Biogen Releases Promising Results for Nusinersen in SMA Treatment
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Positive Study Results from Biogen on Nusinersen for SMA
Biogen Inc. (Nasdaq: BIIB) has recently announced some exciting topline results from their pivotal Phase 2/3 DEVOTE study. This study evaluated a new higher dose regimen of nusinersen aimed at treating spinal muscular atrophy (SMA) in infants diagnosed with the disease. The new dosing regimen has demonstrated considerable potential, suggesting that it may significantly advance the treatment options available for SMA.
Study Design and Findings
The Phase 2/3 DEVOTE study's Part B cohort involved treatment-naïve infants diagnosed with symptomatic SMA. At the core of this study was a higher dose regimen for nusinersen, which combines an accelerated loading phase of two 50 mg doses spaced 14 days apart, followed by a maintenance dose of 28 mg every four months. This regimen is compared to the currently approved dose of 12 mg of nusinersen, known commercially as SPINRAZA.
Within the first six months, the study achieved its primary endpoint, demonstrating a statistically significant enhancement in motor function in infants receiving the higher dose regimen compared to a matched sham control group. Participants who received the new higher dose regimen exhibited a least squares mean difference score of 26.19 on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) test, emphasizing a notable improvement.
Implications for SMA Treatment
“While we have made incredible strides in managing SMA, a considerable unmet need remains,” remarked Stephanie Fradette, Pharm.D., who oversees Biogen's Neuromuscular Development Unit. The study advances the narrative that a higher dose of nusinersen can potentially reduce neurodegeneration more rapidly, as indicated by reduced neurofilament levels observed at day 64 post-treatment. Over time, this regimen is observed to provide a significant clinical benefit for infants suffering from symptomatic SMA.
Overview of the DEVOTE Study
The DEVOTE study is comprehensive, consisting of three parts and enrolling 145 participants across different age groups and types of SMA. The pivotal Part B featured 75 infants who were randomized to either the investigational higher dose regimen or an approved 12 mg regimen comprising four loading doses and subsequent maintenance treatments. Results continue to favor the higher dose regarding not only the primary endpoint but also selected secondary endpoints, where positive trends were observed. Furthermore, the safety monitoring indicated that serious adverse events were less frequent in the higher dose group.
About SPINRAZA
SPINRAZA has become a foundational treatment for SMA, with approval in over 71 countries and more than 14,000 patients treated globally. As an antisense oligonucleotide, this therapy tackles the underlying motor neuron loss by augmenting the production of survival motor neuron (SMN) protein. SPINRAZA is administered directly into the central nervous system, effectively addressing the root cause of SMA. Over the years, extensive clinical monitoring has established its safety and efficacy across various demographics, underscoring the importance of this therapy in the SMA care continuum.
Future Directions
Biogen is committed to providing further detailed data to the broader SMA community and regulatory bodies in the coming months, showcasing the transformative potential of their investigational higher dose regimen of nusinersen. These advancements may lead to enhanced treatment protocols that could significantly improve the quality of life for those affected by this debilitating condition.
Frequently Asked Questions
What is the main finding of Biogen's DEVOTE study?
The study found that a higher dose regimen of nusinersen significantly improved motor function in infants with SMA compared to an untreated control group.
How does the new dosing regimen of nusinersen compare to the approved dose?
The new regimen consists of two higher doses followed by a higher maintenance dose, which contrasts with the previous approved dosage that maintained a 12 mg administration schedule.
What impact does the new dosing have on treatment timelines?
The accelerated loading doses aim to provide earlier and potentially more pronounced clinical benefits, reducing neurological deterioration more quickly in infants diagnosed with SMA.
What is SPINRAZA and how does it function?
SPINRAZA is an FDA-approved therapy designed to treat SMA by increasing the production of survival motor neuron (SMN) protein, delivered directly into the central nervous system.
When can we expect more information on the DEVOTE study results?
More comprehensive results from the DEVOTE study are expected to be presented at upcoming medical conferences, providing greater insight into the efficacy and safety of the higher dose regimen.
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