- New data from the RESPOND study shows that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with Spinraza
- RESPOND efficacy results show improvement in motor function in most participants treated with Spinraza after gene therapy due to previously reported reduction in biomarker complement
CAMBRIDGE, Mass., March 6, 2024 (GLOBE NEWSWIRE) — Biogen, Inc. (Nasdaq: BIIB) has announced six months of interim biomarker data from the first 29 participants in the open-label RESPOND study. Announced.* Phase 4 study evaluates clinical outcomes and safety after two years of treatment with Spinraza in infants and young children with spinal muscular atrophy (SMA) who have unmet clinical need after treatment with Zolgensma .® (Onasemnogen abeparbovec). New data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal damage 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 Science Conference (March 3-6, 2024).
“Our evolving understanding of gene therapy shows that better outcomes are possible,” said Dr. Crystal Proud, a pediatric neurologist at King's Daughters Children's Hospital. “The improvements in motor function and reductions in neurofilament levels seen after treatment with SPINRAZA in response demonstrate the potential to further maximize benefits for patients.”
NfL data from study participants who received Spinraza for 6 months has now been published and shows that:
For participants with two copies of SMN2:
- All participants had elevated baseline NfL levels compared to age-matched healthy children
- Infants (n=11) who were 9 months or younger at the time of first SPINRAZA treatment (mean baseline NfL: 148.3 pg/mL) had an average decrease in NfL levels of 70% from baseline.
- In children 9 months of age and older (n=11) at the first dose of SPINRAZA (baseline mean NfL: 121.8 pg/mL), NfL levels decreased by an average of 78% from baseline.
For participants with three copies of SMN2:
- Baseline NfL levels were elevated in 2 of 3 children (mean: 60.6 pg/mL).
- Decrease in NfL was observed in participants with elevated levels at baseline and remained stable in participants without elevated levels at baseline.
“Biogen is at the forefront of pioneering research aimed at advancing biomarkers to accelerate drug development for people with serious neurodegenerative diseases such as SMA and ALS.” Biogen said Priya Singhal, MD, MPH, development director and interim chief medical officer. “At the start of RESPOND and before receiving Spinraza, we found elevated neurofilament levels in participants compared to healthy children, suggesting ongoing nerve damage. RESPOND Findings highlights the value of neurofilaments as an objective marker to assess remaining unmet needs in SMA patients who have previously undergone gene therapy.”
Increased mean sum score from baseline on the Hammersmith Infant Neurological Examination Section 2 (HINE-2) as reported by the same 29 participants at the June 2023 SMA Research and Clinical Care Conference Improvements in motor function, as measured by , were observed in most participants.1 No new safety concerns have been identified in registered RESPOND participants who received SPINRAZA after Zolgensma. After a median study period of 230.5 days, serious adverse events (AEs) were reported in 13 of 38 participants (34%). AEs were reported in 31 of his 38 (81.6%) participants. There were no serious adverse events associated with Spinraza or leading to study discontinuation, although some were treatment-related.
These data and additional data from the RESPOND study will be presented at conferences later this year, including the 4th International Spinal Muscular Atrophy Congress, hosted by SMA Europe.
About Spinraza® (nusinersen)
Spinraza is approved in more than 71 countries to treat infants, children, and adults with spinal muscular atrophy (SMA). More than 14,000 people around the world have been treated with Spinraza as a cornerstone of the treatment of SMA.2
Spinraza is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.3 It is administered directly to the central nervous system, where motor neurons are located, delivering treatment to where the disease begins.3
Spinraza has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data from patients treated for up to eight years.3Combine it with unparalleled real-world experience. Nusinersen's clinical development program includes more than 10 clinical studies involving more than 460 of his patients from a wide range of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The SHINE and NURTURE open-label extension studies are evaluating the long-term effects 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 nephrotoxicity and coagulation abnormalities, such as acute and severely low platelet counts, which are observed in some patients after administration of ASO.
Biogen has licensed worldwide rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). For important safety information and complete prescribing information for SPINRAZA in the United States, click here or visit the product website for each country.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company pioneering innovative science that delivers new medicines that transform the lives of patients and create value for our shareholders and communities. We apply our deep understanding of human biology and leverage a variety of modalities to advance first-in-class treatments and treatments that deliver superior results. Our approach is to take bold risks while balancing investment returns to achieve long-term growth.
We periodically post information we believe is important to investors on our website (www.biogen.com). Follow us on social media including Facebook, LinkedIn, X, and YouTube.
Biogen Safe Harbor
This news release contains forward-looking statements regarding the potential clinical efficacy of SPINRAZA. Potential Benefits, Safety, and Efficacy of Spinraza. Spinraza clinical development program. Identifying and treating SMA. Research and development program for SMA treatment. the potential of our commercial business and pipeline programs, including Spinraza; risks and uncertainties associated with the development and commercialization of pharmaceutical products; These forward-looking statements include words such as “aim,” “anticipate,” “believe,” “may,” “estimate,” “expect,” “predict,” “intend,” “anticipate,” “anticipate,” “believe,” “may,” “estimate,” “anticipate,” “predict,” “intend,” “anticipate,” “anticipate,” “believe,” “may,” “estimate,” “anticipate,” “project,” “intend,” “anticipate,” “anticipate,” “believe,” “may,” “estimate,” It may be accompanied by words such as “may” and “plan.” , “could,” “potential,” “will,” “would,” and other words and terms of similar meaning. The development and commercialization of pharmaceutical products involves high risks, and only a small number of research and development programs lead to commercialization of products. Results from early-stage clinical trials may not be indicative of the complete results or results of later-stage or larger-scale clinical trials and do not guarantee regulatory approval. Do not place undue reliance on our forward-looking statements.
These statements include, but are not limited to, uncertainties regarding the successful development and commercialization potential of Spinraza, which could cause actual results to differ materially from those reflected in such statements. involves certain risks and uncertainties. the risk that we may not be able to fully enroll in clinical trials or that enrollment will take longer than expected; Unanticipated concerns may arise from additional data, analyzes and results obtained during clinical trials. Regulatory authorities may require additional information or further studies or may deny, deny or delay approval of our drug candidates, including Spinraza. Occurrence of adverse safety events. risk of unanticipated hurdles, costs and delays; failure to protect and enforce our data, intellectual property and other proprietary rights, and uncertainties related to intellectual property claims and contests; Product liability claims. Operating Results and Financial Condition. The foregoing describes many, but not all, of the factors that could cause actual results to differ from our expectations in the forward-looking statements. In addition to this cautionary statement, investors should consider the risk factors identified in the Company's most recent annual or quarterly reports and other reports filed by the Company with the Securities and Exchange Commission. . These statements speak only as of the date of this news release.
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References:
- Proud C. Interim results of the ongoing RESPOND study evaluating nusinersen in children with spinal muscular atrophy previously treated with onasemnogen abeparvovec. June 2023. SMA Research and Clinical Care Conference.Orlando, Florida
- Based on commercial patients, early access patients, and clinical trial participants through December 31, 2022.
- Spinraza US Prescribing Information. Available at:
https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf. Accessed: February 2024. - Core Data Sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.
*Clinical outcomes and NfL were analyzed for 29 participants who had at least 6 months of treatment opportunity at the time of interim analysis. Analysis of mean change in NfL includes participants who took the baseline assessment and his 183rd day assessment. Due to the small number of participants, no mean change from baseline was reported in the SMN2 copy number 3 group. Safety data are reported for all participants (n=38) who received at least one dose of SPINRAZA in the trial.