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During the congress, E-Posters will be accessible to all participants on the congress website 24/7, as well as in the E-poster stations in the congress center.
Preparing your E-Poster
Please review the E-Poster format requirements carefully when preparing your E-Poster. Should your E-Poster not meet the mentioned requirements, it may not be displayed as described above.
E-Poster Submission Deadline
Please prepare and upload your E-Poster no later than March 14, 2026 11.59PM CET. After this date, you will no longer be able to prepare and upload your E-poster and it will not be displayed and accessible on the congress website.
Please follow the instructions below to input your abstract title.
Abstract titles should be brief and reflect the content of the abstract.
Autosomal dominant polycystic kidney disease (ADPKD) is caused by heterozygous loss-of-function mutations in PKD1 or PKD2, leading to progressive renal cystogenesis and kidney failure. miR-17 family suppresses PKD1 and PKD2, providing a therapeutic rationale for miR-17 inhibition to restore polycystin (PC) levels. Farabursen is a potent and selective investigational anti–miR-17 oligonucleotide that de-represses miR-17 mRNA targets, including PKD1 and PKD2, leading to increased PC1 and PC2 and amelioration of preclinical PKD.
A randomized, double-blind placebo-controlled Phase Ib trial evaluated farabursen in patients with ADPKD across 3 weight-based cohorts (1, 2, and 3 mg/kg) and placebo in a 3:1 randomization ratio, and a fixed dose of 300 mg in an open-label cohort. Key entry criteria were Mayo Imaging Class 1C, 1D, or 1E, and estimated glomerular filtration rate (eGFR) 30–90 mL/min/1.73 m2. Patients received subcutaneous injection every 2 weeks (Q2W) for 12 weeks (7 doses), with a 4-week post-treatment follow-up. Primary endpoints were safety and changes in urinary exosomal PC1 and PC2; MRI-measured height-adjusted total kidney volume (htTKV) was a secondary endpoint.
We enrolled 68 patients (58 farabursen,10 placebo) with generally balanced baseline characteristics. The most frequent adverse events were injection site reactions, all of which were mild in severity, except for one that was moderate. Increases in PC1 and PC2 were shown for 2 mg/kg, 3 mg/kg, and 300 mg fixed doses versus pooled placebo. The percent changes in htTKV growth (95% CI) were 1.9% (-1.5; 5.5) for 1 mg/kg, -0.7% (-3.6, 2.3) for 2 mg/kg, -0.3% (-3.4, 2.9) for 3 mg/kg, 0.6% (-1.4, 2.7) for 300 mg fixed dose, and 2.7% (-0.5, 6.0) for pooled placebo (Figure). In a post-hoc analysis pooling the 2 mg/kg, 3 mg/kg, and 300 mg fixed dose groups, the percent change in htTKV growth (95% CI) was -0.1% (-1.6, 1.4) and in the pooled placebo group, it was 2.7% (-0.3, 6.0).
Farabursen was well tolerated with a favorable safety profile at all doses and led to a dose-dependent increase in PC1 and PC2. Treatment with 2 mg/kg, 3 mg/kg and fixed 300 mg doses of farabursen over 12 weeks suggested a mean halting of htTKV growth. These results demonstrate the potential of farabursen as a first-in-class, disease-modifying therapeutic agent for ADPKD. A Phase III registrational trial is planned.
This abstract was also submitted to American Society of Nephrology (ASN) Kidney Week 2025 Congress, November 6-9, in Houston, TX, USA.
