Polycystic Kidney, Autosomal Dominant

Novartis has announced that it will be acquiring Regulus Therapeutics in a deal worth $1.7bn, marking a notable boost to the drugmaker’s renal disease pipeline. The agreement will give Novartis access to farabursen, a candidate in clinical development for autosomal dominant polycystic kidney disease (ADPKD). In ADPKD, the most common genetic cause of renal failure, small fluid-filled cysts gradually develop in the kidneys. Farabursen is an investigational microRNA inhibitor that aims to reduce the growth of cysts and kidney size, as well as delay progression of disease severity in patients with ADPKD. The drug, which targets miR-17, recently demonstrated promising efficacy and safety results in a phase 1b multiple-ascending dose clinical trial. According to Regulus, the candidate showed consistent impact on urinary polycystin, a biomarker of mechanistic response, and height-adjusted total kidney volume, a measure of progressive disease. Shreeram Aradhye, president, development and chief medical officer, Novartis, said farabursen “represents a potential first-in-class medicine with a profile that may provide enhanced efficacy, tolerability and safety versus standard of care”. “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,” Aradhye added. The transaction, which is expected to close in the second half of this year, includes an upfront payment of $800m and a potential $900m payment dependent on the achievement of a future regulatory milestone Regulus’ chief executive officer, Jay Hagan, said: “We are excited to combine with Novartis to potentially bring farabursen to patients living with ADPKD, who currently have limited treatment options. “Novartis’ established global development and commercial capabilities will enable this important new medicine to reach patients if approved.” The announcement comes less than three months after Novartis revealed that it would be expanding its late-stage cardiovascular pipeline by acquiring Anthos Therapeutics for approximately $3.1bn. The deal, expected to close in the first half of 2025, includes abelacimab, which is currently in phase 3 development for the prevention of stroke and systemic embolism in patients with atrial fibrillation.

The upfront cash payment represents a significant premium over Regulus’ recent stock prices. Credit: Judith Linine/Shutterstock. Novartis has agreed to acquire US-based Regulus Therapeutics and its oligonucleotide, farabursen, to bolster its renal disease offerings. The upfront cash payment represents a significant premium over Regulus’ recent stock prices and a contingent value right (CVR) tied to regulatory milestones. Total consideration for the acquisition, including the CVR, could reach $1.7bn if the regulatory milestone is met. The transaction has received unanimous approval from both companies’ boards of directors. Novartis will commence a tender offer through an indirect wholly owned subsidiary to purchase all outstanding Regulus shares. Shareholders of Regulus will receive $7 per share in cash on closure, along with the CVR worth up to an additional $7 per share, contingent upon regulatory approval of the therapy. Post completion, Novartis plans to combine the acquiring subsidiary with Regulus, making it an indirect wholly owned subsidiary of the company. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Anticipated closure will take place in the second half of 2025, contingent on customary closing conditions, including the tender of a majority of Regulus’ common stock outstanding shares and necessary regulatory approvals. Both companies will operate as independent entitiesuntil then. Evercore is the exclusive financial advisor to Regulus, and Latham & Watkins is providing legal counsel. Regulus’ farabursen targets microRNA (miR)-17 with preferential kidney exposure and is intended for treating autosomal dominant polycystic kidney disease (ADPKD). The treatment decreases cyst growth and the size of the kidney, potentially delaying ADPKD’s severity. In March 2025, Regulus completed its Phase Ib multiple-ascending dose trial of the therapy, demonstrating safety and clinical efficacy. Novartis Development president and chief medical officer Shreeram Aradhye stated: “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.” In February 2025, Novartis agreed to acquire Anthos Therapeutics, a Boston-based clinical-stage biopharmaceutical company, for an upfront payment of $925m.

Novartis has identified farabursen as a good fit for its burgeoning kidney disease portfolio.\n Novartis has committed another $1.7 billion to its ambition to become a kidney disease leader. The Swiss drugmaker is making the outlay, around half of which is upfront, to buy Regulus Therapeutics for a rare renal disease drug that is set to enter phase 3 in the third quarter.Regulus published top-line data from a phase 1b trial of its lead candidate, farabursen, in March. The trial enrolled patients with autosomal dominant polycystic kidney disease (ADPKD). People who received the oligonucleotide every other week for three months had statistically significant increases in urinary PC1 and PC2, proteins that inversely correlate with disease severity.Height-adjusted total kidney volume (htTKV), a predictor of renal insufficiency, increased 0.05% over four months in patients on farabursen. HtTKV grew 2.58% in the placebo cohort. Buoyed by the data, Regulus forged ahead with plans to start a phase 3 trial that could support a filing for accelerated approval.Novartis has identified farabursen as a good fit for its burgeoning kidney disease portfolio. The company has agreed to pay $7 a share upfront to acquire Regulus. The upfront payment, which amounts to $800 million, is more than double the closing price of Regulus’ stock Tuesday. Novartis has agreed to pay another $7 a share if Regulus achieves a milestone tied to the regulatory approval of farabursen. The total value, should all these targets be hit, would reach $1.7 billion.  Regulus has designed its phase 3 trial to support a filing for accelerated approval based on htTKV after 12 months. The trial will serve as its own confirmatory study, with the biotech tracking changes in a measure of kidney function, eGFR, out to 24 months to support full approval.If approved, farabursen will enter a market already served by Otsuka’s Jynarque. Otsuka said (PDF) U.S. sales “increased significantly” over the first three months of the year because ADPKD prescriptions rose. Regulus told investors in January that Jynarque is only used in about 7% of the addressable population, a fact the biotech’s CEO Joseph Hagan said is “in large part because of its safety and tolerability profile.”Regulus claimed farabursen is a multibillion-dollar opportunity. Novartis wants to seize the opportunity to advance the kidney care business that it has built out through deals such as the $3.2 billion Chinook Therapeutics acquisition and development of internal programs. The commercial portfolio now includes Fabhalta and Vanrafia.