Arthex Biotech reports central nervous system improvements in DM1 preclinical model

VALENCIA, Spain – Arthex Biotech, a clinical-stage biotechnology company, has published new preclinical data illustrating central nervous system (CNS) activity of its investigational therapy ATX-01 for Myotonic Dystrophy Type 1 (DM1). The study provides the first known scientific evidence linking molecular correction in the brain with functional behavioral improvements in a DM1 animal model.

DM1 is a disabling neuromuscular disorder that affects not only muscles but also the brain, leading to cognitive impairment and behavioral changes. Most therapeutic approaches have historically struggled to effectively reach the CNS. Arthex's antimiR-23b therapy, developed on its BOOST-ON™ platform, demonstrated the ability to cross the blood-brain barrier and engage disease biology in the brain.

In preclinical studies, ATX-01 reduced toxic DMPK transcripts in the brain and increased MBNL1/2 protein levels, correcting disease biology. Importantly, this resulted in the normalization of behavioral alterations, such as exploratory activity, in the animal model. No significant toxicity or neuroinflammation was observed.

These findings suggest the potential for a disease-modifying approach that targets both the muscular and neurological aspects of DM1. "This research represents a major step forward for the DM1 field," said Beatriz Llamusí, Ph.D., Co-founder and Chief Scientific Officer of ARTHEx Biotech. "For the first time, we are seeing a clear connection between improvement of molecular alterations in the brain and meaningful functional improvements."

Arthex Biotech is advancing ATX-01 in its ongoing ArthemiR clinical trial and aims to deliver meaningful benefits to patients across the full spectrum of DM1. The company is working closely with regulatory authorities, clinical investigators, and patient advocacy groups to accelerate progress.