News & Events


<< Back
Viking Therapeutics Announces Initial Results from In Vivo Proof-of-Concept Study of VK2809 in Glycogen Storage Disease Ia (GSD Ia)
Treatment with Novel Thyroid Receptor Beta Agonist Led to Significant Reductions in Liver Triglyceride Levels and Liver Weight Compared to Controls

SAN DIEGO, Feb. 9, 2017 /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 initial results from a proof-of-concept study of VK2809 in an in vivo model of glycogen storage disease Ia (GSD Ia).  GSD Ia is a rare, orphan genetic disease that results in excess accumulation of glycogen and lipids in liver tissue.  Data to date showed that treatment with VK2809 produced rapid and substantial reductions in liver triglyceride content, liver weight and liver weight as a percentage of body weight compared with vehicle-treated controls.  Complete results from the ongoing study, which is being conducted under a sponsored research agreement between Duke University and Viking, will be presented at an upcoming scientific meeting.

Data demonstrated that treatment with VK2809 led to statistically significant reductions in key metabolic markers of GSD Ia.  Mean liver triglyceride content was reduced by more than 60% in VK2809-treated animals relative to vehicle-treated control animals, while average liver weight was reduced by more than 30% vs. controls.  Importantly, average liver weight as a percent of total body weight also declined by approximately 20% in treated vs. control animals.  The study will continue to evaluate the impact of VK2809 on these and other disease markers.

GSD Ia is characterized by an inability to metabolize glucose precursors, resulting in hypoglycemia and increased lipogenesis.  The disease is caused by mutations in the gene for glucose-6-phosphatase (G6PC), a critical enzyme involved in the production of glucose from either glycogen or gluconeogenesis.  Impaired G6PC function leads to dramatically elevated liver triglyceride levels in human patients and in animal models of the disease.  In patients, this may contribute to serious long-term complications, such as severe hepatomegaly, hepatic adenomas, and hepatocellular carcinoma.  Manifestations of the disease begin to appear shortly after birth and continue through adolescence into adulthood.  There is currently no approved therapy for GSD Ia. VK2809's potential to rapidly reduce hepatic triglyceride levels, as demonstrated in this initial evaluation in a GSD Ia model, provide support for the continued investigation of the compound in this indication.

"These initial proof-of-concept data showing VK2809's activity in this model of GSD Ia are very encouraging. Due to its unique liver-targeted activity, we believe that VK2809 has a therapeutic profile that may align well with the treatment goal of reducing triglycerides, steatosis, and mitigating the metabolic complications of GSD Ia," said Brian Lian, Ph.D., chief executive officer of Viking.  "GSD Ia is a rare metabolic disease for which there are no approved therapeutics.  These patients suffer from a range of serious consequences, including an increased risk of liver bleeding, transplant, or cancer.  Our work in this area represents a strategic and complementary addition to our ongoing VK2809 development program, which currently includes a Phase 2 clinical trial in patients with hypercholesterolemia and non-alcoholic fatty liver disease."

The ongoing proof-of-concept study is designed to evaluate the effects of VK2809 in Duke University's G6PC knockout mouse model, which replicates many of the same biochemical and physiological characteristics present in GSD Ia patients.  Study mice are receiving VK2809 or vehicle once-daily and subsequently evaluated for changes in various measures of disease, including liver size, weight, and triglyceride content.  Additional effects on glycogen and genetic markers are also being explored.  Importantly, the preliminary results on hepatic markers are consistent with prior studies demonstrating VK2809's ability to potently reduce liver fat in other models of hepatic steatosis.

"The preliminary findings suggesting the potential triglyceride-lowering activity of VK2809 in GSD Ia are encouraging, particularly when demonstrated in the most studied GSD Ia animal model," said Dwight Koeberl, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine.  "We look forward to continuing the evaluation of VK2809 in GSD Ia."

VK2809 is a novel, orally available small molecule thyroid receptor beta (TRβ) agonist that possesses selectivity for liver tissue, as well as the beta receptor subtype, suggesting promising therapeutic potential in a range of lipid disorders.  Viking is currently evaluating VK2809 in a randomized, double-blind, placebo-controlled, parallel group Phase 2 study designed to assess the drug candidate's efficacy, safety and tolerability in approximately 80 patients with elevated LDL-C and non-alcoholic fatty liver disease.  Previously reported clinical data have demonstrated that treatment with VK2809 leads to significant reductions in plasma triglycerides, LDL cholesterol (LDL-C), and atherogenic protein levels in subjects with mild hypercholesterolemia.

About GSD Ia
Glycogen storage disease Ia (GSD Ia) is a rare, orphan genetic disease caused by a deficiency of glucose-6-phosphatase (G6P), an enzyme responsible for the liver's production of free glucose from glycogen and gluconeogenesis.  The disease, for which there is no approved treatment, results in an excess accumulation of glycogen and lipids in the liver, potentially leading to hepatosteatosis, liver failure, development of hepatic adenomas, and hepatocellular carcinoma.  Increased triglyceride production and elevated hepatic triglyceride levels are characteristic of GSD Ia and associated with many manifestations of the disease.  GSD Ia is estimated to occur in approximately 1 in every 50,000 – 100,000 births in the United States.  As manifestations of the disease begin to present themselves at birth, a sizeable portion of GSD Ia patients are children.

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 in 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.

Follow Viking on Twitter @Viking_VKTX.

Forward-Looking Statements
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 VK2809 and VK2809's prospects. 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, including those for VK2809; risks that prior clinical and pre-clinical results may not be replicated; 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.

For further information: Vida Strategic Partners, Stephanie Diaz (Investors), 415-675-7401,, Tim Brons (Media), 415-675-7402,