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Viking Therapeutics Announces Positive Top-Line Results from Proof-of-Concept Study of VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy (X-ALD)

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Clinical Data The results of this study showed that VK0214 rapidly reduced plasma very long chain fatty acid (VLCFA) levels by more than 25% in treated animals compared with vehicle controls (p < 0.01). The study successfully achieved its primary objective, which was to demonstrate the ability of VK0214 to lower plasma VLCFA levels after six weeks of treatment. Detailed results will be presented at an upcoming scientific meeting.VLCFA reductions in VK0214-treated animals were observed as early as two weeks following initiation of dosing and generally increased through the course of the study. Treatment with VK0214 led to robust effects on multiple VLCFAs and resulted in significantly lower levels of saturated C26, C24, C22, and C20 fatty acids relative to controls. As the accumulation of VLCFAs is believed to contribute to the underlying pathology of X-ALD, today's data provide support for the continued evaluation of VK0214 in this indication.  
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Jul 26, 2016
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Related Keywords Vk0214, In Vivo Model, X-linked Adrenoleukodystrophy, X-ald, Lower Plasma Vlcfa Levels, Thyroid Beta Receptor, Abcd1 Transporter, Vlcfa Metabolism, Adrenoleukodystrophy