Highly regarded renal pathologist and transmission electron microscopy (TEM) expert independently confirms previously reported qualitative assessment by
All three patients treated with ELX-02 showed a visual improvement in podocyte foot process effacement post-treatment in kidney biopsies demonstrating the disease modifying effect of ELX-02
Podocyte foot process effacement is a hallmark of Alport syndrome
Eloxx intends to gain alignment with
Additional independent confirmation has now been reported. The qualitative assessment of images of pre- and post-treatment kidney biopsies from the three patients in the Phase 2 clinical trial noted:
- Patient 4401-01 and Patient 4401-02: All images showed more regions of glomerular basement membranes in post-treatment biopsies covered by intact foot processes, consistent with partial improvement of podocyte injury in those biopsies. As previously announced, Patient 4401-02 achieved remission based on Urine-Protein Creatine ratio (UPCR).
- Patient 4402-01: Post-treatment images showed wider areas of intact foot processes compared with either pre- or post-treatment images for Patient 4401-01 and Patient 4401-02.
These findings are generally consistent with previous assessments, providing stronger evidence of protein restoration and the disease modifying effect of ELX-02 and potential for improvement in proteinuria with longer duration of treatment.
“We are incredibly pleased with this additional confirmation from a globally renowned expert in interpreting the findings from kidney biopsies,” said
Eloxx previously announced achievement of a rapid and sustained remission in one patient in the Phase 2 clinical trial. Based on the results from the Phase 2 trial, Eloxx intends to gain alignment with the FDA on the design of pivotal trial for ELX-02 for the treatment of Alport syndrome with nonsense mutations and the potential for seeking Breakthrough Therapy Designation. Eloxx recently submitted an Investigational New Drug application (IND) to the FDA for ELX-02 for the treatment of Alport syndrome with nonsense mutations. The IND, if allowed, would enable the inclusion of
About Alport syndrome
Alport syndrome is a genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities caused by mutations in the genes (COL4A3, COL4A4, and COL4A5) needed for production of type 4 collagen. Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations. These patients have significantly worse clinical outcomes than other patients with Alport syndrome and have no disease modifying treatment options.
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Source: Eloxx Pharmaceuticals