Triheptanoin (UX007) is being developed for the treatment of long chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is a group of six rare genetic disorders in which the body is unable to convert dietary fatty acids into energy. This inability to produce energy from fat can lead to severe depletion of glucose in the body and serious, unpredictable complications, which can lead to hospitalizations or early death despite the best current care. LC-FAOD has no specifically approved treatments. The current disease management includes avoidance of fasting, maintenance of a low fat diet, and supplementation of diet with oils rich in essential fatty acids.
Triheptanoin is an orally administered synthetic (artificially produced) fat, which is broken down in the liver into substances that can be used to generate energy. Triheptanoin could provide a source of calories and fatty acids for patients with LC-FAOD, potentially improving their muscle function, exercise tolerance, and health-related quality of life. If licenced, triheptanoin could provide a treatment option for paediatric and adult patients with molecularly confirmed LC-FAOD who currently have no approved therapies.
Semaglutide is in clinical development for the treatment of overweight and obese individuals. Excess weight can place stress on both mental and physical health, leading a number of complications such as depression, low self-esteem, and increased risk of heart disease, stroke and type 2 diabetes. Weight can be affected by a number of factors such as diet, physical activity, genetics and general health conditions, however diet and exercise are the two main contributing factors. Being overweight is reversible through lifestyle changes such as increased exercise, healthy diet and a net calorie deficit, along with help through counselling and medication. If weight is not able to be controlled, surgery may be required. Therefore, there is an unmet need for pharmacological therapies that target both weight and glucose control.