Novartis Acquires DTx Pharma for Up to $1 Billion to Advance siRNA Therapies for Neurological Disorders

On July 17, Novartis acquired DTx Pharma, a clinical-stage biotechnology company focused on leveraging its proprietary FALCON platform to develop siRNA therapies for neuroscience indications. Under the terms of the agreement, Novartis will pay $500 million upfront and up to an additional $500 million upon achieving predefined milestones.

DTx's leading project is DTx-1252, which is being developed for the treatment of Type 1A Charcot-Marie-Tooth disease (CMT1A). DTx-1252 targets the root cause of CMT1A — the overexpression of PMP22, a protein that can lead to abnormal myelin function, which supports and insulates nerves in the peripheral nervous system. The FALCON platform facilitates this targeted approach by combining siRNA with naturally occurring fatty acids to enhance biodistribution and cellular uptake in the targeted tissues and cell types.

Charcot-Marie-Tooth Disease (CMT) is a group of hereditary diseases that affect the nervous system. CMT1A is the most common subtype of CMT, impacting approximately 150,000 patients in the United States and Europe. CMT1A is a slowly progressive degenerative disease of the peripheral nervous system, which can lead to progressive muscle atrophy, neuropathic pain, and difficulty walking, significantly affecting patients' quality of life. DTx-1252 has the potential to become the first-in-class therapy for this disease, as there are currently no approved therapies aimed at addressing the underlying genetic factors of CMT1A.

In addition to DTx-1252, the deal also brings Novartis two additional preclinical programs for other neuroscience indications.

This acquisition expands Novartis' siRNA product portfolio and adds DTx's FALCON platform to Novartis' siRNA toolkit.

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