Poxel’s PXL065 granted FDA fast track designation for X-linked adrenoleukodystrophy

Phase 2a clinical proof-of-concept biomarker study for PXL065 in ALD now anticipated to start midyear

POXEL SA, a clinical stage biopharmaceutical company, developing innovative treatments for serious chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to PXL065 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD).

PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone that is preparing to enter into a Phase 2a clinical Proof-of-Concept (POC) biomarker study midyear.

Thomas Kuhn, CEO, Poxel, commented: "By awarding Fast Track Designation to PXL065, the FDA recognizes the drug's potential to address a significant unmet medical need for patients with ALD, where no approved therapies currently exist. This is a very powerful acknowledgement and good news for patients waiting to be treated. Fast Track Designation confers multiple benefits that are of great value and have the potential to substantially accelerate the approval timeline for PXL065 in ALD. We look forward to working closely with the FDA as we prepare to embark on our Phase 2a clinical study for PXL065 now planned to begin midyear, followed by results in early 2023.”

The phase 2a clinical POC biomarker study for PXL065 in X-linked ALD is anticipated to begin midyear, followed by results in early 2023.