Nerandomilast appears to reduce risk of death in IPF, PPF: trial data
Therapy previously met goal of slowing decline in measure of lung function
Treatment with the experimental therapy nerandomilast appears to reduce the risk of death in people with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF), according to a new analysis of clinical trial data presented by the therapy’s developer, Boehringer Ingelheim.
“The new pooled data zoom in on nerandomilast’s potential as monotherapy, pairing efficacy with a nominally significant reduction in the risk of death. While the findings are exploratory, they add to the growing body of evidence and may impact future research directions in pulmonary fibrosis,” Marlies Wijsenbeek, MD, PhD, of Erasmus MC University Medical Center in the Netherlands, said in a press release from Boehringer Ingelheim.
The new analysis comes from two Phase 3 clinical trials: FIBRONEER-IPF (NCT05321069), which enrolled 1,177 people with IPF, and FIBRONEER-ILD (NCT05321082), which enrolled 1,176 people with PPF. In both studies, participants were randomly assigned to receive treatment with low or high doses of nerandomilast, or a placebo, twice daily for about a year.
Both trials hoped to show that nerandomilast slowed the decline in a measure of lung function. Boehringer announced earlier this year that both studies met this goal, and the company has already submitted applications seeking approval of nerandomilast for IPF and PPF in the U.S., the European Union, and China.
Researchers pooled data from both Nerandomilast studies
In the new analysis, researchers pooled data from both FIBRONEER studies to evaluate how nerandomilast affected the risk of death. Results showed that the risk of death was 43% lower in patients given the high dose (18 mg) of nerandomilast relative to those given the placebo. This difference was nominally significant, which means that the available data suggest the effect is unlikely to be random chance, but did not reach statistical significance.
“For people living with pulmonary fibrosis, mortality remains unacceptably high, with every second person dying within 5 years of diagnosis. As the first Phase III trial program to demonstrate a nominally significant reduction in the risk of death in progressive pulmonary fibrosis, FIBRONEER heralds a significant advance for this group of patients, who face a devastating diagnosis and very limited treatment options,” said Shashank Deshpande, chairman of the board of managing directors and head of human pharma at Boehringer.
Many of the participants in the FIBRONEER studies were receiving background therapy with other pulmonary fibrosis treatments, namely Ofev (nintedanib) or Esbriet (pirfenidone). Other study participants were given the study drug as a monotherapy, or on its own. In patients who received monotherapy, the effect on mortality risk was more pronounced, with a 59% reduction in risk of death for those given high-dose nerandomilast.
The new findings go hand-in-hand with nerandomilast’s favorable safety and tolerability profile in previous studies.
There was also a nominally significant reduction in mortality risk for patients given high-dose nerandomilast who were also receiving background Ofev, with data suggesting a 41% lower risk of death.
In line with previously announced findings, safety data from the pooled analysis indicated that nerandomilast was generally well tolerated, with the most common side effect being diarrhea. Rates of discontinuation due to side effects were similar in patients given nerandomilast or the placebo.
“The new findings go [hand in hand] with nerandomilast’s favorable safety and tolerability profile in previous studies,” Wijsenbeek said. “In a disease area with many patients discontinuing treatment, mostly related to limited tolerability of current therapies, this could really improve outcomes for patients.”
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