VK0214

VK0214 is a novel, orally available small molecule that acts as a selective agonist of the thyroid hormone receptor beta (TRβ). Developed by Viking Therapeutics, it is being investigated primarily for the treatment of X-linked adrenoleukodystrophy (X-ALD), particularly the adrenomyeloneuropathy (AMN) form. The drug has demonstrated significant reductions in plasma levels of very long-chain fatty acids (VLCFAs), which are biomarkers and contributors to X-ALD pathology. VK0214 has shown safety and tolerability in early clinical trials and has been granted orphan drug designation by the FDA for X‑ALD[1][5][6][7]. Its mechanism involves upregulation of ABCD2 gene expression via TRβ activation, potentially compensating for defective VLCFA metabolism seen in X‑ALD[8].

Other names

((4-(3-benzyl-4-hydroxybenzyl)-3,5-dimethylphenoxy)methyl)phosphonic acid(((4-(3-benzyl-4-hydroxybenzyl)-3,5-dimethylphenyl)oxy)methyl)phosphonic acidPhosphonic acid, p-((4-((4-hydroxy-3-(phenylmethyl)phenyl)methyl)-3,5-dimethylphenoxy)methyl)