Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Clinical Pharmacology

January 20, 2021
Publication titled: “Phase 1 Renal Impairment Trial Results Enable Targeted Individualized Dosing of ELX-02 in Nephropathic Cystinosis Patients”

WALTHAM, Mass., Jan. 20, 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: “Phase 1 Renal Impairment Trial Results Enable Targeted Individualized Dosing of ELX-02 in Nephropathic Cystinosis Patients,” has been published in the Journal of Clinical Pharmacology.

“We are pleased that our scientific manuscript on the results of our Phase 1 renal impairment trial which defined the relationship of eGFR and drug exposure and urinary clearance in patients with renal impairment has been published in the Journal of Clinical Pharmacology,” said Dr. Thomas Haverty, Chief Medical Officer at Eloxx Pharmaceuticals. “ELX-02 was well tolerated by patients with renal insufficiency and nephropathic cystinosis patients and exhibits a consistent pharmacokinetic profile across increasing degrees of renal insufficiency with reduced clearance, increased exposure, and prolonged renal elimination proportional to eGFR. The data from the renal impairment study enabled the development of an eGFR-PK model of ELX-02 which was successfully used to implement individualized daily dosing of ELX-02 in a Phase 2a study in patients with nephropathic cystinosis to achieve a weekly targeted exposure.” These results further support our ongoing Phase 2 program in cystic fibrosis and IND-enabling studies in autosomal dominant polycystic kidney disease,”

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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Barbara Ryan

SOURCE: Eloxx Pharmaceuticals, Inc.

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Source: Eloxx Pharmaceuticals