Novel Antifibrotic Designated Breakthrough Therapy for Progressive Pulmonary Fibrosis

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to BMS-986278, an investigational lysophosphatidic acid receptor 1 (LPA₁) antagonist, for the treatment of progressive pulmonary fibrosis (PPF).

The designation was based on data from a phase 2 study (ClinicalTrials.gov Identifier: NCT04308681) evaluating the novel antifibrotic therapy in patients with idiopathic pulmonary fibrosis (IPF) and PPF. Study participants were randomly assigned to receive BMS-986278 or placebo orally twice daily. In the PPF cohort, patients were allowed to take background antifibrotics and/or immunosuppressants.

Among PPF patients, treatment with BMS-986278 (60mg taken twice daily) reduced the rate of decline in percent predicted forced vital capacity by 69% compared with placebo. The treatment effect was found to be consistent in the presence or absence of background antifibrotics. As for safety, the rates of adverse events were reported to be similar between the groups.

“People living with pulmonary fibrosis face deteriorating lung function, worsening respiratory symptoms and reduced quality of life, which can ultimately lead to respiratory failure and death,” said Roland Chen, MD, senior vice president and head, Immunology, Cardiovascular and Neuroscience Development, Bristol Myers Squibb. “The FDA’s Breakthrough Therapy designation underscores the potential of BMS-986278 as an innovative, first-in-class treatment that may redefine the standard of care for progressive pulmonary fibrosis.”

The Company is planning a phase 3 study (ClinicalTrials.gov Identifier: NCT06025578) to evaluate the efficacy, safety, and tolerability of BMS-986278 in PPF patients.  

BMS-986278 was previously granted Fast Track and Orphan Drug designations for the treatment of IPF.