Eisai Co., Ltd. and Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of Alzheimer’s disease (AD).

Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. The benefits of a Breakthrough Therapy designation include more intensive guidance on an efficient development program as well as eligibility for rolling review and potentially priority review.

The FDA’s Breakthrough Therapy designation for lecanemab is based on the recently published results of a Phase 2b clinical trial (Study 201) of 856 patients with mild cognitive impairment (MCI) due to AD and mild AD with confirmed presence of amyloid pathology.1 The proof-of-concept Study 201 explored the impact of treatment with lecanemab on reducing brain amyloid beta (Aβ) and clinical decline. In this study, pre-specified analysis showed consistent reduction of clinical decline across several clinical and biomarker endpoints at the highest doses.

In March 2021 Eisai and Biogen completed enrollment of 1,795 patients with early AD in the the Phase 3 Clarity AD study. The study’s primary endpoint is expected to be completed by the end of September 2022. Additionally, the Phase 3 clinical study, AHEAD 3-45, is currently exploring lecanemab in individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains. Open label extension data from Study 201 confirmed time-dependent reduction of brain Aβ in individuals newly treated with lecanemab and was presented at the 2021 Alzheimer’s Disease and Parkinson’s Disease Conference.

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.