Boehringer Ingelheim terminated the phase 1/2 Lenticlair 1 trial for BI 3720931, an inhaled lentiviral vector gene therapy designed to treat cystic fibrosis1
The trial launched in late 2024 and enrolled five CF patients, with the primary readout occurring on February 3, 20261
The company discontinued the program after the clinical data failed to support further development1
The trial assessed safety in phase 1 and lung function improvements (forced expiratory volume) in phase 2, measuring air an individual could forcefully exhale during a single breath compared to baseline at Week 81
The safety profile of BI 3720931 was consistent with expectations for an innovative inhaled genetic therapy, according to a Boehringer spokesperson1
The discontinuation does not impact other gene therapy programs at Boehringer, though the company does not list any other clinical gene therapies on its website1
Boehringer is continuing to evaluate verducatib, a DPP1/Cathepsin C inhibitor, in a phase 3 study for CF-associated and non-CF bronchiectasis1
Cystic fibrosis remains a challenging indication for novel treatment developers; other companies including Arcturus Therapeutics and Vertex Pharmaceuticals have also experienced setbacks with CF gene and mRNA therapies1
Sources:
1. https://www.fiercebiotech.com/biotech/boehringer-axes-inhaled-gene-therapy-after-viewing-cystic-fibrosis-data