Full Press Release Details
Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a
biopharmaceutical company focused on the development of novel
products for rare and ultra-rare diseases, announced the first
patient enrolled in the Phase 2 study of triheptanoin (UX007) for
the treatment of glucose transporter type-1 deficiency syndrome
(Glut1 DS), at Columbia University. Glut1 DS, also known as De Vivo
disease, is a rare, severely debilitating disease characterized by
seizures, developmental delay, and movement disorder.
"Glut1 deficiency was first described by us at the Columbia
University Medical Center in 1991, so we are pleased to have the
opportunity to enroll the first patient in the triheptanoin
clinical trial sponsored by Ultragenyx. It is clear that more
effective symptomatic and disease-modifying treatments are needed
for this rare disease, and we hope this trial is the next step
towards that ultimate goal," said Dr. Darryl De Vivo, the Sidney
Carter Professor of Neurology and Professor of Pediatrics at
Columbia University Medical Center in New York City.
The global, randomized, double-blind, placebo-controlled,
parallel-group Phase 2 clinical trial will evaluate safety and
efficacy in up to 50 Glut1 DS patients between 3 and 17 years of
age inclusive, who are currently not on or not fully compliant with
ketogenic diet and continue to have seizures. The primary efficacy
objective is the reduction in frequency of seizures compared to
placebo following a 6-week baseline period and subsequent 8-week
placebo-controlled treatment period. The blinded treatment period
will be followed by an open-label extension period in which all
patients will be treated with triheptanoin through week 52.
Targeted enrollment may be modified based on an interim analysis.
We anticipate that data from this trial will be available in
"We are advancing triheptanoin for Glut1 DS based on its
anticipated ability to provide an alternative source of energy to
the brain that could result in seizure control and improvement in
other aspects of the disease," commented Emil D. Kakkis, M.D.,
Ph.D., Ultragenyx's Chief Executive Officer. "The
placebo-controlled Phase 2 study is a significant step in the
evaluation of triheptanoin for this rare and devastating
About Glut1 DS and Triheptanoin
Glut1 DS is a severely debilitating disease characterized by
seizures, developmental delay, and movement disorder. Glut1 DS is
caused by a mutation in the gene encoding the Glut1 protein, which
is responsible for transporting glucose across the blood-brain
barrier. Because glucose is the primary source of energy for the
brain, this disorder results in a chronic state of energy
deficiency in the brain. Glut1 DS is a rare disease. Studies
suggest a range of 3,000 to 7,000 Glut1 DS patients in the United
States. There are currently no approved treatments specific to
Triheptanoin is a specially designed synthetic triglyceride
compound intended to provide patients with the medium-length,
odd-chain fatty acid, heptanoate. Heptanoate can circulate and also
be further metabolized to four- and five-carbon ketone bodies in
the liver. All of these metabolites are able to cross the
blood-brain barrier without using the deficient Glut1 transporter,
and can provide an alternative energy source for the brain when
glucose is limited. Heptanoate also crosses the blood-brain barrier
and can be converted to glucose.
Ultragenyx is a development-stage biopharmaceutical company
committed to bringing to market novel products for the treatment of
rare and ultra-rare diseases, with an initial focus on serious,
debilitating metabolic genetic diseases. Founded in 2010, the
company has rapidly built a diverse portfolio of product candidates
with the potential to address diseases for which the unmet medical
need is high, the biology for treatment is clear, and for which
there are no approved therapies.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx's strategy is predicated upon time and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's
website at www.ultragenyx.com.
Forward-Looking Statements
Except for the historical information contained herein, the
matters set forth in this press release, including statements
regarding Ultragenyx's plans, potential opportunities,
expectations, projections, goals, objectives, milestones,
strategies, product pipeline, clinical studies, product development
and the potential benefits of its products under development are
forward-looking statements within the meaning of the "safe harbor"
provisions of the Private Securities Litigation Reform Act of
1995. Such forward-looking statements involve
substantial risks and uncertainties that could cause our clinical
development programs, future results, performance or achievements
to differ significantly from those expressed or implied by the
forward-looking statements. Such risks and uncertainties include,
among others, the uncertainties inherent in the clinical drug
development process, including the regulatory approval process, the
timing of our regulatory filings and other matters that could
affect the availability or commercial potential of our drug
candidate. Ultragenyx undertakes no obligation to update or revise
any forward-looking statements. For a further description of the
risks and uncertainties that could cause actual results to differ
from those expressed in these forward-looking statements, as well
as risks relating to the business of the Company in general, see
Ultragenyx's prospectus filed with the Securities and Exchange
Commission on January 31, 2014, and its future periodic reports to
be filed with the Securities and Exchange Commission.