Novartis has confirmed the formal presentation of its global Phase 3 registrational trial design for farabursen at ERA 2026, held June 3-6 in Glasgow, UK. The oral presentation (Abstract #3398) took place on June 4 and detailed the design of what will be the pivotal trial to bring farabursen to market for ADPKD patients.
Farabursen (formerly RGLS8429/CYX082) is an anti-miR-17 oligonucleotide that works by restoring production of polycystin-1 and polycystin-2 — the proteins whose deficiency causes ADPKD. In Phase 1b (NCT05521191, 68 patients), the drug effectively halted kidney growth: the treatment group showed just 0.05% height-adjusted total kidney volume (htTKV) growth compared to 2.58% in placebo — potentially the most impressive result in PKD drug development in decades.
Novartis acquired Regulus Therapeutics for $1.7 billion in a deal that closed in June 2025, signaling extreme confidence in farabursen's potential. The 300mg subcutaneous dose administered every two weeks was selected based on biomarker data confirming restored polycystin-1 and polycystin-2 protein markers, presented at ASN Kidney Week 2025 and WCN 2026 in Yokohama.
The Phase 3 trial design presentation is a critical milestone: it indicates the study is imminent and will define the endpoints, patient population, and duration that regulators will use to evaluate approval. No NCT ID has been registered for the Phase 3 trial yet on ClinicalTrials.gov, but registration typically follows shortly after design finalization.
For the ADPKD community, farabursen represents a potential paradigm shift — rather than managing symptoms or slowing progression modestly like tolvaptan, it addresses the root genetic cause of the disease. If Phase 3 confirms Phase 1b results, it could become the most effective disease-modifying therapy ever developed for PKD.