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.