Beatriz Llamusi Troísi, Chief Executive Officer & Chief Scientific Officer at ARTHEx Biotech, presented on the preclinical development of a lipophilic conjugated antimir to treat myotonic dystrophy type one. The company, founded in December 2019, focuses on developing treatments based on the modulation of microRNAs for high-end medical needs. At the time of the presentation, ARTHEX was at the preclinical stage but was preparing for the IND submission with the first product expected by the summer.
The drug, ATX-01, is an anti-microRNA against miR-23B with a dual mechanism of action. It demonstrated ten times more potency in muscle, increased durability of effects, and safety with no complement activation or haemolytic potential. The drug works by blocking miR-23B, which is upregulated in myotonic dystrophy type one, contributing to the loss of function of MBNL proteins crucial for splicing. By targeting miR-23B, ATX-01 helps in derepressing the translation of MBNL and decreasing the expression of toxic DMPK RNA.
Preclinical results showed significant outcomes, including a complete rescue of strength in mice and a dose-dependent splicing rescue. The drug also demonstrated a preferential elevation of MBNL levels in tissues affected by the disease, such as muscle, heart, diaphragm, and brain. ARTHEX Biotech had completed the necessary studies requested by the FDA and EMA for the submission of the clinical trial application. The company expected to start the Phase 1/2a trial soon and had obtained orphan drug designation by both the FDA and EMA.
The presentation highlighted the promising potential of ATX-01 in treating myotonic dystrophy type one and the company's progress towards clinical trials.