Arthex is developing ATX-01. An first-in-class treatment for DM1.ATX-01 is a single stranded oligonucleotide, heavily chemically modified to improve its stability and linked to a fatty acid to improve its delivery to the muscles, the main target tissue in this disease in adult patients.
Approximately, 1 Million people suffer from Myotonic Dystrophy type 1 DM1 in the world. Moreover, ATX-01 is part of the vibrant oligonucleotide therapeutics market, where the average value of comparable licensing deals is around €100M.
To date we have risen €7M of funding to complete the preclinical development of ATX—01.
We have been continuously supported by our current financiers and are seeking €20M for further development. Our aims is to complete a Phase I/IIa by year 2025 and chronic preclinical studies that would enable a registrational PhII/III by year 2028. Our development aims for an approval of the FDA (pre-IND meeting, Sep2021).