Roche to share latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association (MDA) 2025 conference
Roche (RHHBY) presented new data at the MDA conference (March 16-19, 2025) showcasing advancements in its neuromuscular portfolio. The company shared significant findings from two key treatments:
Evrysdi for SMA: Five-year SUNFISH study data demonstrated sustained stabilization of motor function improvements in Types 2 or 3 spinal muscular atrophy patients. The study, involving 231 participants aged 2-25 years, showed over 99% treatment adherence and maintained independence in daily activities. Untreated patients typically experienced significant decline over the same period.
Elevidys for Duchenne: Two-year EMBARK trial data and a pooled analysis showed statistically significant and clinically meaningful improvements in motor function. MRI assessments at weeks 52 and 104 indicated stabilization or slowing of disease progression compared to placebo. The treatment demonstrated positive outcomes in patients aged 4-8 years, with no new safety signals observed.
Roche (RHHBY) ha presentato nuovi dati alla conferenza MDA (16-19 marzo 2025) che evidenziano i progressi nel suo portafoglio neuromuscolare. L'azienda ha condiviso risultati significativi da due trattamenti chiave:
Evrysdi per SMA: I dati del studio SUNFISH di cinque anni hanno dimostrato una stabilizzazione sostenuta dei miglioramenti della funzione motoria nei pazienti con atrofia muscolare spinale di tipo 2 o 3. Lo studio, che ha coinvolto 231 partecipanti di età compresa tra 2 e 25 anni, ha mostrato oltre il 99% di aderenza al trattamento e ha mantenuto l'indipendenza nelle attività quotidiane. I pazienti non trattati hanno tipicamente sperimentato un significativo declino nello stesso periodo.
Elevidys per Duchenne: I dati del trial EMBARK di due anni e un'analisi combinata hanno mostrato miglioramenti statisticamente significativi e clinicamente rilevanti nella funzione motoria. Le valutazioni MRI alle settimane 52 e 104 hanno indicato stabilizzazione o rallentamento della progressione della malattia rispetto al placebo. Il trattamento ha dimostrato risultati positivi nei pazienti di età compresa tra 4 e 8 anni, senza nuovi segnali di sicurezza osservati.
Roche (RHHBY) presentó nuevos datos en la conferencia MDA (16-19 de marzo de 2025) que destacan los avances en su cartera neuromuscular. La compañía compartió hallazgos significativos de dos tratamientos clave:
Evrysdi para SMA: Los datos del estudio SUNFISH de cinco años demostraron una estabilización sostenida de las mejoras en la función motora en pacientes con atrofia muscular espinal de tipos 2 o 3. El estudio, que involucró a 231 participantes de entre 2 y 25 años, mostró más del 99% de adherencia al tratamiento y mantuvo la independencia en las actividades diarias. Los pacientes no tratados típicamente experimentaron un declive significativo durante el mismo período.
Elevidys para Duchenne: Los datos del ensayo EMBARK de dos años y un análisis combinado mostraron mejoras estadísticamente significativas y clínicamente relevantes en la función motora. Las evaluaciones de MRI en las semanas 52 y 104 indicaron estabilización o desaceleración de la progresión de la enfermedad en comparación con el placebo. El tratamiento mostró resultados positivos en pacientes de 4 a 8 años, sin nuevos señales de seguridad observadas.
로슈 (RHHBY)는 MDA 컨퍼런스(2025년 3월 16-19일)에서 신경근육 포트폴리오의 발전을 보여주는 새로운 데이터를 발표했습니다. 회사는 두 가지 주요 치료법에서 중요한 발견을 공유했습니다:
SMA를 위한 Evrysdi: 5년 SUNFISH 연구 데이터는 2형 또는 3형 척수성 근위축증 환자에서 운동 기능 개선의 지속적인 안정화를 입증했습니다. 2세에서 25세 사이의 231명의 참여자가 포함된 이 연구는 99% 이상의 치료 순응도를 보여주었고 일상 활동에서의 독립성을 유지했습니다. 치료를 받지 않은 환자들은 같은 기간 동안 상당한 감소를 경험하는 경향이 있었습니다.
듀센느를 위한 Elevidys: 2년 EMBARK 시험 데이터와 통합 분석 결과는 운동 기능에서 통계적으로 유의미하고 임상적으로 의미 있는 개선을 보여주었습니다. 52주 및 104주 차의 MRI 평가에서는 위약과 비교하여 질병 진행의 안정화 또는 둔화가 나타났습니다. 이 치료는 4세에서 8세 사이의 환자에서 긍정적인 결과를 보여주었으며, 새로운 안전성 신호는 관찰되지 않았습니다.
Roche (RHHBY) a présenté de nouvelles données lors de la conférence MDA (16-19 mars 2025) mettant en avant les avancées de son portefeuille neuromusculaire. L'entreprise a partagé des résultats significatifs de deux traitements clés :
Evrysdi pour SMA : Les données de l'étude SUNFISH sur cinq ans ont démontré une stabilisation durable des améliorations de la fonction motrice chez les patients atteints d'atrophie musculaire spinale de types 2 ou 3. L'étude, impliquant 231 participants âgés de 2 à 25 ans, a montré plus de 99 % d'adhérence au traitement et a maintenu l'indépendance dans les activités quotidiennes. Les patients non traités ont généralement connu un déclin significatif au cours de la même période.
Elevidys pour Duchenne : Les données de l'essai EMBARK de deux ans et une analyse groupée ont montré des améliorations statistiquement significatives et cliniquement pertinentes de la fonction motrice. Les évaluations par IRM aux semaines 52 et 104 ont indiqué une stabilisation ou un ralentissement de la progression de la maladie par rapport au placebo. Le traitement a montré des résultats positifs chez les patients âgés de 4 à 8 ans, sans nouveaux signaux de sécurité observés.
Roche (RHHBY) präsentierte neue Daten auf der MDA-Konferenz (16.-19. März 2025), die Fortschritte in seinem neuromuskulären Portfolio zeigen. Das Unternehmen teilte bedeutende Ergebnisse von zwei wichtigen Behandlungen mit:
Evrysdi für SMA: Die fünfjährigen SUNFISH-Studienergebnisse zeigten eine nachhaltige Stabilisierung der Verbesserungen der motorischen Funktion bei Patienten mit spinaler Muskelatrophie der Typen 2 oder 3. Die Studie, an der 231 Teilnehmer im Alter von 2 bis 25 Jahren beteiligt waren, zeigte eine Behandlungsadhärenz von über 99% und bewahrte die Unabhängigkeit in den täglichen Aktivitäten. Unbehandelte Patienten erlebten in der Regel einen signifikanten Rückgang im gleichen Zeitraum.
Elevidys für Duchenne: Die Daten der zweijährigen EMBARK-Studie und eine zusammengefasste Analyse zeigten statistisch signifikante und klinisch relevante Verbesserungen in der motorischen Funktion. MRI-Bewertungen in den Wochen 52 und 104 wiesen auf eine Stabilisierung oder Verlangsamung des Krankheitsverlaufs im Vergleich zu Placebo hin. Die Behandlung zeigte positive Ergebnisse bei Patienten im Alter von 4 bis 8 Jahren, ohne dass neue Sicherheitszeichen beobachtet wurden.
- Evrysdi showed sustained motor function stabilization over 5 years in SMA patients
- Exceptional treatment adherence rate of over 99% for Evrysdi
- Elevidys demonstrated statistically significant motor function improvements vs control groups
- MRI data confirmed Elevidys' effectiveness in slowing disease progression
- None.
- New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA)
- Late-breaking oral on Elevidys’ Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD)
- Muscle pathology as measured by MRI continued to generally favour Elevidys across muscle groups in boys with Duchenne up to two years
Basel, 17 March 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present new data at the Muscular Dystrophy Association (MDA) conference, 16-19 March, 2025, in Dallas, Texas, from its neuromuscular portfolio, including 12 oral and poster presentations. These data demonstrate the breadth of Roche’s neuromuscular portfolio across spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).
“These longer-term results are encouraging for people living with SMA and Duchenne, as they demonstrate sustained improvements or stabilisation in mobility and independence for those receiving our disease-modifying treatments,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. “The positive SUNFISH five-year results encompass one of the broadest SMA populations to be studied to date. We are also very encouraged by the data being shared across our Elevidys studies, affirming the potential of the first gene therapy for the treatment of Duchenne to stabilise or slow disease progression.”
Five-year exploratory data from the pivotal SUNFISH study of Evrysdi in people with Types 2 or 3 SMA
After up to five years of treatment in the SUNFISH trial (NCT02908685, n=231), SMA patients on Evrysdi demonstrated overall long-term stabilisation of motor function improvements from baseline that were observed during the first year, as measured by Motor Function Measure 32 (MFM-32). This marks the final readout from SUNFISH, which has reinforced the efficacy, safety and longer-term impact of Evrysdi in a broad population of people with Types 2 and 3 SMA, aged 2-25 years at enrollment. Untreated natural history data presented together with the SUNFISH five-year data show that without treatment, patients from a similar population can experience a significant decline in motor function over the same five-year period.
Patients (>12 years of age) and caregivers both reported continuous improvement or stabilization in levels of independence for performing daily activities, such as dressing, picking up objects and washing, as measured by the SMA Independence Scale (SMAIS-ULM).
Additionally, mean treatment adherence with Evrysdi over the multi-year period was over
Muscle health and longer-term functional outcomes from individuals with Duchenne’s disease after treatment with Elevidys, vs. well-matched external control groups
Elevidys showed statistically significant and clinically meaningful improvements across key measures of motor function two years after treatment in EMBARK (Phase III, NCT05096221) and three years after treatment in a pooled analysis of Study 101 ( Phase I/II, NCT03375164, n=4), Study 102 (Phase II, NCT03769116, n=26), and ENDEAVOR Cohort 1 (Phase Ib, NCT04626674, n=20), compared to well-matched external control groups. Collectively, these data demonstrate that treatment with Elevidys results in long-term stabilisation or slowing of disease progression in individuals aged four through eight years of age at the time of treatment.
Topline data from year two of the EMBARK trial were announced in January 2025. No new safety signals were observed in the EMBARK study over the two-year duration.
To further evaluate the effect of Elevidys on disease progression, muscle health and changes in muscle pathology were assessed by magnetic resonance imaging (MRI) in a subset of individuals in EMBARK part one. At week 52, results showed stabilisation or slowing of disease progression in Elevidys-treated patients compared to placebo-treated patients. At week 104, MRI changes from baseline continued to generally favour Elevidys versus placebo at week 52 across muscles and muscle groups.
Further information on the abstracts that Roche will present at MDA 2025 can be found below.
| Topic | Abstract Title | Presentation Number/Presentation Details |
| DMD | Long-term functional outcomes, safety, and micro-dystrophin expression following delandistrogene moxeparvovec treatment in DMD: EMBARK two-year results | #169 oral presentation |
| Three-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls | #167 oral presentation | |
| Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy | #73 poster presentation | |
| Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 | #168; poster presentation | |
| Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical Trials | #89 poster presentation | |
| Impact of satralizumab on bone strength and muscle function in Duchenne muscular dystrophy (DMD): design of the SHIELD-DMD study | #82 poster presentation | |
| Assessing biomarkers of bone metabolism and the role of the interleukin (IL)-6 signalling pathway in patients with Duchenne muscular dystrophy | #146 poster presentation | |
| Natural history of bone health in Duchenne muscular dystrophy: A systematic review and implications for the design of a clinical trial | #145 poster presentation | |
| Longitudinal Stride-Level Evaluation of Ambulatory Function with Ankle Wearable Technology in Ambulant DMD Patients Below 4 Years Old | #92 poster presentation | |
| SMA | SUNFISH Parts 1 and 2: Five-year efficacy and safety data of risdiplam in Types 2 and 3 spinal muscular atrophy | #94 poster presentation |
| Real-world risdiplam effectiveness in adults with spinal muscular atrophy (SMA) from the Pediatric Neuromuscular Clinical Research (PNCR) registry | #391 poster presentation | |
| RAINBOWFISH: Two-year efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA) | #281 oral presentation Session: Clinical Trial Updates |
About Duchenne muscular dystrophy
Duchenne is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Approximately one in 5,000 boys worldwide are born with Duchenne, while Duchenne in girls is very rare. Everyone who has Duchenne will lose the ability to walk, upper limb, lung and cardiac function and mean life expectancy is 28 years. A diagnosis of Duchenne will require full-time caregiving which is most often provided by parents, the majority of whom will find it difficult to carry out usual work or household activities and suffer from depression and physical pain.
Duchenne is caused by mutations of the DMD gene, which affects the production of the muscle protein, dystrophin. Dystrophin is a critical component of a protein complex that strengthens muscle fibers and protects them from injury during muscle contraction. Due to a genetic mutation in the DMD gene, people with Duchenne do not make functional dystrophin; their muscle cells are more sensitive to injury and muscle tissue is progressively replaced with scar tissue and fat. As dystrophin is also deficient in vital organ systems such as the cardiovascular and respiratory systems, the effect is thus inevitably fatal, with an average survival limited to the third decade of life.
About spinal muscular atrophy
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation or deletion of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of functional SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Currently approved drugs, including Evrysdi, prevent degeneration or death of these cells, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth. In the United States a room-temperature stable Evrysdi 5 mg tablet formulation is also now available.
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions such as swallowing, speaking and breathing.
Evrysdi is currently approved in more than 100 countries, with more than 18,000 people with SMA treated globally.
Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
About ELEVIDYS™ (delandistrogene moxeparvovec, SRP-9001)
Elevidys is the first approved disease-modifying gene therapy for Duchenne and is designed to address the underlying cause of Duchenne through targeted skeletal, respiratory and cardiac muscle expression of shortened dystrophin produced by Elevidys. Elevidys is a one-time treatment administered through a single intravenous dose. Elevidys is contraindicated in individuals with any deletion in exons 8 and/or 9 in the DMD gene.
Elevidys is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the US, United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. Elevidys is also approved for the treatment of ambulatory individuals aged four through seven years in Brazil and Israel. Filings have also been submitted to the European Medicines Agency (EMA) and regulatory authorities in Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia. To date, more than 600 patients have been treated with Elevidys.
About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.
Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045.
Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.
For more information, please visit www.roche.com.
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FAQ
What were the key findings from Roche's (RHHBY) 5-year SUNFISH study for Evrysdi?
How did Elevidys perform in RHHBY's EMBARK trial for Duchenne muscular dystrophy?
What age groups were studied in RHHBY's neuromuscular treatment trials?
Were there any safety concerns in RHHBY's Elevidys EMBARK trial?
