NOVATO -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE) said it obtained a new license for one of its products in development, covering hard-to-treat epilepsy and other rare brain disorders.
The agreement with UniQuest Pty Limited, one of Australia's largest commercializers of academic research, will allow Ultragenyx to market its triheptanoin formulation, which the Novato biopharmacutical company calls UX007, worldwide for the treatment of refractory epilepsy and other seizure-related brain conditions. The University of Queensland had conducted research on the use of triheptanoin for those conditions.
"The completion of the license agreement with UniQuest and the issuance of additional composition patent claims mark the continued strengthening of our triheptanoin portfolio," said Emil Kakkis, Ph.D., M.D., chief executive officer and president of Ultragenyx (ultragenyx.com). "We are excited about the potential for triheptanoin to be used in certain difficult-to-treat seizure disorders and hope it will provide a new treatment option for affected patients."
Triheptanoin is a specially crafted type of fat -- to be precise, a medium odd-chain triglyceride of three seven-carbon fatty acids -- that helps patients whose disorders block their bodies from effectively converting fat to energy, which can cause glucose sugar levels in the body to fall too low and damage the brain, liver, muscle and heart, according to a recent Ultragenyx stock prospectus.
Ultragenyx in August 2012 licensed certain research on triheptanoin by Baylor Research Institute. The fat has been studied clinically for 13 years on 130 patients with various conditions, including 60 with long-chain fatty acid oxidation disorders, or LC-FAOD, which hampers conversion of fat to glucose. About 2,000--3,500 have that condition in the U.S., the company said, based on National Newborn Screening Information System data.
Ultragenyx has participated in an open trial of triheptanoin on those 60 patients, and results of that phase 2 trial are expected next year.
In March of this year, the company started phase 2 trials of triheptanoin to treat glucose transporter type 1 deficiency syndrome, or Glut1 DS, a genetic defect in the protein that carries glucose to the brain. Lack of that sugar in the brain can lead to seizures, delayed development and problems controlling muscle movement, so sufferers are often prescribed a certain high-fat diet to replace the sugar as a brain food source. Epilepsy drugs don't work for controlling seizures on about one-third of patients with that condition, Ultragenyx said.
Results from treatments on 50 patients in that trial are expected also next year. About 3,000--7,500 are estimated to have Glut1 DS in the U.S., the company said.
Ultragenyx also announced it received a U.S. patent for achieving a certain purity level of compositions of triheptanoin. The patent expires in October 2025. The company secured a patent almost a year ago for a dosage unit of formulations of triheptanoin, and it expires in June 2020.
Ultragenyx's stock price Tuesday was $48.50 a share, up 6.24 percent from Monday.