Boehringer Ingelheim announced that the Phase III FIBRONEER-IPF trial of nerandomilast met its primary endpoint, demonstrating a statistically significant absolute change from baseline in Forced Vital Capacity (FVC) at week 52 compared to placebo. This outcome marks a potential advancement in the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and often fatal lung disease. The full efficacy and safety data will be presented in the first half of 2025.
Clinical Significance
The FIBRONEER-IPF trial is the largest study conducted to date in IPF, involving over 1177 patients across more than 30 countries and 330 sites. The primary endpoint was the absolute change from baseline in FVC (mL) at Week 52. FVC is a key measure of lung function, and its preservation is a critical goal in managing IPF. A key secondary endpoint was the time to the first occurrence of any of the components of the composite endpoint: time to first acute IPF exacerbation; first hospitalization for respiratory cause; or death (whichever occurs first) over the duration of the trial.
Ioannis Sapountzis, Head of Global Therapeutic Areas at Boehringer Ingelheim, stated, "This is the first IPF phase-III-trial in a decade to meet its primary endpoint. Today’s announcement represents the next step in our long history in the research of this disease. IPF has a high unmet need for patients, and we are continuously fostering our research activities to develop more options for one of the most common interstitial lung diseases."
Nerandomilast: A Novel PDE4B Inhibitor
Nerandomilast (BI 1015550) is an investigational oral, preferential inhibitor of phosphodiesterase 4B (PDE4B). It is currently under investigation as a potential treatment for both IPF and progressive pulmonary fibrosis (PPF). The FDA granted nerandomilast Breakthrough Therapy Designation for IPF in February 2022. The efficacy and safety of nerandomilast have not been established, as it is still an investigational agent.
The FIBRONEER program includes two Phase III randomized, double-blind, placebo-controlled trials: FIBRONEER-IPF (NCT05321069) and FIBRONEER-ILD (NCT05321082). These trials are designed to evaluate the efficacy, safety, and tolerability of nerandomilast over at least 52 weeks in patients with IPF and PPF, respectively.
Trial Design and Dosing
In the FIBRONEER-IPF trial, participants were randomized to receive either oral nerandomilast at twice-daily doses of 9 mg or 18 mg, or placebo, for at least 52 weeks. The inclusion of both 9 mg and 18 mg twice-daily doses allows for evaluation of the benefit-risk profile at different dose levels, as well as providing further dose-response and exposure-response data. The 18 mg twice-daily dose is supported by results from a Phase II study.
About IPF and PPF
Idiopathic pulmonary fibrosis (IPF) is a progressive fibrosing interstitial lung disease (ILD) affecting approximately 3 million people worldwide. Symptoms include breathlessness, a dry cough, chest discomfort, fatigue, and weakness. It primarily affects individuals over the age of 50 and is more common in men.
Progressive pulmonary fibrosis (PPF) can develop in patients with certain types of fibrosing ILDs other than IPF. PPF is characterized by worsening respiratory symptoms, physiological evidence of disease progression, and radiological evidence of disease progression.
Boehringer Ingelheim's Commitment
Boehringer Ingelheim, a biopharmaceutical company, is dedicated to developing innovative therapies to improve and extend lives in areas of high unmet medical need. The company's focus on research and development aims to provide more options for patients with IPF and other respiratory diseases.
