Viking Therapeutics has announced positive results from its Phase Ib clinical trial of VK0214, a thyroid hormone receptor beta agonist, in patients with X-linked adrenoleukodystrophy (X-ALD).
The multicentre, double-blind, randomised, placebo-controlled trial aimed to assess the safety, tolerability and pharmacokinetics of VK0214 when administered once daily over 28 days.
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It enrolled adult male patients with the adrenomyeloneuropathy (AMN) form of X-ALD across three cohorts.
The safety and tolerability of VK0214 were the primary objectives of the trial.
Secondary objectives included assessing the pharmacokinetics of the asset after 28 days of dosing.
An exploratory objective assessed VK0214’s effects on plasma levels of very long-chain fatty acids (VLCFAs), the biomarkers of disease in X-ALD patients.
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By GlobalDataTrial results indicated that VK0214 was safe and well-tolerated following the dosing period.
Significant reductions in plasma levels of VLCFAs were observed, as well as other lipids, when compared to the placebo.
Both the 20 mg/day and 40 mg/day doses of VK0214 led to significant decreases in mean VLCFA levels, including the 26 carbon lysophosphatidyl choline (C26:0-LPC) derivative – a key diagnostic marker for X-ALD.
Subjects receiving VK0214 demonstrated reductions in other plasma lipids, including low-density lipoprotein cholesterol, apolipoprotein B and lipoprotein.
These results were relative to both baseline and placebo following 28 days of treatment.
Treatment-emergent adverse events were found to be mostly mild to moderate with one severe adverse event – a wrist fracture – reported in the placebo cohort.
Viking Therapeutics CEO Brian Lian stated: “Patients receiving VK0214 demonstrated progressive improvement in plasma levels of very long chain fatty acids in the relatively brief treatment period evaluated in this study.
“In addition, VK0214 continued to show benefits on broader plasma lipids, such as LDL-C, important for overall cardiometabolic health. Consistent with prior clinical results in healthy volunteers, VK0214 was shown to be safe and well-tolerated in this 28-day study.”
In March 2024, the company reported positive data from its Phase I trial of VK2735 for the treatment of metabolic disorders such as obesity.
