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PXL770 is a novel, firstin-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione.
PXL065 is a novel, proprietary deuteriumstabilized R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione molecules.
ER004 is a pioneering in-utero therapy designed to replace the function of endogenous Ectodysplasin A1 (EDA1), a protein key to the normal development of ectodermal structures in the foetus.
PXL065 is the deuterated-stabilized R stereoisomer (single R-isomer) of pioglitazone, its parent molecule. It is currently being evaluated in a Phase 2 study (DESTINY-1) for NASH, with results expected in Q3 2022.
PXL065 (deuterium-stabilized R-pioglitazone) was observed to ameliorate key features of ALD in preclinical models, including both patient-derived cells and a classical rodent disease model.
The results show that odiparcil was associated with decreased glycosaminoglycan (GAG) accumulation as well as increased GAG excretion, and highlight its distribution in MPS VI disease-relevant tissues and organs.
The Phase IIa clinical study results in MPS VI adult patients showed the capacity of odiparcil to improve key clinical parameters such as corneal clouding and cardiac and respiratory functions.