Boehringer Succeeds in Phase III Pulmonary Fibrosis Trial, Eyes FDA Application

Boehringer Ingelheim's investigational drug nerandomilast has achieved a key milestone by meeting its primary endpoint in a Phase III clinical trial for idiopathic pulmonary fibrosis (IPF). The German pharmaceutical company announced that the oral medication significantly improved lung function in patients with IPF, a chronic and progressive lung disease, over a 52-week period when compared to a placebo.

The results come from the Phase III FIBRONEER-IPF study, which enrolled nearly 1,200 patients across more than 30 countries. Participants received either a high or low dose of nerandomilast or a placebo, and their lung function was assessed over the course of a year. While Boehringer Ingelheim has yet to disclose specific data, the company confirmed that the primary endpoint—improvement in forced vital capacity—was met. Full data and analyses are expected to be presented in the first half of 2025.

Idiopathic pulmonary fibrosis is a serious condition characterized by persistent cough, fatigue, chest discomfort, and breathlessness during physical activities. The disease falls under the broader category of interstitial lung diseases and poses a significant unmet need for effective treatments. According to Ioannis Sapountzis, head of global therapeutic areas at Boehringer Ingelheim, the successful outcome of the Phase III trial is a crucial step in addressing this high unmet need.

Nerandomilast works by targeting and inhibiting the phosphodiesterase 4B (PDE4B) enzyme, which plays a significant role in lung fibrosis and inflammation. By disrupting these biological pathways, the drug is designed to exert both anti-fibrotic and anti-inflammatory effects, thereby improving lung function and potentially altering the disease course. The FDA recognized the potential of nerandomilast by granting it Breakthrough Therapy designation for IPF in 2022.

The Phase III FIBRONEER-IPF study was a double-blinded, randomized, placebo-controlled trial. In addition to measuring lung function, the study assessed several secondary outcomes such as the time to the first acute IPF exacerbation or death, and the time to first respiratory hospitalization or death. This comprehensive approach aims to provide a robust evaluation of the drug’s efficacy and safety profile.

Sapountzis highlighted that FIBRONEER-IPF is the first Phase III trial for IPF in a decade to meet its primary endpoint, underscoring the significance of these findings. Beyond IPF, Boehringer Ingelheim is also investigating nerandomilast for progressive pulmonary fibrosis through the Phase III FIBRONEER-ILD study, further broadening the scope of potential applications for the drug.

The recent success in the Phase III trial positions nerandomilast as a promising candidate to potentially succeed Boehringer Ingelheim’s current IPF treatment, Ofev (nintedanib). Ofev has been a strong performer for the company, generating over $3.9 billion in revenue in 2023 with a 12.8% year-over-year growth. The positive results from the FIBRONEER-IPF study could pave the way for nerandomilast to become a key player in the treatment landscape for IPF and possibly other fibrotic lung diseases.

Boehringer Ingelheim plans to leverage the findings from FIBRONEER-IPF to support regulatory submissions for nerandomilast worldwide, including with the FDA. If approved, the drug could offer a new and effective treatment option for patients suffering from idiopathic pulmonary fibrosis, addressing a critical unmet medical need.