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Eloxx Presents Two Preclinical Posters at the 2020 North American Cystic Fibrosis Virtual Conference

WALTHAM, Mass., Oct. 22, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that it presented data from two scientific abstracts at the North American Cystic Fibrosis Virtual Conference (NACFC). The two abstracts were also showcased in the NACFC virtual poster gallery and electronically published as a supplement to Pediatric Pulmonology. The live sessions and discussions will take place through October 23rd, 2020. These virtual posters are available to registered attendees on the NACFC online conference platform.

“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 Europe, Israel and the U.S., and we look forward to reporting top line data from the Phase 2 clinical trial program as quickly as possible.”

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.

About Eloxx Pharmaceuticals

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