Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA modulation drug candidates designed to be Eukaryotic Ribosomal Selective Glycosides (ERSG) for the treatment of rare and ultra-rare premature termination codon diseases. Premature termination codons are point mutations that disrupt protein synthesis in messenger RNA. Eloxx’s lead investigational candidate, ELX-02, is a small molecule drug candidate aimed at restoring the production of full-length functional proteins. ELX-02 is currently in Phase 2 clinical trials for cystic fibrosis. ELX-02 is an investigational drug not yet approved by any global regulatory authority. Eloxx’s preclinical library of drug candidates includes a novel ERSG library identified based on read-through potential. Eloxx is currently conducting IND-enabling studies for kidney diseases such as Autosomal Dominant Polycystic Kidney Disease (ADPKD) and rare inherited ocular conditions such as Usher Syndrome.