials (86 healthy adult males), with single

SUBMITTER: Hoffman EP 

PROVIDER: S-EPMC6136660 | biostudies-literature | 2018 Jun

REPOSITORIES: biostudies-literature