DTx Pharma, a preclinical biotechnology company tackling the challenges of delivering oligonucleotide therapies with a fatty acid ligand-conjugated oligonucleotide (FALCON) platform, has been acquired by Novartis.
The FALCON platform enables delivery and therapeutic activity of small interfering RNA (siRNA) to tissues outside the liver, enhancing cellular biodistribution and uptake. DTx Pharma’s flagship program is currently in preclinical development, under 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 lifelong loss of muscle function and disability.
Currently, there are no approved therapies that treat PMP22, the underlying genetic cause of CMT1A, for the approximately 150,000 patients living with debilitating disease in the US and Europe. DTx Pharma’s key asset, DTx-1252, is a new and potential class one FALCON siRNA candidate targeting PMP22. This asset offers a robust preclinical package, demonstrating disease reversal in preclinical rodent models and translation to higher species with studies that enable IND to thrive.
“I am excited that Novartis will be moving forward with our CMT1A therapy program and the FALCON platform. With its resources and capabilities in neuromuscular disease, Novartis is well positioned to accelerate development of DTx-1252 and provide hope to patients who most need therapy,” said Artie Suckow, co-founder and CEO of DTx Pharma.
“I am also very proud of the commitment and passion of our team, which has made DTx Pharma a leader in extra-liver siRNA delivery, as demonstrated by our work advancing research into the first FALCON siRNA designed for peripheral delivery. nervous system to treat genetic causes 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 Novartis’ commitment to bringing life-changing medicine to the fore for patients with neuromuscular diseases and other disorders of the nervous system, said Robert Baloh, global head of neuroscience for Novartis Institutes for BioMedical Research.
“We are excited to advance this promising preclinical program and explore the potential of the FALCON platform.”
“The Novartis team’s in-depth knowledge of CMT1A biology and solid understanding of patient burden was evident from our first interactions, and we are very pleased with their support to advance DTx-1252 development,” said Peter Condon, CBO of DTx Pharma.
“This agreement is also a strong validation of our FALCON platform and its potential to open up new therapeutic areas by delivering siRNA outside the liver.”
Under the terms of the agreement, Novartis will make an upfront payment of $500 million and an additional payment of up to $500 million upon completion of predetermined milestones.
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