SAN DIEGO, June 5, 2018 /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 that enrollment has been completed in the company's ongoing Phase 2 clinical trial of VK2809 in patients with primary hypercholesterolemia and non-alcoholic fatty liver disease (NAFLD). VK2809 is a novel, orally available small molecule thyroid receptor agonist that possesses selectivity for liver tissue, as well as the beta receptor subtype, suggesting promise in this patient population.
The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled, parallel-group study designed to evaluate the efficacy, safety and tolerability of VK2809 in patients with elevated LDL cholesterol (LDL-C) and NAFLD. Patients have been randomized to receive once-daily oral doses of VK2809 or placebo for 12 weeks followed by a four-week off-drug phase. The trial's primary endpoint will evaluate the effect of VK2809 treatment on LDL-C after 12 weeks compared to placebo. Secondary and exploratory endpoints include assessments of changes in liver fat content, triglycerides, and other lipid markers. Viking expects to announce results from this trial in the second half of 2018.
"We are pleased to have completed patient enrollment in this important study and remain on track to report top-line results during the second half of this year. We believe VK2809's potent activity at the thyroid beta receptor may provide benefit to patients with fatty liver disease, such as non-alcoholic steatohepatitis (NASH)," said Brian Lian, Ph.D., chief executive officer of Viking. "In vivo studies have shown that treatment with VK2809 leads to significant improvement in markers related to NASH development, severity and progression. In addition, prior clinical data in subjects with mild hypercholesterolemia have demonstrated that treatment with VK2809 results in significant reductions in LDL cholesterol, triglycerides and atherogenic proteins."
In a Phase 1b study in patients with mild hypercholesterolemia, treatment with VK2809 resulted in placebo-adjusted reductions in low-density lipoprotein ranging from 15% to 40%. Patients also experienced significant reductions in triglycerides, as well as the atherogenic proteins lipoprotein-a and apolipoprotein B. Consistent with its liver- and receptor-selective mechanism of action, treatment with VK2809 has also demonstrated rapid reduction of liver fat in animal models of hepatic steatosis. Furthermore, results from an in vivo evaluation of VK2809 in a model of diet-induced NASH demonstrated statistically significant reductions in fibrosis, steatosis and the NAFLD activity score (NAS) in animals treated with VK2809 compared with vehicle-treated controls. Gene expression analyses demonstrated significant improvements in genes related to fibrosis, steatosis, insulin sensitivity and metabolic control.
These preclinical and clinical study results suggest a differentiated therapeutic profile for VK2809 relative to existing development programs targeting hypercholesterolemia and fatty liver disease, such as NASH. The potential markets for these indications are significant. In the U.S., approximately 33% of adults, or 71 million people, have elevated LDL cholesterol. Additionally, NASH is becoming recognized as a leading cause of cirrhosis and liver failure and affects an estimated 6 to 15 million Americans.
VK2809 is an orally available, tissue and receptor-subtype selective agonist of the thyroid beta receptor (TRβ) that possesses selectivity for liver tissue, as well as the beta receptor subtype, suggesting promising therapeutic potential in a range of lipid disorders. The compound is in Phase 2 development for the treatment of patients with hypercholesterolemia and non-alcoholic fatty liver disease (NAFLD) and Phase 1 development for the treatment of patients with GSD Ia. VK2809 belongs to a family of novel prodrugs which are cleaved in vivo to release potent thyromimetics. Selective activation of the TRß receptor in liver tissue is believed to favorably affect cholesterol and lipoprotein levels via multiple mechanisms, including increasing the expression of low-density lipoprotein (LDL) receptors and increasing mitochondrial fatty acid oxidation.
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 (SARM) in Phase 2 development for the treatment and prevention of lean body mass loss in patients who have undergone hip fracture surgery; and VK2809, a small molecule thyroid beta agonist in Phase 2 development for the treatment of hypercholesterolemia and non-alcoholic fatty liver disease (NAFLD). Viking is also developing novel and selective agonists of the thyroid beta receptor for glycogen storage disease type 1a (GSD 1a) and X-linked adrenoleukodystrophy (X-ALD), as well as programs targeting diabetes, metabolic diseases and anemia.
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This press release contains forward-looking statements regarding Viking Therapeutics, Inc., under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, 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 its prospects. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially and adversely 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 preclinical results may not be replicated; risks regarding regulatory requirements; and other risks that are described in Viking's most recent periodic reports filed with the Securities and Exchange Commission, including Viking's Annual Report on Form 10-K for the year ended December 31, 2017 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2018, including the risk factors set forth in those filings. These forward-looking statements speak only as of the date hereof. Viking disclaims any obligation to update these forward-looking statements except as required by law.
SOURCE Viking Therapeutics, Inc.