New Data for Nusinersen Underscore Biogen’s Commitment to Advancing Clinical Research to Improve Outcomes in SMA
Biogen (NASDAQ:BIIB) announced new clinical data for nusinersen (SPINRAZA®) in treating spinal muscular atrophy (SMA). The DEVOTE Part C study, evaluating a higher dose regimen, showed improvements in motor function across different patient groups. The new dosing comprises two 50 mg loading doses followed by 28 mg maintenance doses every four months.
In DEVOTE Part C, participants previously treated with 12 mg SPINRAZA showed functional improvements after transitioning to the higher dose. Non-ambulatory participants improved by +2.5 on the HFMSE scale. The safety profile remained consistent with the known 12 mg dosing.
The final NURTURE study results demonstrated that 92% of presymptomatic infants achieved independent walking, with all participants surviving and maintaining clinical benefits over eight years. The higher dose regimen is currently under regulatory review globally.
Biogen (NASDAQ:BIIB) ha annunciato nuovi dati clinici su nusinersen (SPINRAZA®) nel trattamento dell'atrofia muscolare spinale (SMA). Lo studio DEVOTE Parte C, che valuta un regime posologico più elevato, ha mostrato miglioramenti nella funzione motoria in diversi gruppi di pazienti. Il nuovo schema prevede due dosi di carico da 50 mg seguite da dosi di mantenimento da 28 mg ogni quattro mesi.
Nel DEVOTE Parte C, i partecipanti precedentemente trattati con 12 mg di SPINRAZA hanno mostrato miglioramenti funzionali dopo il passaggio alla dose più alta. I partecipanti non deambulanti hanno registrato un miglioramento di +2,5 nella scala HFMSE. Il profilo di sicurezza è rimasto coerente con quello noto per la dose da 12 mg.
I risultati finali dello studio NURTURE hanno dimostrato che il 92% degli infanti presintomatici ha raggiunto la deambulazione autonoma, con tutti i partecipanti ancora vivi e con benefici clinici mantenuti per oltre otto anni. Il regime posologico più elevato è attualmente in fase di revisione regolatoria a livello globale.
Biogen (NASDAQ:BIIB) anunció nuevos datos clínicos sobre nusinersen (SPINRAZA®) para el tratamiento de la atrofia muscular espinal (AME). El estudio DEVOTE Parte C, que evalúa un régimen de dosis más altas, mostró mejoras en la función motora en diferentes grupos de pacientes. La nueva dosificación consiste en dos dosis de carga de 50 mg seguidas de dosis de mantenimiento de 28 mg cada cuatro meses.
En DEVOTE Parte C, los participantes previamente tratados con 12 mg de SPINRAZA mostraron mejoras funcionales tras pasar a la dosis más alta. Los participantes no ambulantes mejoraron en +2,5 en la escala HFMSE. El perfil de seguridad se mantuvo consistente con la dosis conocida de 12 mg.
Los resultados finales del estudio NURTURE demostraron que el 92% de los bebés presintomáticos lograron caminar de forma independiente, con todos los participantes sobreviviendo y manteniendo beneficios clínicos durante más de ocho años. El régimen de dosis más altas está actualmente bajo revisión regulatoria a nivel mundial.
Biogen (NASDAQ:BIIB)는 척수 근위축증(SMA) 치료를 위한 누신ersen(SPINRAZA®)의 새로운 임상 데이터를 발표했습니다. 고용량 요법을 평가한 DEVOTE 파트 C 연구에서는 다양한 환자 그룹에서 운동 기능 개선이 확인되었습니다. 새로운 투여법은 50mg 적재용량 2회 후 4개월마다 28mg 유지용량으로 구성됩니다.
DEVOTE 파트 C에서 이전에 12mg SPINRAZA를 투여받은 참가자들은 고용량으로 전환 후 기능적 개선을 보였습니다. 보행이 불가능한 참가자들은 HFMSE 척도에서 +2.5 향상을 기록했습니다. 안전성 프로파일은 12mg 용량과 일관성을 유지했습니다.
NURTURE 연구의 최종 결과는 증상 전 영아의 92%가 독립 보행을 달성했으며, 모든 참가자가 8년 이상 생존하며 임상적 혜택을 유지하고 있음을 보여주었습니다. 고용량 요법은 현재 전 세계적으로 규제 심사 중입니다.
Biogen (NASDAQ:BIIB) a annoncé de nouvelles données cliniques concernant le nusinersen (SPINRAZA®) dans le traitement de l'amyotrophie spinale (SMA). L'étude DEVOTE Partie C, évaluant un schéma posologique plus élevé, a montré des améliorations de la fonction motrice chez différents groupes de patients. La nouvelle posologie comprend deux doses de charge de 50 mg suivies de doses d'entretien de 28 mg tous les quatre mois.
Dans DEVOTE Partie C, les participants précédemment traités avec 12 mg de SPINRAZA ont montré des améliorations fonctionnelles après être passés à la dose plus élevée. Les participants non ambulatoires ont progressé de +2,5 sur l'échelle HFMSE. Le profil de sécurité est resté conforme à celui connu pour la dose de 12 mg.
Les résultats finaux de l'étude NURTURE ont démontré que 92 % des nourrissons présymptomatiques ont atteint la marche autonome, avec tous les participants survivants et bénéficiant d'avantages cliniques sur plus de huit ans. Le schéma posologique plus élevé est actuellement en cours d'examen réglementaire à l'échelle mondiale.
Biogen (NASDAQ:BIIB) gab neue klinische Daten zu Nusinersen (SPINRAZA®) bei der Behandlung der spinalen Muskelatrophie (SMA) bekannt. Die DEVOTE Teil C-Studie, die ein höheres Dosierungsschema evaluiert, zeigte Verbesserungen der motorischen Funktion in verschiedenen Patientengruppen. Die neue Dosierung umfasst zwei 50 mg Ladedosen, gefolgt von 28 mg Erhaltungsdosen alle vier Monate.
In DEVOTE Teil C zeigten Teilnehmer, die zuvor mit 12 mg SPINRAZA behandelt wurden, funktionelle Verbesserungen nach dem Wechsel zur höheren Dosis. Nicht gehfähige Teilnehmer verbesserten sich um +2,5 auf der HFMSE-Skala. Das Sicherheitsprofil blieb mit der bekannten 12 mg Dosierung konsistent.
Die endgültigen Ergebnisse der NURTURE-Studie zeigten, dass 92 % der präsymptomatischen Säuglinge eigenständig gehen konnten, wobei alle Teilnehmer über acht Jahre überlebten und klinische Vorteile beibehielten. Das höhere Dosierungsschema befindet sich derzeit weltweit in der behördlichen Prüfung.
- None.
- Some adverse events reported including pneumonia, respiratory failure, and COVID
- 15% of participants in Part C experienced serious adverse events
Insights
Positive clinical data from DEVOTE and NURTURE studies strengthens nusinersen's efficacy profile, potentially expanding Biogen's SMA market position.
The new data from Biogen's clinical studies represents significant advancement in SMA treatment optimization. The DEVOTE Part C results demonstrate that patients previously stabilized on the standard 12mg SPINRAZA regimen for approximately four years can achieve additional functional improvements when transitioned to the higher dose regimen (50mg loading, 28mg maintenance).
Particularly noteworthy is the functional improvement in non-ambulatory participants, who gained +2.5 points on the Hammersmith Functional Motor Scale (HFMSE). This exceeds the commonly accepted threshold of 2 points for clinical meaningfulness in SMA, suggesting genuine therapeutic benefit rather than statistical artifact.
The NURTURE study results are even more compelling. With 92% of presymptomatic infants achieving independent walking and 100% survival after eight years, these outcomes dramatically exceed the natural history of even the mildest forms of untreated SMA. The absence of permanent ventilation requirements further confirms the profound neurological protection provided by early intervention.
The consistent safety profile across both studies is reassuring, especially considering the higher dosing in DEVOTE. The biomarker data showing rapid reduction in neurofilament light chain (NfL) levels provides objective evidence of reduced neuronal damage, complementing the clinical assessments.
These findings have significant implications for clinical practice. If approved, the higher dose regimen could offer improved outcomes for patients with partially treated disease, while the NURTURE data further cements the critical importance of newborn screening and early intervention in SMA.
- New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who transitioned to the investigational higher dose regimen of nusinersen from 12 mg SPINRAZA® (nusinersen)
- Final results from the landmark NURTURE study highlight the profound impact of early treatment with 12 mg SPINRAZA in clinically presymptomatic SMA with
92% of children achieving the ability to walk independently
CAMBRIDGE, Mass., June 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). These latest findings from Part C of the DEVOTE trial evaluating a higher dose regimen of nusinersen and the NURTURE trial which evaluated the approved 12 mg regimen (SPINRAZA®) in clinically presymptomatic SMA were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. Biogen’s applications for the higher dose regimen of nusinersen are currently under review in the U.S., Europe, Japan and other global markets. The higher dose regimen of nusinersen comprises a more rapid loading regimen – two 50 mg doses 14 days apart – and a higher maintenance regimen – 28 mg every four months.
“As the SMA treatment landscape continues to evolve, we remain steadfast in our commitment to address the unmet needs of the community. The findings from Part C of the DEVOTE study further strengthen the growing body of evidence supporting the potential benefits of the higher dose regimen of nusinersen,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen.
Improvements Observed With Higher Dose Regimen of Nusinersen in Previously Treated Patient Population
Detailed results from Part C of the DEVOTE study highlight the potential clinical benefits of an investigational higher dose of nusinersen in a broad range of individuals (n=38) who had been previously treated with nusinersen at the approved 12 mg dose for approximately four years (median: 3.9 years). Participants were 4 to 65 years of age and half (n=19) were ambulatory. Participants in Part C received one loading dose (50 mg) and two maintenance doses (28 mg each) of open-label higher dose nusinersen during the study period.
Most participants experienced improvements on the Hammersmith Functional Motor Scale – Expanded (HFMSE), Revised Upper Limb Module (RULM), and/or Clinical Global Impression of Change (CGI-C; assessed by investigator or caregiver) after transitioning to the higher dose regimen. These improvements were observed across phenotypes, functional status and age. For example, non-ambulatory participants improved by +2.5 (
“These emerging data indicate that additional gains in function might be possible even in those with established disease who have been on therapy for years,” said Richard Finkel, M.D., director, Center for Experimental Neurotherapeutics (CENT) at St. Jude Children’s Research Hospital. “This effort to optimize the dosing of SPINRAZA is very exciting for the field and could fundamentally change how we treat our patients.”
The safety profile of the higher dose regimen of nusinersen is broadly consistent with the known safety profile of 12 mg SPINRAZA. In the DEVOTE study overall, reported adverse events (AEs) included pneumonia, respiratory failure, pyrexia, COVID, upper respiratory tract infection, procedural pain and procedural headache. In Part C (n=40), AEs were reported in 37/40 participants, the majority of which were mild or moderate in severity. Serious AEs were reported by six participants (
Final NURTURE Data Redefine Expectations for Early Treatment
Final data from the eight-year, open-label NURTURE study highlight the impact of early intervention with 12 mg SPINRAZA in clinically presymptomatic infants with SMA.
At the study conclusion, all children who participated in NURTURE (n=25) were alive and experienced continued clinical benefits over the course of the study. No participants required permanent ventilation, and the majority (20 of 25 participants) went without any ventilatory support throughout the study. Ninety-two percent of participants achieved the ability to walk independently, many within normal developmental timeframes. Participants with elevated levels of neurofilament light chain (NfL) at baseline experienced rapid and sustained reductions in NfL after initiation of nusinersen, reinforcing the potential utility of NfL as an objective biomarker of disease activity and treatment response in SMA.
Nusinersen was generally well tolerated with no new safety concerns identified with eight years of follow-up. All participants had at least one AE, the majority of which were mild to moderate in severity; no AEs led to treatment discontinuation or study withdrawal.
About SPINRAZA
SPINRAZA (nusinersen) 12 mg/5 mL injection is approved in more than 71 countries to treat infants, children and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.1 The currently approved 12 mg regimen for SPINRAZA is comprised of four loading doses administered over approximately 60 days, followed by maintenance dosing every four months thereafter.
SPINRAZA is an antisense oligonucleotide (ASO) that targets the underlying cause of motor neuron loss by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.2
SPINRAZA has shown sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years,3,4 combined with unsurpassed real-world experience. The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube.
Biogen Safe Harbor
This news release contains forward-looking statements, including related to the potential clinical effects of a higher dose regimen of nusinersen; the potential benefits, safety and efficacy of higher dose regimen of nusinersen; the clinical development program for higher dose regimen of nusinersen; the identification and treatment of SMA; our research and development program for the treatment of SMA; the potential of our commercial business and pipeline programs, including SPINRAZA; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will,” “would” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on our forward-looking statements.
These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of higher dose regimen of nusinersen; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including SPINRAZA; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release.
We do not undertake any obligation to publicly update any forward-looking statements.
References:
- Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022.
- SPINRAZA U.S. Prescribing Information. Available at:
https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: June 2025. - Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.
- Finkle et al. Cure SMA 2024. “Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA.”
| MEDIA CONTACT: Biogen Jack Cox + 1 781 464 3260 public.affairs@biogen.com | INVESTOR CONTACT: Biogen Tim Power +1 781 464 2442 IR@biogen.com |
FAQ
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Where is Biogen's higher dose nusinersen currently under review?
