Novartis Acquires DTx Pharma and Expands Its Neuroscience Pipeline

On July 17, Novartis announced the acquisition of the preclinical-stage biotechnology company DTx Pharma for $500 million. This acquisition will serve to bolster the Swiss healthcare company’s neuroscience pipeline. DTx Pharama’s lead program is for the treatment of Charcot-Marie-Tooth disease(CMT), a neuromuscular disease. 

Related Article: Novartis Plans to Acquire Chinook Therapeutics in a Potential $3.5B Deal, Revolutionizing IgAN Treatment Landscape

Novartis Takes a Leap Forward in Neurological Therapeutics

According to the agreement, Novartis will acquire DTx Pharma for $500 million, and upon the completion of specified milestones, they will make an additional payment of up to $500 million. Novartis will have the rights to DTx’s FALCON platform for siRNA therapeutic delivery. Additionally, they will also gain the rights to two early-stage programs for central nervous system and neuromuscular indications. But more importantly, they will gain access to DTx’s lead program for Charcot-Marie-Tooth Disease Type 1A (CMT1A). 

This represents Novartis’s second acquisition in the past few months, following its acquisition of Chinook Therapeutics for $3.2 billion. It will strengthen their neuroscience pipeline as well as their therapeutic platforms for the development of medicines. The Global Head of Neuroscience for the Novartis Institutes for BioMedical Research, Robert Baloh, said, “This acquisition underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system.” 

Advancing Treatment for Charcot-Marie-Tooth Disease

CMT is an inherited disorder that causes nerve damage. There is currently no cure for CMT. The available treatments consist mainly of physiotherapy and occupational therapy. This shows a clear need for the development of therapies that address the underlying cause of CMT. CMT1A is the most common subtype of CMT, occurring in about two-thirds of all CMT cases. This disease is characterized by muscle weakness and atrophy and mostly affects a patient’s lower legs, feet, hands, and forearms. CMT1A is the result of a point mutation of the PMP22 gene, which is crucial for the maintenance and creation of the myelin sheath. Therefore, CMT1 results in damage to the myelin sheath, which covers nerves. 

DTx’s lead program, called DTx-125, is a potential first-in-class siRNA candidate that targets PMP22. DTx-1252 was recently granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). This candidate uses DTx’s Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform to repress the overexpression of PMP22. It essentially reverses the effects of PMP22 overexpression by remyelating the axons to normal levels. DTx-1252 is in the preclinical stage and is anticipated to move into clinical development in 2023. 

Their FALCON platform combines siRNAs with fatty acids to deliver RNA therapeutics to tissues across the body. This combination improves the RNAs’ biodistribution and cellular uptake in target tissues. One of the main problems with the use of siRNAs is that they are cleared quickly by the kidneys. FALCON siRNAs are able to silence genes that cause diseases beyond the liver. This technology builds on Novartis’s existing xRNA technology. The purchase of DTx Pharma by Novartis is an important milestone in the company’s expansion of its neuroscience portfolio and in addressing the unmet needs of patients suffering from neuromuscular disorders.

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GeneOnline Team