Viking Therapeutics (NASDAQ:VKTX) announced positive top-line results from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD).
The results showed that VK0214 rapidly reduced very long chain fatty acid (VLCFA) levels in plasma by more than 25% in treated animals, compared with vehicle controls. The study successfully achieved its primary objective, which was to demonstrate the ability of VK0214 to lower plasma VLCFA levels after six weeks of treatment.
VLCFA reductions in VK0214-treated animals were observed as early as two weeks following initiation of dosing and generally increased through the course of the study.
In a statement, CEO, Brian Lian, said these results provide an encouraging proof-of-concept signal for VK0214 in the setting of X-ALD, which is a devastating and progressively debilitating disease with a lack of therapeutic options.
“Patients with X-ALD experience elevated VLCFAs due to metabolic defects that may potentially be addressed via VK0214’s novel mechanism of action on the thyroid beta receptor,” he added. “This early dataset helps validate our approach to reducing VLCFAs via selective receptor activation.”
Along with the company’s Phase 2 VK5211 program in hip fracture and Phase 2 VK2809 program in fatty liver disease, VK0214 represents another important pipeline asset with the potential to create significant value, Mr. Lian said.
