Farabursen for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study Overview

A Phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating farabursen, a novel first-in-class anti-miR-17 oligonucleotide, in patients with ADPKD at risk of disease progression. The study aims to assess whether farabursen can decrease total kidney volume (TKV) growth and slow kidney function decline. Farabursen targets miR-17, which is upregulated in ADPKD and plays a key role in cystogenesis.

Inclusion Criteria

• ✓Adults aged 18 years or older with confirmed ADPKD diagnosis
• ✓At risk of progression defined by Mayo Imaging Classification (MIC) 1C, 1D, or 1E
• ✓eGFR 25-90 mL/min/1.73 m² for ages 18-59, or eGFR 25-45 for ages 60+
• ✓Young severe population (18-30) with MIC 1E and eGFR 91-140 mL/min/1.73 m²

Exclusion Criteria

• ✗Use of tolvaptan or somatostatin analogue within 3 months of screening
• ✗Prior exposure to farabursen
• ✗Etiology of kidney disease other than ADPKD
• ✗Solitary kidney, anatomical anomalies, or kidney transplant recipient
• ✗Any cyst reduction therapy expected in the upcoming 2 years