VALENCIA, Spain, April 19, 2024 /PRNewswire/ — ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, today announced an oral presentation at the upcoming Oligonucleotide & Peptide Therapeutics (TIDES) Conference 2024, being held in a hybrid format May 14-17, 2024 in Boston and online. 

Details of the oral presentation are as follows:

Presenter: Dr. Frédéric Legros, ARTHEx Biotech Executive Chairman and CEO

Title: “Use of a Lipophilic-conjugation to Deliver antimiR-23b into Skeletal Muscle and Nervous System as a Dual Therapeutic Approach in DM1“

Session: Oligonucleotide Discovery, Preclinical and Clinical

Date/Time: Friday, May 17, 9:30am 

For more information about TIDES, please visit the conference website here.

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.

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.

ARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs.  The Company’s lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR™ trial.  ARTHEx is also advancing its in-house discovery engine to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetically-driven diseases like DM1.  The Company headquarters are in Valencia, Spain.

For more information, please visit www.arthexbiotech.com and engage with us on LinkedIn.

 

 

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SOURCE ARTHEx Biotech

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