Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that
the first patients have been dosed in both of its late-stage
clinical trials evaluating setrusumab in pediatric and young adult
patients with OI sub-types I, III and IV. The Phase 3 portion of
the pivotal Phase 2/3 Orbit study is evaluating the effect of
setrusumab compared to placebo on annualized clinical fracture rate
in patients aged 5 to <26 years. The newly initiated Phase 3
Cosmic study is an active-controlled study evaluating setrusumab
compared to intravenous bisphosphonate (IV-BP) therapy on
annualized total fracture rate in patients aged 2 to <5 years.
"I am extremely encouraged by the recent data from the Phase 2
portion of the Orbit study, which includes improvement in
biochemical markers and bone density measures that reflect the
clinical response we have observed in study participants," stated
Thomas Carpenter, M.D., professor of Pediatrics (Endocrinology) and
of Orthopaedics and Rehabilitation, Yale School of Medicine. "We
are looking forward to evaluating the full clinical potential of
setrusumab as this important Phase 3 program moves forward, with
hopes for improving therapeutic outcomes in OI."
“Data from the Phase 2 portion of the Orbit study demonstrated
increases in bone formation and bone mineral density, which are
important markers of bone strength, as well as early indications of
improved bone health from our investigators,” said Eric Crombez,
M.D., chief medical officer at Ultragenyx. “Our comprehensive Phase
3 program is designed to study the impact of setrusumab on clinical
fracture risk reduction. The two Phase 3 trials will evaluate
patients over a broad age range, including the younger pediatric
population, where the risk of fracture is higher and where we can
potentially have the greatest impact on their future health.”
Ultragenyx is leading the clinical development of setrusumab as
part of a collaboration and license agreement with Mereo BioPharma
Group plc (NASDAQ: MREO), a clinical-stage biopharmaceutical
company focused on rare diseases. The companies recently announced
positive data from the dose-selection Phase 2 portion of the Orbit
study showing that setrusumab rapidly induced bone production in
OI-affected patients.
U.S. residents can learn more by visiting
ultraclinicaltrials.com.
The Setrusumab Phase 3 Program
The global, seamless Phase 2/3 Orbit study is evaluating the
effect of setrusumab compared to placebo on clinical fracture rate
in patients aged 5 to <26 years. In the Phase 2 portion, 24
patients were randomized 1:1 to receive setrusumab at one of two
doses to determine the optimal dosing strategy for Phase 3. The
pivotal Phase 3 portion of the study will include approximately 195
patients at more than 40 sites across 12 countries, randomized 2:1
to receive setrusumab or placebo, with a primary efficacy endpoint
of annualized clinical fracture rate. All patients will transition
to an extension period and receive open-label setrusumab after the
Phase 3 primary analysis is complete.
The global Phase 3 Cosmic study is an open-label, randomized,
active-controlled study in patients aged 2 to <5 years
evaluating setrusumab compared to intravenous bisphosphonates
(IV-BP) therapy on reduction in total fracture rate, including
morphometric vertebral fractures. The Cosmic study will enroll
approximately 65 patients at more than 20 sites across 8
countries.
About Osteogenesis Imperfecta (OI)
Osteogenesis Imperfecta (OI) includes a group of genetic
disorders impacting bone metabolism. Approximately 85% to 90% of OI
cases are caused by mutations in the COL1A1 or COL1A2 genes,
leading to either reduced or abnormal collagen and changes in bone
metabolism. The collagen mutations in OI can result in increased
bone brittleness, which contributes to a high rate of fractures,
including at atypical sites. Patients with OI also exhibit
increased bone resorption (breakdown of old bone) and inadequate
production of new bone, which leads to decreased bone mass, bone
fragility and weakness. OI can also lead to bone deformities,
abnormal spine curvature, pain, decreased mobility, and short
stature. No treatments are approved for OI, which affects
approximately 60,000 people in the developed world.
About Setrusumab (UX143)
Setrusumab is a fully human monoclonal antibody that inhibits
sclerostin, a protein that acts on a key bone-signaling pathway
that inhibits the maturation and activity of bone-forming cells.
The goal of blocking inhibitory effects of sclerostin is to
increase new bone formation, bone mineral density and bone
strength. Sclerostin inhibition also reduces excessive bone
resorption, further enhancing its impact on bone density. In mouse
models of OI, the use of anti-sclerostin antibodies was shown to
stimulate bone formation, improve bone mass and density, and
increase bone strength against fracture force testing.
Mereo BioPharma’s Phase 2b study (ASTEROID) treatment phase of
the dose-finding study of setrusumab for the treatment of OI in 112
adults was concluded in 2019. The ASTEROID study demonstrated
treatment with setrusumab resulted in a clear, dose-dependent and
statistically significant effect on bone formation and bone density
at multiple anatomical sites among adult participants with OI.
Ultragenyx and Mereo BioPharma are collaborating on the
development of setrusumab globally based on the collaboration and
license agreement between the parties. The companies have developed
a comprehensive late-stage program to continue development of
setrusumab in pediatric and young adult patients across OI
sub-types I, III and IV.
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 UX143, expectations regarding the tolerability
and safety of UX143, and future clinical and regulatory
developments for UX143 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 and Mereo BioPharma to successfully develop UX143, 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, the potential for any license
or collaboration agreement, including the company’s collaboration
agreement with Mereo to be terminated, 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-/).
Contacts
Ultragenyx Pharmaceutical, Inc.InvestorsJoshua
Higa415-475-6370IR@ultragenyx.com
MediaJeff Blake415-612-7784media@ultragenyx.com
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