A multinational clinical trial found that the drug candidate did not significantly slow the progression of idiopathic pulmonary fibrosis compared to a placebo.

RT’s Three Key Takeaways:

  1. No Difference in Lung Function: The study found no significant difference in lung function decline, measured by forced vital capacity (FVC), between patients treated with pamrevlumab and those given a placebo after 48 weeks.
  2. No Impact on Symptoms or Survival: There were no significant differences in symptom exacerbations, hospitalizations, or death rates between the pamrevlumab group and the placebo group.
  3. Recommendations for Future Trials: The study’s lead author emphasized the need for future IPF trials to focus more on patient-centered outcomes, such as symptoms, quality of life, and survival, rather than relying heavily on lung function tests.

An investigational drug for idiopathic pulmonary fibrosis (IPF) proved to be no better than a placebo at decreasing the rate of disease progression among participants in a multinational clinical trial. 

In earlier trials, the drug pemrevlumab appeared to slow disease progression. The trial’s results were published in JAMA, the Journal of the American Medical Association. 

Current Treatment Landscape

Only two drugs, pirfenidone and nintedanib, have been approved by the US Food and Drug Administration for IPF. Both slow patients’ decline in lung function, but not everyone who takes the drugs reports relief of symptoms.

The drug evaluated in the new study, pamrevlumab, is a monoclonal antibody that binds to and inhibits a molecule called connective tissue growth factor, which plays a key role in the development of fibrosis. In phase 2 trials, the drug slowed the decline in lung function and was well tolerated.

Clinical Trial Findings

In the new trial, 356 patients were randomly assigned to receive either pamrevlumab or an inactive placebo intravenously every three weeks for 48 weeks. Changes in lung function were assessed with a test called forced vital capacity (FVC), which assesses the elasticity of lungs and their ability to expand. With IPF, elasticity declines as lung scarring progresses.

The researchers found that after 48 weeks of treatment there was no difference in the FVCs of patients who received pamrevlumab and those who received the placebo. There also were no significant differences in exacerbations of symptoms, hospitalizations or death. 

Future Directions for IPF Trials

The paper’s lead author Ganesh Raghu, MD, professor of medicine and director of the Center for Interstitial Lung Diseases at the University of Washington School of Medicine in Seattle, says in a release that “the failure of a drug that looked so promising” underscores the recommendations of a recent report he coauthored.

To speed the identification and evaluation of new drugs, the report urged researchers to decrease the emphasis of lung function tests as primary outcomes and give more weight to improvements in patients’ symptoms and quality of life.

“IPF trials should focus on patients’ experiences—how they feel, function, and how long they survive—not just measures like FVC,” he says in a release.

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