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ACU401 is an investigational bispecific antibody being developed by Acumen Pharmaceuticals in collaboration with JCR Pharmaceuticals for the treatment of Alzheimer's disease, specifically early symptomatic Alzheimer's disease. Developed under Acumen's Enhanced Brain Delivery (EBD) program, ACU401 utilizes JCR's proprietary J-Brain Cargo blood-brain barrier (BBB)-penetrating technology. It is a bispecific antibody that combines a novel next-generation amyloid beta oligomer (AβO)-selective antibody (ACU234) with anti-transferrin receptor (TfR) antibody fragments. This design enables receptor-mediated transcytosis across the BBB, targeting soluble amyloid beta oligomers in the brain. In preclinical non-human primate studies, ACU401 demonstrated up to 40-fold greater frontal cortex exposure compared to the native anti-AβO antibody, with a favorable safety profile and no hematological signals suggestive of anemia. IND-enabling activities are ongoing, with an IND submission targeted for mid-2027.
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