The phase 2 APPLAUD study has shown that LAU-7b reduces loss of lung function in adults with cystic fibrosis (CF).

The results, announced by news release, were presented in poster and oral sessions at the North American Cystic Fibrosis Conference in Philadelphia, Pennsylvania, on November 3.

“The potential clinical benefits of reducing the loss of lung function demonstrated with LAU-7b in the APPLAUD study, especially when administrated on top of CFTR modulators, are very promising and represent a first with a drug candidate addressing inflammation in adults with CF,” said Michael Konstan, MD, vice dean for Translational Research at Case Western Reserve University School of Medicine in Cleveland, and co-lead investigator for the study.

LAU-7b (oral fenretinide) modulates inflammation and protein trafficking in CF by restoring cell membrane lipid balance, which is believed to improve the resolution of pulmonary inflammation and help stabilize CF transmembrane conductance regulator (CFTR) expression during periods of inflammation.

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The APPLAUD study was a 6-month, double-blind, placebo-controlled trial on 166 adults in the US, Canada, and Australia with CF who were randomized to LAU-7b once daily or placebo, in addition to the standard CFTR modulator regimen. The primary objectives were to assess the safety of the medication and its effect on lung function loss by reducing inflammation.

The primary outcome measure was change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at 6 months, and secondary endpoints included pulmonary exacerbations, inflammatory and lipidomic markers, and quality of life.

The results revealed a significant change in ppFEV1 in favor of LAU-7b over the 24-week period, as well as a clinically meaningful reduction of 49% in lung function loss over the same period compared with placebo. The results also showed LAU-7b to have an acceptable safety profile with no unexpected serious adverse events reported.

Reference

Laurent Pharmaceuticals presents results from phase 2 clinical study of LAU-7b in cystic fibrosis at the NACFC 2022. News release. Laurent Pharmaceuticals; November 3, 2022.