The European Commission has granted orphan drug designation (ODD) for Poxel’s PXL770 and PXL065 for the treatment of adrenoleukodystrophy (ALD).
The decision follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).
These molecules have separate and distinct mechanisms of action. PXL770 is a novel, first-in-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 molecules.
Both compounds are preparing to enter into Phase 2a clinical Proof-of-Concept (POC) biomarker studies in ALD patients with adrenomyeloneuropathy (AMN) as soon as possible, subject to financing.
“Orphan Drug Designation in adrenoleukodystrophy for both PXL770 and PXL065 further strengthens the value of these clinical assets where we are preparing to initiate Phase 2 proof-of-concept studies, pending additional financing,” noted Thomas Kuhn, CEO of Poxel. “We are in active discussions to restructure our current debt obligations and secure the funding to execute our strategy in rare metabolic diseases.”