Date: 2015-01-07
Type of information: Licensing agreement
Compound: triheptanoin (UX007)
Company: Ultragenyx Pharmaceutical (USA - CA) Inserm Transfert (France) Institut du Cerveau et de la Moelle Epiniere (ICM) (France)
Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases
Type agreement: licensing
Action mechanism: Triheptanoin is a purified, pharmaceutical-grade, specially designed synthetic triglyceride compound created via a multi-step chemical process. Triheptanoin is metabolized to and intended to provide patients with heptanoate, which can diffuse across the blood-brain barrier and be converted into glucose. Heptanoate can also be further metabolized to four- and five-carbon ketone bodies in the liver that also cross the blood-brain-barrier and provide an additional energy source to the brain. Heptanoate and five-carbon ketone bodies can also regenerate new glucose in the brain, which is deficient in these patients. Ultragenyx is conducting a Phase 2 study in the U.S. and Europe (France, Italy, UK) to evaluate the potential of triheptanoin to treat glucose transporter type-1 deficiency syndrome patients who have failed the ketogenic diet and who continue to have breakthrough seizures. (NCT02036853 and NCT01993186). An Inserm-sponsored trial is also ongoing in France. The purpose of this phase 2 open label trial is to study the efficacy of triheptanoin oil in patients with Glucose Type 1 Transporter Deficiency (NCT02014883)
Disease: Huntington\'s disease
Details: * On January 7, 2015, Ultragenyx Pharmaceutical announced a license agreement with Inserm Transfert SA and Institut du Cerveau et de la Moelle Epiniere (ICM) for intellectual property related to the treatment of Huntington\'s disease with triheptanoin (UX007). \"Huntington\'s is a severe and lethal rare genetic disease,\" commented Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and President of Ultragenyx. \"Energy deficiency is believed to play a role in the pathophysiology of Huntington\'s disease, and we are encouraged by data from a small pilot study suggesting improvement in brain energy metabolism after treatment with triheptanoin.\" Ultragenyx is supporting a second investigator-sponsored clinical study being planned by ICM to evaluate the safety and efficacy of triheptanoin in patients with Huntington\'s disease.
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