Date: Jul 17, 2023 Author: Lisa Munger Source: Biospace (
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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."