Recognizing that severe myotonic dystrophy involves CNS impairment, Arthex deliberately invested in a lipid conjugation delivery system for its RNA therapeutic. This strategic choice was made specifically to cross the blood-brain barrier, enabling the treatment of both muscular and neurological symptoms of the disease.
Instead of targeting the DMPK gene like competitors, Arthex's ATXO1 targets miR23B. This indirectly increases MBNL protein levels to compensate for sequestration while also destabilizing the toxic DMPK foci. This dual mechanism addresses both the downstream protein deficiency and the upstream genetic cause of the disease.
By raising an $87 million Series B, Arthex secured a cash runway projected to last until its final Phase 1/2 data readout in early 2027. This long-term funding allows the company to fully execute its current clinical trial and prepare for Phase 3 without the immediate pressure of further fundraising.