Novartis acquires Regulus for $800M for phase 3 kidney drug

Novartis is strategically expanding its presence in the kidney disease market with a significant investment aimed at acquiring Regulus Therapeutics, a move that aligns with its commitment to becoming a leader in this medical field. The Swiss pharmaceutical giant has earmarked $1.7 billion for this acquisition, with approximately $800 million as an upfront payment. This initial payment equates to $7 per share, more than doubling the recent market price of Regulus’ stock. An additional payment of $7 per share is contingent on the regulatory approval of Regulus' lead drug candidate, farabursen.

Farabursen is designed to treat autosomal dominant polycystic kidney disease (ADPKD), a rare renal disorder. This oligonucleotide-based therapy showed promising results in a phase 1b clinical trial conducted by Regulus. The study, completed in March, demonstrated that patients receiving farabursen bi-weekly for three months had significant increases in urinary proteins PC1 and PC2, which inversely correlate with the severity of the disease. Furthermore, height-adjusted total kidney volume (htTKV), an important predictor of renal insufficiency, increased minimally by 0.05% over four months in patients treated with farabursen, compared to a 2.58% increase in the placebo group.

Buoyed by these positive outcomes, Regulus plans to commence a phase 3 trial in the third quarter to potentially secure accelerated approval. The trial will focus on changes in htTKV after 12 months and will also track changes in eGFR, a measure of kidney function, over a 24-month period to support the case for full approval.

If successful, farabursen will enter a competitive market currently served by Otsuka's Jynarque, which has already seen a significant rise in U.S. sales due to increased prescriptions for ADPKD. Despite this, Jynarque is used by only a small fraction of the eligible patient population, primarily due to concerns over its safety and tolerability, as noted by Regulus’ CEO, Joseph Hagan. This opens a potential market opportunity for farabursen, which Regulus claims could reach multibillion-dollar revenues.

For Novartis, securing farabursen complements its existing kidney disease portfolio, which includes recent acquisitions like Chinook Therapeutics for $3.2 billion, as well as internal developments. The company’s commercial offerings in this domain now feature Fabhalta and Vanrafia.

Overall, Novartis' acquisition of Regulus and its promising drug candidate, farabursen, reflects its strategic intent to broaden its impact in the kidney disease market, leveraging both innovative therapies and strategic acquisitions to drive growth and address unmet medical needs.