CSIMarket.com
In recent announcements, Biogen Inc. and Ionis Pharmaceuticals, Inc. have made significant decisions regarding their drug development programs. The termination of BIIB105 (ION541), an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS), based on topline results from the Phase 1/2 ALSpire study, has been reported. Additionally, Biogen Inc. has provided updates on their SPINRAZA (nusinersen) treatment for spinal muscular atrophy (SMA) and the realignment of resources for their Alzheimer’s Disease franchise.
Regarding BIIB105, the Phase 1/2 ALSpire study showed statistically significant reductions in cerebrospinal fluid (CSF) ataxin-2 (ATXN2) protein levels, suggesting a potential benefit. However, over the six-month placebo-controlled period, treatment with BIIB105 did not demonstrate a reduction in plasma neurofilament light chain (NfL) levels, a biomarker associated with neurodegeneration and neuronal damage. Furthermore, BIIB105 did not show an impact on clinical outcome measures of function, breathing, and strength. As a result, Biogen and Ionis Pharmaceuticals have decided to terminate the development of BIIB105 for ALS.
In another study, interim 6-month biomarker data from the open-label RESPOND study evaluating SPINRAZA treatment in infants and toddlers with SMA were shared by Biogen Inc. The data revealed that nearly all study participants experienced a reduction in plasma neurofilament light chain (NfL) levels after treatment. This reduction is an encouraging sign as NfL levels are associated with axonal injury and neurodegeneration. These findings will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
Furthermore, Biogen Inc. has outlined its plans to reprioritize resources in their Alzheimer’s Disease franchise to focus on growth opportunities. The company will continue to advance LEQEMBI (lecanemab-irmb), the first anti-amyloid beta treatment with FDA traditional approval in the United States. They also plan to accelerate the development of potential new treatment modalities, including their antisense oligonucleotide (ASO) targeting tau (BIIB080), and an oral small molecule inhibitor of tau aggregation (BIIB113). In line with this strategic shift, Biogen will discontinue the development and commercialization of ADUHELM (aducanumab-avwa) 100 mg/mL injection for intravenous use and terminate the ENVISION clinical study. This decision has been made independent of any safety or efficacy concerns.
In conclusion, Biogen and Ionis Pharmaceuticals are terminating the development of BIIB105 for ALS due to its lack of efficacy in reducing plasma neurofilament light chain levels and clinical outcome measures. Meanwhile, positive interim biomarker data from the RESPOND study show promise for SPINRAZA in reducing NfL levels in infants and toddlers with SMA. Biogen’s strategic decision to realign its resources towards Alzheimer’s Disease therapies aims to drive near and long-term growth by advancing LEQEMBI and developing potential new treatment modalities.
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