Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
January 25 2024 - 4:05PM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that
all patients have been dosed with UX701 across the three
dose-escalation cohorts in Stage 1 of its pivotal Phase 1/2/3
Cyprus2+ study. 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.
“With the support of patients, physicians and the Wilson disease
community, we’ve completed Stage 1 enrollment in the Cyprus2+
program, which moves us one step closer to beginning Stage 2, the
pivotal, randomized placebo-controlled stage of the study,” said
Eric Crombez, M.D., chief medical officer at Ultragenyx. “Beyond
the seamless nature of this study, another important differentiator
of this program is that it leverages our Pinnacle PCL™ platform,
which enabled a single run to support Stage 1, demonstrating that
the productivity improvements generated by our platform are able to
support larger-scale clinical programs.”
Data presented in October 2023 at a Company Analyst Day
demonstrated that UX701 has been well tolerated in the first dose
cohort, with no unexpected related treatment emergent adverse
events observed as of the data cut-off date. Four of five patients
enrolled in Cohort 1 had started tapering standard-of-care
treatment, including two that came completely off of chelators
and/or zinc therapy. Additional interim data from all three Stage 1
dose cohorts are expected in the first half of 2024, which will be
followed by dose selection and initiation of Stage 2 in the second
half of 2024.
U.S. residents can learn more by visiting
www.ultraclinicaltrials.com.
Phase 1/2/3
Cyprus2+ study
designThis 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 were 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 medication
by Week 52. After the initial 52-week study period, all patients
will have long-term follow up in Stage 3.
About Wilson DiseaseWilson 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 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 UX701UX701 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) 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 the company's
website at: www.ultragenyx.com.
Ultragenyx Forward-Looking Statements and Use of Digital
MediaExcept 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 November 3, 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-/).
ContactsUltragenyx Pharmaceutical
Inc.InvestorsJoshua
Higa+1-415-475-6370ir@ultragenyx.com
MediaCarolyn Wang+1-415-225-5050media@ultragenyx.com
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