Eloxx Pharmaceuticals Presents New Positive Data for ELX-02 at the American Society of Nephrology (ASN) Kidney Week 2019
“We are pleased to have presented the results of our completed renal impairment study at Kidney Week, which have been important in defining the appropriate doses for patients with renal insufficiency. These data support our current clinical programs as well as the expansion of our research in the kidney beyond nephropathic cystinosis into other areas such as autosomal dominant polycystic kidney disease where there is a high prevalence of nonsense mutation patients,” said Dr.
In a poster session titled “An open label-single dose, parallel-group study to evaluate the effects of renal impairment on the pharmacokinetics of ELX-02: Results from subjects with mild and moderate renal impairment,” Dr.
- As degree of renal impairment increased, the exposure to ELX-02 increased and its clearance decreased.
- There were no significant differences in plasma ELX-02 concentrations between the control group and the mildly impaired renal groups. AUC0-24 was higher in the moderate and severe groups relative to the control group.
- The observed changes in plasma concentrations enable dose adjustment based on eGFR/renal function.
- Urinary ELX-02 clearance was similar to plasma clearance, with decreased rate in subjects with more severe renal impairment.
- To date, ELX-02 has been generally well tolerated in clinical studies, with 105 volunteers exposed, no reported SAEs or renal findings.
- Collectively, these data support the future evaluation of ELX-02 in Phase 2 trials with nonsense mediated diseases.
In a poster session titled “Cystinosis nonsense mutation read-through mediated by ELX-02 restores protein function using in vitro and in vivo models,” Dr.
- ELX-02 read-through is sufficient to produce functional CTNS protein and increase CTNS mRNA.
- ELX-02 demonstrated that the expressed cystinosin protein reduced the accumulated lysosomal cystine by one third in the time frame of the experiment at the given dose of 10 mg/kg.
- Kidney exposure and demonstration of efficacy in vivo support dose-range selection for a Phase 2 clinical trial of ELX-02 in Nephropathic Cystinosis.
- Completion of a Phase 1 study in renal insufficient participants provides modeling necessary for dose adjustments based on renal function.
- These results support the continued development of ELX-02 for the potential treatment of nephropathic cystinosis and other nonsense mutation mediated diseases of the kidney, such as Autosomal Dominant Polycystic Kidney Disease (ADPDK).
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Source: Eloxx Pharmaceuticals