Novartis Acquires Rare Disease Therapeutics Developer DTx Pharma

Rare Daily Staff

Novartis said it will acquire DTx Pharma, a preclinical stage company addressing the delivery challenges of oligonucleotide therapeutics and a preclinical pipeline of rare disease therapeutics.

Under the terms of the agreement, Novartis will make an upfront payment of $500 million and additional payments of up to $500 million upon completion of pre-specified milestones.

DTx Pharma’s FALCON (Fatty Acid Ligand Conjugated OligoNucleotide platform enables the delivery and activity of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake. The company’s lead program is currently in preclinical development, with FDA Orphan Drug designation, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A), a progressive, neuromuscular, autosomal-dominant disease that can lead to life-long loss of muscle function and disability.

CMT1A is driven by overexpression of the PMP22 gene in Schwann cells, which prevents myelination of peripheral nerves and causes progressive muscle wasting, neuropathic pain, difficulty walking and, eventually for many, an inability to live independently, leading to a significant and debilitating impact on patient lives. Currently, there are no approved therapeutics addressing PMP22, the underlying genetic cause of CMT1A, for the estimated 150,000 patients living with the debilitating disease in the United States and Europe.

DTx Pharma’s lead asset, DTx-1252, is a novel, potential first-in-class, FALCON siRNA candidate targeting PMP22. By repressing PMP22, DTx-1252 reverses CMT1A in a mouse model that faithfully recapitulates the genetic and clinical manifestations of the disease. DTx-1252 treatment induces remyelination of axons to normal levels, improves relevant electrophysiological measurements and increases muscle mass, grip strength, coordination and agility in preclinical studies.

In addition to the CMT1A program, Novartis has acquired full rights to the FALCON platform and two other early-stage programs in neuromuscular and central nervous system indications.

“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,” said Artie Suckow, co-founder and CEO of DTx Pharma.

Photo: Artie Suckow, co-founder and CEO of DTx Pharma