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Kidney Week

Abstract: TH-PO368

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

Session Information

Category: Pharmacology (PharmacoKinetics, -Dynamics, -Genomics)

  • 1800 Pharmacology (PharmacoKinetics, -Dynamics, -Genomics)


  • Williams, Gregory, Eloxx Pharmaceuticals, Inc, Waltham, Massachusetts, United States

ELX-02 is an investigational synthetic eukaryotic ribosomal selective glycoside (ERSG) that induces read-through of nonsense mutations through interaction with the ribosome resulting in full-length functional proteins. ELX-02 is being developed as a therapy for genetic diseases caused by nonsense mutations such as nephropathic cystinosis and polycystic kidney disease. It is well established that kidney disease is characterized by multiple physiologic effects, which induce clinically significant changes in pharmacokinetics.


Eloxx Pharmaceuticals, Inc. is presently conducting a Phase 1 study designed to determine the effect of various severities (i.e., mild [eGFR 60-89 mL/min/1.73m2], moderate [eGFR 30-59 mL/min/1.73m2], and severe eGFR [<30 mL/min/1.73m2, not requiring dialysis] of renal impairment, compared to a control group with normal renal function [eGFR ≥ 90 mL/min/1.73m2) on the PK of ELX-02 after a single dose of 1 mg/kg. Six subjects are planned to be enrolled in each category of renal failure, and 6-8 subjects enrolled in the control group.


To date, treatment of subjects with mild and moderate renal impairment has been completed. In subjects with mild renal impairment the mean eGFR at baseline was 74.7 mL/min/1.73m2, Cmax was 2,993 ng/mL, Tmax was 1.1 hr, AUC0-inf was 16,999 ng*h/mL, t1/2 was 3.3 hr, and Cl/F was 4.4 L/h. In the moderate group the mean baseline eGFR was 41.7 mL/min/1.73m2, Cmax was 3,688 ng/mL, Tmax was 1.3 hr, AUC0-inf was 35,172 ng*h/mL, t1/2 was 6.5 hr, and Cl/F was 2.4 L/h. These results indicate that the degree of renal impairment has effects on the PK of ELX-02, with an increase in Cmax, Tmax, AUC, and t1/2 and a decrease in Cl/F in subjects with moderate renal impairment when compared to subjects with mild renal impairment. No adverse events have been reported in this study.


Results from the severe and control groups will be available at the meeting. Based on the preliminary results available to date, ELX-02 can be used safely in patients with renal dysfunction and dose adjustments may be considered for patients with varying degrees of renal impairment. These results support the continued development of ELX-02 for the potential treatment of nonsense mutation mediated kidney diseases.


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