Boehringer Seeks Approval for Lung Fibrosis Drug After Phase III Success

Boehringer Ingelheim is moving forward with regulatory approval for its drug nerandomilast after a successful Phase III trial in idiopathic pulmonary fibrosis (IPF). This trial, named FIBRONEER-IPF (NCT05321069), achieved its primary endpoint by improving forced vital capacity, a key measure of lung function, over a 52-week period. Full efficacy and safety data from this study are anticipated to be presented in the first half of 2025.

This achievement marks a significant milestone as it is the first late-stage IPF trial in a decade to meet its primary endpoint. Boehringer Ingelheim has a history of success in this therapeutic area. In 2014, their tyrosine kinase inhibitor (TKI) Ofev (nintedanib) was approved by the US Food and Drug Administration (FDA) for the treatment of IPF. Since then, Ofev has also been approved for treating interstitial lung disease associated with systemic sclerosis or scleroderma (SSc-ILD) and chronic interstitial lung disease. The drug has been commercially successful, generating €3.5 billion ($3.89 billion) in global sales last year.

The FIBRONEER-IPF trial enrolled 1,177 patients with IPF. Participants were randomized to receive either nerandomilast or a placebo, taken twice daily. The study's secondary endpoint includes measuring the time to the first occurrence of acute IPF exacerbation, first hospitalisation for respiratory causes, or death.

Nerandomilast functions as a preferential inhibitor of phosphodiesterase 4B (PDE4B) and received breakthrough therapy designation from the US FDA for IPF treatment in 2022. Additionally, Boehringer is conducting a Phase III FIBRONEER-ILD trial (NCT05321082) to investigate nerandomilast's efficacy in patients with progressive fibrosing interstitial lung diseases. This trial is expected to be completed by the end of the year.

IPF remains a condition with significant unmet medical needs, as it currently has only two FDA-approved medications: Boehringer's Ofev and Roche’s Esbriet (pirfenidone). While Roche's drug also received FDA approval in 2014, it has faced stiff competition from generic versions. Consequently, Esbriet's sales have declined, dropping from SFr718 million ($752 million) in 2022 to CHF202 million ($239 million) last year.

Most treatments for IPF that are presently in development are in Phase II clinical trials. For example, in May, Vicore Pharma announced that its Phase IIa trial of the angiotensin II type 2 receptor (AT2R) agonist buloxibutid met both its primary and secondary endpoints. Similarly, Bridge Biotherapeutics is conducting a Phase II trial for its autotoxin inhibitor, BBT-877, which is expected to yield topline data in the first half of 2025.

In summary, Boehringer Ingelheim's progress with nerandomilast offers hope for more effective treatments for IPF, a disease with limited therapeutic options. The successful Phase III trial positions nerandomilast as a promising candidate to address the high unmet needs in the treatment of IPF.