Viking Therapeutics Presents Positive Phase 1b Clinical Data on VK2809 in Hypercholesterolemic Subjects

SAN DIEGO, April 11, 2016 /PRNewswire/ -- Viking Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today highlighted positive data from a Phase 1b clinical trial of VK2809 in subjects with mild hypercholesterolemia, presented at the 65^th Annual Scientific Session & Expo of the American College of Cardiology (ACC).  The results demonstrated substantial and clinically meaningful reductions in subjects' low-density lipoprotein cholesterol (LDL-C), triglycerides, and atherogenic proteins lipoprotein-a and apolipoprotein B following 14 days of treatment.  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 promising therapeutic potential in this patient population. 

The randomized, double-blind, placebo-controlled Phase 1b study was designed to evaluate the safety, tolerability and pharmacokinetics of VK2809, at a range of doses, in 56 subjects with elevated serum cholesterol (n = 6 per drug-treated cohort).  Following 14 days of VK2809 treatment, subjects demonstrated clinically and statistically significant placebo-adjusted reductions in LDL-C ranging from 15.2% at the 5.0 mg dose (p=0.026) to 41.2% at the 20 mg dose (p<0.0001).  In addition, subjects experienced placebo-adjusted reductions in triglycerides ranging from 34.8% at 5.0 mg dose (p=0.052) to 78.6% at the 40 mg dose (p=0.0001).  Significant reductions in lipoprotein-a and apolipoprotein B were also observed, with declines of 30% or more reported at all doses above 2.5 mg. 

Treatment with VK2809 was also shown to be safe and well-tolerated at all doses studied. No serious adverse events were reported and no treatment- or dose-related trends were observed for abnormal vital signs, electrocardiograms, cardiac rhythm or physical examination assessments.  Consistent with liver-targeted thyroid receptor activation, mild, asymptomatic elevations in liver enzymes and decreased thyroid hormone levels were observed at higher doses.  Metabolically, VK2809 was not eliminated intact through the kidneys, and less than 3% of the administered dose was eliminated through the kidneys as the drug's active metabolite, VK2809A. 

"We believe these results provide compelling preliminary evidence of VK2809's efficacy in this important indication.  The observed statistically significant effects in a trial of this size and duration are quite promising and demonstrate the potential therapeutic benefits of thyroid receptor modulation for lipid dysregulation," said Brian Lian, Ph.D., chief executive officer of Viking.  "These data, combined with VK2809's novel liver-targeted delivery and mechanism of action, as well as prior in vivo data demonstrating robust reductions in hepatic fat content, provide a strong rationale for applications in settings such as hypercholesterolemia and fatty liver disease.  We believe that receipt of a Best Poster award from a conference of this stature reinforces the potential importance of the results.  We are focused on further establishing the safety and efficacy profile of VK2809 and continue to prepare for our upcoming Phase 2 clinical trial, which we expect to initiate mid-year."

Viking is currently planning to initiate a Phase 2 clinical trial of VK2809 in patients with hypercholesterolemia and fatty liver disease.  The Phase 2 clinical trial will be a randomized, double-blind, placebo-controlled, parallel group study designed to evaluate the efficacy, safety and tolerability of VK2809 in patients with elevated LDL-C and fatty liver disease. 

About VK2809
VK2809 is an orally available, tissue and receptor-subtype selective agonist of the thyroid beta receptor entering Phase 2 development for the treatment of patients with hypercholesterolemia and fatty liver disease.  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.  These characteristics suggest a highly differentiated therapeutic profile relative to existing oral options for patients with hypercholesterolemia and fatty liver disease, such as nonalcoholic steatohepatitis (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 rapidly becoming a leading cause of cirrhosis and liver failure and affects an estimated 6 to 15 million Americans. 

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.

Forward Looking Statements
This press release contains forward-looking statements regarding Viking Therapeutics, including statements about Viking's expectations regarding the company's proposed Phase 2 clinical trial for VK2809, as well as VK2809's potential to produce therapeutic benefits. 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.