Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis, inherited retinal disorders, and other diseases caused by nonsense mutations limiting production of functional proteins, announced that it has initiated a Phase 2 clinical trial (Study 003) for ELX-02 in cystinosis in Canada.
Study 003 is a single arm, open label study designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) ELX-02 in patients with nephropathic cystinosis with at least 1 nonsense mutation in the cystinosin gene. Six patients will be enrolled in the trial, and will be treated with ELX-02 for four weeks with three escalating dose levels.
“I am excited to have the opportunity to offer ELX-02 to my cystinosis patients, a new potential treatment option which has exhibited positive results in cellular and animal models with dose-dependent reductions in white blood cell cystine levels, a biomarker used to monitor cysteamine therapy and to predict clinical benefit,” said Dr. Paul Goodyer, Professor of Pediatrics at McGill University. “There remains a high unmet medical need in cystinosis, which is diagnosed in infancy, and new therapeutic options with the potential to alter the course of this progress disease could dramatically improve the lives of patients and their families.”
“We are very pleased to have initiated a Phase 2 clinical trial for ELX-02 in cystinosis patients with nonsense mutations in Canada and we are grateful for the support and non-dilutive funding we have received for this trial from Genome Quebec and Genome Canada,” said Dr. Greg Williams, COO of Eloxx Pharmaceuticals. “We look forward to reporting top line data from this trial early in the fourth quarter of this year. We believe that the results to date, as well as the preliminary results from our renal impairment study provide support for both continuing our clinical development programs and evaluating the suitability of our ERSG library for development in additional renal disorders, including autosomal dominant polycystic kidney disease and cystinuria.”
Dr. Goodyer previously reported data at the WORLDSymposium which showed that ELX-02 decreases the cystine content in cellular and animal models.
Dr. Goodyer extended his research to show ELX-02 cystine decreases when used in combination with cystamine. These data were presented in March at the Cystinosis Research Foundation “Day of Hope” meeting in Irvine, California.
In support of the cystinosis program, where preserving renal function is a major concern, Eloxx has completed a renal study in mild, moderate, and severe renal impairment cohorts. Previously, the U.S. Food and Drug Administration granted orphan drug designation for ELX-02 for the treatment of cystinosis and an IND is currently open. ELX-02 is an investigational drug that has not been approved by any global regulatory body.
About 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.