Vamorolone

Recent/key publications and posters

An open-label study to collect long-term safety and efficacy data from boys with DMD who have completed prior studies with vamorolone (GUARDIAN Study). De Vera Ana et al. WMS 2024 conference. View here.

Evaluation of behavioural problems using PARS III in the VISION-DMD study of vamorolone vs prednisone in Duchenne muscular dystrophy (DMD). Henricson Eric et al. EAN conference 2024. View here.

Long-term dose-titration experience with vamorolone in subjects with Duchenne muscular dystrophy (DMD) enrolled in expanded access programs. De Vera Ana et al. ICCBH conference 2024. View here.

Impact of vamorolone, prednisone and placebo on linear growth in the VISION-DMD study, as measured by changes in height over 24 weeks. De Vera A et al. MDA conference 2024, poster M190. View here.

Vamorolone dose titration in expanded access protocols and its impact on rates of weight change in subjects with Duchenne muscular dystrophy (DMD). De Vera A et al. MDA conference 2024, poster M191. View here.

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy. Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. View here.

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy. Guglieri M et al. JAMA Neurology. doi:10.1001/jamaneurol.2022.2480. View here.

Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment. Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293. View here.

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Heier CR et al (2019). Life Science Alliance DOI:10.26508. View here.

Vamorolone has shown less impact than daily prednisone or deflazacort on height and body mass index in patients with Duchenne muscular dystrophy (DMD). Ward et al., WMS 2022, FP.27 - Poster 71. View here.

Feasibility of switch from prednisone to vamorolone in patients with DMD in the VBP15-004 study. Hasham S et al., MDA 2022 Poster presentation. View here

The spine fracture burden in boys with DMD treated with the novel dissociative steroid vamorolone versus deflazacort and prednisone – Leanne Ward, MD, WMS 2022 #FP.03 View here

Results of a double-blind cross-over trial of vamorolone in Duchenne muscular dystrophy (DMD): an alternative to traditional corticosteroids – Eric Hoffman, PhD, WMS 2022 #FP.27 View here

Daily regimens of prednisone, deflazacort, and vamorolone have shown to improve motor function similarly in patients with Duchenne muscular dystrophy – McDonald C et al, WMS 2022 #P.133 View here

Earlier papers and posters

Efficacy and safety of vamorolone during 48-week treatment in patients with Duchenne Muscular Dystrophy (DMD) in the VBP15-004 study. Leinonen M et al. View here

Delayed-start analysis of efficacy outcomes in placebo-to-vamorolone crossover participants in VBP15-004. Dang UJ et al. View here

2.5-years of vamorolone treatment in Duchenne muscular dystrophy: Results of an open label long-term extension. Hoffman E et al. View here

Vamorolone versus corticosteroid real-world experience: Comparisons of 2-year treatment period with NorthStar UK Network and CINRG Duchenne Natural History. Mah J K et al. View here

Evolution of time to stand velocity in glucocorticoid using and non-using patients with DMD. MacDonald CM et al. View here

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy - A 30-Month Nonrandomized Controlled Open-Label Extension Trial. Mah J.K. et al. JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178.

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Hoffman EP et al. (2019). Neurology 93: e1312-e1323.

Population pharmacokinetics of vamorolone (VBP15) in healthy men and boys with Duchenne muscular dystrophy. Mavroudis PD et al. (2019). J Clin Pharmacol. 59 :979-988.

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Heier CR et al. (2019). Life Science Alliance https://doi.org/10.26508/lsa.2... vol 2 | no 1 | e201800186

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Hoffman EP et al. (2018). Steroids 134: 43–52.

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Conklin LS et al. (2018). Pharmacological Research 136: 140–150.

Membrane stabilization by modified steroid offers a potential therapy for muscular dystrophy due to dysferlin deficit. Streetama SC et al. (2018). Molecular Therapy 26: 2231-2242.

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. Heier CR et al. (2013). EMBO Mol Med. 5: 1569–1585

VBP15: Preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Reeves EKM et al. (2013). Bioorg Med Chem. 21: 2241–2249.