Date: 2017-09-07
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 13th International Congress of Inborn Errors of Metabolism (ICIEM)
Company: Viking Therapeutics (USA - CA)
Product: VK2809
Action
mechanism:
Disease: glycogen storage disease type Ia (GSD Ia)
Therapeutic
area: Rare diseases - Genetic diseases - Liver diseases
Country:
Trial
details:
Latest
news:
- • On September 7, 2017, Viking Therapeutics announced the presentation of positive final results from a proof-of-concept study of VK2809 in an in vivo model of glycogen storage disease type Ia (GSD Ia). Final results from the study were summarized in a poster titled, "Evaluation of the Thyroid Hormone Agonist VK2809 in an In Vivo Model of Glycogen Storage Disease Type Ia," presented at the 13th International Congress of Inborn Errors of Metabolism (ICIEM), in Rio De Janeiro, Brazil.
- In this study, treatment with VK2809 led to statistically significant reductions in key metabolic markers of glycogen storage disease Ia after just four days, highlighting the molecule's potent, rapid-acting effect in liver tissue. Researchers utilized the glucose-6-phosphatase (G6PC) knockout mouse model, which is intended to replicate the impairment of this enzyme's activity in patients with glycogen storage disease Ia. Highlights of the data presented include:
Mean total liver triglycerides were reduced by 69.0% in VK2809-treated animals relative to vehicle-treated control animals. Total liver triglyceride content in VK2809-treated animals was reduced to within the range of the study's wild type control mice.
- Mean liver weights were reduced by 35.7% in VK2809-treated animals as compared to controls. Liver weights in treated animals were reduced to within the range of wild type controls. Mean liver triglyceride concentration was reduced by 54.0%. In addition, liver mass as a percent of body mass was significantly reduced among treated animals compared with controls, in line with the expected benefit of VK2809. Additional evaluation of VK2809 on markers of autophagy and genes associated with lipid metabolism in this model is ongoing.
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.
The proof-of-concept study was conducted under a sponsored research agreement between Duke University and Viking Therapeutics and designed to evaluate the effects of VK2809 in Duke University's G6PC knockout mouse model.
Is
general: Yes
