Investors and Media

Scientific publications


World Muscle Society 2022 - Flash poster presentations and posters

The spine fracture burden in boys with DMD treated with the novel dissociative steroid vamorolone versus deflazacort and prednisone – Leanne Ward, MD, #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, #FP.27 View here

Vamorolone has less impact than daily prednisone or deflazacort on height and body mass index in patients with Duchenne muscular dystrophy (DMD) – Ward L et al, #P.71 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, #P.133 View here

Papers and earlier posters

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.

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

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

Delayed-start analysis of efficacy outcomes in placebo-to-vamorolone crossover participants in VBP15-004. Dank 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 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.

Lonodelestat (POL6014)

Barth P et al. (2020). Single dose escalation studies with inhaled POL6014, a potent novel selective reversible inhibitor of human neutrophil elastase, in healthy volunteers and subjects with cystic fibrosis. J Cyst Fibros; 19: 299-304

Sellier Kessler O et al. (2018). Effect of POL6014, a potent and selective inhaled neutrophil elastase inhibitor, in a rat model of lung neutrophil activation. Am J Respir Crit Care Med; 197: A2988

Lagente V et al. (2009). A novel protein epitope mimetic (PEM) neutrophil elastase (NE) inhibitor, POL6014, inhibits human NE-Induced acute lung injury in mice. Am J Respir Crit Care Med; 179: A5668