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 Khalil, Nasreen
A1 Manganas, Helene
A1 Ryerson, Christopher J.
A1 Shapera, Shane
A1 Cantin, Andre M.
A1 Hernandez, Paul
A1 Turcotte, Eric E.
A1 Parker, Joseph M.
A1 Moran, John E.
A1 Albert, Gary R.
A1 Sawtell, Renata
A1 Hagerimana, Aline
A1 Laurin, Pierre
A1 Gagnon, Lyne
A1 Cesari, Frank
A1 Kolb, Martin
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