Novartis has announced its acquisition of DTx Pharma, a preclinical stage biotech focused on developing siRNA therapies for neuroscience indications, in a deal worth up to $1bn.
The transaction gives Novartis access to DTx’s fatty acid ligand-conjugated oligonucleotide (FALCON) platform as well as its lead preclinical therapy for Charcot-Marie-Tooth disease type 1A (CMT1A).
Approximately 150,000 people in the US and Europe are affected by CMT1A, a slow-progressing and degenerative disease of the peripheral nervous system.
There are currently no approved treatments to address the underlying genetic cause of the condition: the over-expression of PMP22, a protein that causes the myelin sheath that supports and insulates nerves in the peripheral nervous system to function abnormally.
DTx-1252, which was recently granted Orphan Drug Designation by the US Food and Drug Administration, decreases the expression of this protein.
“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 a statement.
In addition to the CMT1A programme, Novartis has gained two other early-stage programmes for undisclosed neuromuscular and central nervous system indications.
Fiona Marshall, president of the Novartis Institutes for BioMedical Research, said: “We look forward to continuing the development of DTx’s therapeutic programmes and bringing new hope to patients with neuromuscular and other neurological disorders for which there have historically been few treatment options.
“We are also excited to bring DTx’s FALCON technology to Novartis and explore its potential to deliver drugs to extrahepatic tissues.”
Under the terms of the agreement, Novartis will make an upfront payment of $500m to DTx, which will be eligible for additional payments of up to $500m upon completion of pre-specified milestones.
Artie Suckow, co-founder and chief executive officer of DTx, commented: “I am thrilled that Novartis will be moving forward with our CMT1A therapeutic programme and the FALCON platform.
“With its resources and capabilities in neuromuscular diseases, Novartis is well positioned to accelerate the development of DTx-1252 and provide hope to patients, who are desperately in need of therapy.”