Swiss pharmaceutical giant Novartis has acquired DTx Pharma, a preclinical stage biotechnology company based in California renowned for its Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform, which addresses the delivery challenges of oligonucleotide therapeutics.

This acquisition, including the revolutionary FALCON platform, will enhance the delivery of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, improving biodistribution and cellular uptake. DTx Pharma’s lead program, aiming to treat Charcot-Marie-Tooth Disease Type 1A (CMT1A), is currently in preclinical development, with FDA Orphan Drug Designation.

CMT1A is a progressive, neuromuscular, autosomal-dominant disease that affects around 150,000 patients in the United States and Europe. It leads to a lifelong loss of muscle function and disability. To date, there are no approved therapeutics that address PMP22, the genetic cause of CMT1A. However, DTx Pharma’s lead asset, DTx-1252, a novel, potential first-in-class, FALCON siRNA candidate, is designed to target PMP22. Preclinical trials show promising results, indicating the reversal of disease in rodent models, with Investigational New Drug (IND) enabling studies progressing well.

Artie Suckow, co-founder and CEO of DTx Pharma, expressed excitement at the acquisition, confident in Novartis’ capabilities to accelerate the development of DTx-1252, and provide a potential therapeutic solution to CMT1A patients. Apart from the CMT1A program, Novartis also gained full rights to the FALCON platform and two other early-stage programs in neuromuscular and central nervous system (CNS) indications.

Robert Baloh, Global Head of Neuroscience for the Novartis Institutes for BioMedical Research, shared that this acquisition highlights Novartis’ commitment to developing life-changing treatments for neuromuscular diseases and other nervous system disorders. Peter Condon, DTx Pharma’s CBO, expressed enthusiasm over Novartis’ support and its recognition of the FALCON platform’s potential to expand new therapeutic areas by delivering siRNA beyond the liver.

The acquisition agreement entails an upfront payment of $500 million from Novartis, with additional payments of up to $500 million pending the achievement of pre-specified milestones.

  Artie Suckow, California, Central nervous system, Charcot-Marie-Tooth Disease Type 1A, DTx Pharma, Novartis, Pharma acquisition news, Robert Baloh