- DTx Pharma's Fatty Acid Ligand Conjugated Oligonucleotide (FALCON™) platform facilitates extra-hepatic delivery of siRNA therapeutics
- The acquisition includes DTx-1252 with the potential to deliver a transformative medicine to Charcot-Marie-Tooth Disease Type 1A (CMT1A) patients
- The acquisition also includes additional early-stage programs in neuromuscular and CNS indications
- Novartis will make an upfront payment of $500M and additional payments of up to $500M upon completion of pre-specified milestones
SAN DIEGO, July 17, 2023 -- DTx Pharma, invested and incubated by Viva BioInnovator (VBI), is a preclinical stage biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotide (FALCON™) platform. Recently they announced 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 was 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."
Dr. Han Dai, CIO of Viva Biotech and head of Viva BioInnovator, said, "Relying on the founding team's years of deep cultivation and accumulation of siRNA drugs, DTx has successfully developed the innovative RNA drug delivery platform FALCON, which is used to deliver RNA drugs to Organs other than the liver and has solved the pain point of RNA drug delivery. We are pleased to witness and accompany the growth of DTx Pharma from proof of concept to preclinical research and participated in its multiple rounds of financing. The acquisition has once again verified the strategic vision and professional post-investment support of the Viva investment team. With Novartis' accumulation in the field of rare diseases, we believe that the research and development of the DTx pipeline is expected to be further accelerated and better develop the field of rare diseases."
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.
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.
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 a 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 www.dtxpharma.com and follow DTx on LinkedIn and Twitter @DTxPharma.