Investigational New Drug (IND) application submitted to U.S. Food and Drug Administration (FDA) for ELX-02 for the treatment of Alport syndrome with nonsense mutations

Rebound in average UPCR 3-months post treatment provides further evidence that proteinuria remission in one out of three patients was drug related

All 3 Alport patients (100% response rate) treated with ELX-02 had biopsy confirmed disease regression, suggesting clinical benefit likely with longer treatment duration

Significant strategic interest in ZKN-013 following FDA approval to start Phase 1 single ascending dose (SAD) trial

WATERTOWN, MA, USA I September 07, 2023 I Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today 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.

Alport syndrome is a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, characterized by podocyte injury and impaired kidney filter function leading to proteinuria.

“We have shared the results of our Phase 2 study with several key opinion leaders and key stakeholders within the Alport syndrome community. They have expressed uniform and overwhelming strong support for the potential of ELX-02 to treat this devasting indication and the need to advance into a pivotal study,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

IND Submitted to U.S. FDA for Alport Syndrome

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 (100% response rate) treated with ELX-02 in its proof-of-concept Phase 2 open-label clinical trial (NCT05448755) 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 (loss) 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 today 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.

Patient Average UPCR (mg/g) at baseline Average UPCR (mg/g) during treatment over 8 weeks Average UPCR (mg/g)
3 months post end of treatment
Patient 4401-01 1,299 1,799 2,328
Patient 4401-02* 1,646 850 1,495
Patient 4402-01 1,659 2,209 1,559

*Patient who achieved remission.

Strategic Interest in ZKN-013

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 (RDEB) and junctional epidermolysis bullosa (JEB).

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.

About Eloxx Pharmaceutical

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit

SOURCE: Eloxx Pharmaceuticals