Apaxen’s lead compound, MFC-1040, is a novel, first-in-class, orally bioavailable, highly potent and selective small molecule inhibitor of NLRP3 Inflammasome activation. It interferes with the binding of intracellular MIF (Macrophage migration Inhibitory Factor) to NLRP3 units, which is required for interaction with cytoskeleton protein Vimentin and for the construction of active NLRP3 inflammasomes.

As a first step towards treatment of the many inflammatory and auto-immune diseases that depend on NLRP3 inflammasome activation, MFC-1040 is being developed for the acute treatment of Gout flares and for chronic treatment of pulmonary arterial hypertension (PAH).

MFC-1040 has shown robust in vivo efficacy in multiple rodent models of PAH (J. Med. Chem. 2018, 61, 2725-2736), namely monocrotaline (MCT), Sugen/chronic hypoxia (SuHx) and Bleomycin (BLM) (Int. J. Mol. Sci. 2018, 19, 4105) both as monotherapy and/or as a combination with other PAH commercialized drugs.

The mechanism of action of MFC-1040 combines potent anti-inflammatory and anti-fibrotic effects. It has the potential to revolutionise the standard of care of PAH, by halting or reverting the progression of the disease while available therapies only address the symptoms of this disease.

Pre-clinical development of MFC-1040 is currently in progress. Apaxen expects to start first-in-human (FIH) clinical trials for MFC-1040 by early 2023.