FDA Grants Fast Track Designation to Biogen’s Novel Alzheimer Disease Drug Candidate

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The FDA has granted Fast Track designation to Biogen’s BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau for the treatment of Alzheimer disease (AD). In a press release, Biogen noted that BIIB080 is the first ASO targeting tau to enter clinical development. It is currently being evaluated in the Phase II CELIA study for early-stage AD.¹

“We are encouraged by the FDA’s Fast Track designation for BIIB080, which highlights the urgent need for innovative treatments targeting tau pathology in Alzheimer’s disease,” said Priya Singhal, MD, MPH, head of development, Biogen, in a press release. “Alzheimer’s is a complex and fatal disease that we believe will require multiple therapeutic approaches to address its diverse pathologies. BIIB080, an investigational antisense therapy, is a differentiated approach to targeting tau, with promising potential for patients. We are advancing this program with urgency on behalf of people living with Alzheimer’s and their families.”

In October 2023, Biogen reported new data from a Phase Ib study evaluating the safety and tolerability of multiple doses of BIIB080 in patients with mild dementia due to AD. Patients were randomly assigned into five cohorts, receiving either BIIB080 in doses of 10 mg once every four weeks (Q4W), 30 mg Q4W, 60 mg Q4W, 115 mg once every 12 weeks, or a placebo. In a long-term extension study, participants received either 60 mg or 115 mg every 12 weeks.2 The primary endpoint of the study was safety and tolerability of the treatment.³

Building on previous findings, favorable trends were observed in high-dose groups based on the global Clinical Dementia Rating Sum of Boxes, Mini-Mental State Exam cognitive scales, and Functional Activities Questionnaire at week 100. These findings included dose-dependent reductions in soluble tau in cerebrospinal fluid (CSF), decreased aggregated tau pathology observed via PET imaging, and favorable trends in exploratory cognitive and functional measures.^1,2 Previous results, which were announced in March 2023, demonstrated a 60% reduction from baseline CSF tau levels at the end of the long-term extension study.³

BIIB080 was generally well tolerated, with most adverse events (AEs) being mild to moderate. The most common AEs included headache, back pain, pain in extremity, post-lumbar puncture syndrome, and procedural pain.²

According to the Alzheimer’s Association, an estimated 7 million people in the United States are currently living with AD, with 73% over the age of 73 years. Approximately 10.9% of people over 65 years of age in the United States have AD, and the number of cases is projected to grow to 12.7 million by 2050 due to an aging population. Globally, 10 million new cases of dementia are reported annually, and as of 2020, an estimated 55 million people were living with AD or another form of dementia.

Currently, 60% of all dementia cases are in low- or middle-income countries, a figure expected to rise to 71% by 2050. The global annual cost of dementia was approximately $1.3 trillion in 2020 and is projected to reach $2.8 trillion by 2030, encompassing informal care, direct care, and medical expenses.^4,5

The Phase II CELIA study is fully enrolled, with results anticipated in 2026.¹

References

1. Biogen’s Investigational Tau-Targeting Therapy BIIB080 Receives FDA Fast Track Designation for the Treatment of Alzheimer’s Disease. Biogen. April 2, 2025. Accessed April 2, 2025. https://investors.biogen.com/news-releases/news-release-details/biogens-investigational-tau-targeting-therapy-biib080-receives

2. New Data from Biogen’s Investigational Antisense Oligonucleotide (ASO) Targeting Tau Shows Promise for Potential New Generation of Treatments in Early Alzheimer’s Disease. Biogen. October 25, 2023. Accessed April 2, 2025. https://investors.biogen.com/news-releases/news-release-details/new-data-biogens-investigational-antisense-oligonucleotide-aso

3. New Data Presented at AD/PD™ 2023 Show Biogen’s BIIB080 (MAPT ASO) Substantially Reduced Tau Protein Levels in Patients with Early-stage Alzheimer’s Disease. Biogen. March 29, 2023. Accessed April 2, 2025. https://investors.biogen.com/news-releases/news-release-details/new-data-presented-adpdtm-2023-show-biogens-biib080-mapt-aso

4. Alzheimer's Disease Facts and Figures. Alzheimer’s Association. Accessed April 2, 2025. https://www.alz.org/alzheimers-dementia/facts-figures#:~:text=navigate%20dementia%20care.-,Prevalence,other%20dementias%20as%20older%20Whites.

5. Dementia statistics. ADI. Accessed April 2, 2025. https://www.alzint.org/about/dementia-facts-figures/dementia-statistics/