THERAPEUTICS

Leading the Field Toward The Future of Treatment

Two scientists collaborating in a laboratory

Momentum in drug development continues to advance at a faster speed than ever before. This year, we saw FDA approval of Eli Lilly’s Kisunla, expanding the first class of amyloid-targeting drugs. These offer options for patients in the immediate term while the ADDF continues to lead the development of additional therapeutics based around the biology of aging, which could be combined with amyloid-targeting drugs for greater effect in the future.

In January, the ADDF convened an expert advisory board around combination therapies, offering guidance on how best to design the next generation of biomarker-powered clinical trials to transform the field.

Guiding and Shaping the Science

We are driving research toward the ultimate goal of combination therapies and precision medicine for Alzheimer’s, the same approach which has proven successful for cancer. This year, we supported publication of a special issue of The Journal of Prevention of Alzheimer’s Disease, which featured pioneering research from seven ADDF-funded scientists and offered a roadmap for the next generation of therapeutics.

The ADDF has been seeding the pipeline with a diverse array of novel drugs from day one, shaping the trajectory of the field, and as the science matures, we are seeing the pace of progress increase. Our diverse and robust portfolio is a testament to the ADDF’s leadership.

OUR PIPELINE AT A GLANCE

ACTIVE CLINICAL TRIALS
ACTIVE CLINICAL TRIALS BY
Drug Target
26
%
Inflammation
15
%
Mitochondria 
and Metabolic Function
15
%
Neuroprotection
15
%
Synaptic Activity and Neurotransmitters
11
%
Other
9
%
Genetics & Epigenetics
9
%
Misfolded Proteins
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ACTIVE CLINICAL TRIALS BY
Phase
Phase 1
20
%
Phase 2
68
%
Phase 3
12
%

Driving Innovation Through Venture Philanthropy

Our unique venture philanthropy model re-invests returns from successful investments into additional research, allowing us to keep our funds and focus aimed at advancing the next great idea. The ADDF’s returns in 2024 totaled more than $3 million and this will only continue to grow. This model means we can assume risks others cannot, supporting unique research approaches that have great potential but are in danger of going overlooked or underfunded. A sampling of these innovative projects is below.

Fred Grossman, D.O., FAPA
Fred Grossman, D.O., FAPA

Advancing a New Combination Therapy Approach

With the ADDF’s support, Coya Therapeutics is running a phase 2 clinical trial of their novel combination therapy, which targets inflammation and abnormal immune function to treat frontotemporal degeneration (FTD), a rare form of dementia most common in people under 60. Clinical trials for FTD can also unlock insights into Alzheimer’s, and combination therapy approaches are especially promising because they allow us to target multiple causes of the disease at once.

Tony Giordano, PhD
Tony Giordano, PhD

Addressing a Promising Target: Inflammation

ADDF-funded Neurotherapia plans to conduct a phase 2a trial of their novel drug, which aims to protect neurons from the damaging effects of inflammation caused by over-activation of microglia, immune cells in the brain, thereby improving cognition. Recent research has deepened our understanding of inflammation and established it as a particularly promising target for treating Alzheimer’s disease.

Paul Edison, MBBS, PhD, FRCPI
Paul Edison, MBBS, PhD, FRCPI

Using GLP-1s to Fight Alzheimer’s

Results presented at the 2024 Alzheimer’s Association International Conference from a phase 2b study on liraglutide, a GLP-1 drug also known as Victoza and Saxenda, found the drug promoted neuroprotection and slowed cognitive decline by 18% more than placebo. GLP-1 is a hormone that regulates blood sugar, appetite, and digestion. Drugs that mimic the effects of GLP-1 have recently been popularized for weight loss. This study, supported by the ADDF and initially presented in 2020, adds to the growing body of evidence that GLP-1 drugs are a promising pathway for the treatment and prevention of Alzheimer’s.