Viking Therapeutics announced positive data from its Phase 1b clinical trial of VK0214, a novel small molecule agonist of the thyroid hormone receptor beta (TRβ), in patients with X-linked adrenoleukodystrophy (X-ALD) on October 9, 2024. The study demonstrated that VK0214 was safe and well-tolerated following once-daily dosing over a 28-day period. This outcome is consistent with prior clinical results observed in healthy volunteers.
The trial showed significant reductions in plasma levels of very long-chain fatty acids (VLCFAs), which are biomarkers of disease in X-ALD patients. Specifically, mean plasma levels of C22:0, C24:0, C26:0, and C26:0-LPC decreased significantly at both 20 mg/day and 40 mg/day doses compared to placebo. These reductions indicate a positive biological effect of the drug.
Furthermore, subjects receiving VK0214 also demonstrated reductions in other plasma lipids, including low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (ApoB), and lipoprotein (a) [Lp(a)]. These broader lipid benefits are important for overall cardiometabolic health, adding to the therapeutic potential of VK0214 beyond its primary target in X-ALD.
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