Ultragenyx, a privately held, clinical-stage biotechnology company committed to bringing to market life-transforming therapeutics for patients with rare and ultra-rare metabolic genetic diseases, has expanded its exclusive license from Baylor Research Institute (BRI) in Dallas, Texas, to develop and  commercialize triheptanoin outside of 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 a number of diseases including fatty acid oxidation disorders (FAOD), the lead indication being developed by the company.

The company plans to initiate a phase 2 clinical trial with UX007 later this year in  patients with long-chain FAOD.  The company is also completing a retrospective study of triheptanoin in 24 long-chain FAOD patients who have been receiving triheptanoin through an investigator-sponsored compassionate-use protocol for up to 13 years. Data from the retrospective study will be presented this fall.  

“We are encouraged by the data we have seen to date from investigator-initiated  studies, and are excited to expand the potential utility of UX007 in a global development programme,” said Emil D. Kakkis,  chief executive officer of Ultragenyx. “Our next  step is to build on these investigator programs with clinical data from sponsored trials of UX007 in patients with these devastating metabolic disorders.”

In addition to advancing its own development program toward potential approval, 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. UX007 is a pharmaceutical-grade product being developed by Ultragenyx under an exclusive supply agreement for triheptanoin with an experienced European manufacturer.

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.

Fatty acid oxidation disorders are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to break down fatty acids into energy. Several thousand patients are afflicted with FAOD in the US and a  number of fatty acid oxidation disorders are now included in newborn screening panels.

Several thousand additional patients outside of the US are also affected, and newborn screening for these disorders is becoming more common.

UX007 is a specially designed synthetic compound intended to provide patients with a  medium-length, odd-chain fatty acid that is metabolized to replace intermediate substrates in fatty acid oxidation and in the tricarboxylic acid (TCA) cycle, a key energygenerating process. UX007, a highly purified form of triheptanoin, is produced using a GMP-compliant process intended to reduce taste and odor with the goal of enhancing patient compliance.


Established in 1984 in Dallas, Texas, Baylor Research Institute (BRI) promotes and  supports research to bring innovative treatments from the laboratory workbench to the patient bedside.