New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
Biogen Inc. announced positive interim 6-month biomarker data from the open-label RESPOND study, showing reductions in neurofilament levels in most participants treated with SPINRAZA. The Phase 4 study focuses on infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after Zolgensma treatment. The data indicate improvements in motor function and reductions in neurodegeneration, highlighting the potential benefits of SPINRAZA in maximizing patient outcomes.
Positive
Positive interim biomarker data from the RESPOND study showed reductions in neurofilament levels in most SPINRAZA-treated participants.
The study focuses on infants and toddlers with SMA who have unmet clinical needs after Zolgensma treatment.
Improvements in motor function and decreases in neurodegeneration were observed, suggesting potential benefits of SPINRAZA in maximizing patient outcomes.
The interim findings from the RESPOND study indicating a reduction in neurofilament light chain (NfL) levels in SMA patients treated with SPINRAZA are significant for several reasons. Firstly, the reduction of NfL, a biomarker for axonal injury and neurodegeneration, suggests that SPINRAZA may be effective in reducing ongoing neuronal injury in this patient population. This is particularly relevant for infants and toddlers who have unmet clinical needs after gene therapy with Zolgensma, as it opens the possibility of additional treatment avenues to improve their condition.
Furthermore, the data showing improvements in motor function as measured by the HINE-2 score reinforces the potential of SPINRAZA as a therapeutic intervention post-gene therapy. While the reported serious adverse events in 34% of participants and any adverse events in 81.6% of participants warrant attention, the lack of serious adverse events directly related to SPINRAZA or leading to study withdrawal is encouraging from a safety perspective.
These findings could influence the treatment protocols for SMA and potentially lead to an increase in the use of SPINRAZA in patients who have previously received Zolgensma but still exhibit unmet clinical needs. This could have implications for Biogen's market share and revenue in the neurodegenerative disease treatment sector.
The RESPOND study results presented by Biogen have important implications for the competitive landscape in the SMA treatment market. The observed decrease in NfL levels and improved motor function post-treatment with SPINRAZA may strengthen Biogen's position in the market, especially given the current interest in biomarkers for neurodegenerative diseases. As investors and stakeholders evaluate the potential market impact, they will consider the fact that SPINRAZA could become a standard follow-up treatment after gene therapy, potentially increasing its adoption rate.
From an industry perspective, the focus on biomarkers like NfL is part of a broader trend towards personalized medicine and could set a precedent for other neurodegenerative diseases. The ability to demonstrate objective improvements in biomarkers linked to disease progression is a powerful tool for drug development and regulatory approval processes. Biogen's emphasis on pioneering biomarker research could not only benefit patients with SMA but also enhance the company's reputation as a leader in neurodegenerative disease research.
It is also worth noting that the presentation of these results at international conferences increases the visibility of SPINRAZA and may influence prescribing patterns. This could lead to an uptick in investor confidence if the market perceives the data as a sign of continued growth and innovation from Biogen.
The financial implications of the RESPOND study for Biogen are multifaceted. A reduction in NfL levels and improved motor function in SMA patients could mean a broader treatment indication for SPINRAZA, potentially expanding the drug's target demographic beyond those who have not received gene therapy. This expansion could drive revenue growth for Biogen, as SPINRAZA is one of its flagship products.
However, it is essential to consider the cost implications of long-term treatment with SPINRAZA, especially in the context of its use after an expensive gene therapy like Zolgensma. Payers, including insurance companies and government healthcare programs, will weigh the cost-effectiveness of such a treatment regimen. The long-term market uptake will depend on the balance between the clinical benefits to patients and the financial burden on the healthcare system.
Investors should monitor subsequent data presentations and peer-reviewed publications for a more comprehensive understanding of SPINRAZA's efficacy and safety profile. Additionally, regulatory responses to these findings could affect Biogen's stock performance, making it important to track FDA feedback and potential label expansions.
03/06/2024 - 07:30 AM
New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA Reductions 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 age In 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 aimed at advancing biomarkers to accelerate development of drugs for people living with devastating neurodegenerative diseases like SMA and ALS,” said Priya Singhal, M.D., M.P.H., Head of Development and interim Chief Medical Officer at Biogen. “Prior to receiving SPINRAZA at the start of RESPOND, we saw that participants had elevated neurofilament levels, as compared to healthy children suggesting ongoing neuronal injury. The RESPOND findings underscore the value of neurofilament as an objective marker for assessing remaining unmet needs in SMA patients who have previously received gene therapy.”
As reported at the SMA Research & Clinical Care Meeting in June 2023 from the same 29 participants, improvements in motor function were observed in most participants as measured by increased mean total Hammersmith Infant Neurological Examination Section 2 (HINE-2) score from baseline.1 No new emerging safety concerns have been identified in enrolled RESPOND participants who received SPINRAZA after Zolgensma. After a median of 230.5 days in the study, serious adverse events (AEs) were reported in 13/38 (34% ) participants. Any AEs were reported in 31/38 (81.6% ) participants. No serious AEs were considered related to SPINRAZA or led to study withdrawal, although some were related to administration.
These data and additional data from the RESPOND study will be presented at subsequent conferences this year including the 4th International Congress on Spinal Muscular Atrophy hosted by SMA Europe.
About SPINRAZA® (nusinersen) 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 into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.3
SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 8 years,3 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 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.
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.
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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 SPINRAZA; 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.
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References:
Proud C. Interim results from the ongoing RESPOND study evaluating nusinersen in children with spinal muscular atrophy previously treated with onasemnogene abeparvovec. June 2023. SMA Research & Clinical Care Meeting. Orlando, Fla. 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: February 2024. Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA. * Clinical outcomes and NfL were analyzed in the 29 participants who had the opportunity for at least six months of treatment at the time of the interim analysis. Analysis of mean change in NfL includes participants with baseline and Day 183 assessments; a mean change from baseline was not reported in the 3 SMN2 copies group, due to the small number of participants. Safety data are reported in all participants (n=38) who received at least one dose of SPINRAZA in the trial.
The new data from the RESPOND study revealed reductions in neurofilament levels in nearly all study participants treated with SPINRAZA.
The Phase 4 RESPOND 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 Zolgensma treatment.
The study participants showed improvements in motor function and reductions in neurofilament levels after treatment with SPINRAZA, indicating potential benefits for maximizing patient outcomes.
Nearly all study participants treated with SPINRAZA showed reductions in neurofilament levels, highlighting the positive impact of the treatment.
No new emerging safety concerns were identified in the enrolled RESPOND participants who received SPINRAZA after Zolgensma treatment, indicating the treatment's safety profile.
