Ultragenyx Announces Initiation of Dosing in Second Cohort of Pivotal Phase 1/2/3 Cyprus2+ Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
31 Julho 2023 - 9:00AM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced it
has begun dosing the second dose-escalation cohort in its pivotal
Phase 1/2/3 Cyprus2+ study following completion of dosing and
safety review in the first cohort. The company's investigational
AAV9 gene therapy is designed to deliver stable expression of the
ATP7B copper transporter following a single intravenous infusion,
with the goal of normalizing copper metabolism in patients with
Wilson disease.
In the first dose cohort, UX701 has been well tolerated with no
unexpected related treatment emergent adverse events observed as of
July 11, 2023. The Data Safety Monitoring Board recommended that
the company proceed with dosing patients at the higher dose of 1.0
x 10^13 GC/kg. Investigators have dosed the first patient and have
identified and screened the remaining four patients for dosing in
Cohort 2. The company is on track to complete enrollment in Stage 1
of the Phase 1/2/3 trial this year and expects to share initial
data in the first half of 2024.
“We are encouraged by the safety data and early signals of the
establishment of normal trafficking of copper observed in Cohort 1
and with acceleration of enrollment following improvements in study
design and entry criteria,” said Eric Crombez, M.D., chief medical
officer at Ultragenyx. “The shift to open label design and
broadening of inclusion criteria allows us to enroll subjects who
may not be well managed on current standard of care with chelators
and/or zinc.”
U.S. residents can learn more by visiting
ultraclinicaltrials.com.
Cyprus2+ Phase 1/2/3
study design
This study evaluating UX701 for the potential treatment of
Wilson disease is designed with three stages. During the first
stage, the safety and efficacy of up to three dose levels of UX701
will be evaluated over the course of 52 weeks and a dose will be
selected for further evaluation in Stage 2. In this first stage, 15
patients will be enrolled into three sequential dosing cohorts to
evaluate doses of 5.0 x 10^12 GC/kg, 1.0 x 10^13 GC/kg, and 2.0 x
10^13 GC/kg.
In Stage 2, a new cohort of patients will be randomized 2:1 to
receive the selected dose of UX701 or placebo. The primary safety
and efficacy analyses will be conducted at Week 52 of Stage 2. The
primary efficacy endpoints are change in 24-hour urinary copper
concentration and percent reduction in standard of care (SOC)
medication by Week 52. After the initial 52-week study period, all
patients will have long term follow up in stage 3.
About Wilson Disease
Wilson disease is a rare inherited disorder caused by mutations
in the ATP7B gene, which results in deficient production of ATP7B,
a protein that transports copper. Loss of function of this
copper-binding protein results in the accumulation of copper in the
liver and other tissues, most notably the central nervous system,
and also the failure to properly distribute copper by
ceruloplasmin. Patients with Wilson disease experience hepatic,
neurologic and/or psychiatric problems. Those with liver disease
can experience such symptoms as fatigue, lack of appetite,
abdominal pain and jaundice, and can progress to fibrosis,
cirrhosis, life-threatening liver failure and death. Wilson disease
can be treated by reducing copper absorption or removing excess
copper from the body using life-long chelation therapy, but unmet
needs exist because some treated patients experience clinical
deterioration and severe side effects. Wilson disease affects more
than 50,000 individuals in the developed world.
About UX701
UX701 is an investigational AAV9 gene therapy designed to
deliver stable expression of the ATP7B copper transporter following
a single intravenous infusion. It has been shown in preclinical
studies to normalize copper trafficking and excretion from the
body. The U.S. Food and Drug Administration (FDA) has granted
Orphan Drug Designation to UX701.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing
novel products to patients for the treatment of serious rare and
ultrarare genetic diseases. The company has built a diverse
portfolio of approved therapies and product candidates aimed at
addressing diseases with high unmet medical need and clear biology
for treatment, for which there are typically no approved therapies
treating the underlying disease.
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
ultragenyx.com.
Ultragenyx Forward-Looking Statements and Use of Digital
Media
Except for the historical information contained herein, the
matters set forth in this press release, including statements
related to Ultragenyx's expectations and projections regarding its
future operating results and financial performance, business plans
and objectives for UX701, expectations regarding the tolerability
and safety of UX701, and future clinical and regulatory
developments for UX701 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, collaboration with third parties,
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 uncertainty
of clinical drug development and unpredictability and lengthy
process for obtaining regulatory approvals, the ability of the
company to successfully develop UX701, the company’s ability to
achieve its projected development goals in its expected timeframes,
risks related to adverse side effects, risks related to reliance on
third party partners to conduct certain activities on the company’s
behalf , smaller than anticipated market opportunities for the
company’s products and product candidates, manufacturing risks,
competition from other therapies or products, and other matters
that could affect sufficiency of existing cash, cash equivalents
and short-term investments to fund operations, the company’s future
operating results and financial performance, the timing of clinical
trial activities and reporting results from same, and the
availability or commercial potential of Ultragenyx’s products and
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 (SEC) on May 5, 2023, and its
subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public
conference calls, Ultragenyx uses its investor relations website
and social media outlets to publish important information about the
company, including information that may be deemed material to
investors, and to comply with its disclosure obligations under
Regulation FD. Financial and other information about Ultragenyx is
routinely posted and is accessible on Ultragenyx’s Investor
Relations website (https://ir.ultragenyx.com/) and LinkedIn website
(https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).
Contacts
Ultragenyx Pharmaceutical, Inc.InvestorsJoshua
Higa415-475-6370IR@ultragenyx.com
MediaJeff Blake415-612-7784media@ultragenyx.com
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