Drugs / ATX-01
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06300307 | Oct 2024 → Jul 2027 expected | myotonic dystrophy type 1 | ARTHEx Biotech S.L. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-07-23 | ARTHEx Biotech S.L. | Results ARTHEx Biotech Publishes First Demonstration of Improvement in a DM1-related Behavioral Alteration in a Preclinical Model of Myotonic Dystrophy Type 1 (DM1) arthexbiotech.com ↗
today announced the publication of new preclinical data demonstrating central nervous system (CNS) activity of its investigational antimiR-23b therapy, ATX-01, in Myotonic Dystrophy Type 1 (DM1). |
| 2026-05-27 | ARTHEx Biotech S.L. | Results ARTHEx Biotech Delivers Multiple Presentations at IDMC-15 Highlighting Clinical Progress of ATX-01 and Mechanistic Insights for Myotonic Dystrophy Type 1 (DM1) and 2 (DM2) arthexbiotech.com ↗
The presentations showcased the clinical progress of ATX-01 in the ongoing ArthemiR™ study and new mechanistic insights for DM1 and DM2. |
| 2026-03-11 | ARTHEx Biotech S.L. | Regulatory Arthex Biotech Granted FDA Fast Track Designation for ATX-01 for the Treatment of Myotonic Dystrophy Type 1 (DM1) arthexbiotech.com ↗
the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ATX-01 for the treatment of Myotonic Dystrophy type 1 (DM1). |
| 2026-02-25 | ARTHEx Biotech S.L. | Results ARTHEx Biotech Announces Publication in The American Journal of Human Genetics Highlighting the Discovery and Preliminary Preclinical Profile of ATX-01 for Myotonic Dystrophy Type 1 (DM1) arthexbiotech.com ↗ |
| 2024-11-07 | ARTHEx Biotech S.L. | Regulatory ARTHEx Biotech Announces FDA Rare Pediatric Designation (RPD) of ATX-01 for the Treatment of Myotonic Dystrophy Type 1 (DM1) arthexbiotech.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder. |
| 2024-02-28 | ARTHEx Biotech S.L. | Regulatory ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1) arthexbiotech.com ↗
ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). |
| 2022-12-15 | ARTHEx Biotech S.L. | Regulatory Arthex announces regulatory milestones met in its program to develop ATX-01 in Myotonic Dystrophy Type 1 arthexbiotech.com ↗
In separate applications, Arthex has obtained Orphan Drug Designation (ODD) from both the FDA and EMA for ATX-01 in the treatment of DM1, a rare disease. |
| 2022-07-20 | ARTHEx Biotech S.L. | Regulatory ARTHEx Biotech announces ATX-01 has been granted Orphan Drug Designation by the FDA arthexbiotech.com ↗
Arthex Biotech’s lead investigationalproduct ATX-01 has been granted Orphan Drug Designation ( ODD ) by the U.S. Food and Drug Administration ( FDA ). |
All press releases naming this drug 14 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ATX-01 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06300307 ↗ |
| Known as | X82108 | “ATX-01 is a formulation of the anti-microRNA 23b (anti-miR-23b), known as X82108, a novel type of antisense oligonucleotide” NCT06300307 ↗ |
| Action | Inhibit | “ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.” NCT06300307 ↗ |
| Route | Intravenous | “IV Administration of ATX-01” NCT06300307 ↗ |
| Target | MIR23B | “ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.” NCT06300307 ↗ |