Novartis acquires rare kidney disease drugs prepared in stage 3 through $800 million acquisition

Novartis has made kidney disease a priority area, and now it is using a deal to get Regulus Therapeutics, a biotechnology, and is ready for critical testing in rare kidney disease with limited treatment options.

The Swiss pharmaceutical giant said on Wednesday it agreed to pay $800 million in advance to buy Regulus. If shareholders of San Diego Biotech shareholders may get more lead assets, it is a genetic medicine called Farabursen that can be called through regulation.

Farabursen is developing treatments for autosomal dominant polycystic kidney disease (ADPKD). This rare disease causes the development of cysts in the organs, mainly the kidneys. As the disease progresses, fluid-filled cysts can enlarge the kidneys, thereby impairing their function. Symptoms include back and side pain, high blood pressure, blood in the urine, and urinary tract infections. In some patients, ADPKD develops into end-stage renal disease.

Standard ADPKD treatment is managing symptoms and trying to slow disease progression. The only treatment is Tolvaptan, brand name Jynarque, an Otsuka Pharmaceutical drug approved by the FDA in 2018 to reduce cyst growth in ADPKD. However, this once-daily drug introduces the risk of severe hepatotoxicity.

Regulus' research focuses on MicroRNAs, or non-coding RNAs, which play a role in regulating gene expression. Biotechnology drugs are oligonucleotides designed to inhibit disease-related microRNAs. Farabursen targets miR-17, preferentially blocking microRNA in the kidneys. This approach aims to reduce cyst growth and kidney size, thereby delaying disease progression.

ADPKD is caused by mutations in the PKD1 or PKD2 gene that reduces the function of their respective protein products. In a placebo-controlled, multi-stabilized dose 1B study, the results showed an increased level of these protein products in the urine. The results also show improvements in other kidney health measures. In addition, Farabursen was tolerated by study participants. In January, Regulus said it had reached a meeting with the FDA about what needs to be shown in the Phase 3 study in order to seek accelerated approval from Farabursen in the ADPKD. In late March, Regulus said it plans to start key research in the third quarter of this year.

“Given the treatment options for patients with ADPKD, Farabursen represents a potentially best-in-class drug that may provide enhanced efficacy, tolerance and safety with standards of care,” Novartis President and Chief Medical Officer Shreeram Aradhye said in a prepared statement.

Joseph Schwartz, an analyst at Leerink Partners, said in a note sent to investors that Novartis was a “logical partner” with Farabursen, especially given the growing presence of the drug giant in rare kidney disease. In early April, Novartis drug Vanrafia won accelerated FDA approval to treat immunoglobulin A nephropathy (IGAN). That small molecule comes from the $3.2 billion acquisition of Chinook's therapeutic drug in 2023.

Novartis found and developed a complement protein inhibitor, Fabhalta, internally, which won FDA approval last August as a treatment for Igan. In March, the twice-daily pill expanded its label, becoming the FDA-approved first treatment for C3 glomerulopathy, an ultra-rare kidney disease that can progress to kidney failure. Novartis is conducting other research that could support Fabhalta's further expansion to other kidney diseases.

Established in 2008, Regulus is a MicroRNA joint venture between Alnylam Pharmaceuticals and ISIS Pharmaceuticals (now known as Ionis Pharmaceuticals). When Regulus went public in 2012, its stock price was $4.
The financial terms branch of Regulus' acquisition is $7 in cash per share of biotech share, totaling $800 million. The price is 108% premium to Tuesday's biotech closing price. Regulus shareholders may receive $7 per share in a transaction related to legal approval.

Schwartz noted that regulatory regulatory applications for state Faraberson must obtain regulatory approval by the end of 2034 to trigger the right, but the document does not specify whether this is an accelerated approval or a traditional approval. The Leerink project may be accelerated in 2029, leaving a lot of time if there is a delay or the FDA requests more data. While the Regulus acquisition still requires approval from antitrust regulators, Schwartz has not seen much attention from the Federal Trade Commission, as Farabursen has no signs of kidneys showing Novartis is speaking for Fabharta or studying kidneys.

The Novartis and the statute board approved the acquisition, which is expected to close in the second half of this year.

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