Ultragenyx Announces Completion of Enrollment in Global Phase 1/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome
03 1월 2024 - 10:00PM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
therapies for serious rare and ultrarare genetic diseases, today
announced the completion of patient enrollment in its Phase 1/2
clinical trial of GTX-102 for the treatment of pediatric patients
with Angelman syndrome (AS). The dose-expansion cohorts (Cohorts
A-E) have enrolled 53 patients for a total of 74 patients enrolled
globally in the Phase 1/2 trial. 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).
“With completion of enrollment in the Phase 1/2 trial, we remain
on track to report results in the first half of 2024 from at least
20 expansion cohort patients on therapy for a minimum of 6 months.
We are confident that the cumulative safety and efficacy data will
allow for dose and endpoint selection to support our Phase 3
program,” said Eric Crombez, M.D., chief medical officer at
Ultragenyx. “We appreciate the support of the Angelman community,
including the patients, families and healthcare providers, as we
urgently work together to develop a new treatment option that may
be able to improve the quality of life of those impacted by this
devastating disease.”
In October 2023, interim data from the extension cohorts
(Cohorts 4-7) of the ongoing Phase 1/2 study were announced and
showed improvements across multiple domains compared to natural
history data, where available, and clinical changes were associated
with quantitative changes in EEG. Long-term data showed patients
who stopped and restarted treatment reacquired previously gained
developmental skills when they were re-dosed with the current
regimen. As of the data cut-off, there have been no additional
treatment-related SAEs, including lower extremity weakness, since
November 2022.
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 Syndrome
Angelman 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 1 in
12,000 to 1 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. While 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-102
GTX-102 is an investigational antisense oligonucleotide
delivered via intrathecal administration and designed to target and
inhibit expression of UBE3A-AS. Nonclinical studies show that
GTX-102 reduces the 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.
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, 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, 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
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 Higa415-475-6370ir@ultragenyx.com
MediaCarolyn Wang415-225-5050media@ultragenyx.com
Ultragenyx Pharmaceutical (NASDAQ:RARE)
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Ultragenyx Pharmaceutical (NASDAQ:RARE)
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