Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference

  • First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam)
  • Baseline characteristics indicate all nine infants and toddlers enrolled in RESPOND had suboptimal clinical status in ≥2 areas after receiving Zolgensma® (onasemnogene abeparvovec); there were no new safety findings with subsequent SPINRAZA® (nusinersen) treatment
  • New NURTURE results continue to show the potential long-term benefit of early treatment before SMA symptom onset, with 92 percent of participants now able to walk alone, most in age-appropriate timelines

Biogen recently announced new data and updates from its SPINRAZA® (nusinersen) and spinal muscular atrophy (SMA) research program aimed at improving clinical outcomes for people impacted by the disease, including the ASCEND, RESPOND and NURTURE studies.

First Patient Treated in Phase 3b ASCEND Study
The ASCEND study is currently enrolling with the first patient treated in Q1 2022. The primary endpoint in ASCEND is the total change from baseline in the Revised Upper Limb Module (RULM) score. The study will integrate smartphone-based digital assessments as an exploratory endpoint using Konectom™ NMD, a mobile application developed by Biogen Digital Health that will allow teen and adult participants to quantitatively and remotely self-assess motor function in their daily lives. ASCEND aims to enroll approximately 135 children, teens and adults previously treated with Evrysdi (a nusinersen-naïve group and a nusinersen-experienced group). All participants will receive higher dose nusinersen in the study.

Information on the ASCEND study (NCT05067790) is available at

Ongoing Research Aims to Inform SMA Treatment Decisions
Biogen will also present baseline characteristics from the RESPOND study investigating the efficacy and safety of SPINRAZA in infants and toddlers who still have unmet clinical needs following treatment with Zolgensma® (onasemnogene abeparvovec). All study participants (enrolled as of August 2021, n=9) who previously received the gene therapy showed suboptimal clinical status in two or more domains at baseline, the most common being motor and respiratory function. Initial safety findings indicate none of the adverse events (AEs) or serious AEs (parainfluenza virus infection and viral upper respiratory tract infection) reported were considered related to SPINRAZA treatment.

Additionally, the latest results from NURTURE, a study in infants treated in the presymptomatic stage of SMA, demonstrate that early and sustained treatment with SPINRAZA for up to 5.7 years (median 4.9 years) helped participants to maintain and make progressive gains in motor function. After 11 months of additional follow-up since the 2020 interim analysis, all children who were able to walk alone maintained this ability and one child gained the ability to walk alone, increasing the total percentage to 92 percent (23/25). Most children achieved motor milestones within age-appropriate timelines1 and no major motor milestones were lost. The safety of SPINRAZA over this extended follow-up period was consistent with previously reported findings.

*Nusinersen is currently commercialized under the brand name SPINRAZA® and the U.S. Food and Drug Administration-approved dose is 12 mg.

*Data presented at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 13-16, 2023).

About SPINRAZA® (nusinersen)
The SPINRAZA clinical development program encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations,2 including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. 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.

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