Novartis, which recently unveiled significant investments in U.S.-based infrastructure, has entered into an agreement to acquire Regulus Therapeutics, a San Diego-based, clinical-stage biopharmaceutical company focused on developing microRNA therapeutics, for a total consideration of as much as approximately $1.7 billion.

Regulus’ lead asset, farabursen, is a potential first-in-class, next-generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Farabursen is an investigational microRNA inhibitor designed to target miR-17 with preferential kidney exposure, aiming to reduce the growth of cysts and kidney size, as well as delay progression of disease severity in ADPKD. In March 2025, Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen. The Phase 1b trial data showed promising clinical efficacy and safety, including consistent impact on urinary polycystin (PC), a biomarker of mechanistic response, and height-adjusted total kidney volume (htTKV), a measure of progressive disease.

“With limited treatment options currently available for patients suffering from ADPKD, farabursen represents a potential first-in-class medicine with a profile that may provide enhanced efficacy, tolerability and safety versus standard of care,” said Shreeram Aradhye, President, Development and Chief Medical Officer, Novartis. “ADPKD is the most common genetic cause of renal failure worldwide. The team at Regulus has done meaningful foundational work with farabursen, and we look forward to investigating its potential further as we aim to bring a better treatment option to patients in need.”

“We are excited to combine with Novartis to potentially bring farabursen to patients living with ADPKD, who currently have limited treatment options,” added Jay Hagan, CEO of Regulus Therapeutics. “Novartis’ established global development and commercial capabilities will enable this important new medicine to reach patients if approved.”

Other Pharma Industry Acquisitions

Check out Contract Pharma’s 2024 Pharmaceutical Industry Mergers & Acquisitions Roundup.