Reasons for Hope

Compared to a decade ago, there are substantially more drugs (a 35% increase) being tested for Alzheimer’s disease in more clinical trials (a 40% increase), targeting a greater number of aspects of the disease, according to a new analysis of Alzheimer’s disease drug development as reflected in clinical trials registered on clinicaltrials.gov.

“The current drug development pipeline provides the basis for optimism regarding the emergence of new therapies for patients with Alzheimer’s. There are a robust number of trials, and agents in trials target a variety of disease processes,” said Dr. Jeffrey Cummings, Joy Chambers-Grundy professor of Brain Science in the Department of Brain Health at the University of Nevada, Las Vegas, and lead author of the paper. “This reflects our improved understanding of the biology of Alzheimer’s and the success of developing disease targeted therapies — starting with the anti-amyloid monoclonal antibodies.

“Alzheimer’s is no longer an untreatable disease. It is now a disease with treatments that successfully interfere in the disease process,” Cummings added. “Progress is also evident in clinical trial design, integration of biomarkers into trials, and emergence of promising candidate therapies. Biomarkers are increasingly used for trial eligibility as well as being integrated as trial outcomes.”

“Alzheimer’s Disease Drug Development Pipeline: 2026” was recently published by Alzheimer’s and Dementia: Translational Research & Clinical Interventions, a journal of the Alzheimer’s Assoc. Cummings and colleagues’ annual review of the Alzheimer’s drug development pipeline began in 2016.

Dr. Jeffrey Cummings

“Alzheimer’s is no longer an untreatable disease. It is now a disease with treatments that successfully interfere in the disease process.”

Looking at the most active areas of drug development revealed that the Alzheimer’s drug pipeline has become significantly more diverse. For example, over the last 10 years, the percentage of the pipeline devoted to:

• Inflammation/immune dysfunction has increased from 6% to approximately 20%.

• Tau targeted agents have increased from 6% to approximately 20%.

• Amyloid targeted agents have decreased from 33% to approximately 20%.

“It is clear that Alzheimer’s is a complex disease with many contributing elements,” Cummings said. “Inflammation is consistently present in the brain of Alzheimer’s patients, and reducing the inflammatory response promises to slow the disease process. Researchers are seeking ways to complement the anti-amyloid therapies, and there are proportionately fewer amyloid drugs in the pipeline.”

The researchers identified 192 clinical trials for Alzheimer’s, assessing 158 drugs. This included 54 trials assessing 36 drugs in phase 3, 89 trials assessing 84 drugs in phase 2, and 49 trials assessing 45 drugs in phase 1. The 192 clinical trials and 158 novel agents in 2026 expand on the 182 clinical trials assessing 138 drugs in the 2025 pipeline.

Disease-targeting therapies (DTTs) account for 73% of agents in trials. Cognition-enhancing symptom-targeted therapies contribute 18%, and drugs targeting neuropsychiatric symptoms comprise 10%.

Furthermore, 2026 may be an exciting and busy year for Alzheimer’s drug news as eight phase 3 trials will reach their
primary completion date, and 29 phase 2 clinical trials will be completed.

Not only are there more drugs and more trials, but a varied and comprehensive array of Alzheimer’s disease processes is being addressed by investigational drugs. The researchers identified 17 aspects of Alzheimer’s impact on the brain that are targeted by at least one drug in current clinical trials.

Repurposed agents approved for non-Alzheimer’s indications include 56 drugs and 73 currently active trials. Repurposing plays an important role in Alzheimer’s drug development, and repurposed agents comprise almost half of phase 2 drugs. The benefits of investigating drugs that are already approved for other indications include significantly reduced development time, lower development costs, higher probability of success, and a well-known safety profile.

Addressing an Unmet Need

“While the current FDA-approved treatments for early Alzheimer’s are a game-changing breakthrough, there is still a great, unmet need for drug development to address the needs of the growing population of individuals with Alzheimer’s in all communities and across all stages of the disease,” said Maria Carrillo, chief science officer and medical affairs lead for the Alzheimer’s Assoc.

For example, according to the Alzheimer’s & Dementia paper, there are no DTTs approved for pre-clinical Alzheimer’s disease or for moderate to severe Alzheimer’s dementia; no new classes of cognition-enhancing agents have been approved since 2004; and there are no approved treatments for symptoms such as Alzheimer’s-related psychosis, depression, or apathy.

Maria Carrillo

“Compounds for people who show early biological signs of Alzheimer’s but have no detectable clinical symptoms are now in clinical trials. If these studies are positive, that could quickly change how the disease is managed.”

The Alzheimer’s Assoc., through its Part the Cloud program, is advancing Alzheimer’s treatments by providing critical funding for early-phase clinical trials that bridge the gap between laboratory research and final-stage human trials. Part the Cloud has funded 83 research projects with more than $90 million to support diverse, high-risk, high-reward approaches aimed at stopping or slowing the disease.

Recently, Part the Cloud announced more than $11 million in new investments, focusing on tauopathy therapeutics, improving synaptic connectivity, and combination therapies. The association funds studies on neuroinflammation, metabolism, and immune response to target the disease from multiple angles.

Alzheimer’s Network for Treatment and Diagnostics (ALZ-NET) is a voluntary, nationwide network sponsored by the Alzheimer’s Assoc. that collects real-world clinical, safety, and imaging data from patients receiving new FDA-approved Alzheimer’s therapies. It aims to improve treatment, monitor long-term outcomes, and enhance care. ALZ-NET is enrolling clinical sites across the country.

Meanwhile, the Alzheimer’s Assoc. is leading a pivotal shift in early detection and treatment of Alzheimer’s disease, from responding to symptoms after they appear to identifying risk of cognitive decline, quick and accurate diagnosis, and much earlier intervention.

“Treatments that slow progression of early Alzheimer’s and offer meaningful benefits have been approved by the FDA and other agencies around the world,” Carrillo said. “At the same time, compounds for people who show early biological signs of Alzheimer’s but have no detectable clinical symptoms are now in clinical trials. If these studies are positive, that could quickly change how the disease is managed.

“Plus, we are at a turning point in what we know about brain health,” she added. “The results from the Alzheimer’s Association U.S. POINTER trial demonstrate with confidence that engaging in a structured, multi-component healthy lifestyle program can protect brain health and improve cognition for many people at risk for dementia in the U.S.”

Continued Investment

The growth of the Alzheimer’s drug development pipeline reflects the impact of sustained federal investment in Alzheimer’s and dementia research at the National Institutes of Health (NIH), which the Alzheimer’s Assoc. has championed alongside bipartisan leaders in Congress.

To continue the scientific momentum reflected in this year’s pipeline, the association is working to secure the NIH director’s professional judgment budget request of a $187.21 million increase for Alzheimer’s and dementia research at NIH in FY 2027.