VALENCIA, Spain, October 18, 2024 -
ARTHEx Biotech S.L., a biotechnology company in the clinical-stage, announced that the initial participant has received a dose in their Phase I-IIa ArthemiR™ Trial. This trial investigates
ATX-01 for
Myotonic Dystrophy Type 1 (DM1), a rare
neuromuscular disorder leading to
muscle weakness and other severe complications. At present, no treatments exist that modify the course of the disease.
ATX-01 represents the first anti-microRNA therapeutic explored in DM1, targeting
microRNA 23b (miR-23b). This microRNA influences the expression of
MBNL proteins, vital for correct splicing of many mRNAs and subsequent protein production. The pathogenesis of DM1 involves the sequestration of MBNL by toxic DMPK mRNA in the nucleus and the repression of MBNL production due to miR-23b overexpression. Both processes result in a net reduction of MBNL, essential for normal regulatory functions. ATX-01’s dual mechanism increases MBNL production and destabilizes toxic DMPK foci, reducing DMPK mRNA and releasing sequestered MBNL. This dual action increases active MBNL levels and corrects splicing.
Dr. Frédéric Legros, Chairman and CEO of ARTHEx, expressed pride in reaching this milestone, emphasizing the significance of this trial for DM1 patients and their families. Dr. Beatriz Llamusí, Chief Scientific Officer and Co-Founder, highlighted that ATX-01 is a unique molecule developed entirely by ARTHEx, with substantial potential to improve patient outcomes due to its dual mechanism of action.
The ArthemiR™ trial is a global, double-blind, placebo-controlled study involving single and multiple ascending doses in participants with classic DM1. Its primary goal is to assess the safety and tolerability of ATX-01. Additionally, ARTHEx will examine target engagement at the muscle level using biomarkers such as MBNL levels and RNA splicing index. Clinical endpoints will include measures of muscle function, patient-reported outcomes, and quality of life assessments. The trial aims to enroll fifty-six participants to evaluate ATX-01's performance comprehensively.
Dr. Judy Walker, Chief Medical Officer of ARTHEx, noted the well-tolerated safety profile of ATX-01 in non-clinical studies. She expressed excitement about advancing the clinical trial, which offers a novel approach to treatment due to its dual mechanism. Dr. Walker hopes that ATX-01 will provide functional benefits, a favorable safety profile, and improved quality of life for DM1 patients. She emphasized the importance of continuing participant enrollment and initiating additional study sites globally.
Dr. Valeria Sansone, Professor of Neurology at the University of Milan, remarked on the significance of dosing the first participant in the ArthemiR trial. She highlighted that new clinical studies with innovative treatments like ATX-01 are crucial for DM1 patients, families, and the broader community to gain knowledge that could lead to better clinical outcomes and potentially an approved treatment.
ATX-01 is designed to target miR-23b, implicated in DM1 pathogenesis. In human DM1 myoblast cell lines and two murine models, ATX-01 has demonstrated a unique dual mechanism: reducing toxic DMPK mRNA and increasing MBNL protein production. ARTHEx’s in-house discovery engine, developed to identify and optimize gene expression modulators, discovered ATX-01.
Myotonic dystrophy type 1 (DM1) affects over a million people globally, causing muscle and other tissue issues, including respiratory problems, fatigue, cardiac abnormalities, gastrointestinal complications, and cognitive impairments. The disease, which commonly manifests in adulthood as classic DM1, significantly reduces a patient’s ability to perform daily activities and shortens lifespan. Currently, there is no approved treatment to slow its progression.
ARTHEx Biotech, headquartered in Valencia, Spain, focuses on developing innovative gene expression modulation medicines. Their lead compound, ATX-01, is under evaluation for DM1 in the Phase I-IIa ArthemiR™ trial. The trial is co-funded by the EIC Accelerator program. ARTHEx continues to advance its discovery engine to develop nucleic acid-based therapies for other genetic disorders with high unmet medical needs.
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