REGENXBIO Announces Presentation at the World Muscle Society
REGENXBIO (Nasdaq: RGNX) announced that Chief Medical Officer Steve Pakola, M.D., will present new analysis of 12-month functional data from the Phase I/II trial of RGX-202 at the International Congress of the World Muscle Society in Vienna, Austria, October 7-11, 2025.
The presentation will highlight that RGX-202 demonstrated a favorable safety profile with no serious adverse events in the Phase I/II study. Notably, pivotal dose participants showed superior performance compared to baseline-matched external natural history controls across all functional measures, suggesting RGX-202's potential as a best-in-class gene therapy for Duchenne muscular dystrophy.
REGENXBIO (Nasdaq: RGNX) a annoncé che il Chief Medical Officer Steve Pakola, M.D., presenterà una nuova analisi dei dati funzionali a 12 mesi dal trial di fase I/II di RGX-202 al Congresso Internazionale della World Muscle Society a Vienna, in Austria, dal 7 all'11 ottobre 2025.
La presentazione evidenzierà che RGX-202 ha mostrato un profilo di sicurezza favorevole, senza eventi avversi gravi nello studio di fase I/II. Da notare, i partecipanti trattati con dose chiave hanno mostrato prestazioni superiori rispetto ai controlli esterni di storia naturale normalizzati per la baseline, su tutte le misure funzionali, suggerendo il potenziale di Duchenne muscular dystrophy come terapia genica leader nella sua classe.
REGENXBIO (Nasdaq: RGNX) anunció que el Director Médico Steve Pakola, M.D., presentará un nuevo análisis de los datos funcionales a 12 meses del ensayo de fase I/II de RGX-202 en el Congreso Internacional de la World Muscle Society en Viena, Austria, del 7 al 11 de octubre de 2025.
La presentación destacará que RGX-202 mostró un perfil de seguridad favorable sin eventos adversos graves en el estudio de fase I/II. En particular, los participantes con dosis clave mostraron un rendimiento superior en comparación con controles históricos externos normalizados por línea de base en todas las medidas funcionales, lo que sugiere el potencial de Duchenne muscular dystrophy como una terapia génica líder en su clase.
REGENXBIO (Nasdaq: RGNX)는 최고의학책임자 Steve Pakola 박사(M.D.)가 RGX-202의 1/2상 12개월 기능 데이터에 대한 새로운 분석을 2025년 10월 7-11일 오스트리아 비엔나에서 열리는 국제 World Muscle Society 학술대회에서 발표할 것이라고 발표했다.
발표는 RGX-202가 1/2상 연구에서 중대한 이상반사 없이 안전성 프로파일이 우수했음을 강조할 것이며, 핵심 용량 참가자들이 모든 기능 지표에서 기저선 매칭 외부 자연사 대조군 대비 우수한 성과를 보였다고 하여 Duchenne muscular dystrophy를 최고의 유전자 치료제로 가능성을 시사한다.
REGENXBIO (Nasdaq: RGNX) a annoncé que le directeur médical Steve Pakola, M.D., présentera une nouvelle analyse des données fonctionnelles sur 12 mois de l’essai de phase I/II de RGX-202 lors du Congrès international de la World Muscle Society à Vienne, en Autriche, du 7 au 11 octobre 2025.
La présentation mettra en évidence que RGX-202 a démontré un profil de sécurité favorable sans événements indésirables graves dans l’étude de phase I/II. Notamment, les participants à dose clé ont montré une performance supérieure par rapport aux contrôles historiques externes appariés à la ligne de base sur toutes les mesures fonctionnelles, suggérant le potentiel de Duchenne muscular dystrophy comme thérapie génique de référence dans sa catégorie.
REGENXBIO (Nasdaq: RGNX) hat bekannt gegeben, dass der Chief Medical Officer Steve Pakola, M.D., neue Analysen der 12-monatigen Funktionsdaten aus der Phase-I/II-Studie von RGX-202 beim Internationalen Kongress der World Muscle Society in Wien, Österreich, vom 7. bis 11. Oktober 2025 vorstellen wird.
Die Präsentation wird hervorheben, dass RGX-202 im Phase-I/II-Studie ein günstiges Sicherheitsprofil ohne schwerwiegende unerwünschte Ereignisse zeigte. Bemerkenswert ist, dass Teilnehmer mit der vorgesehenen Dosis im Vergleich zu baseline-normalisierten externen Natural-History-Kontrollen über alle Funktionsmaße hinweg eine überlegene Leistung zeigten, was das Potenzial von Duchenne muscular dystrophy als best-in-class Gentherapie nahelegt.
REGENXBIO (Nasdaq: RGNX) أعلنت أن المدير الطبي ستيف باكولا، دكتور في الطب، سيلقي عرضاً لتحليل جديد لبيانات وظيفية لمدة 12 شهراً من تجربة المرحلة الأولى/الثانية لـ RGX-202 في المؤتمر الدولي لـ World Muscle Society في فيينا، النمسا، من 7 إلى 11 أكتوبر 2025.
سيبرز العرض أن RGX-202 أظهر ملفاً أمانياً مفضلاً بدون حدوث أحداث جانبية خطيرة في دراسة المرحلة الأولى/الثانية. ومن الجدير بالذكر أن المشاركين المعطاة بالجرعة الأساسية أظهروا أداءً متفوقاً مقارنةً بضمائر التاريخ الطبيعي الخارجية المطابقة للخط الأساس عبر جميع المقاييس الوظيفية، مما يوحي بإمكان Duchenne muscular dystrophy أن تكون علاجاً وراثياً جينياً رائداً في فئتها.
REGENXBIO (Nasdaq: RGNX)宣布，首席医疗官Steve Pakola博士将于2025年10月7日至11日在奥地利维也纳举行的世界肌肉学会国际大会上，就< b>RGX-202的 Phase I/II 试验的12个月功能数据，发表新的分析结果。
该报告将强调< b>RGX-202在I/II期研究中表现出有利的安全性特征，未出现严重不良事件。值得注意的是，关键剂量的参与者在所有功能指标上相对于基线匹配的外部自然历史对照组显示出更出色的表现，表明< b>Duchenne muscular dystrophy作为一种同类中的最佳基因治疗潜力。
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The podium presentation will share new analysis of previously presented 12-month functional data from the Phase I/II trial of RGX-202, including individual patient improvement on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model from the Collaborative Trajectory Analysis Project. As reported, RGX-202 demonstrated a favorable safety profile with no serious adverse events or adverse events of special interest observed in the Phase I/II study. Pivotal dose participants exceeded baseline-matched external natural history controls on all functional measures.
Using multiple models of natural history disease progression, these results further demonstrate the potential of RGX-202 to serve as a differentiated, best-in-class gene therapy for the treatment of Duchenne muscular dystrophy.
Presentation: RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data
Presenter: Steve Pakola, M.D., Chief Medical Officer, REGENXBIO
Session: Short Oral Presentations 1: Updates on SMA and DMD Trials
- Accompanying Poster: P425
Date/Time: October 8, 3:30 CET
The presentation will be available on the Publications page of REGENXBIO's website, www.REGENXBIO.com.
About RGX-202
RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain.
Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress® suspension-based platform process.
ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.
FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
George E. MacDougall
Investor Relations
IR@regenxbio.com
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SOURCE REGENXBIO Inc.
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