Boehringer to File NDA for Pulmonary Fibrosis Drug Post-Phase 3 Results

A Phase 3 clinical trial evaluating Boehringer Ingelheim’s nerandomilast for idiopathic pulmonary fibrosis (IPF) has achieved its primary endpoint, according to topline results disclosed on Monday. This outcome marks a significant milestone, prompting Boehringer to plan a New Drug Application (NDA) submission for the oral medication to the FDA and other regulatory bodies, although the company did not provide a specific timeline for these submissions.

The FIBRONEER-IPF trial focused on the primary endpoint, which was the absolute change from baseline in forced vital capacity (FVC) at 52 weeks when compared to a placebo. Forced vital capacity is a critical measure of lung function, and achieving this endpoint indicates a positive effect of nerandomilast on lung performance in those with IPF.

However, an investor note from Leerink Partners highlighted that Boehringer’s announcement lacked details about the specific dose(s) that met the primary endpoint, as well as information on the safety and tolerability of the drug. Despite these omissions, the positive outcome is seen as beneficial not only for Boehringer but also for other companies in the same therapeutic area, such as Pliant Therapeutics and PureTech Health, both of which are also developing treatments for IPF.

In May, Pliant Therapeutics reported promising data from a mid-stage trial, which suggested their drug might have the potential to reverse fibrosis. Leerink analysts interpreted Boehringer’s success as a promising sign for the broader IPF treatment landscape, which has faced significant challenges in recent years. "In our opinion, this can be seen as a clearing event demonstrating that Phase 3 trials in IPF can succeed, providing a positive counter to the tough track record in IPF trials over the last several years,” the analysts wrote. Nevertheless, they expressed concerns about whether the safety profile of nerandomilast would prove acceptable.

Boehringer’s global head of therapeutic areas, Ioannis Sapountzis, emphasized the significance of the trial’s success, noting that this is the first IPF Phase 3 trial to meet its primary endpoint in a decade. The company plans to disclose the full dataset, including a crucial secondary endpoint, in the first half of next year. This secondary endpoint comprises the time to the first occurrence of acute IPF exacerbation, the first hospitalization for a respiratory cause, or death.

This achievement offers a ray of hope for patients suffering from idiopathic pulmonary fibrosis, a disease characterized by the progressive scarring of lung tissue, which leads to debilitating respiratory issues. Successful Phase 3 trials in this area have been notoriously difficult to achieve, making Boehringer’s recent success a notable breakthrough in the ongoing quest to find effective treatments for this severe and life-threatening condition.