Inhaled pirfenidone solution (AP01) for IPF: a randomised, open-label, dose-response trial

Area of Science:

• Pulmonary Medicine
• Pharmacology
• Clinical Trials

Background:

• Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease.
• Oral pirfenidone is effective but associated with significant systemic side effects.
• Lower doses of oral pirfenidone may not be sufficiently effective.

Purpose of the Study:

• To assess the safety, tolerability, and efficacy of inhaled pirfenidone (AP01) in IPF patients.
• To evaluate dose-response relationship of inhaled pirfenidone.
• To compare inhaled pirfenidone with oral antifibrotic treatments.

Main Methods:

• Phase 1b, randomized, open-label, dose-response trial.
• 25 sites in six countries; ACTRN12618001838202.
• 91 IPF patients received nebulized AP01 (50 mg once daily or 100 mg twice daily) for up to 72 weeks.

Main Results:

• Most treatment-related adverse events were mild/moderate (cough, rash, nausea).
• Week 48: Forced vital capacity (FVC) decline was -4.9 mL in the 50 mg group and -34 mL in the 100 mg group.
• The 100 mg twice-daily dose group showed stable mean FVC % predicted.

Conclusions:

• Inhaled pirfenidone (AP01) demonstrated a favorable safety profile with less frequent side effects compared to oral pirfenidone.
• The 100 mg twice-daily dose of inhaled pirfenidone appears to stabilize lung function in IPF patients.
• Further investigation of inhaled pirfenidone for IPF treatment is warranted.