CTX03 is a proprietary nanoformulation of encapsulated Cyclosporine A (CsA) being developed by Cardiol for the treatment of heart failure.
CsA is an immunosuppressant more commonly used for the prevention of organ transplant rejection, including for heart transplantation. The current evidence suggests that the mechanism of CsA relates to its activity at the mitochondrial membrane, specifically at pores in the membrane (the mitochondrial permeability transition pores, MPTP), where the action of CsA reduces the rupture of the mitochondrial membrane. Rupture of the mitochondrial membrane leads to cell death, and, in the heart, death of heart muscle cells (cardiomyocytes). Cardiomyocyte cell death is an important component of the pathology of heart failure causing largely irreparable damage and is therefore a key clinical target in patients with heart failure. Cardiol aims to use encapsulated CsA to target the drug directly to the failing heart, an approach supported by a Position Paper from the European Society of Cardiology which, in 2014, stated that “Targeting therapies to the heart… will almost certainly be required for these and other therapies.”