Eloxx Pharmaceuticals Presents Positive New Data at ARVO


Eloxx Pharmaceuticals, Inc. recently presented new findings related to preclinical inherited retinal disease therapeutic development at the ARVO 2019 Annual Meeting in Vancouver, BC, on May 2, 2019.

“We are very pleased with the preclinical results for several of our ERSG compounds  demonstrating they are suitable for intravitreal injection, well-tolerated in a sensitive model species, active against Usher Syndrome mutations and capable of reaching the retina. We look forward to advancing IND-enabling studies in inherited retinal diseases, with an initial focus on Usher Syndrome,” said Dr. Matthew Goddeeris, Director of Research, Eloxx Pharmaceuticals. Dr. Susan Schneider, Eloxx’s SVP, Clinical Development, Ophthalmology added, “There is a significant unmet medical need and high prevalence of nonsense mutations across inherited retinal diseases, including Usher Syndrome, and we look forward to working closely with key opinion leaders in ophthalmology and the Foundation Fighting Blindness to develop potential treatments for these patients.”

In a Poster presentation at the ARVO Annual Meeting on May 2, 2019 titled Instituting a read-through therapeutic approach to nonsense-mutation based inherited retinal disorders, ELX-03, a translational nonsense mutation read-through agent demonstrates tolerability and activity for use in inherited retinal disorders, presented by Dr. Matthew Goddeeris, Director of Research, at Eloxx, it was reported that:

-Eloxx has screened multiple compounds from its ERSG library of read-through agents for potential use in the treatment of inherited retinal disorders with an initial focus on Usher Syndrome, beginning with USH2A.

-Multiple Eloxx compounds in preclinical studies have demonstrated: Dose-dependent activity against Usher mutations; Restoration of missing Usher Syndrome protein; Favorable tolerability profile at high doses in sensitive species; preserved electroretinogram (ERG) and no compound-related histopathological changes; and Encouraging pharmacokinetics demonstrating retina exposure by   intravitreal injection.

-IND-enabling studies ongoing.

-Next steps include evaluation of patient-derived cell models and sustained release formulations.

At the request of the Foundation Fighting Blindness, Dr. Matthew Goddeeris, also made a presentation at the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit in Vancouver, BC., on April 26, 2019, to review Eloxx Pharmaceuticals’ read-through therapeutic approach to inherited retinal disorders with its library of ERSG molecules. Eloxx compounds are investigational agents not approved by any regulatory agency for therapeutic use.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are designed 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 moving to Phase 2 clinical development focusing on cystic fibrosis and cystinosis. 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 is also advancing a new program focused on rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel. For more information, visit www.eloxxpharma.com.

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