A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

Patients must meet all of the following criteria to participate in this study: 1. Male or female patients who, at the time of screening, are 18 years of age or older (Cohort 1) or ≥12 years of age (Cohort 2) 2. A diagnosis of nephropathic cystinosis and biallelic CTNS mutations, including at least one nonsense mutation 3. Patients should have a mild to moderate disease estimated glomerular filtration rate ≥40 mL/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration CKD-EPI formula 4. Body mass index of 19.0 to 30.0 kg/m2. Patients with a lower BMI may be entered into the study at the discretion of the Investigator following consultation with the Sponsor 5. Renal transplant permitted with stable graft function (serum creatinine) for 3 months prior to Screening Patients with any of the following characteristics/conditions will not be included in the study: 1. Participation in clinical study including administration of any investigational drug or device in the last 30 days or 5 half-lives (whichever is longer) prior to investigational product dosing in the current study 2. Concomitant use of cysteamine bitartrate from 7 days prior to baseline until 7 days following final administration ELX-02 3. An average systolic blood pressure and/or diastolic blood pressure ≥95th percentile for sex, age, and height on 3 or more occasions during the screening period 4. Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been…