The US FDA Clears ARTHEx Biotech’s IND Application to Commence the P-I-IIa (ArthemiR) Study of ATX-01 for Myotonic Dystrophy Type 1 (DM1)
Shots:
- ARTHEx Biotech’s IND application to begin P-I-IIa (ArthemiR) study of ATX-01 to treat Myotonic Dystrophy Type 1 (DM1) has been cleared by the US FDA. It is expected to begin first in the US, followed by Canada & the EU
- The P-I-IIa (ArthemiR) dose-escalation study assesses the safety & tolerability of ATX-01’s single & multiple ascending doses in patients with DM1. Target engagement at the muscle level through MBNL levels & splicing index will also be evaluated along with measures related to muscle function, patient-reported outcomes & quality of life
- ATX-01 is an antimiR that targets microRNA 23b (miR-23b) which regulates MBNL proteins expression responsible for the pathogenesis of DM1
Ref: ARTHEx Biotech | Image: ARTHEx Biotech
Related Post:- Vertex Collaborated with Entrada Therapeutics to Develop Endosomal Escape Vehicle Therapies for Myotonic Dystrophy Type 1
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
