DTx Pharma, an innovative biotechnology company incubated and invested in by Viva BioInnovator (VBI), recently made headlines when it was acquired by Novartis. Their remarkable FALCON™ platform allows for the delivery and activity of small interfering RNA (siRNA) beyond the liver – a feat that is sure to revolutionize the therapeutic landscape.
With their lead program currently in preclinical development and with FDA Orphan Drug Designation, DTx Pharma is pushing the boundaries on researching a potential treatment for 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. With this acquisition, Novartis and DTx Pharma are now poised to bring new hope to patients suffering from CMT1A and beyond.
For the estimated 150,000 people who suffer from the life-debilitating CMT1A, caused by the underlying genetic cause of PMP22, hope is finally on the horizon. DTx Pharma is developing a first-of-its-kind FALCON siRNA candidate targeting PMP22, named DTx-1252.
Pre-clinical data has demonstrated the reversal of the disease in rodent models and it has been proven to translate to higher species. And with IND-enabling studies progressing well, a therapy may soon be within reach.
I am overflowing with excitement that Novartis has chosen to launch forward with our CMT1A therapy program and the FALCON platform. With specialized expertise and experience in neuromuscular diseases, Novartis is capable of driving DTx-1252’s development in an expeditious manner, bringing hope to the many patients in need of treatment.
Looking back on our team’s achievements, I’m overwhelmed with pride and admiration for our commitment to extra-hepatic delivery of siRNA, particularly for our efforts in developing the first investigational FALCON siRNA that can be administered to the peripheral nervous system to cure CMT1A at its genetic root.
Novartis has acquired the entire FALCON platform, along with two additional programs for neuromuscular and CNS indications. This addition to their already established CMT1A program provides expanded possibilities for exploring treatments in the varying fields of neurological conditions.
Novartis is thrilled to add the groundbreaking FALCON platform to its arsenal, furthering its ambition to discover life-changing treatments for patients with neurologic diseases. With this acquisition, Novartis demonstrates its commitment to advancing medicines and therapies for neurological disorders, and its dedication to the exploration of the FALCON platform’s potential. The Novartis Institutes for BioMedical Research will be at the forefront of driving this progress forward, offering hope to patients and families worldwide.
DTx Pharma is thrilled by the partnership with Novartis, which will open up new avenues for the development of DTx-1252. The expertise and experience of the Novartis team in CMT1A biology, as well as their insight into the needs of patients, was apparent from the outset. Peter Condon, DTx Pharma’s Chief Business Officer, also emphasized the role of the FALCON platform in unlocking treatment opportunities through the delivery of siRNA beyond the liver.
Dr. Han Dai, CIO of Viva Biotech and head of Viva BioInnovator, is thrilled to have witnessed the growth of DTx Pharma from proof of concept to preclinical research, and its multiple rounds of financing. Throughout this journey, Viva’s investment team have provided an unwavering strategic vision and post-investment support to DTx.
Through this successful acquisition, DTx’s innovative RNA drug delivery platform – FALCON, which is used to deliver RNA drugs to organs other than the liver – has solved the pain point of RNA drug delivery. With Novartis’ accumulation in the field of rare diseases, we are confident that DTx’s research and development pipeline will be further accelerated and better develop the field of rare diseases.
Novartis and representatives have reached an exciting agreement: Novartis will make an initial payment of half a billion dollars, plus up to $500 million more once certain objectives have been satisfied.
About CMT1A
CMT1A is an all-too-common inherited neuromuscular disorder that affects an estimated 150,000 people in the U.S. and Europe. The condition is caused by overexpressing the PMP22 gene in the Schwann cells, which affects the myelination of peripheral nerves and worsens over time, leading to an inability to walk independently and impacting quality of life significantly. Unfortunately, there are no FDA-approved therapies that address its root cause directly, leaving patients without an appropriate answer to their suffering.
About DTx-1252
DTx-1252 is a revolutionary FALCON™ siRNA therapeutic that could provide the first-ever treatment for CMT1A. DTx-1252 works by repressing the PMP22 gene, and has been clinically validated in preclinical studies to successfully reverse CMT1A features such as remyelination of axons to normal levels, improved electrophysiological measures, increased muscle mass, grip strength, coordination and agility. This exciting breakthrough treatment could potentially lead to a new hope for CMT1A sufferers.
About DTx Pharma
DTx Pharma, Inc. is a privately held biotechnology company harnessing the power of its revolutionary Fatty Acid Ligand Conjugated OligoNucleotide (FALCON™) technology platform to deliver RNA-based therapeutics to tissues and cell types throughout the body.
In exciting preclinical studies, FALCON siRNAs have been proven to effectively and durably silence target genes in the peripheral nervous system (PNS), skeletal muscle, heart, skin and CNS when administered intravenously, subcutaneously and intrathecally.
This cutting-edge research has prompted an influx of more than $115M in investment from an impressive array of healthcare investors such as RA Capital Management, Access Biotechnology, Surveyor Capital (a Citadel company), Eli Lilly and Company, Friedman Bioventure, Viva Biotech Holdings, and research foundations like the CMT Research Foundation (CMTRF) and the National Institutes of Health. DTx Pharma’s pipeline is currently focused on PNS, CNS and neuromuscular diseases, bringing life-changing treatments to people around the world.
About Viva Biotech Holdings
Viva Biotech, a force of leading scientific innovation and global biopharmaceutical development since 2008, provides comprehensive solutions, from early-stage Structure-Based Drug R&D to commercial drug delivery, to help bring new treatments to life.
With our state-of-the-art equipment in X-ray crystallization, Cryo-EM, ASMS, SPR, HDX, CADD, and highly experienced chemistry team that support drug design, medicinal chemistry, custom synthesis, chemical analysis and purification, kilogram scale-up, peptide synthesis and corresponding bioassays, Viva’s integrated CMC (Chemical, Manufacturing, and Control) service, provided through our subsidiary Langhua Pharma, allows us to meet the needs of pharmaceutical and biotech partners worldwide, from preclinical to commercial manufacturing.
To accelerate innovation and impact, we also invest in promising startup companies through our equity for service (EFS) model.