Phase 2 clinical trial of PBI-4050 in patients with idiopathic pulmonary fibrosis Academic Article uri icon

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abstract

  • 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.

authors

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

publication date

  • March 2019