“We were pleased to have the opportunity to present additional preclinical study results in cystic fibrosis at the 2020 NACFC virtual conference that demonstrate ELX-02’s selectivity for read-through of premature stop codons versus native stop codons and its ability to restore production of functional CFTR in patient-derived organoids,” said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. “We believe that these results de-risk the current Phase 2 proof of concept clinical trials for ELX-02 in cystic fibrosis. We are continuing to advance our trials in
The details for the two ELX-02 poster presentations are:
Poster Session Presentation Title: “ELX-02 Generates Protein Via Premature Stop Codon Read-through Without Inducing Native Stop Codon Read-through Proteins”
Poster #: 433
Presenter: Dr. Dan Crawford, Eloxx Pharmaceuticals
- ELX-02 produces significant read-through of premature stop codons leading to full length proteins, demonstrated using DMS-114 cells with the R213X nonsense mutation in the TP53 gene.
- Using three complementary techniques, no evidence of native stop codon read-through products could be detected. These data suggest that ELX-02 does not promote native stop codon read-through at concentrations relevant to premature stop codon read-though.
- The results of studies are consistent with the acceptable tolerability profile of ELX-02 across preclinical and clinical studies to date.
Poster Session Presentation Title: “CFTR Restoration By ELX-02 Across CF Nonsense Genotypes: Utilizing Patient-Derived Organoids to Survey Responsive Alleles”
Poster #: 383
Presenter: Dr. Matthew Goddeeris, Eloxx Pharmaceuticals
- The patient-derived organoid CFTR FIS assay has enabled the screening of a wide selection of cystic fibrosis nonsense alleles representing >75% of the cystic fibrosis nonsense population. Using this method, we continue to identify new responsive genotypes.
- The response of W1282X patient-derived organoids to ELX-02 mediated through read-through positively correlates with CFTR mRNA expression.
- Increasing the available CFTR mRNA pool through inhibition of nonsense mediated decay has a synergistic effect on ELX-02 mediated functional CFTR read-through.
- These results help guide the interpretation of the patient-derived organoid CFTR FIS assay data by highlighting the importance of considering CFTR expression differences across patient-derived organoids for the applicability of ELX-02 as a potential therapeutic option for cystic fibrosis patients with nonsense alleles.
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