Eloxx Pharmaceuticals Plans To Take ELX-02 Into Pivotal Trial For Alport Syndrome

  • Eloxx Pharmaceuticals Inc ELOX intends to advance ELX-02 into a pivotal trial for treating Alport syndrome with nonsense mutations following the achievement of remission in one patient in its Phase 2 study
  • Alport syndrome is a rare genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss, and eye abnormalities.
  • The patient demonstrated a significant reduction in urine protein creatinine ratio (UPCR) from baseline. 
  • Consistent with prior clinical studies, ELX-02 was well tolerated in this trial.
  • Eloxx has dosed three patients with ELX-02 in the ongoing proof-of-concept Phase 2 open-label clinical trial. Two patients have completed dosing, and the third is still receiving ELX-02.
  • One patient who completed dosing achieved a remission, defined as a UPCR decline greater than or equal to 50%. 
  • Five out of eight UPCR readings produced an average of 53% below baseline for this patient. 
  • Spontaneous reductions in proteinuria are not expected in this population, adding weight to this result. 
  • The patient achieving remission also demonstrated a significant reduction (-49%) (-p-value p=0.009) in UPCR from baseline versus values collected over the treatment period.
  • No significant change in UPCR was seen in the other patient, who completed eight weeks of dosing.
  • Eloxx intends to provide more detailed results from the trial at an upcoming medical meeting.
  • Price Action: ELOX shares are down 5.12% at $8.15 on the last check Wednesday.
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