Idiopathic Pulmonary Fibrosis
What is Idiopathic Pulmonary Fibrosis (IPF)?
IPF is a chronic, devastating, and ultimately fatal disease characterized by a progressive decline in lung function. It is a specific type of interstitial lung disease in which the small air sacs of the lung, the “alveoli,” gradually become replaced by fibrotic (scar) tissue, reducing the ability of the lung to function properly. The term ‘idiopathic’ is used when the cause of the pulmonary fibrosis is unknown. IPF usually occurs in adult individuals of between 50 and 70 years of age, particularly those with a history of cigarette smoking, and it affects men more often than women.
Patients with IPF generally have a poor prognosis with a mean survival between two to five years, and there is a high unmet medical need for safe and well-tolerated medicines that can halt, and potentially reverse, its progression.
Prevalence of IPF:
IPF is a chronic, relentlessly progressive fibrotic disorder of the lungs that typically affects adults over the age of 40. There are approximately 200,000 patients with IPF in the U.S. and Europe, with 75,000 newly diagnosed patients per year. As such, IPF is considered a rare disease.
Orphan drug development for IPF:
In January 2017, we completed an open-label, single arm, exploratory, observational Phase 2 trial of fezagepras, both as a monotherapy and in combination with either nintedanib or pirfenidone, in 41 patients with IPF. The primary endpoints of this trial were safety and tolerability and the key secondary endpoints of this trial included change in pulmonary function and change in inflammatory and fibrotic markers. Nine patients received 800 mg doses of fezagepras alone, 16 patients received 800 mg oral doses of fezagepras in combination with nintedanib and 16 patients received 800 mg doses of fezagepras in combination with pirfenidone, with 40 subjects completing the trial as planned (n= 9, 16 and 15) respectively; all administered orally on a daily basis for 12 weeks. The full results of this trial were published in the European Respiratory Journal in 2018.
Fezagepras (800 mg) was well tolerated alone and in combination with either nintedanib or pirfenidone. Importantly, there were no serious adverse events requiring fezagepras’ discontinuation.
Fezagepras has been granted Orphan Drug Designation by the FDA and the EMA for the treatment of IPF.