THERAPEUTICS

Advancing Treatments Through Our Signature Focus On Diverse Targets

a woman in a lab coat looking through a microscope

Using a 360-Degree View to Seek New Answers

This year, we have seen significant progress in the field, with breakthroughs for amyloid-targeting drugs like Leqembi and donanemab. At the ADDF, our focus is advancing novel drugs that can be used in combination with anti-amyloids in a precision medicine approach – which has proven successful in cancer treatment – to address the unique causes of each patient's disease.

We believe every possible cause of Alzheimer’s is worthy of research because each aging-related target presents another potential pathway to a cure. As a result, our portfolio is one of the largest and most diverse in the world.

OUR PIPELINE AT A GLANCE

ACTIVE CLINICAL TRIALS
Colorful ring pie chart showing the distribution of active clinical trials segmented by drug target categories
ACTIVE CLINICAL TRIALS BY
Drug Target
18%
Inflammation
18%
Synaptic Activity & Neurotransmitters
16%
Mitochondria & Metabolic Function
16%
Other
9%
Misfolded Proteins
5%
Biomarkers
5%
Cellular Senescence
5%
Genetics & Epigenetics
5%
Neuroprotection
3%
Proteostasis
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Colorful ring pie chart illustrating the proportions of active clinical trials in different phases
ACTIVE CLINICAL TRIALS BY
Phase
Phase 0
3%
Phase 1
21%
Phase 2
71%
Phase 3
5%

Innovative Ideas + Strategic Investments = Impact

Our venture philanthropy financing model funnels profits from successful investments back into supporting additional research. This enables us to assume risks others cannot and support unique research approaches that have great potential but are in danger of going overlooked or underfunded. The influence of our approach is reflected in today’s clinical trials pipeline, where three quarters of trials are aimed at targets other than amyloid and tau.

“The ADDF has developed a global presence. They are not just about making investments, but also shaping the field.”
Jeffrey Cummings, MD, ScD, Director of the Chambers-Grundy Center for Transformative Neuroscience at the University of Nevada, Las Vegas

A Promising New Target: “Zombie” Cells

Headshot of Mirando Orr wearing glasses and a pink jacket
Miranda Orr, PhD

A Promising New Target: “Zombie” Cells

Miranda Orr, PhD, Assistant Professor of Gerontology and Geriatric Medicine at the Wake Forest University School of Medicine and winner of the 2022 Goodes Prize, has identified toxic senescent “zombie” cells in the brain that contribute to the neurodegenerative decline associated with Alzheimer’s disease. They develop mechanisms to avoid the natural death cycle that should come to all cells, and instead live on, causing brain inflammation and releasing damaging toxins. Dr. Orr’s work was featured in National Geographic earlier this year and is now in a phase 2 clinical trial with support from the ADDF.

Early ADDF Support Leads to Large Grants for Developing Drugs

Headshot of Lisa Ricciardi smiling
Lisa Ricciardi

Early ADDF Support Leads to Large Grants for Developing Drugs

In February 2023, Cognition Therapeutics, a biotech the ADDF has supported for nearly 15 years, reached a significant milestone in the development of their oral Alzheimer’s treatment, CT1812. Cognition was granted $81 million from the NIA and cleared by the FDA to conduct a large, multi-site phase 2 trial which has now completed enrollment. CT1812 uses a novel mechanism to prevent amyloid-caused damage to neurons and synapses, a primary driver of Alzheimer’s disease. We are particularly excited about the potential of CT1812 because it is a small molecule, meaning it comes in tablet form, which is more accessible for patients and less invasive and expensive than other anti-amyloid treatments coming to the market.

Novel Drug Shows Promise

Headshot of Jeffrey Stavenhagen in a suit and glasses smiling
Jeffrey Stavenhagen, PhD

Novel Drug Shows Promise

With the ADDF’s support, Therini Bio is developing a drug to block fibrin, a blood-clotting factor that has been implicated in the toxic inflammation that damages neurons during Alzheimer’s disease. Therini is designing its drug not to affect fibrin’s blood clotting function, which is essential for normal blood clotting after an injury, and is instead targeting the way fibrin can abnormally seep into the brain because of blood vessel changes that occur in Alzheimer’s. In May 2023, the company initiated its phase 1 clinical trial for the promising new therapeutic.