ELX-02 demonstrates dose-responsive pronounced increases in functional CFTR and read-through in organoids, human bronchial epithelial (HBE), and Ussing chamber systems
ELX-02 increases CFTR mRNA to healthy control levels
ELX-02 has shown increased CFTR function in organoids bearing nonsense alleles representing >75% of the cystic fibrosis nonsense genotype population
Eloxx to report top line data from Phase 2 clinical trial in cystic fibrosis in 2019
Phase 2 protocol has been reviewed and approved by the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN) and given a score of “high priority”
Eloxx Pharmaceuticals, Inc. (“Eloxx”), (ELOX:Nasdaq), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis, inherited retinal disorders, and other diseases caused by nonsense mutations limiting production of functional proteins, today announced positive data demonstrating that ELX-02 increases functional CFTR protein in organoid, human bronchial epithelial, and Ussing chamber systems and restores CFTR mRNA to healthy control levels. These data were presented at the 42nd European Cystic Fibrosis Society Conference on June 5-8, 2019 in Liverpool, UK.
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