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FDA Clears ARTHEx Biotech for Phase I-IIa ArthemiR™ Trial of ATX-01 in Myotonic Dystrophy Type 1 (DM1)

Thursday, February 29, 2024

ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, has announced that the U.S. Food and Drug Administration (FDA) has given clearance for the Phase I-IIa ArthemiR™ study of ATX-01 for treating Myotonic Dystrophy Type 1 (DM1).

ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic being investigated for DM1 and marks the first therapeutic from ARTHEx's pipeline to enter clinical trials. miR-23b is known to regulate the expression of MBNL proteins involved in the pathogenesis of DM1, a rare neuromuscular disorder causing muscle weakness and other complications. Currently, there are no treatments that modify the progression of DM1.

Arthex, expressed the significance of FDA clearance, stating, "Receiving FDA clearance to initiate our first-in-human study, ArthemiR™, for ATX-01 in DM1 is a major milestone for ARTHEx and for DM1 patients and their families who are in need of an approved therapeutic option." Dr. Walker continued, "ATX-01 holds significant potential to deliver therapeutic benefit to DM1 patients, based on its dual mechanism of action that targets both the toxic DMPK mRNA and the reduced active MBNL levels. We plan to launch the ArthemiR™ study first in the US, followed by Canada and Europe."

The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1). The primary objective is to determine the safety and tolerability of single and multiple ascending doses of ATX-01 in DM1 participants. ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index. Additionally, the trial will include clinical endpoints related to muscle function, patient-reported outcomes, and quality of life measures.

Dr. Nicholas Johnson, Professor and Vice Chair of Research in the Department of Neurology at Virginia Commonwealth University, and the lead investigator for the ArthemiR™ trial, commented on the significance of new agents entering clinical trials, stating, "It is exciting to see new agents coming to clinical trials, especially compounds with different mechanisms of action. This increases our hope of one day having effective and safe, disease-modifying treatments for this multisystemic condition. Patients are eager for new trials, and we are delighted to start enrolment in the coming months."

ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b (miR-23b), associated with regulating MBNL proteins involved in the pathogenesis of DM1. It has been shown 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 will shortly be 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.

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.



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