ADX-038

ADX-038 is an investigational small interfering RNA (siRNA) duplex oligonucleotide developed by ADARx Pharmaceuticals for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and severe disease characterized by uncontrolled activation of the complement system leading to destruction of red blood cells and severe anemia. The drug specifically targets CFB mRNA, resulting in selective and durable inhibition of Factor B (Complement factor B), a key component in the alternative complement pathway. By inhibiting Factor B, ADX-038 aims to prevent both intravascular and extravascular hemolysis associated with PNH. The drug is currently being evaluated in Phase 1/2 clinical trials for safety, tolerability, pharmacokinetics, and pharmacodynamics in both healthy volunteers and patients with PNH[1][2][3][4][5][6].