Ultragenyx to Present GTX-102 Angelman Syndrome Program Update at the ASF Family Conference and Research Symposium
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will present updates on its GTX-102 Angelman syndrome program at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on July 24, 2024. The presentations will include:
1. A review of positive interim Phase 1/2 results previously disclosed at the American Academy of Neurology Meeting in April.
2. An update on plans to initiate a Phase 3 pivotal trial by the end of 2024.
3. Insights on patient progress, with continued development of new skills across multiple domains and no new serious adverse events reported.
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, expressed enthusiasm about working with the ASF and the broader community as they prepare for a global randomized study. The presentations will be livestreamed on the ASF website.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) presenterà aggiornamenti sul suo programma GTX-102 per la sindrome di Angelman durante la Conferenza Familiare e il Simposio di Ricerca della Angelman Syndrome Foundation (ASF) che si terrà il 24 luglio 2024. Le presentazioni includeranno:
1. Una revisione dei risultati positivi intermedi della Fase 1/2 precedentemente divulgati durante la Riunione dell'Accademia Americana di Neurologia ad aprile.
2. Un aggiornamento sui piani per avviare uno studio clinico di Fase 3 cruciale entro la fine del 2024.
3. Approfondimenti sui progressi dei pazienti, con lo sviluppo continuo di nuove abilità in vari ambiti e nessun nuovo evento avverso grave riportato.
Il Dr. Eric Crombez, Direttore Medico di Ultragenyx, ha espresso entusiasmo per la collaborazione con l'ASF e con la comunità più ampia mentre si preparano per uno studio randomizzato globale. Le presentazioni saranno trasmesse in diretta sul sito web dell'ASF.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) presentará actualizaciones sobre su programa GTX-102 para el síndrome de Angelman en la Conferencia Familiar y Simposio de Investigación de la Angelman Syndrome Foundation (ASF) el 24 de julio de 2024. Las presentaciones incluirán:
1. Una revisión de los resultados intermedios positivos de la Fase 1/2 divulgados anteriormente en la Reunión de la Academia Americana de Neurología en abril.
2. Una actualización sobre los planes para iniciar un ensayo clínico pivotal de Fase 3 a finales de 2024.
3. Perspectivas sobre el progreso de los pacientes, con un desarrollo continuo de nuevas habilidades en múltiples áreas y sin nuevos eventos adversos graves reportados.
El Dr. Eric Crombez, Director Médico de Ultragenyx, expresó entusiasmo por trabajar con la ASF y la comunidad en general mientras se preparan para un estudio aleatorio global. Las presentaciones se transmitirán en vivo en el sitio web de la ASF.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)는 2024년 7월 24일에 개최되는 Angelman Syndrome Foundation (ASF) 가족 회의 및 연구 심포지엄에서 GTX-102 앙겔만 증후군 프로그램에 대한 업데이트를 발표할 예정입니다. 발표 내용은 다음과 같습니다:
1. 4월에 열린 미국 신경학 아카데미 회의에서 공개된 긍정적인 중간 1/2상 결과 검토.
2. 2024년 말까지 3상 주요 시험을 시작할 계획에 대한 업데이트.
3. 여러 영역에서 새로운 기술의 지속적인 개발과 보고된 심각한 부작용이 없는 환자 진행 상황에 대한 통찰.
Ultragenyx의 최고 의학 책임자 Eric Crombez 박사는 글로벌 무작위 연구를 준비하면서 ASF 및 더 넓은 커뮤니티와 협력할 수 있어 기쁘다고 전했습니다. 발표는 ASF 웹사이트에서 생중계될 것입니다.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) présentera des mises à jour sur son programme GTX-102 pour le syndrome d'Angelman lors de la Conférence familiale et du Symposium de recherche de la Angelman Syndrome Foundation (ASF) le 24 juillet 2024. Les présentations incluront :
1. Un examen des résultats intermédiaires positifs de la Phase 1/2 précédemment divulgués lors de la Réunion de l'Académie Américaine de Neurologie en avril.
2. Une mise à jour sur les plans de lancement d'un essai pivot de Phase 3 d'ici la fin de 2024.
3. Des perspectives sur les progrès des patients, avec un développement continu de nouvelles compétences dans plusieurs domaines et aucun nouvel événement indésirable grave signalé.
Le Dr Eric Crombez, directeur médical d'Ultragenyx, a exprimé son enthousiasme à travailler avec l'ASF et la communauté élargie alors qu'ils se préparent pour une étude randomisée mondiale. Les présentations seront diffusées en direct sur le site Web de l'ASF.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) wird am 24. Juli 2024 Updates zu seinem GTX-102-Programm für das Angelman-Syndrom auf der Familienkonferenz und dem Forschungssymposium der Angelman Syndrome Foundation (ASF) präsentieren. Die Präsentationen umfassen:
1. Eine Überprüfung der zuvor auf dem Treffen der American Academy of Neurology im April mitgeteilten positiven Zwischenresultate der Phase 1/2.
2. Ein Update zu den Plänen zur Einleitung einer entscheidenden Phase-3-Studie bis Ende 2024.
3. Einblicke in den Fortschritt der Patienten, mit fortlaufender Entwicklung neuer Fähigkeiten in mehreren Bereichen und ohne Meldung neuer schwerwiegender Nebenwirkungen.
Dr. Eric Crombez, Chief Medical Officer von Ultragenyx, äußerte Begeisterung über die Zusammenarbeit mit der ASF und der breiteren Gemeinschaft, während sie sich auf eine globale randomisierte Studie vorbereiten. Die Präsentationen werden live auf der Website der ASF übertragen.
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Presentations will include an encore of the positive interim Phase 1/2 results presented in April at the 76th Annual American Academy of Neurology (AAN) Meeting
NOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio.
“Since our positive interim Phase 1/2 data presentation at AAN in April, we have continued to see the patients in our study develop new skills across multiple domains with no new serious adverse events,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “We look forward to working with the ASF and the broader community as we prepare to initiate a global randomized study by the end of this year.”
The ASF data presentation will review the previously disclosed Phase 1/2 results presented at AAN and an update on plans to initiate a Phase 3 pivotal trial by the end of the year.
ASF 2024 Presentation Details:
Title: Developing Treatments for Complex Rare Diseases
Session: Industry Introduction with Ultragenyx
Presenter: Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx
Date / Time: Wednesday, July 24, 2024, at 3:10 pm Eastern Time
Title: GTX-102 Angelman Program Update
Session: Industry Overview and Updates
Presenter: Kim Goodspeed, M.D., medical director, global clinical development at Ultragenyx
Date / Time: Wednesday, July 24, 2024, at 3:40 pm Eastern Time
Both presentations will be livestreamed from the ASF website: https://asfconference.org/family-conference/
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 have shown that GTX-102 reduces levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the central nervous system (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 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 approximately 60,000 people in commercially accessible geographies.
Individuals with Angelman syndrome have a lifelong neurodevelopmental disorder including cognitive impairment, motor impairment, balance issues 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 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 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 the clinical benefit, tolerability and safety of GTX-102 and the corresponding impact on patients, the anticipated dosing of the Phase 2 study for GTX-102 and the timing for initiation of a Phase 3 study for GTX-102 and associated 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, the risk that results from earlier studies may not be predictive of future study results, 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 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 May 3, 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-/).
Contacts
Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
+1-415-475-6370
ir@ultragenyx.com
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Carolyn Wang
+1-415-225-5050
media@ultragenyx.com
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