Eloxx Pharmaceuticals provides updates on ELX-02, ZKN-013

Eloxx Pharmaceuticals provided an update on the continued advancement of ELX-02 for the treatment of Alport syndrome with nonsense mutations, including additional positive results from its Phase 2 clinical study evaluating ELX-02, as well as an update on ZKN-013. Based on the encouraging clinical results to date, Eloxx has submitted an IND application with the U.S. FDA for ELX-02 for the treatment of Alport syndrome with nonsense mutations. Allowance of the IND will allow for the inclusion of U.S.-based sites in the planned pivotal trial. Biopsy Results from ELX-02 Phase 2 Alport Trial Support Protein Restoration: All three patients treated with ELX-02 in its proof-of-concept Phase 2 open-label clinical trial showed an improvement in podocyte foot process effacement, a hallmark of proteinuric kidney diseases like Alport syndrome. Electron microscopy images of biopsy samples showed an improvement in the glomerular basement membrane and a re-attachment of podocyte foot processes in all treated patients. This confirms protein restoration and the disease modifying effect of ELX-02 and potential for improvement in proteinuria with longer duration of treatment. Podocytes are specialized cells that bind to the glomerular basement membrane and form finger-like extensions called foot processes that enable efficient ultrafiltration. Podocyte injury leads to the effacement of podocyte foot processes and proteinuria in nearly all cases of Alport syndrome. Eloxx previously announced achievement of remission in one patient Eloxx is announcing 3 month post treatment results. The increase in UPCR following withdrawal of therapy confirms likely on-treatment remission in one out of three patients, providing additional evidence of drug activity. As we previously announced, the FDA has cleared the company to begin a SAD trial in healthy volunteers for ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa and junctional epidermolysis bullosa. Eloxx has since received significant strategic interest in ZKN-013, which the company is actively pursuing. Should these discussions lead to a transaction, it will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform.