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New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with
About this update from Biogen Inc.
[{"type":"text","content":"New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZAReductions in biomarker complement previously reported RESPOND efficacy results showing improved motor function in most participants treated with SPINRAZA after gene therapy CAMBRIDGE, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced interim 6-month biomarker data from the initial 29 participants in the open-label RESPOND study.* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec). The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA. These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024). “Our evolving understanding of gene therapy indicates there may be an opportunity for better outcomes,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters. “Improvements in motor function together with decreases in neurofilament levels seen after treatment with SPINRAZA in RESPOND show that we may be able to further maximize benefits for patients.” Today NfL data are being presented from study participants treated with SPINRAZA for 6 months showing: Among participants with 2 SMN2 copies: All participants had elevated baseline NfL levels relative to healthy children of similar ageIn infants (n=11) who were 9 months or younger at first SPINRAZA dose (mean baseline NfL: 148.3 pg/mL), NfL levels decreased by a mean of 70% from baseline.In children (n=11) over 9 months of age at first SPINRAZA dose (mean baseline NfL: 121.8 pg/mL), NfL levels decreased by a mean of 78% from baseline. Among participants with 3 SMN2 copies: Baseline NfL levels were elevated in 2 of 3 children (mean: 60.6 pg/mL).NfL reductions were observed in those with elevated levels at baseline and remained stable in the participant without an elevated level at baseline. “Biogen is at the forefront of pioneering research ai...