ELX-02 shows significant increases in CFTR protein expression and localization correlating with functional activity in Cystic Fibrosis patient-derived organoids bearing CFTR nonsense mutations
In previous studies, the increased CFTR functional activity associated with ELX-02 was shown to correlate with increases in CFTR mRNA with elevations to wild-type levels
ELX-02 appears to increase CFTR mRNA stability, suggesting that ELX-02 may be modulating nonsense mediated decay
Eloxx Pharmaceuticals, Inc. (“Eloxx”), (NASDAQ: ELOX), 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, for the first time in organoids, that ELX-02 significantly increases CFTR protein expression and localization on the apical surface in organoids with nonsense mutations. These findings were presented today at the ECFS Basic Science Conferencein Croatia.
“ELX-02 is the first read-through agent to demonstrate significant increases in CFTR protein expression, localization on the apical surface, and functional activity in a dose responsive manner in organoids derived from cystic fibrosis patients with nonsense mutations” said Dr. Matthew Goddeeris, Research Director at Eloxx Pharmaceuticals. Robert Ward, Chairman and CEO, added, “We believe these ground-breaking new data establish a solid basis for understanding the activity of ELX-02 and it’s potential for development in the treatment of the high unmet medical need cystic fibrosis patients with nonsense mutations. We look forward to reporting top line data in 2019 from our planned Phase 2 cystic fibrosis clinical trial.”
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