FIGHT-DM1 is a project aimed at developing, ATX-01, a first-in-class oligo therapy for myotonic dystrophy type 1 (DM1). In order to relieve the health-related pains of patients, our project aims to clinically validate our proposed product, ATX-01, as a disruptive disease-modifying therapy for myotonic dystrophy type 1. We aim to impact the quality of life of DM1 patients by relieving their symptoms and slowing the disease progression, while also contributing to significant economic savings for their families and healthcare systems.
About ATX-01
ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), which is associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1. It has been demonstrated in human DM1 myoblast cell lines that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein levels. Toxic DMPK and reduced levels of MBNL have been identified as the molecular underpinnings of DM1. ATX-01 is being evaluated in the Phase I-IIa ArthemiR™ trial for the treatment of DM1. ATX-01 has received Orphan Drug Designation for ATX-01 in DM1 from the US and European authorities.
ATX-01 was discovered through ARTHEx’s in-house discovery engine, which is designed to identify and optimize novel microRNA modulators and ensure their preferential delivery to target tissues, for the treatment of diseases in which microRNAs are involved in the disease pathogenesis.
About Myotonic Dystrophy Type 1 (DM1)
Myotonic dystrophy type 1 (DM1) is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other tissues (causing respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment). Most commonly, it manifests during adulthood (classic DM1), although DM1 can develop at birth in a congenital form, or during childhood. Although signs and symptoms vary among affected individuals, sadly, with progression of the disease, DM1 patients experience a reduction in the ability to perform activities of daily living. Moreover, patients have a significantly shortened lifespan and there is currently no approved treatment to slow the progression of the disease.
