A Potential Breakthrough Therapy for Chronic Metabolic Disorders, including NASH
PXL770 directly activates adenosine monophosphate-activated protein kinase (AMPK), which plays a key role as a master regulator of cellular energy, which turns on pathways that replenish energy and turns off pathways that consume it. Through its unique mechanism of action that directly activates AMPK, PXL770 acts on a very important biological target, which has the potential to treat numerous chronic metabolic diseases, including diseases that affect the liver, such as non-alcoholic steatohepatitis or NASH. This target has the potential to trigger benefits on the three key pathophysiology processes involved in NASH development, including:
- Liver steatosis
PXL770 is advancing into Phase 2a development for the treatment of NASH.
In a Phase 1a study, PXL770 exhibited good safety, tolerance and pharmacokinetics after single administration up to the highest dose tested.
PXL770 exhibited a favorable safety and pharmacokinetic profile in the Phase 1b part-study that included a multiple ascending dose (MAD) trial and a drug-drug interaction study. PXL770 was well tolerated up to the highest dose tested and there were no serious adverse events or adverse events leading to withdrawal.
PXL770 has the potential to be studied in several metabolic disorders, including liver disease, such as NASH. Having successfully completed the Phase 1 trials with favorable results, we anticipate initiating a Phase 2a clinical proof-of-efficacy study in patients with NAFLD/NASH in early 2019. This study will include 12 weeks of treatment with a primary endpoint of change in liver fat mass based on MRI-PDFF. We are also considering additional proof-of-concept studies in other metabolic indications.
Our Development and Commercialization Strategy
PXL770, as a first-in-class novel mechanism, provides a unique opportunity for patients with chronic metabolic disorders, including NASH.