ARTHEx Biotech S.L. is a spin-off company of the University of Valencia that is developing antimiR treatments against degenerative muscular disorders. The first target of the company will be to investigate anti-microRNAs for the treatment of myotonic dystrophy Type 1 (DM1).
DM1 is a genetic disease caused by abnormal DNA expansion. Which is an orphan disease affecting more than 900.000 people worldwide (estimated prevalence 1/8000). DM1 is a disease that affects the muscles and other body systems. The disease is characterized by progressive muscle loss and weakness.
Our research has revealed new therapeutic targets to treat the disease. The target and the mechanism of action of our molecules is new, and no other competitor has developed a similar approach.
In this year that ends, the company completed the Discovery phase and have begun the preclínical studies. This has included the selection of the lead compound, known as ATX-01.
The selection of the lead was carried out in three different phases: the first phase consisted of an “in vitro” screening, the second phase was a preselection, and the third phase consisted of an “in vivo” screening.
Once the lead has been selected, activities of the CMC (Chemical Manufacturing Control) plan have been developed, such as the characterization of the Drug Substance and the necessary specification for the release.
These results that allowed the selection of the lead were discussed with the U.S. Food and Drug Administration (FDA) in a successful meeting prior to the application of IND (Investigational New Drug) application.
In the first week of September the company, ARTHEx Biotech met the U.S. Food and Drug Administration (FDA) in a teleconference pre-IND meeting and reached alignment on activities to complete prior to IND (Investigational New Drug) submission.
“We are very pleased with the outcome of the meeting, we have clear guidance and are on track to submit the IND. '' said Beatriz Llamusí, PhD. Arthex Biotech co-founder and CEO. “We look forward to completing the preclinical development of ATX-01 in 2022”.
This year has been very fruitful, and the ARTHEx team has grown in all aspects, including the hiring of new highly experienced employees that will allow us to move forward antimiR therapeutics for the patients from the bench to the patients’ bed.
In addition to these international recognitions, at the national level we have been recognized as one of the best firms of the year by Bioval, the biotech hub of the Region of Valencia.
We have also got more than €1M of non-dilutive funding from public competitive grants to launch new R&D projects in the next 3 years. This will contribute to expanding our pipeline to other musculoskeletal diseases. Thanks CDTI (Neotec and Misiones), Generalitat Valenciana (IVACE and AVI) and the Spanish Ministry of Science (Torres Quevedo) for trusting us.
The ARTHEx team has also participated in various conferences such as TIDES USA, one of the largest conferences in the world on oligonucleotides and peptide therapies, were the CEO talks about Development of an antimiR-Based Therapy to Treat Myotonic Dystrophy Disease.
The end of the year brings no greater joy than the opportunity to express to you the season's greetings and good wishes for the new year 2022.