DTx Pharma is engaged in developing small interfering RNA (siRNA) therapeutics for neuroscience indications, leveraging its FALCON platform, which deliver the siRNA therapies to tissues beyond the liver with enhanced biodistribution and cellular uptake
Novartis Campus and Banting 1 in the background. (Credit: Novartis AG.)
Swiss pharmaceutical company Novartis has acquired DTx Pharma, US-based preclinical stage biotechnology company, which develops siRNA therapies for neuroscience indications.
Novartis has agreed to make an upfront payment of $500m, along with additional milestone payments contingent to the completion of certain pre-specified milestones.
DTx develops the siRNA therapies leveraging its unique fatty acid ligand-conjugated oligonucleotide (FALCON) platform.
The FALCON platform helps deliver the small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing its biodistribution and cellular uptake.
The acquisition includes the US company’s lead programme DTx-1252, which targets the overexpression of PMP22, a protein that causes the abnormal functioning of myelin sheath.
DTx-1252 works to decrease the expression of PMP22 in Schwann cells, the target cell type for the development, maintenance, and function of peripheral nerves.
In addition to DTx-1252, Novartis will also acquire two additional preclinical programs for other neuroscience indications.
The acquisition will expand its siRNA toolkit, based on its xRNA capabilities, said Novartis.
Novartis Institutes for BioMedical Research (NIBR) president Fiona Marshall said: “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.
“We are also excited to bring DTx’s FALCON technology to Novartis and explore its potential to deliver drugs to extrahepatic tissues.”
The US Food and Drug Administration (FDA) has recently granted Orphan Drug Designation to DTx-1252, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).
Charcot-Marie Tooth disease (CMT) is a group of inherited disorders that affect nervous system, and CMT1A is the most prevalent subtype of CMT.
It is a progressive, neuromuscular, autosomal-dominant disease that causes gradual muscle wasting, neuropathic pain, and difficulty walking, and significantly impacts the quality of life.
DTx-1252 is potentially the first treatment for CMT1A, as there are currently no approved treatments that address the underlying genetic cause of CMT1A.
DTx Pharma co-founder and CEO Artie Suckow said: “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.
“I am also extremely proud of the commitment and passion of our team, which has established DTx Pharma as a leader in extra-hepatic delivery of siRNA, as demonstrated by our work to advance the first investigational FALCON siRNA designed to be delivered to the peripheral nervous system to treat the genetic cause of CMT1A.”