Acumen’s Anti-Amyloid Beta Oligomer Antibody Granted FDA Fast Track Designation for Alzheimer's Disease

Acumen Pharmaceuticals, Inc. recently announced ACU193, the first clinical-stage monoclonal antibody that selectively targets toxic soluble amyloid beta oligomers (AβOs), has been granted Fast Track designation for the treatment of early Alzheimer’s disease by the U.S. Food and Drug Administration (FDA). ACU193 is currently being studied in the Phase 1 INTERCEPT-AD trial designed to assess safety and proof of mechanism of ACU193.

ACU193 is a humanized monoclonal antibody discovered and developed based on its selectivity for soluble AβOs, which scientific evidence shows are the most toxic and pathogenic form of Aβ, relative to Aβ monomers and amyloid plaques. Some types of toxic soluble AβOs have been found to interact within synapses which leads to altered neuronal function, and can initiate and perpetuate the process of neurodegeneration, ultimately leading to cell death. ACU193 binds AβOs that bind to synapses, and due to its unique binding profile, ACU193 has potential to provide therapeutic benefit with low risk of amyloid-related imaging abnormalities (ARIA), because ACU193 blocks the toxic effects of AβOs without directly targeting amyloid plaques.

“We are encouraged to receive Fast Track designation for ACU193, reflecting its potential clinical utility to treat Alzheimer’s disease,” said Daniel O’Connell, President and Chief Executive Officer of Acumen. “We look forward to collaborating with the FDA to advance the development of ACU193. Treating Alzheimer’s disease ultimately requires therapies that target different components of the disease pathway, and we are developing ACU193 with the goal of providing patients with more treatment options.”

The FDA Fast Track program is designed to facilitate the development and expedite the review of new drugs intended to treat serious or life-threatening conditions with the potential to fill an unmet medical need. The Fast Track designation allows Acumen to have more frequent engagement with the FDA to discuss development plans and clinical study design for ACU193 to ensure collection of appropriate data to support evaluation for approval.

ACU193 is a humanized monoclonal antibody (mAb) discovered and developed based on its selectivity for soluble AβOs, which Acumen believes are the most toxic and pathogenic form of Aβ, relative to Aβ monomers and amyloid plaques. Soluble AβOs have been observed to be potent neurotoxins that bind to neurons, inhibit synaptic function and induce neurodegeneration. By selectively targeting toxic soluble AβOs, ACU193 aims to directly address what a growing body of evidence indicates is a primary underlying cause of the neurodegenerative process in AD.

Approximately 62 individuals with early AD (mild cognitive impairment or mild dementia due to AD) are expected to be randomized into this double-blind, placebo-controlled, first-in-human study of ACU193. INTERCEPT-AD is designed to establish safety and proof of mechanism. It consists of single-ascending-dose (SAD) and multiple-ascending-dose (MAD) cohorts and is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and target engagement of intravenous doses of ACU193. The study is enrolling at multiple investigative sites located in the United States. More information can be found on www.clinicaltrials.gov, NCT identifier NCT04931459.

Acumen, headquartered in Charlottesville, VA, with clinical operations based in Carmel, IN, is a clinical-stage biopharmaceutical company developing a novel disease-modifying approach to treat Alzheimer’s disease. Acumen’s scientific founders pioneered research on AβOs, which a growing body of evidence indicates are primary triggers of Alzheimer’s disease pathology. Acumen is currently focused on advancing its investigational product candidate, ACU193, a humanized monoclonal antibody that selectively targets toxic soluble AβOs in INTERCEPT-AD, a Phase 1 clinical trial involving early Alzheimer’s disease patients. For more information, visit www.acumenpharm.com.