Investigational New Drug (IND) application submitted to
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
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
IND Submitted to
Based on the encouraging clinical results to date, Eloxx has submitted an IND application with the
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.
|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 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
For more information, please visit www.eloxxpharma.com.
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Source: Eloxx Pharmaceuticals