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Reductions in Very Long-Chain Fatty Acids and Plasma Lipid Levels Observed After 28 Days of Once Daily Dosing
VK0214 Shown to be Safe and Well-Tolerated in 28-Day Study
SAN DIEGO, Oct. 9, 2024 /PRNewswire/ -- Viking Therapeutics, Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced positive data from the company's 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). Results from this study showed VK0214 to be safe and well-tolerated following once-daily dosing over the 28-day study period. In addition, significant reductions were observed in plasma levels of very long-chain fatty acids (VLCFAs) and other lipids, as compared to placebo.
Highlights from the study results include:
Reductions in VLCFAs
In addition to safety and tolerability, the study included an exploratory assessment of changes in plasma levels of VLCFAs after 28 days of dosing. VLCFAs are considered biomarkers of disease in patients with X-ALD. Treatment with VK0214 resulted in significant reductions in mean VLCFA levels at both doses evaluated, 20 mg/day and 40 mg/day, compared to placebo. Importantly, cohorts receiving VK0214 demonstrated reductions in mean plasma levels of the 26 carbon lysophosphatidyl choline (C26:0-LPC) derivative, a key diagnostic marker.
Percent Change from Baseline in VLCFAs Following 28 Days of Treatment of VK0214
Placebo1 (n=6) | 20 mg (n=8) | 40 mg (n=9) | |
C22:0 | 4.0 % | -16.6 % | -19.5 % |
p-value vs. placebo2 | - | 0.0097 | 0.0042 |
C24:0 | 5.2 % | -12.8 % | -18.0 % |
p-value vs. placebo | - | 0.0162 | 0.0029 |
C26:0 | 20.8 % | -17.4 % | -17.6 % |
p-value vs. placebo | - | 0.0145 | 0.0117 |
C26:0-LPC3 | 23.1 % | -8.4 % | -14.8 % |
p-value vs. placebo | - | 0.0427 | 0.0105 |
Notes: 1) Least squares mean change from baseline to Day 28. 2) Two-sided t-test using mixed model for repeated measures. 3) C26:0-LPC data for 20 mg, 40 mg cohorts include results from n=7, n=8 subjects, respectively. |
Reductions in Plasma Lipids
In addition to VLCFA changes, subjects who received VK0214 demonstrated reductions in other plasma lipids. Mean reductions relative to baseline and placebo were observed for low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (ApoB), and lipoprotein (a) [Lp(a)] following 28 days of treatment.
Percent Change in Lipid Markers Following 28 Days of Treatment of VK0214
Placebo1 (n=6) | 20 mg (n=9) | 40 mg (n=9) | |
LDL-C | 4.7 % | -19.4 % | -20.2 % |
p-value vs. placebo2 | - |
