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 as soon as possible, subject to financing. 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.

In early 2022, the U.S. Food and Drug Administration (FDA) granted Fast Track and Orphan Drug Designation to PXL065 and PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD). Orphan Drug Designation (ODD) is granted by the FDA to novel therapeutics for diseases or conditions that affect fewer than 200,000 individuals in the U.S. ODD gives a company a potential seven-year window of exclusive marketing rights following FDA approval, along with a reduction in certain application fees, and tax credits for expenses related to qualified clinical trials conducted after orphan designation is received. Fast Track Designation (FTD) is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy. The FDA notes that 'the purpose of the Fast Track program is to get important new drugs to the patient earlier”. FTD must be requested by the sponsor company and must be accompanied by a detailed review of both preclinical and clinical data. The key benefits of FTD comprise enhanced access to the FDA, with regular and more frequent opportunities for consultation and discussion. In addition, drugs with FTD may be eligible for Accelerated Approval, in which a new medicine is approved prior to the availability of definitive data, and Priority Review, in which the standard 10-month review process is reduced to six months. Drugs with FTD may also enter a 'rolling review' of their NDA submission, in which sections are submitted and reviewed as they become available, substantially expediting the approval process.

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”.