The pharmaceutical drug, named ATX-01, has just been authorised to start clinical trials in the US. This new therapeutic advance gives hope to those suffering from Myotonic Dystrophy type 1, an hereditary degenerative disease currently lacking of effective treatment.
This new pharmaceutical drug, developed by the biotech company ARTHEx Biotech S.L., is based on initial research carried out by the Translational Human Genomics group of the Institute of Biotechnology and Biomedicine (BIOTECMED). It will be the first in the category of microRNAs blockers trialled in Myotonic Dystrophy type 1 (DM1), and the first to reach clinical stages in ARTHEx Biotech.
The Food and Drug Administration of the United States (FDA) has authorised the UV spin-off to start clinical trials in two stages to test the safety of the pharmaceutical drug and its activity. It is expected that the first patient will be inscribed in mid 2024.
The ArthemiR™ trial is a double-blind study, controlled by a placebo and with dose increase that is expected to inscribe patients with classic myotonic dystrophy type 1 (DM1) The main objective is to determine the safety and tolerance to rising single and multiple doses of ATX-01 in participants with DM1. ARTHEx will also research the response at muscle level through biomarkers. Furthermore, the clinical evaluation criteria of the trial will include measures related to muscle function, patient-informed results and the measurements of quality of life.
“Receiving the authorisation of the FDA to start our first study on human beings, ArthemiR™, for ATX-01 in DM1 is an important milestone for ARTHEX and for the patients with DM1 and their families that need an approved therapeutic option”, stated Dr Judith Walker, medical director of Arthex. “We plan on launching the ArthemiR™ study in the US first, followed by Canada and Europe”, she added.
Arthex Biotech
Arthex Biotech is, since 2020, a spin-off of the Universitat de València founded in late 2019 from the Translational Genomics Research Group, led by Full University Professor of Genetics Rubén Artero. His team had previously discovered a new therapeutic target for DM1 and invented molecules that allowed symptoms of the disease to be improved. The invent was patented and the proof of concept, in an animal model, was published in the Nature Communications magazine. The UV later licensed the patent to ARThex for its use.
ARTHEx also works in the discovery of new pharmaceutical drugs to identify and develop modulators of microRNA for other diseases with high non-covered medical necessities, including genetic diseases such as DM1. This spin-off of the Universitat de València is headquartered at the Science Park of the academic institution.
