Clinical Trials in ALD

Clinical Development in X-linked adrenoleukodystrophy (ALD)

In ALD, two identical Phase 2a clinical POC biomarker studies, one each with PXL065 and PXL770, are planned to initiate mid-2022, with data expected early 2023. The initial focus will be on patients with adrenomyeloneuropathy (AMN), the largest subtype of ALD. The studies will enroll adult male AMN patients and assess the effect of PXL065 and PXL770 over 12 weeks of treatment on pharmacokinetics, safety, and efficacy using relevant biomarkers, including the impact on elevated very long-chain fatty acids (VLCFA), the hallmark plasma marker of disease.

Early 2022, the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PXL065 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD). The Fast Track Designation is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.

Community Engagement

Poxel has established relationships with Key Opinion Leaders and collaborations with the following important patient advocacy associations:


For any questions related to our clinical trials, please contact us through our contact form by selecting “Clinical Trials”.