Eisai initiates rolling submission for the US FDA Biologics license application of lecanemab for early Alzheimer's disease under the accelerated approval pathway

STOCKHOLM, Sept. 28, 2021 /PRNewswire/ -- BioArctic AB's (publ) (Nasdaq Stockholm: BIOA B) partner Eisai today announced that they have initiated a rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application (BLA) for lecanemab (BAN2401), an investigational anti-amyloid beta (Aβ) protofibril antibody, for the treatment of early Alzheimer's disease (early AD). The BLA is being submitted under the accelerated approval pathway and is primarily based on clinical, biomarker and safety data from the Phase 2b clinical trial in people with early Alzheimer's disease and confirmed amyloid pathology. The lecanemab Phase 2b trial results demonstrated a high degree of Aβ plaque lowering and consistent reduction of clinical decline across several clinical endpoints. The correlation between the extent of Aβ plaque reduction and effect on clinical endpoints in the Phase 2b study further supports Aβ as a surrogate endpoint that is reasonably likely to predict clinical benefit. AD is a serious, progressive and devastating disease with few treatment options. Eisai is utilizing the accelerated approval pathway after discussions with the FDA and aims to bring a new potential treatment option to people living with early AD, their families and healthcare professionals.

In June 2021, lecanemab was granted Breakthrough Therapy designation, which is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. Eisai has an agreement with the FDA to submit the BLA for lecanemab as a rolling submission. This agreement allows completed portions of the application to be submitted to the FDA for review on an ongoing basis. After all portions are submitted to the FDA and the agency accepts the BLA, the Prescription Drug User Fee Act (PDUFA) action date (target date for completion of examination) will be set. 

The BLA submission for lecanemab is primarily based on the results of the proof-of-concept Phase 2b study in 856 patients with mild cognitive impairment (MCI) due to AD and mild AD (collectively known as early AD) with confirmed presence of amyloid pathology. The results were published in a peer-reviewed journal in April 2021.  The Phase 2b study explored the impact of treatment with lecanemab on reducing brain Aβ and clinical decline. At 18 months of treatment, 10-mg/kg biweekly lecanemab reduced brain amyloid by 0.306 SUVr units (from a baseline mean of 1.37), and over 80% of subjects became amyloid negative by visual read. Furthermore, the extent of reduction in amyloid was correlated with slower clinical decline on ADCOMS (Alzheimer's Disease Composite Score), CDR-SB (Clinical Dementia Rating-Sum-of-Boxes), and ADAS-cog (Alzheimer Disease Assessment Scale-Cognitive Subscale) at the treatment group and patient level. The rate of amyloid-related imaging abnormalities-edema/effusion (ARIA-E), an adverse event associated with amyloid targeted therapies, for the 10 mg/kg biweekly dosing was 9.9%.