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> Insights > Comprehensive Analysis of the Alzheimer's Disease Drug Development Pipeline: 2025 Landscape 
Alzheimer’s disease (AD) poses a significant global public health challenge. While several monoclonal antibody therapies targeting beta-amyloid (Aβ) have gained approval, there is still an urgent and unmet demand for more effective and precise therapeutic approaches. The recent comprehensive review “Alzheimer’s Disease Drug Development Pipeline: 2025” by Professor Jeffrey Cummings and his colleagues provides valuable insights into the evolving AD drug development. Our analysis distills their findings through three critical dimensions: (1) pipeline expansion, (2) target diversification, and (3) technological innovation — providing a strategic overview of the latest trends and advancements in AD research and development.
As of January 1, 2025, 138 AD candidate drugs are being evaluated across 182 clinical trials worldwide—representing a significant increase from the prior-year period (127 drugs, 164 trials). In terms of development stages, the pipeline currently includes: 48 Phase III (31 drugs), 86 Phase II (75 drugs), and 48 Phase I (45 drugs). Notably, the number of Phase I trials has increased by 85% compared to 2024 (from 26 to 48), reflecting the industry’s growing focus on novel mechanisms of action.
Regarding treatment strategies, disease-targeting therapies (DTTs) continue to dominate the pipeline, accounting for 74% of the pipeline (60 small-molecules and 42 biologics). Additionally, 14% of therapies (19 in total) aim to enhance cognitive function by modulating the cholinergic system or glutamate receptors to improve memory decline. Another 11% (15 drugs total) target neuropsychiatric symptoms (NPS) such as agitation, psychosis, and apathy.
As of January 1, 2025, drugs in clinical trials for AD treatment registered on clinicaltrials.gov
The Common Alzheimer's Disease Research Ontology (CADRO) classifies drug targets and mechanisms into 15 AD-related pathological categories. At least one drug is currently in development for each category, indicating a shift in AD drug development toward multi-mechanistic and multi-dimensional approaches.
Classic pathological mechanisms such as Aβ deposition and Tau hyperphosphorylation still hold a central position in AD drug research, with 18% and 11% of current investigational drugs targeting these two core pathologies, respectively. Emerging mechanisms, including neurotransmitter receptors (22%), neuroinflammation/immune response (17%), synaptic plasticity/neuroprotection (6%), energy metabolism (6%), growth factor, lipid metabolism, protein homeostasis, vascular function, and circadian rhythm, are gradually becoming hotspots in AD research. More cutting-edge directions, such as drugs targeting the gut-brain axis and epigenetic regulation, further reflect the deepening exploration of AD’s complex etiology.
Number of AD drug candidates by mechanism of action across different stages of development
Compared with traditional AD clinical trial designs that primarily rely on symptom scores as the main endpoints, current AD drug clinical research has seen significant advancements in the application of biomarkers. Among 182 AD clinical trials, 57% incorporated biomarkers as inclusion or exclusion criteria, while 27% used them as primary endpoints. Magnetic Resonance Imaging (MRI) and Aβ positron emission tomography (PET) remain the most widely used imaging biomarkers. Concurrently, the adoption of fluid biomarkers is rising, with highly sensitive markers such as p-tau217 and the Aβ42/40 ratio being adopted in some trials for screening and efficacy evaluation.
In addition, the exploration of combination therapies is increasingly active. Currently, 20 clinical trials are evaluating various combination strategies, including synergistic pharmacodynamic combinations—such as dasatinib and quercetin, which target neuroinflammation in the treatment of AD—and pharmacokinetically optimized combinations, such as dextromethorphan combined with a CYP2D6 inhibitor to enhances its efficacy by slowing down the hepatic metabolism.
The AD drug development pipeline is characterized by a trend of expanding scale, target diversification, and technological innovation. Research has broadened from the classical Aβ and Tau-targeting approaches to multiple parallel therapeutic directions, including inflammation, metabolism, and neurotransmission. The increasing use of biomarkers and the exploration of combination therapy strategies are also driving clinical research toward greater precision and efficiency. The diversification of therapeutic targets and technologies marks an exciting new era in AD drug development, paving the way for more personalized and effective patient care.
As the brand focused on neuroscience of ACROBiosystems, Aneuro offers cutting-edge solutions, including target proteins, pre-formed fibrils (PFFs), brain organoids, and p-tau antibodies, to accelerate AD research and drug development.
Cummings J L, Zhou Y, Lee G, et al. Alzheimer's disease drug development pipeline: 2025[J]. Alzheimer's & Dementia: Translational Research & Clinical Interventions, 2025, 11(2): e70098. doi: 10.1002/trc2.70098
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