Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the T...
April 23 2018 - 8:30AM
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical
company focused on the development of novel products for rare and
ultra-rare diseases, today announced that the U.S. Food and Drug
Administration (FDA) has cleared the Investigational New Drug (IND)
application for DTX401, an adeno-associated virus vector based gene
therapy for the treatment of glycogen storage disease type Ia
(GSDIa). Enrollment in the Phase 1/2 study is expected to begin in
the first half of 2018, with data from the first cohort expected in
the second half of 2018.
“GSDIa is a devastating disease that requires patients to adhere
to a strict and burdensome cornstarch feeding protocol to maintain
normal blood glucose levels and prevent hypoglycemia. Failure of
dietary therapy can lead to episodes of severe hypoglycemia
resulting in seizures and death,” said Emil D. Kakkis, M.D., Ph.D.,
Chief Executive Officer and President of Ultragenyx. “We look
forward to initiating our clinical program for DTX401, our gene
therapy designed to replace the deficient enzyme in the liver to
improve glucose control and prevent the devastating short and
long-term consequences of this disease.”
The open-label, multicenter Phase 1/2 study will evaluate the
safety, tolerability and therapeutic response of DTX401 in adults
with GSDIa. Key efficacy assessments include time to hypoglycemia,
impact on biomarkers such as lipids, uric acid, and measurement of
glycogen in liver. There are three potential dosing cohorts in the
study, and three patients will be enrolled in each cohort.
About GSDIa
GSDIa is the most common genetically inherited glycogen storage
disease. It is caused by a defective gene for the enzyme
glucose-6-phosphatase-α (G6Pase-α), resulting in the inability to
regulate blood sugar (glucose). Hypoglycemia in GSDIa patients can
be life-threatening, while the accumulation of the complex sugar
glycogen in certain organs and tissues can impair the ability of
these tissues to function normally. If chronically untreated,
patients can develop severe lactic acidosis, progress to renal
failure, and potentially die in infancy or childhood. There are no
approved pharmacological therapies. An estimated 6,000 patients
worldwide are affected by GSDIa.
About DTX401
DTX401 is an investigational adeno-associated virus vector (AAV)
type 8 gene therapy designed to deliver stable expression and
activity of G6Pase-α following a single intravenous infusion and
has been shown in preclinical studies to improve G6Pase-α activity
and reduce hepatic glycogen levels, a well-described biomarker of
disease progression. DTX401 has been granted Orphan Drug
Designation in both the United States and Europe.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing
to patients novel products for the treatment of rare and ultra-rare
diseases, with a focus on serious, debilitating 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
relating to Ultragenyx's expectations regarding the timing of
release of additional data for its product candidates, and plans
for its clinical programs and its clinical studies, 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,
such as the regulatory approval process, the timing of regulatory
filings, and other matters that could affect sufficiency of
existing cash, cash equivalents and short-term investments to fund
operations and the availability or commercial potential of our drug
candidates. 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 Ultragenyx in general, see
Ultragenyx's Annual Report on Form 10-K filed with the Securities
and Exchange Commission on February 21, 2018, and its subsequent
periodic reports filed with the Securities and Exchange
Commission.
Contact Ultragenyx Pharmaceutical Inc.Investors &
MediaDanielle Keatley415-475-6876
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