Ultragenyx Receives PRIME Designation from European Medicines Agency (EMA) for GTX-102 for the Treatment of Angelman Syndrome
2024年2月6日 - 6:05AM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that
the European Medicines Agency (EMA) has granted Priority Medicine
(PRIME) designation to GTX-102 for the treatment of Angelman
syndrome (AS). 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). The EMA granted this designation in response
to compelling early clinical data from the extension cohorts in the
Phase 1/2 study of GTX-102 demonstrating clinically meaningful
improvements in several neurodevelopmental domains including
cognition, receptive communication and gross motor skills in
individuals with Angelman syndrome.
"By granting PRIME designation, the EMA is recognizing the
potential for GTX-102 to address the critical need for new
treatments for children and families impacted by Angelman syndrome
in the EU,” said Eric Crombez, M.D., chief medical officer at
Ultragenyx. “Our team is working with urgency on the development of
GTX-102 for Angelman syndrome and looks forward to working closely
with regulators in the U.S. and EU to bring this innovative
treatment to patients.”
PRIME designation is granted by the EMA to provide early and
proactive support to developers of promising medicines that may
offer a major therapeutic advantage over existing treatments or
benefit to patients without treatment options. These medicines are
considered priority medicines by the EMA, whose aim is to optimize
development plans and speed up evaluations so these medicines that
address significant unmet medical needs can reach patients
faster.
About the Phase 1/2 study
The Phase 1/2, open-label, multiple-dose, dose-escalating study
is evaluating the safety and tolerability of GTX-102 administered
by intrathecal (IT) injection to pediatric patients with Angelman
syndrome with a genetically confirmed diagnosis of full maternal
UBE3A gene deletion. The study is also assessing clinical response
as measured by a panel of efficacy assessments for the functional
domains impacted in Angelman syndrome. Patients in the earlier
extension cohorts (Cohorts 4-7) of the study have moved into
long-term maintenance dosing, and the study has completed
enrollment for the new expansion cohorts to verify the GTX-102 dose
range and treatment regimen that will be used in the Phase 3
program.
About Angelman SyndromeAngelman syndrome is a
rare, neurogenetic disorder caused by loss-of-function of the
maternally inherited allele of the UBE3A gene. The
maternal-specific inheritance pattern of Angelman syndrome is due
to genomic imprinting of UBE3A in neurons of the central nervous
system (CNS), a naturally occurring phenomenon in which the
maternal UBE3A allele is expressed and the paternal UBE3A is not.
Silencing of the paternal UBE3A allele is regulated by the UBE3A
antisense transcript (UBE3A-AS), the intended target of GTX-102. In
almost all cases of Angelman syndrome, the maternal UBE3A allele is
either missing or mutated, resulting in limited to no protein
expression. This condition is generally not inherited but instead
occurs spontaneously. It is estimated to affect one in 12,000 to
one in 20,000 people globally.
Individuals with Angelman syndrome have developmental delay,
balance issues, motor impairment and debilitating seizures. Some
individuals with Angelman syndrome are unable to walk and most do
not speak. Anxiety and disturbed sleep can be serious challenges in
individuals with Angelman syndrome. Although individuals with
Angelman syndrome have a normal lifespan, they require continuous
care and are unable to live independently. Angelman syndrome is not
a degenerative disorder, but the loss of the UBE3A protein
expression in neurons results in abnormal communications between
neurons. Angelman syndrome is often misdiagnosed as autism or
cerebral palsy. There are no currently approved therapies for
Angelman syndrome; however, several symptoms of this disorder can
be reversed in adult animal models of Angelman syndrome suggesting
that improvement of symptoms can potentially be achieved at any
age.
About GTX-102GTX-102 is an investigational
antisense oligonucleotide delivered via intrathecal administration
and designed to target and inhibit expression of UBE3A-AS.
Nonclinical studies have shown that GTX-102 reduces levels of
UBE3A-AS and reactivates expression of the paternal UBE3A allele in
neurons of the CNS. Reactivation of paternal UBE3A expression in
animal models of Angelman syndrome has been associated with
improvements in some of the neurological symptoms associated with
the condition. GTX-102 has been granted Orphan Drug Designation,
Rare Pediatric Disease Designation, and Fast Track Designation from
the FDA, and Orphan Designation and PRIME designation from the
EMA.
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,
future clinical and regulatory developments for GTX-102, the
clinical benefit, tolerability and safety of GTX-102, timing for
enrollment, dosing and data for GTX-102 and the company’s other
investigational therapies and regulatory meetings 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, risk that
access to PRIME for our product candidates, if granted, may not
lead to faster development or regulatory review or approval
process, and does not increase the likelihood that our product
candidates will receive marketing approval; 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, 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 product
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
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ContactsUltragenyx Pharmaceutical
Inc.InvestorsJoshua
Higa+1-415-475-6370ir@ultragenyx.com
MediaCarolyn Wang+1-415-225-5050media@ultragenyx.com
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 5 2024 まで 6 2024
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 6 2023 まで 6 2024