Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Cystic Fibrosis
Publication titled: “Targeting G542X CFTR Nonsense Alleles With ELX-02 Restores CFTR Function in Human-Derived Intestinal Organoids”
WALTHAM, Mass., Feb. 09, 2021 (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 a scientific manuscript titled: “Targeting G542X CFTR Nonsense Alleles With ELX-02 Restores CFTR Function in Human-Derived Intestinal Organoids” was published in the Journal of Cystic Fibrosis.
“We are very pleased that our scientific manuscript on the results of our evaluation of ELX-02 mediated read-though, using the CFTR-dependent Forskolin-induced swelling (FIS) assay across a selection of G542X homozygous and heterozygous patient-derived organoids, was published in the Journal of Cystic Fibrosis,” said Dr. Matthew Goddeeris, PhD, Vice President of Research, Eloxx Pharmaceuticals. “ELX-02 increased CFTR activity in a dose-dependent fashion across a variety of forskolin induction concentrations.The functional increases are similar to those obtained with tezacaftor/ivacaftor in an F508del homozygous organoid. Additionally, ELX-02 treatment of these patient-derived organoids results in a 5-fold increase in CFTR mRNA when compared with vehicle treated, resulting in normalization of CFTR mRNA as measured using Nanostring. These data support our current Phase 2 clinical trial for ELX-02 in cystic fibrosis patients with G542X nonsense alleles, for whom there are few, if any, treatment options available. We look forward to reporting top line data in the first half of 2021.”
Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx’s preclinical candidate pool consists of a library of novel drug candidates designed to be eukaryotic ribosomal selective glycosides identified based on read-through potential. Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with operations in Rehovot, Israel and Morristown, NJ. For more information, please visit www.eloxxpharma.com.
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Contact:
Barbara Ryan
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SOURCE: Eloxx Pharmaceuticals, Inc.
