Oral NRF-2 activator programme
C4XD has designed and discovered novel potent activators of the NRF-2 pathway, which is important in mediating lung diseases such as Sickle Cell Disease (“SCD”), chronic obstructive pulmonary disease (“COPD”), pulmonary arterial hypertension (“PAH”), and other inflammatory diseases. Multiple C4XD compounds have shown >12 hour NRF-2 activation in the lungs and other target tissues following low dose oral administration in pre-clinical studies. These novel compounds are being optimised for solubility and pharmacokinetic properties for pre-clinical candidate short-list selection.
Pre-clinical data suggests that the anti-oxidant and anti-inflammatory activity provided by NRF-2 activators may ameliorate haemolysis-related complications in SCD such as severe pain episodes, organ damage, heart attacks and stroke and the $41 million COPD market represents an area of substantial unmet medical need.