Viking Therapeutics, a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, has announced an agreement with PoC Capital, LLC to fund Viking's initial clinical development of VK2809 in glycogen storage disease type Ia (GSD Ia). Viking plans to file an Investigational New Drug (IND) application for VK2809 for the treatment of GSD Ia and initiate a human proof-of-concept study in the second half of 2017.
Under the terms of the agreement, PoC Capital will be responsible for paying up to $1.8 million in expenses associated with VK2809 clinical studies, including a planned proof-of-concept trial in patients with GSD Ia. In exchange, up to $1.8 million in Viking shares will be issued to PoC Capital.
Viking's agreement with PoC Capital follows the company's recent announcement of positive top-line results from a proof-of-concept study of VK2809 in an in vivo model of GSD Ia. Data from the study, conducted under a sponsored research agreement between Duke University and Viking, 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 per cent vs. controls. In addition, average liver weight as a percent of total body weight declined by approximately 20 per cent in treated vs. control animals. The ongoing study is continuing to evaluate the impact of VK2809 on these and other disease markers. Detailed data will be submitted for presentation at an appropriate scientific conference.
GSD Ia is a rare, orphan genetic disease 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 pharmacologic therapy for GSD Ia.
"We are pleased to enter into this agreement with PoC Capital as it will allow us to conduct the initial clinical work evaluating VK2809 in GSD Ia in a capital efficient manner. We view GSD Ia as an attractive development opportunity for VK2809, where we can leverage prior human data showing the drug candidate's robust effect on plasma triglycerides, as well as our recent results demonstrating promising efficacy in an animal model of the disease," said Brian Lian, Ph.D., chief executive officer of Viking. "GSD Ia is an indication with a high unmet medical need where we believe VK2809 could yield important benefits to patients who have otherwise limited therapeutic options. As such, we are interested in evaluating the drug candidate's potential effects in a clinical proof-of-concept study."
"In collaboration with researchers from Duke University, the team at Viking has generated encouraging data in a model of GSD Ia, a rare genetic condition. We are very pleased to support Viking's plan to run a proof-of-concept study with VK2809, a selective thyroid hormone receptor beta agonist," said Daron Evans, managing director of PoC Capital. "Viking's strong research team has the potential to open the door to this and other potential orphan indications with their deep pipeline of therapeutic products."
VK2809 is a novel, orally available small molecule thyroid receptor beta (TRß) 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.