FDA Approves ARTHEx's Phase I-IIa Trial for DM1 Drug ATX-01

Spanish clinical-stage biotechnology firm ARTHEx Biotech S.L. has been granted approval by the U.S. Food and Drug Administration (FDA) to commence the Phase I-IIa clinical trial of ATX-01, a novel microRNA therapeutic candidate for the treatment of Myotonic Dystrophy Type 1 (DM1). ATX-01, an antimiR oligonucleotide, is aimed at microRNA 23b (miR-23b), which plays a role in the regulation of MBNL proteins, a key factor in the development of DM1. This rare neuromuscular disorder is characterized by muscle weakness and various life-altering complications, with no current disease-modifying treatments available.

Dr. Judith Walker, the Chief Medical Officer of ARTHEx, has expressed that the FDA's clearance is a significant step forward for the company and for patients with DM1 who are in desperate need of an effective treatment. The unique dual mechanism of ATX-01, which targets both the toxic DMPK mRNA and the reduced active MBNL levels, is expected to provide substantial therapeutic benefits to DM1 patients. The company plans to initiate the study in the United States, with subsequent expansion to Canada and Europe.

The ArthemiR™ trial is designed as a double-blind, placebo-controlled study involving dose escalation. It is anticipated to include participants diagnosed with classic DM1. The primary goal is to assess the safety and tolerability of ATX-01 in single and multiple ascending doses. Additionally, the trial will explore the drug's target engagement at the muscle level through biomarkers such as MBNL levels and splicing index. Clinical endpoints will encompass muscle function assessments, patient-reported outcomes, and quality of life metrics.

Dr. Nicholas Johnson, a prominent figure in neurology research and the lead investigator for the trial, has highlighted the importance of new agents with distinct mechanisms of action in clinical trials, as they raise the prospect of developing effective and safe treatments for DM1, a multisystemic condition. The patient community is eager for new therapeutic options, and the trial's initiation is eagerly anticipated.

ATX-01 has been developed using ARTHEx's proprietary discovery platform, which is dedicated to identifying and optimizing microRNA modulators for diseases where microRNAs play a role in pathogenesis. The compound has received Orphan Drug Designation from both U.S. and European authorities for its potential use in DM1.

Myotonic dystrophy type 1 (DM1) is a debilitating disease that impacts over a million people globally. It affects muscles and other tissues, leading to respiratory issues, fatigue, hypersomnia, cardiac problems, severe gastrointestinal issues, and cognitive and behavioral impairments. The disease typically presents in adulthood but can also manifest congenitally or during childhood. Unfortunately, as DM1 progresses, patients face a decline in their ability to perform daily activities and have a significantly reduced lifespan.

ARTHEx Biotech, headquartered in Valencia, is dedicated to the advancement of innovative medicines through microRNA modulation. The company is not only focused on the Phase I-IIa ArthemiR™ trial for DM1 but is also working on identifying and developing microRNA modulators for other diseases with significant unmet medical needs, particularly genetically-driven conditions like DM1.