SAN DIEGO, CA, USA I July 17, 2023 I DTx Pharma, a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotide (FALCON™) platform, announced today that it 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 United States 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, Ph.D., 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.”

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 a 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 $500M and additional payments of up to $500M upon completion of pre-specified milestones.

Evercore served as financial advisor to DTx Pharma, while Wilson Sonsini Goodrich & Rosati served as legal counsel.

About CMT1A
CMT1A is the most common inherited neuromuscular disease, affecting approximately 150,000 patients in the U.S. and Europe. Currently, there are no approved therapies directly addressing the underlying cause of the disease for patients. 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.

About DTx-1252
DTx-1252 is a potential first-in-class FALCON™ siRNA therapeutic for the treatment of CMT1A. 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.

About DTx Pharma
DTx Pharma, Inc. is a privately held biotechnology company, addressing the challenges associated with delivering RNA-based therapeutics beyond the liver with the company’s proprietary Fatty Acid Ligand Conjugated OligoNucleotide (FALCON™) technology platform. The FALCON platform leverages fatty acids for enhanced biodistribution and cellular uptake to tissues and cell types throughout the body. In preclinical studies, FALCON siRNAs have demonstrated potent and durable repression of target genes in the peripheral nervous system (PNS), skeletal muscle, heart, skin and CNS. FALCON siRNAs can be delivered by intravenous, subcutaneous and intrathecal routes of administration and can be manufactured at relatively low cost. The company has a pipeline focused on PNS, CNS and neuromuscular diseases. DTx Pharma has raised more than $115M in combined investment from several of the world’s leading healthcare investors including RA Capital Management, Access Biotechnology, Surveyor Capital (a Citadel company), Eli Lilly and Company, Friedman Bioventure, Viva Biotech Holdings, as well as support from research foundations including the CMT Research Foundation (CMTRF) and the National Institutes of Health.

To learn more about DTx Pharma, please visit and follow DTx on LinkedIn and Twitter @DTxPharma.

SOURCE: DTx Pharma