Investors and Media

Scientific publications


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

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

Heier CR, Yu Q, Fiorillo AA, et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance vol 2 | no 1 | e201800186

Hoffman EP, Riddle V, Siegler MA, 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, Damsker JM, Hoffman EP, 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.

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

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

Reeves EKM, Hoffman EP, Nagaraju K, et al. (2013). VBP15: Preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. 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