This week, Alnylam and Biogen took a significant step toward redefining the landscape of Alzheimer’s disease treatment by revealing promising early-phase data from their groundbreaking RNAi and antisense approaches. The innovative techniques employed by these two companies offer hope for patients battling this neurodegenerative ailment.
Diverging Paths: Alnylam and Biogen are spearheading separate avenues in their quest to combat Alzheimer’s. Alnylam, in collaboration with Regeneron, is diligently exploring the RNAi approach. Their primary goal is to suppress the production of amyloid precursor protein (APP), a key factor in the development of amyloid deposits within the brain. This endeavor represents the inaugural utilization of Alnylam’s advanced technology designed to deliver treatments directly to the central nervous system.
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Biogen’s venture, on the other hand, hinges on antisense oligonucleotides. Their strategy centers on silencing the gene responsible for encoding tau, a protein intricately linked to Alzheimer’s. Since acquiring this asset from Ionis in 2019, Biogen has been diligently collecting clinical data showcasing the remarkable potential of their treatment in reducing total tau levels.
Showcasing Progress: Alnylam and Biogen chose the Clinical Trials on Alzheimer’s Disease (CTAD) conference as the platform to unveil their latest findings. Alnylam presented updated data concerning the initial phase of their phase 1 trial for the RNAi candidate, ALN-APP, involving 20 patients with early-onset Alzheimer’s. Notably, the updated data revealed that reductions in soluble APPα and soluble APPβ persisted over time. Alnylam initially reported median decreases of both biomarkers exceeding 70% for at least three months. The six-month mark saw reductions of over 55% for APPα and 65% for APPβ. In the latest 10-month data, mean reductions were observed at 33% for APPα and 39% for APPβ.
It’s worth noting that Alnylam achieved these responses after a single dose, with a multiple-dose segment now in progress in Canada and soon to commence in the Netherlands and the UK. However, this particular stage of the study remains on hold in the US due to data from nonclinical chronic toxicology studies.
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Biogen’s CTAD session showcased data from a phase 1b trial of their antisense oligonucleotide, BIIB080, a drug candidate that has garnered increased attention and resources under the leadership of CEO Christopher Viehbacher. The phase 1b results were previously documented in a Nature Medicine paper earlier this year.
During their CTAD presentation, Biogen offered a glimpse of early data concerning measures of cognition and function. Encouragingly, the trends favor BIIB080, with Biogen referencing external studies as a point of comparison, illustrating the candidate’s potential positive effects on various clinical assessments, including the clinical dementia rating scale, mini mental state examination, and functional activities questionnaire. Biogen is actively enrolling patients for a phase 2 trial, further cementing their commitment to advancing the fight against Alzheimer’s disease.
