SWITZERLAND — Novartis, the Swiss pharmaceutical company has acquired DTx Pharma, a preclinical biotech based in San Diego, for US$500 million.
The deal also includes the potential for additional payments of up to US$500 million upon the achievement of certain milestones.
DTx Pharma specializes in the development of therapies that target disorders affecting the nervous system, which aligns with Novartis’ strategic efforts to expand its neuroscience pipeline.
The lead program of DTx Pharma, called DTx-1252, focuses on addressing the root cause of Charcot-Marie-Tooth (CMT) disease type 1, which is the overexpression of a protein called PMP22 in Schwann cells.
This overexpression leads to abnormal functioning of the myelin sheath, which supports and insulates nerves in the peripheral nervous system.
CMT is a group of inherited disorders that impact the nervous system, with CMT1A being the most common subtype affecting approximately 150,000 patients in the United States and Europe.
DTx-1252 has the potential to become a first-in-class treatment for this disease, as there are currently no approved therapies that target its underlying genetic cause.
In recognition of its potential, the U.S. Food and Drug Administration (FDA) granted DTx-1252 Orphan Drug Designation last month.
As part of the acquisition, Novartis will also gain access to DTx’s proprietary Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform, which is designed to deliver small interfering RNA (siRNA) therapeutics to tissues beyond the liver for improved biodistribution and cellular uptake.
Peter Condon, Chief Business Officer of DTx Pharma, expressed enthusiasm about the acquisition, highlighting Novartis’ deep knowledge of CMT1A biology and their commitment to advancing the development of DTx-1252.
He also emphasized the validation of DTx’s FALCON platform and its potential to expand therapeutic areas by enabling the delivery of siRNA beyond the liver.
Novartis stated that the acquisition will enhance its capabilities in RNA-based therapeutics by integrating DTx’s FALCON platform into its existing siRNA toolkit.
Fiona Marshall, President of the Novartis Institutes for BioMedical Research, expressed excitement about the opportunity to continue developing DTx’s therapeutic programs and bringing new hope to patients with neuromuscular and neurological disorders that have historically had limited treatment options.
Marshall also highlighted the potential of DTx’s FALCON technology to deliver drugs to tissues beyond the liver, opening up new avenues for therapeutic intervention.
Through this acquisition, Novartis aims to strengthen its position in the field of neuroscience and advance the development of innovative therapies for patients with neurological conditions, particularly those affecting the neuromuscular system.
Neuroscience is one of Novartis’ five core therapeutic areas, with the company looking to use new technology platforms to discover new treatments.
Last year, the drugmaker outlined its reduced focus alongside the technologies it is prioritizing: these include ‘xRNA,’ which covers RNA targeting siRNA and mRNA cancer vaccines.
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