Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting
2024年4月12日 - 9:00PM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that
preclinical and clinical data, including new clinical efficacy and
safety data from the expansion cohorts in the Phase 1/2 study of
GTX-102 for the treatment of Angelman syndrome, will be presented
at the 76th Annual American Academy of Neurology Meeting (AAN)
being held April 13-18 in Denver and virtually. GTX-102 is an
investigational antisense oligonucleotide delivered via intrathecal
administration and is designed to target and inhibit expression of
UBE3A antisense transcript (UBE3A-AS).
Abstracts are available to view online at
www.aan.com/events/annual-meeting-abstracts. The GTX-102 emerging
science presentation will include data beyond what is currently
available online.
AAN 2024 Presentation Details:
Title: Clinical Activity and Safety of GTX-102,
an Investigational Antisense Oligonucleotide for the Treatment of
Patients With Angelman SyndromeFormat: Oral
(abstract #192, presentation #008)Session:
Emerging Science 2Presenter: Kemi A. Olugemo,
M.D., FAAN, Ultragenyx Date/ Time: Tuesday, April
16, 6:12-6:18 p.m. MDT
Title: Proteomic Profiling of Angelman Syndrome
for Disease-Associated Biomarker DiscoveryFormat:
Poster (#8.001)Session: Child Neurology and
Developmental Neurology: Neurogenetics 2
Presenter: Rachael Elizabeth Hawtin, Ph.D.,
Ultragenyx Date/ Time: Tuesday, April 16,
5:30-6:30 p.m. MDT
Title: Clinical Characteristics of Creatine
Transporter Deficiency (CTD): Final Results of the Vigilan
Observational StudyFormat: Poster
(#8.002)Session: Child Neurology and Developmental
Neurology: Neurogenetics 2 Presenter:
Melanie Brandabur, M.D., Ultragenyx Date/ Time:
Tuesday, April 16, 5:30-6:30 p.m. MDT
Conference Call and Webcast
InformationUltragenyx will host a conference call at 8:00
a.m. ET on Wednesday, April 17, 2024, to discuss the new efficacy
and safety data from the GTX-102 Phase 1/2 clinical study being
presented at the conference.
The live and replayed webcast of the call will be available
through the company’s website at
https://ir.ultragenyx.com/events-presentations. To participate in
the live call, please register by clicking on the following link
(registration link) and you will be provided with dial-in
details.
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.
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 GTX-102, expectations regarding the tolerability
and safety of GTX-102, and future clinical and regulatory
developments for GTX-102 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 GTX-102, 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 Annual Report
on Form 10-K filed with the Securities and Exchange Commission
(SEC) on February 21, 2024, 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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