Eloxx Pharmaceuticals reports results of patient biopsy assessment by NIPOKA

Eloxx Pharmaceuticals reported results from an assessment of patient biopsies by NIPOKA GmbH. They have developed a highly accurate method for the quantification of podocyte foot process morphology. These results confirm previously reported positive biopsy results from the proof-of-concept Phase 2 open-label clinical trial of ELX-02 for the treatment of Nonsense Mutation Alport syndrome patients. Analysis of formalin-fixed paraffin-embedded biopsy samples by Nipoka show ELX-02 treatment improved podocyte foot process morphology with lower effacement in all three patients at the end of the 8-week study period. NIPOKA GmbH have developed a Podocyte Exact Morphology Procedure to quantify podocyte foot process morphology accurately and precisely in an unbiased and reproducible manner. PEMP utilizes immunostaining for foot-process specific protein markers followed by 3D-SIM imaging to quantify Filtration Slit Density for 15 to 20 glomeruli per sample. FSD is a quantitative measure of the degree of podocyte foot process effacement. Higher FSD correlates with better podocyte health and lower podocyte foot process effacement. Healthy patients have an FSD of approximately 3.0 to 4.0. This analysis has been validated in multiple glomerular diseases. PEMP analysis confirmed that ELX-02 treatment improved podocyte foot process effacement in all three patients with an average post-treatment increase in FSD of 60% as compared to baseline levels. These findings are also consistent with previous Transmission Electron Micrograph image assessments. Differences in Urine Protein-Creatinine ratio changes across patients during treatment were correlated to severity of disease at baseline. Therefore, improvement in UPCR was assessed both during and 2 months after treatment to evaluate clinical benefit and capture the full effect of the 45-day protein half-life. All patients had proteinuria stabilization or improvement. This is consistent with clinical benefit and with the improvement in kidney morphology. Renowned key opinion leaders have reviewed these data and overwhelmingly believe that they provide strong evidence of the potential of the disease modifying effect of ELX-02 and warrant advancement into a pivotal trial.