RT Journal Article SR Electronic T1 Phase 2 clinical trial of PBI-4050 in patients with idiopathic pulmonary fibrosis JF European Respiratory Journal JO Eur Respir J FD European Respiratory Society SP 1800663 DO 10.1183/13993003.00663-2018 VO 53 IS 3 A1 Nasreen Khalil A1 Helene Manganas A1 Christopher J. Ryerson A1 Shane Shapera A1 Andre M. Cantin A1 Paul Hernandez A1 Eric E. Turcotte A1 Joseph M. Parker A1 John E. Moran A1 Gary R. Albert A1 Renata Sawtell A1 Aline Hagerimana A1 Pierre Laurin A1 Lyne Gagnon A1 Frank Cesari A1 Martin Kolb YR 2019 UL http://erj.ersjournals.com/content/53/3/1800663.abstract AB PBI-4050 is a novel orally active small-molecule compound with demonstrated anti-fibrotic activity in several models of fibrosis, including lung fibrosis. We present results from our first clinical study of PBI-4050 in patients with idiopathic pulmonary fibrosis (IPF).This 12-week open-label study explored the safety, efficacy and pharmacokinetics of daily oral doses of 800 mg PBI-4050 alone and in combination with nintedanib or pirfenidone in patients with predominantly mild or moderate IPF. Nine patients received PBI-4050 alone, 16 patients received PBI-4050 with nintedanib and 16 patients received PBI-4050 with pirfenidone.PBI-4050 alone or in combination with nintedanib or pirfenidone was well tolerated. Pharmacokinetic profiles for PBI-4050 were similar in the PBI-4050 alone and PBI-4050+nintedanib groups but reduced in the PBI-4050+pirfenidone group, suggesting a drug–drug interaction. There were no significant changes in forced vital capacity (FVC), either in % predicted or mL, from baseline to week 12 for PBI-4050 alone or PBI-4050+nintedanib. In contrast, a statistically significant reduction (p<0.024) in FVC % pred was seen for PBI-4050+pirfenidone after 12 weeks.There were no safety concerns with PBI-4050 alone or in combination with nintedanib or pirfenidone in IPF patients. The stability of FVC between baseline and week 12 looked encouraging for PBI-4050 alone and in combination with nintedanib.PBI-4050 alone and in combination with nintedanib demonstrated no safety concerns and showed encouraging results for lung function in IPF patients http://ow.ly/olQD30myD0E