KEY TAKEWAYS
- The Alzheimer’s drug pipeline is rapidly evolving and gaining momentum with a growing, diverse set of 138+ drugs targeting multiple pathways.
- Key trends include real-world validation of anti-amyloid drugs, advanced biomarker use for earlier diagnosis, and new approaches such as dual-target vaccines, cell therapies, and novel antibodies with better brain delivery.
What is the current state of the drug development pipeline in Alzheimer's disease?
As of 2025, the Alzheimer's disease (AD) drug pipeline is characterized by significant growth and a broadening of therapeutic targets. There are currently 138 unique drugs being assessed in 182 clinical trials, representing a record level of activity and a notable increase from 2024.
The pipeline is divided across three clinical stages, with a marked nearly 100% increase in early-stage (Phase 1) activity since 2024.
- Phase 3: 31 drugs in 48 trials (focusing on large-scale efficacy).
- Phase 2: 75 drugs in 86 trials (focusing on proof-of-concept and safety).
- Phase 1: 45 drugs in 48 trials (initial safety and dosing).
Key Therapeutic Categories
The pipeline has shifted away from a singular focus on amyloid to a more diverse set of 15 different disease processes.
- Disease-Targeted Therapies (DTTs): These comprise 74% of all pipeline drugs. They aim to slow or halt the disease rather than just manage symptoms.
- Amyloid and Tau: Traditional targets remain prominent, with amyloid-related therapies making up 18% and tau-related therapies making up 11% of the pipeline.
- Symptomatic Agents: Drugs targeting cognitive enhancement (14%) and neuropsychiatric symptoms like agitation or psychosis (11%) remain vital components.
- Repurposed Drugs: Approximately 33% of drugs in the pipeline are already approved for other conditions (e.g., cancer or diabetes) and are being tested for AD.
Major Drugs and Recent Developments
Recent regulatory milestones and late-stage results have shaped the 2025 landscape.
- Recent Approvals: Lecanemab (Leqemb®i) and Donanemab (Kisunla) have received traditional FDA approval to slow cognitive decline in early-stage AD. A subcutaneous version (self-injectable) of lecanemab (Lequembi IQLIK™) was approved by the FDA in August 2025. Several Phase 3 trials are expected to be completed in the coming months.
- Emerging Mechanisms: New trials are investigating innovative targets such as the gut-brain axis, circadian rhythm disturbances, and vascular health.
- Next-Generation Delivery: "Brain shuttle" technologies that use specially engineered molecules to are being tested to help drugs penetrate the blood-brain barrier more effectively with fewer side effects.
The Role of Biomarkers
Biomarkers have become central to drug development; 57% of current clinical trials use them for participant eligibility, and 27% use them as primary outcome measures. This includes a significant move toward plasma-based (blood) tests like p-tau217 for easier.
Learn More:
Annual Alzheimer’s Disease Drug Development Pipeline Report – this annual report has been running since 2016 and is led by Dr. Jeffrey Cummings a neurologist and research professor at the Department of Brain Health – Kirk Kerkorian School of Medicine. University of Nevada. The most recent report is:
- Cummings JL, Zhou Y, Lee G, et al. Alzheimer's disease drug development pipeline: 2025. Alzheimer’s Dement. 2025;11:e70098. https://doi.org/10.1002/trc2.70098
National Institute on Aging. 2025 NIH Alzheimer's Disease and Related Dementias Research Progress Report: Advances and Achievements.
TREAT-AD Drug Discovery Center – Indiana University School of Medicine/Emory-Sage-Structural Genomics Consortium – Jackson Laboratory. Target Portfolio and Progress Dashboard.
NEWDIGS Issue Briefs
This Issue Brief is part of a series of reports from the NEWDIGS Consortium on strategy to expand patient access to the new generation of disease modifying therapies (DMTs) for early Alzheimer’s disease.
The NEWDIGS project on Alzheimer’s Disease (AD) is organized around a hypothesis that ensuring safe, effective, and equitable patient access to DMTs for AD will require a shift toward a more primary care-centered model of care including detection, diagnosis, treatment, and monitoring.
AD is the first case study in the Biomedical Health Efficiency (BHE) Project of NEWDIGS, launched in 2026. BHE is focused on re-engineering life science innovation to streamline access for all patients to biomedical products in ways that optimize outcomes while minimizing the use of resources.
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October 11, 2023About the Center for Biomedical System Design
The NEWDIGS Consortium is dedicated to improving health by accelerating appropriate, timely, and equitable patient access to biomedical products in ways that work for all stakeholders.
Based at the Center for Biomedical System Design at Tufts Medical Center in Boston, NEWDIGS aims to help the health care system catch up with the science of biomedical innovation by removing barriers and designing methods to ensure that cutting-edge treatment is made available to patients. The consortium’s collaborators include patients, clinicians, payers, biopharmaceutical companies, regulators, and investors, among others.
Launched at MIT in 2009, the organization moved to Tufts Medical Center in 2022 to be closer to patient care and to longstanding collaborators. Among its successes are payment innovations for durable cell and gene therapies, and regulatory innovations that inspired a European-wide pilot led by the European Medicines Agency focused on Adaptive (Licensing) Pathways.
Its current work integrates insights from all prior projects to advance “Biomedical Health Efficiency” - a new system innovation methodology focused on optimizing outcomes with fewer resources for all patients through improved alignment of stakeholder goals, strategies, incentives, and metrics.