Eloxx Pharmaceuticals (NASDAQ:ELOX) initiated a Phase 2 trial of ELX-02 for the treatment of nephropathic cystinosis.
Cystinosis is a genetic disease caused by a mutation in the cystinosin gene, which limits production of functional cystinosin proteins. Patients experience an abnormal build-up of cysteine that can damage organs, including the kidneys.
ELX-02 is a small molecule drug candidate designed to restore production of cystinosin and preserve renal function.
Dr. Paul Goodyer, the study’s principal investigator, said that ELX-02 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.”
Six patients will be enrolled in the trial and receive ELX-02 for four weeks at three escalating dose levels. Eloxx plans to report topline data from the trial early in the fourth quarter of 2019.
