Alport syndrome is a rare progressive hereditary glomerular kidney disease caused by variants in COL4A and patients have significant unmet medical needs, with no disease modifying treatments currently available
Eloxx announced additional results from its Phase 2 ELX-02 trial showing the patient that achieved a remission rebounded one month after withdrawing treatment, providing additional evidence of drug activity
Eloxx intends to advance into a pivotal trial of ELX-02 for the treatment of Alport syndrome with nonsense mutations
The event featured two globally renowned Alport syndrome experts:
Detlef Bockenhauer, MD., Ph.D., Professor of Paediatric Nephrology, University Hospitaland KU Leuven
Rachel Lennon, Ph.D., Professor of Nephrology, Consultant Paediatric Nephrologist at the Royal Manchester Children's Hospital, Director of the Wellcome Centre for Cell-Matrix Researchat the University of Manchester, Director of the Stoneygate and Kidney Research UKAlport Research Hub
“Alport Syndrome is a progressive disease caused by a genetic defect in Collagen Type IV protein. There's no approved therapy currently and our current standard of care is supportive care,” said Professor
“In one of these three patients, there has been a substantial reduction in the proteinuria, pretty much half of it. So it is consistent with the remission of proteinuria,” said
“The two drugs that were previously tested but failed in Alport did not have the biological plausibility that is as strong as we have here [with ELX-02],” added
Eloxx provided topline data for 8-weeks of treatment for the third patient in the study and data after 4, and 8-weeks after end of treatment in all three patients that have completed treatment.
|Patient||Average change in Urine Protein to Creatinine (UPCR) during treatment over 8 weeks||UPCR levels in patients at 4 weeks and 8 weeks after end of treatment|
|Patient 4401-01||-49% (Achieved remission) (p=0.009)||Regressed to baseline (+97% vs. end of treatment)|
|Patients 4401-01 and 4402-01||No change||No change|
The rapid increase in UPCR after end of treatment in one patient who achieved remission during the trial provides additional evidence of biological activity of ELX-02 in this population. As observed in prior clinical studies, ELOX-02 was well tolerated in this trial. Based on these results, Eloxx intends to advance ELX-02 into a pivotal trial for the treatment of Alport syndrome with nonsense mutations.
“Eloxx’s recent announcement that one Alport syndrome patient in its Phase 2 trial of ELX-02 achieved remission is an extremely important milestone in addressing the needs of these patients have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options,” said
A replay of the webcast and presentation is available on www.eloxxpharma.com under "Events & Presentations" in the Investors section of the website for 30 days.
“We were delighted to bring more attention to the debilitating unmet medical needs of Alport syndrome patients during this event,” said
About the Phase 2 Clinical Study
This Phase 2 trial included Alport syndrome patients with nonsense mutations in the COL4 gene. Patients were dosed with ELX-02 for two months with a three month follow-up. In addition to the primary endpoint of safety, the key secondary efficacy endpoint of proteinuria was measured every two weeks. Treatment effect on proteinuria is a well-validated endpoint for several renal indications and a good predictor of treatment outcomes. For eligible patients, induction of COL4 was also measured at the end of two months.
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 Alport patients and have no disease modifying treatment options.
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Source: Eloxx Pharmaceuticals