Efficacy and safety of LAU-7b in a Phase 2 trial in adults with cystic fibrosis

Document Type

Article

Publication Date

1-1-2025

Journal Title

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society

MeSH Headings

Humans; Cystic Fibrosis (drug therapy, physiopathology); Male; Female; Double-Blind Method; Adult; Treatment Outcome; Forced Expiratory Volume; Cystic Fibrosis Transmembrane Conductance Regulator (genetics); Middle Aged; Respiratory Function Tests (methods); Young Adult

Abstract

BACKGROUND: Lung inflammation is associated with tissue damage in cystic fibrosis (CF). LAU-7b, a novel oral drug candidate, was shown to control inflammation and stabilize CFTR protein in the epithelial membrane during inflammatory stress in preclinical models of CF. METHODS: A double-blind, randomized, placebo-controlled Phase 2 study was conducted to evaluate efficacy and safety of LAU-7b in adults with CF. LAU-7b or placebo was administered over 24 weeks as six 21-day treatment cycles each separated by 7 days. The primary efficacy endpoint was the absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV) at 24 weeks. RESULTS: A total of 166 subjects received at least one dose of study drug (Intent-To-Treat population, ITT), of which 122 received ≥5 treatment cycles (Per-Protocol population, PP). Both treatment arms showed a mean lung function loss at 24 weeks of 1.18 ppFEV points with LAU-7b and 1.95 ppFEV with placebo, a 0.77 ppFEV (40 s) difference, p=0.345, and a 0.95 ppFEV (49 %) difference in the same direction in PP population, p=0.263. Primary analysis of mean ppFEV through 24 weeks showed differences of 1.01 and 1.23 ppFEV, in the ITT (65 % less loss, p=0.067) and PP populations (78 % less loss, reaching statistical significance p=0.049), respectively. LAU-7b had an acceptable safety profile. CONCLUSION: Although the study did not meet its primary efficacy endpoint in the ITT population, LAU-7b was generally well tolerated and showed evidence of preservation of lung function to support further development.

First Page

83

Last Page

90

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