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SAN DIEGO, July 26, 2016 /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 top-line results from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD). The results of this study showed that VK0214 rapidly reduced plasma very long chain fatty acid (VLCFA) levels by more than 25% in treated animals compared with vehicle controls (p < 0.01). 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. Detailed results will be presented at an upcoming scientific meeting.
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. Treatment with VK0214 led to robust effects on multiple VLCFAs and resulted in significantly lower levels of saturated C26, C24, C22, and C20 fatty acids relative to controls. As the accumulation of VLCFAs is believed to contribute to the underlying pathology of X-ALD, today's data provide support for the continued evaluation of VK0214 in this indication.
"We believe 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," said Brian Lian, Ph.D., chief executive officer of Viking. "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. This early dataset helps validate our approach to reducing VLCFAs via selective receptor activation. Along with our 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."
VK0214 is a novel, orally available thyroid receptor beta (TRβ) agonist that selectively regulates the expression of genes believed to be relevant to the manifestation of X-ALD. The proof-of-concept study, conducted at the Kennedy Krieger Institute under a sponsored research agreement with Viking, was designed to evaluate changes in VLCFA levels in the ABCD1 knockout mouse model. This model is intended to mirror the loss of ABCD1 transporter activity that is considered the hallmark of X-ALD. Mice received VK0214 or vehicle once-daily for six weeks. VLCFA levels were determined by measuring unsaturated lysophosphatidylcholine fatty acid esters, which are biomarkers for VLCFAs in X-ALD.
X-ALD is a rare and often fatal metabolic disorder characterized by a breakdown in the protective barriers surrounding brain and nerve cells; a process known as demyelination. The disease, for which there is no approved treatment, is caused by mutations in a peroxisomal transporter of VLCFAs, known as ABCD1. As a result, transporter function is impaired and patients are unable to efficiently metabolize VLCFA. The resulting accumulation can trigger a rapid, inflammatory demyelination, which leads to cognitive impairment, motor skill deterioration, and even death. X-ALD is estimated to occur in approximately 1 in 17,000 births.
The thyroid beta receptor is known to regulate expression of an alternative VLCFA transporter, known as ABCD2. Various preclinical models have demonstrated that increased expression of ABCD2 can lead to normalization of VLCFA metabolism.
About Viking Therapeutics, Inc.
Viking Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders. The company's research and development activities leverage its expertise in metabolism to develop innovative therapeutics designed to improve patients' lives. Viking has exclusive worldwide rights to a portfolio of five therapeutic programs in clinical trials or preclinical studies, which are based on small molecules licensed from Ligand Pharmaceuticals Incorporated. The company's clinical programs include VK5211, an orally available, non-steroidal selective androgen receptor modulator, or SARM, in Phase 2 development for the treatment and prevention of lean body mass loss in patients who have undergone hip fracture surgery, VK2809, a small molecule thyroid beta agonist entering Phase 2 development for hypercholesterolemia and fatty liver disease, and VK0612, a first-in-class, orally available drug candidate in Phase 2 development for type 2 diabetes. Viking is also developing novel and selective agonists of the thyroid beta receptor for adrenoleukodystrophy, as well as two earlier-stage programs targeting metabolic diseases and anemia.
This press release contains forward-looking statements regarding Viking Therapeutics, including statements about Viking's expectations regarding its development activities, timelines and milestones, as well as the company's goals and plans regarding VK0214. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: risks associated with the success, cost and timing of Viking's product candidate development activities and clinical trials; and risks regarding regulatory requirements, among others. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements.
SOURCE Viking Therapeutics, Inc