Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that
data from its ongoing late-stage program evaluating setrusumab
(UX143) in osteogenesis imperfecta (OI) will be presented at the
American Society for Bone and Mineral Research 2023 Annual Meeting
(ASBMR) October 13-16, 2023, in Vancouver, British Columbia.
Ultragenyx and its collaborators will present late-breaking
presentations on new, updated data from the Phase 2 portion of the
Phase 2/3 Orbit study evaluating setrusumab on annualized clinical
fracture rate in patients aged 5 to <26 years, and data on the
burden of fractures for individuals living with OI. The company
will also present information on the Phase 3 Cosmic study design
comparing setrusumab to intravenous bisphosphonate (IV-BP) in
patients aged 2 to <5 years.
Details of the setrusumab presentations are as
follows:
Abstract Title: Evaluating Setrusumab for the
Treatment of Osteogenesis Imperfecta: Phase 2 Data from the Phase
2/3 ORBIT Study (Lewiecki, E.M. et al.)Session:
Late-breaking poster session ISession Date and
Time: Saturday, Oct 14, 2023, 13:30–15:00
PDTPresentation Number: LB SAT-650
Abstract Title: Burden of Fractures for
Individuals Living with Osteogenesis Imperfecta: Integrating
Real-world Claims Data in the United States and Caregiver Panel
Interviews (Byers, H. et al.)Session:
Late-breaking poster session ISession Date and
Time: Saturday, Oct 14, 2023, 13:30–15:00
PDTPresentation Number: LB SAT-648
Abstract Title: Cosmic: An open-label,
randomized, active-controlled, phase 3 study of setrusumab compared
with bisphosphonates in pediatric subjects with osteogenesis
imperfecta (Krolczyk, S. et al.)Session: Poster
session ISession Date and Time: Saturday, Oct 14,
2023, 13:30–15:00 PDTPresentation Number:
SAT-500
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 previously
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.
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. 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 UltragenyxUltragenyx 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
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 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 August 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-/).
ContactsInvestorsJosh Higair@ultragenyx.com
MediaJeff Blake+1-415-612-7784media@ultragenyx.com
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