Novartis Institutes for BioMedical Research GmbH & Co. KG

Novartis will gain full rights to the San Diego-based biotech’s fatty acid ligand-conjugated oligonucleotide (FALCON) platform. On Novartis’ previous earnings call, CEO Vas Narasimhan, M.D., insisted that despite a “relatively quiet” year, the Swiss Big Pharma was “very actively” looking at bolt-on M&A to boost the business. With less than a day to go before the company’s next quarterly call, Novartis will certainly have something to point to—having just signed off its second acquisition in as many months. The latest deal sees Novartis push further into neuroscience courtesy of a $500 million upfront payment for siRNA-focused DTx Pharma. For its money, the Big Pharma is getting full rights to the San Diego-based biotech’s fatty acid ligand-conjugated oligonucleotide (FALCON) platform as well as a preclinical therapy for Charcot-Marie-Tooth disease type 1A (CMT1A), a neuromuscular disorder for which there are currently no approved meds. In addition, Novartis will gain two other preclinical programs for undisclosed neuromuscular and central nervous system indications. Should those three programs develop as planned, DTx will be eligible to double its money thanks to up to $500 million in milestone payments. Coming the same morning that Novo Nordisk announced an RNA-focused collaboration with Eleven Therapeutics, Novartis said its acquisition of DTx will expand its own “capabilities in RNA-based therapeutics, adding DTx’s FALCON platform to the Novartis siRNA toolkit.” The CMT1A program, called DTx-1252, is designed to target the “root cause” of the disorder by tackling the overexpression of a protein called PMP22 that affects the myelin sheath that should normally support nerves in the peripheral nervous system. “The FALCON platform facilitates this targeted approach by conjugating siRNAs to naturally occurring fatty acids to improve the biodistribution and cellular uptake to tissues and cell types of interest,” Novartis explained in this morning’s release. “The growing arsenal of therapeutic platforms available to us continues to enable the development of novel, high-value medicines—particularly in cases where the underlying biology of disease is well defined,” Robert Baloh, global head of neuroscience for the Novartis Institutes for BioMedical Research, said in the release. “We look forward to continuing the development of DTx’s potential first-in-class medicine for CMT1A and are hopeful we can bring a therapeutic option to patients living with this debilitating neuromuscular condition,” Baloh said. DTx Pharma’s chief business officer Peter Condon said the Novartis team's “deep knowledge of CMT1A biology and strong understanding of the patient burden were evident from our first interactions.” “This agreement is also a strong validation of our FALCON platform and its potential to break open new therapeutic areas by delivering siRNA beyond the liver,” Condon added. This opportunity to unlock the potential of RNA-targeted therapies beyond the liver, where these therapies have often been limited to until now, has attracted interest from other Big Pharmas like GSK, which has signed a number of oligonucleotide-focused deals in recent months. For Novartis, the acquisition of DTx comes just a month after the Swiss drugmaker handed over a far larger sum, $3.2 billion, to buy Chinook Therapeutics for a pair of late-stage programs targeting a rare, progressive chronic kidney disease. Setting out Novartis’ M&A strategy on its first-quarter earnings call in April, Narasimhan said forthcoming deals would “focus primarily in the sub-$5 billion range.” With the company’s next earnings call scheduled for Tuesday, it will be interesting to see whether those ambitions have expanded now that the company has rediscovered its taste for acquisitions.

Pictured: Novartis/Courtesy of Adobe Stock, Yingko Novartis has acquired San Diego-based preclinical biotech DTx Pharma for $500 million, the Swiss pharma announced Monday. The deal also includes additional potential payments of up to $500 million upon completion of certain milestones. DTx Pharma focuses on developing therapies targeting disorders affecting the nervous system, which aligns with Novartis’ efforts to expand its neuroscience pipeline. DTx-1252, DTx Pharma’s lead program, targets the root cause of Charcot-Marie-Tooth (CMT) disease type 1—the overexpression of PMP22, a protein that causes the myelin sheath that supports and insulates nerves in the peripheral nervous system to function abnormally. CMT is a group of inherited disorders that affect the nervous system. CMT1A, the most prevalent subtype, affects approximately 150,000 patients in the U.S. and Europe, according to the companies. DTx-1252 can potentially be a first-in-class treatment for this disease, as currently, no approved treatments target its underlying genetic cause. The FDA last month granted DTx-1252 Orphan Drug Designation. In the acquisition, Novartis will also nab DTx’s proprietary Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform intended to deliver small interfering RNA (siRNA) therapeutics to tissues beyond the liver for better biodistribution and cellular uptake. “The Novartis team’s deep knowledge of CMT1A biology and strong understanding of the patient burden were evident from our first interactions, and we are excited by their support to advance the development of DTx-1252,” Peter Condon, DTx Pharma’s chief business officer, said in a prepared statement. “This agreement is also a strong validation of our FALCON platform and its potential to break open new therapeutic areas by delivering siRNA beyond the liver.” Novartis said that the acquisition will expand its capabilities in RNA-based therapeutics, adding DTx’s FALCON platform to the Swiss pharma’s siRNA toolkit. “We look forward to continuing the development of DTx’s therapeutic programs and bringing new hope to patients with neuromuscular and other neurological disorders for which there have historically been few treatment options,” Fiona Marshall, president of the Novartis Institutes for BioMedical Research, said in a statement. “We are also excited to bring DTx’s FALCON technology to Novartis and explore its potential to deliver drugs to extrahepatic tissues.” Lisa Munger is a senior editor at BioSpace. You can reach her at lisa.munger@biospace.com. Follow her on LinkedIn.