The Charcot-Marie-Tooth Association July 17 hailed the news that global healthcare giant Novartis is acquiring DTx Pharma and its CMT1A therapy DTx-1252 as a significant step forward for the acceleration of novel CMT treatments capable of reaching the peripheral nervous system. Under the terms of the agreement, Novartis will make an upfront payment of $500 million and additional payments of up to $500 million upon completion of pre-specified milestones.

DTx-1252 is a first-in-class FALCON siRNA therapeutic that represses PMP22, the causative gene for CMT1A. The FALCON (fatty acid ligand conjugated oligonucleotide) platform enables the delivery and activity of small interfering RNA (siRNA) therapeutics to tissues beyond the liver, enhancing biodistribution and cellular uptake. By repressing PMP22, DTx-1252 reversed CMT1A in a mouse model that faithfully recapitulated the genetic and clinical manifestations of the disease. DTx-1252 treatment induced remyelination of axons to normal levels and increases muscle mass, grip strength, coordination and agility.

Artie Suckow, PhD, co-founder and CEO of DTx Pharma, said, “I am thrilled that Novartis will be moving forward with our CMT1A therapeutic program and the FALCON platform. With its resources and capabilities in neuromuscular diseases, Novartis is well positioned to accelerate the development of DTx-1252 and provide hope to patients, who are desperately in need of therapy.” He added, “DTx Pharma owes a lot of its success to the CMT1A community; patients initially inspired our efforts to tackle this debilitating disease several years ago and continue to inspire us today by sharing their stories with the DTx Pharma team. DTx is excited to pass the baton to Novartis and Bob Baloh to continue development of DTx-1252.”

Robert Baloh, MD, PhD, global head of neuroscience for the Novartis Institutes for BioMedical Research, said the acquisition “underscores the Novartis commitment to bringing life-changing medicines forward for patients with neuromuscular diseases and other disorders of the nervous system,” adding, “We are excited to drive forward these promising preclinical programs and explore the potential of the FALCON platform.”

According to CMTA Chief Research Officer Katherine Forsey, PhD, the acquisition highlights the progress made in positioning CMT as an attractive proposition to pharma, the founding principle of the CMTA’s Strategy to Accelerate Research (STAR).The CMTA has been increasing its collaborative work with DTx Pharma team members as they prepare for the commencement of first-in-human clinical trials of DTx-1252, Forsey said, adding, “We are excited by the prospect of continuing this work with the Novartis team and applaud their commitment to bringing life-changing medicines forward for patients with neuromuscular diseases like CMT.”

Noting the CMTA’s long history with Baloh, who served on its Scientific Advisory Board from 2015 to 2020, Forsey said “We look forward to working with Dr. Baloh and Novartis to further accelerate progress. The CMTA is committed to supporting clinical trial readiness and roll-out for CMT and we look forward to the next chapter of this treatment’s development.”

DTx Pharma announced June 7 that it had been granted orphan drug designation for DTX-1252, signifying the Food and Drug Administration’s recognition of the drug as a promising treatment option for CMT1A.

Read the press release from DTx Pharma.

Read the press release from Novartis.

Published: July 17, 2023