Boost for Charcot-Marie-Tooth patients as Novartis acquires DTx Pharma

Charcot-Marie-Tooth disease

DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its fatty acid ligand conjugated oligonucleotide (FALCON) platform, has been acquired by Novartis. 

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

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 U.S. and Europe. DTx Pharma’s lead asset, DTx-1252, is a novel, potential first-in-class, FALCON siRNA candidate targeting PMP22. The asset boasts a robust preclinical package, demonstrating the reversal of disease in preclinical rodent models and translation to higher species with IND-enabling studies progressing well.

“I am thrilled that Novartis will be moving forward with our CMT1A therapeutic program 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,” said Artie Suckow, co-founder and CEO of DTx Pharma. 

“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.”

Neuromuscular diseases

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 (CNS) indications.

“This acquisition underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system,” said Robert Baloh, global head of neuroscience for the Novartis Institutes for BioMedical Research. 

“We are excited to drive forward these promising preclinical programs and explore the potential of the FALCON platform.”

“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,” said Peter Condon, DTx Pharma’s CBO. 

“This agreement is also strong validation of our FALCON platform and its potential to break open new therapeutic areas by delivering siRNA beyond the liver.”

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.

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