ARTICLE | Translation in Brief

Viking’s heart bypass

Viking navigates thyroid receptor β agonist tissue specificity for X-ALD

October 20, 2016 7:00 AM UTC

Viking Therapeutics Inc. (NASDAQ:VKTX) has presented data showing its preclinical thyroid hormone receptor β agonist, VK0214, cut down pathogenic accumulation of very long chain fatty acids (VLCFAs) in a mouse model of X-linked adrenoleukodystrophy (X-ALD). The company thinks VK0214’s cardiac-sidestepping chemistry is a good match for the disease and a safeguard against the heart toxicity that has made the receptor a difficult target.

X-ALD is a genetic disease caused by mutations in ATP binding cassette transporter D member 1 (ABCD1) that lead to poor peroxisome clearance of VLCFAs, resulting in neural and endocrine defects. By stimulating thyroid hormone receptor β signaling, Viking’s compound induces up-regulation of ABCD2, which compensates for ABCD1 deficiency and promotes peroxisomal clearance of VLCFAs...