ARTHEx Biotech

ARTHEx Biotech S.L. is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs.

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 advancing into clinical development for the treatment of myotonic dystrophy type 1 (DM1), a rare progressive muscle wasting disease. ATX-01 is an antimiR designed to target microRNA 23b (miR-23b), which is associated with regulating the expression of MBNL protein 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 targets toxic DMPK and MBNL proteins. ARTHEx is focused on advancing ATX-01 into a Phase 1/2a clinical trial for DM1 called ArthemiR™. In April 2023, ARTHEx announced the close of a €42 Series B to advance these efforts. 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.

• Small molecules
• Musculoskeletal Disorders
• Neurology
• Platform Technology