On Wednesday, Viking Therapeutics Inc. (NASDAQ:VKTX) released data from the Phase 1b trial of VK0214, a novel small molecule agonist of the thyroid hormone receptor beta (TRβ), in patients with X-linked adrenoleukodystrophy (X-ALD).
X-linked adrenoleukodystrophy is a genetic disorder that mainly affects the nervous system and the adrenal glands, which are located on top of each kidney.
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Results showed VK0214 to be safe and well-tolerated following once-daily dosing over the 28-day study period.
In addition, plasma levels of very long-chain fatty acids (VLCFAs) and other lipids were significantly reduced compared to placebo.
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.
Cohorts receiving VK0214 demonstrated reductions in mean plasma levels of the 26 carbon lysophosphatidyl choline (C26:0-LPC) derivative, a key diagnostic marker.
A 14.8% reduction was observed in the 40 mg cohort (p=0.0105) and a reduction of 8.4% in the 20 mg cohort (p=0.0427). 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.
Treatment-emergent adverse events were reported as mild to moderate. Gastrointestinal adverse events were slightly higher among placebo subjects (33%) compared with VK0214-treated subjects (11%).
William Blair highlights that the positive progress of VK0214 provides investors with additional opportunities beyond Viking's existing programs, including the subcutaneous and oral formulations of VK2735 for obesity, VK2809 for metabolic-dysfunction-associated steatohepatitis (MASH), and its preclinical amylin program, which is expected to enter clinical trials next year.
Since the analyst believes most investors have yet to factor in the potential revenue from VK0214, its continued successful development could offer an upside to current projections. Moreover, the focus on treating an orphan disease broadens Viking's potential revenue streams by expanding from the larger obesity and MASH markets into the rare disease space.
Price Action: VKTX stock is down 2.67% at $64.90 at the last check on Wednesday.
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