Ultragenyx, a privately held, clinical-stage biotechnology company, has expanded its exclusive license from Baylor Research Institute (BRI) in Dallas, Texas, to develop and commercialize triheptanoin outside North America. The global license includes rights to patents, patent applications and other intellectual property related to the composition and formulation of UX007, as well as its use in treating diseases including fatty acid oxidation disorders (FAOD), the lead indication being developed by the company.
The company plans to initiate a phase II clinical trial with UX007 later this year in patients with long-chain FAOD. It also is completing a retrospective study of triheptanoin in 24 FAOD patients who have been receiving triheptanoin through an investigator-sponsored compassionate-use protocol for up to 13 years.
Ultragenyx is supporting multiple independent investigator-sponsored and compassionate-use clinical trials in FAOD and other indications and is providing drug free of charge for these studies. Ultragenyx is planning to develop other potential indications for UX007, including GLUT1 deficiency syndrome (also known as De Vivo disease), a genetic disorder that impairs brain energy metabolism.