Ultragenyx Announces Completion of Patient Dosing in First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornit...
November 16 2017 - 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 it has completed patient
dosing in the first cohort of three patients enrolled in the Phase
1/2 study of DTX301, an adeno-associated virus (AAV) gene therapy
for the treatment of ornithine transcarbamylase (OTC) deficiency.
Initial data from this first cohort of three patients is expected
in early 2018.
The phase 1/2 clinical study is an open-label, dose-finding and
safety study of single ascending doses of DTX301 in adults with
late-onset OTC Deficiency who are clinically stable and under good
metabolic control at time of dosing. Patients in the first cohort
received a single dose of 2.0 × 10^12 GC/kg. To evaluate
therapeutic response of DTX301, the study measures the change in
the rate of ureagenesis, the pathway for the metabolism of ammonia
which is deficient in OTC patients. This is determined using a
well-established stable 13C-acetate labeling approach.
Ammonia levels, neurocognitive assessment, biomarkers, and safety
will also be evaluated. The decision to proceed to the next, higher
dose cohort will be made after the data monitoring committee
evaluates the safety data for all patients in the previous dosing
cohort.
About OTC Deficiency
OTC deficiency, the most common urea cycle disorder, is caused
by a genetic defect in a liver enzyme responsible for
detoxification of ammonia. Individuals with OTC deficiency can
build up excessive levels of ammonia in their blood, potentially
resulting in acute and chronic neurological deficits and other
toxicities. It is estimated that more than 10,000 patients are
affected by OTC deficiency worldwide, of which approximately 80%
are classified as late-onset. In the late onset form of the
disease, elevated ammonia can lead to significant medical issues
for patients who are in need of new disease-modifying therapies.
The greatest percentage of patients, including males and females,
experience late-onset disease, representing a clinical spectrum of
disease severity. Neonatal onset disease occurs in males, presents
as severe disease, and can be fatal at an early age. Approved
therapies, which must be taken multiple times a day for the
patient's entire life, do not eliminate the risk of future
metabolic crises. Currently, the only curative approach is liver
transplantation.
About DTX301
DTX301 is an investigational AAV type 8 gene therapy designed to
deliver stable expression and activity of OTC following a single
intravenous infusion and has been shown in preclinical studies to
normalize levels of urinary orotic acid, a marker of ammonia
metabolism. DTX301 was granted Orphan Drug Designation in both the
United States and Europe.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing
to market novel products for the treatment of rare and ultra-rare
diseases, with a focus on serious, debilitating genetic diseases.
The Company has rapidly built and advanced 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, plans for
its clinical programs, and the potential market size and the size
of the patient populations for its product candidates, if approved
for commercial use, 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 Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on November 3, 2017, 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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