PXL770, a first in class direct adenosine monophosphate activated protein kinase (AMPK) activator is
the only compound in this class that has been tested in human to treat metabolic diseases. AMPK is a
well known cellular energy sensor, and its activation has been shown to improve insulin sensitivity, to
decrease glycemia, de novo lip ogenesis, liver steatosis, inflammation, fibrosis in numerous preclinical
models and publications . PXL770 has confirmed all these benefits in a large number of preclinical
models. Interestingly, most of the preclinical effects have translated into clinical benefits confirming
the potential of direct AMPK activation.
An extensive scientific literature also support s A MPK activation a s a target of choice for the treatment
of two rare metabolic disease s : X linked adrenoleukodystrophy (ALD) and autosomal dominant
polycystic kidney disease ( Preclinical evidences have demonstrat e d the benefit of PXL770 for
the treatment of these diseases. T ranslation from preclinical to patients, as observed for metabolic
parameters, remains to be demonstrated. C linical proof of concept studies are under active
preparation, to be initiated as soon as possible .