Novartis announced Wednesday it will acquire San Diego-based Regulus Therapeutics for $800 million upfront, significantly expanding its kidney disease portfolio with a novel microRNA-targeting therapy. The deal includes potential additional payments of up to $900 million contingent upon regulatory milestones, potentially valuing the acquisition at $1.7 billion.
The Swiss pharmaceutical giant will pay $7 per share for Regulus—representing a substantial 108% premium over the biotech's Tuesday closing price. Shareholders will also receive a $7-per-share contingent value right, payable upon regulatory approval of farabursen, Regulus' lead candidate for autosomal dominant polycystic kidney disease (ADPKD).
Strategic Expansion into Oligonucleotide Therapeutics
The acquisition aligns with Novartis CEO Vas Narasimhan's stated strategy of pursuing "bolt-on acquisitions" to strengthen the company's growth prospects beyond 2030. By acquiring Regulus, Novartis gains immediate entry into the expanding field of oligonucleotide therapeutics targeting microRNAs.
Farabursen represents a novel approach to treating ADPKD, a genetic disorder affecting approximately 160,000 patients in the United States alone. The condition is characterized by the development of fluid-filled cysts throughout the kidneys, progressively impairing renal function and potentially leading to kidney failure.
Innovative Mechanism Targeting Disease Pathology
ADPKD is caused by mutations in either the Pkd1 or Pkd2 genes, which lead to upregulation of miR-17, a microRNA molecule. This upregulation suppresses the production of polycystin 1 (PC1) and polycystin 2 (PC2) proteins, which are essential for normal kidney function.
Farabursen works by binding to miR-17, disrupting this pathological pathway and restoring PC1 and PC2 levels. This mechanism aims to address the underlying cause of ADPKD rather than merely managing symptoms, potentially reducing cyst growth and preserving kidney function.
The drug is currently in Phase Ib clinical trials, with study completion expected later this year. The acquisition agreement has been unanimously approved by the boards of both companies and is expected to close in the second half of 2024, subject to customary closing conditions.
Broader Pipeline Opportunities
Beyond farabursen, the acquisition brings additional assets to Novartis, including another nephrology candidate in Regulus' pipeline and a program targeting an undisclosed central nervous system indication. These additional programs further enhance the value proposition for Novartis.
Dr. Jay Bradner, President of the Novartis Institutes for BioMedical Research, commented on the acquisition: "Regulus has pioneered important work in microRNA therapeutics, particularly for kidney diseases where there remains significant unmet need. Farabursen represents a potentially transformative approach to treating ADPKD by addressing the fundamental genetic drivers of the disease."
Market Implications
The substantial premium Novartis is paying for Regulus—more than triple its market capitalization prior to the announcement—may signal renewed interest in acquisitions of innovative small biotechs. This could provide encouragement to investors who have witnessed biotech valuations decline significantly in recent years.
Industry analysts note that the deal demonstrates major pharmaceutical companies' continued willingness to pay substantial premiums for novel therapeutic approaches addressing diseases with high unmet needs, particularly when these approaches have the potential to modify disease progression rather than simply manage symptoms.
The transaction is expected to have minimal impact on Novartis' 2024 financial outlook but could contribute meaningfully to the company's growth in the latter part of the decade if farabursen achieves regulatory approval and commercial success.
