FDA Clears ARTHEx's ATX-01 Phase I-IIa Trial for DM1 Treatment

ATHEx Biotech, a company at the forefront of microRNA modulation for medical innovation, has received approval from the FDA to commence clinical trials for ATX-01, a pioneering microRNA-based treatment for Myotonic Dystrophy Type 1 (DM1). This marks a significant step, as ATX-01 is the first of its kind to be tested for DM1 and the inaugural drug candidate from the company's pipeline to reach clinical evaluation.
Scheduled for the second quarter of 2024, the first patient is anticipated to be enrolled in the Phase I-IIa ArthemiR™ study. ATX-01 is engineered to target miR-23b, a microRNA linked to the regulation of MBNL proteins, which are integral to the development of DM1. This neuromuscular condition leads to muscle weakness and other severe complications, and currently, no treatments are available to alter its course.
Dr. Judith Walker, ARTHEx's Chief Medical Officer, highlighted the importance of the FDA's clearance, emphasizing ATX-01's potential to offer therapeutic benefits through its dual-action mechanism that addresses both the toxic DMPK mRNA and the diminished levels of active MBNL. The company plans to initiate the study in the United States, with subsequent expansion to Canada and Europe.
The ArthemiR™ study is designed as a double-blind, placebo-controlled trial that will assess the safety and tolerability of ATX-01 in patients with classic DM1. It will also measure biomarker engagement at the muscle level and clinical endpoints related to muscle functionality, patient-reported outcomes, and quality of life.
Dr. Nicholas Johnson, the principal investigator for the trial and a prominent figure in neurology research, expressed enthusiasm for the trial, noting the importance of new agents with distinct mechanisms of action in the pursuit of effective treatments for DM1.
ATX-01's development was facilitated by ARTHEx's proprietary discovery platform, aimed at identifying and refining novel microRNA modulators for diseases where microRNAs play a role in pathogenesis. The drug has been granted Orphan Drug Designation for DM1 by both the US and European authorities.
Myotonic Dystrophy Type 1 is a debilitating disease that impacts over a million individuals globally, affecting not only muscles but also other tissues, leading to a range of serious health issues. The disease's onset varies, but its progression invariably results in a decreased capacity for daily activities and a reduced life expectancy.
ARTHEx Biotech, headquartered in Valencia, Spain, is dedicated to advancing microRNA-based therapeutics for diseases with significant medical needs, including genetically influenced conditions like DM1. The company continues to innovate and develop new treatments using its cutting-edge discovery engine.

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