- Regulus Therapeutics is a clinical-stage biopharmaceutical company focused on developing microRNA therapeutics, with an emphasis on autosomal dominant polycystic kidney disease (ADPKD), a serious renal condition.
- Farabursen, a main investigational drug for the treatment of ADPKD, is a novel next-generation oligonucleotide targeting miR-17. It has recently completed a Phase 1b multiple ascending dose clinical trial.
- The deal includes an $800 million upfront payment and an additional $900 million that may be paid upon reaching regulatory milestones in the future. The transaction is expected to be completed in the second half of 2025, subject to the fulfillment of customary closing conditions.
Recently, Novartis announced that it has reached an agreement to acquire Regulus Therapeutics, a publicly traded company based in San Diego. This clinical-stage biopharmaceutical company focuses on developing microRNA therapeutic drugs. REGULUS’s lead drug candidate, Farabursen, is a potential first-in-class next-generation oligonucleotide targeting the miR-17 site for the treatment of autosomal dominant polycystic kidney disease (ADPKD). This agreement aligns fully with Novartis' therapeutic priorities and will leverage Novartis' strengths and expertise in renal diseases.
Dr. Shreeram Aradhye, President of Novartis Pharmaceuticals Development and Chief Medical OfficerSaid: "Currently, the treatment options for ADPKD patients are limited. Farabursen, as a potential first-in-class drug, is expected to outperform existing standard treatment regimens in terms of efficacy, tolerability, and safety. ADPKD is the most common hereditary cause of renal failure worldwide."1"The Regulus team has laid an important foundation in the development of Farabursen, and we look forward to further exploring its potential to bring better treatment options to patients in need."
Farabursen is an investigational microRNA inhibitor targeting the miR-17 site, with preferential activity in the kidney, aiming to reduce cyst growth and kidney volume while slowing the progression of ADPKD disease severity. In March 2025, Regulus announced the successful completion of the Phase 1b multiple ascending dose clinical trial of Farabursen. The Phase 1b trial data demonstrated promising clinical efficacy and safety, including consistent effects on two key measures: urinary polycystin (PC) and height-adjusted total kidney volume (htTKV).
Statement
1. The purpose of this material is to convey cutting-edge pharmaceutical information and research progress, not for advertising purposes. The information contained in this material is for reference only; please follow the advice or guidance of doctors or other healthcare professionals.
2. The drug Farabursen mentioned in this material has not been approved in mainland China.