Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from patient quality of life.
In November 2018, Santhera acquired from Idorsia the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide (except Japan and South Korea).
Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with participation in funding and design of studies by several international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program. For more information about the vamorolone and the development program click here.
Following single and multiple ascending dose clinical pharmacology studies (VBP15-001, clinicaltrials.gov id NCT02415439) in healthy volunteers3 vamorolone completed a Phase IIa study (VBP15-002; clinicaltrials.gov id NCT02760264) in 48 boys with DMD aged 4 to <7 years. Vamorolone was reported to be safe and well tolerated up to 6.0mg/kg/day, around 10 times the standard corticosteroid dose4. A 6-month extension study (VBP15-003; clinicaltrials.gov id NCT02760277) also demonstrated dose-dependent improvement in timed function tests which was comparable to standard corticosteroid treatment5.
The ongoing Phase IIb VISION-DMD study (VBP15-004; clinicaltrials.gov id NCT03439670) builds on the available promising preliminary safety and efficacy data from Phase IIa and is designed to bridge exploratory biomarker data to clinical outcomes. This pivotal study will enroll approximately 120 boys aged 4 to <7 with DMD that have not yet been treated with corticosteroids, randomized to one of four groups: low dose vamorolone (2 mg/kg/day), high dose vamorolone (6 mg/kg/day), prednisone (0.75 mg/kg/day), or placebo. Additional information can be viewed on the study website.
Vamorolone has received Orphan Drug Designation in the US and in Europe and fast-track status and rare pediatric disease designation from the FDA in the US.
Heier CR at al. (2013). VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 5: 1569–1585
Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186
Hoffman E et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids 134: 43-52
Conklin LS et al. (2018). Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first in-class dissociative steroidal anti-inflammatory drug. Pharmacological Research 136:140-150.
Hoffman et al. (2019) Neurology