Alzheimer's foundation statement on FDA panel's support for donanemab

NEW YORK, June 10, 2024. The FDA Peripheral and Central Nervous System Drugs Advisory Committee has unanimously voted that donanemab demonstrates clinical benefit for treating early Alzheimer’s disease. Should donanemab receive approval, it will become the second disease-modifying drug approved for Alzheimer's, offering new possibilities for combination therapies.

Dr. Howard Fillit, Co-Founder and Chief Science Officer of the Alzheimer's Drug Discovery Foundation (ADDF), remarked that the vote is promising for donanemab’s imminent approval. He emphasized that this development should be viewed within the broader context of Alzheimer’s treatment which increasingly involves combination therapies and precision medicine. According to Dr. Fillit, donanemab would contribute to the first class of disease-modifying drugs, laying the groundwork for future treatments. While anti-amyloids are not a cure-all, they provide an opportunity to alter the disease’s progression as new therapies are developed targeting the underlying biology.

The donanemab trials highlight the field's success in conducting innovative trials using biomarkers to determine a drug's efficacy. The TRAILBLAZER-ALZ 2 trial employed a "goldilocks strategy," using amyloid and tau imaging to identify patients in the early stages of Alzheimer’s who would benefit most from the treatment.

Dr. Fillit noted that some patients achieved full amyloid clearance with donanemab and did not experience significant plaque buildup for nearly four years. This success underscores the value of biomarker tests, which can detect, quantify, and monitor plaque buildup in the brain. As Alzheimer's research progresses, biomarkers will continue to play a crucial role in clinical trial design, guiding the development of drugs targeting novel pathways based on the biology of aging.

This achievement is timely, coinciding with a shift in drug development driven by the biology of aging. Currently, about 75% of Alzheimer’s drugs under development target novel pathways related to aging, such as inflammation, metabolic disturbances, and vascular dysfunctions. Notable contributions come from ADDF-funded companies like Coya Therapeutics, Therini Bio, and PharmatrophiX. Each is developing new therapies that address various aging-related mechanisms contributing to Alzheimer’s.

The diversity in the drug pipeline, alongside advancements in biomarkers—particularly the development of accessible blood-based biomarkers—marks a new era in Alzheimer’s care. This progress enables early detection and intervention, making it possible to identify patients in the preclinical phase and reduce their risk based on individual biomarker profiles.

Dr. Fillit concluded that the advisory committee’s endorsement is a significant milestone for researchers, patients, families, and caregivers who have long worked to advance new treatment options. However, he stressed that the journey is not over. Ongoing innovation and progress are essential to eventually treating Alzheimer’s on an individual basis through precision medicine, aiming to halt disease progression or prevent its onset entirely.

The Alzheimer's Drug Discovery Foundation (ADDF), founded in 1998 by Leonard A. and Ronald S. Lauder, is dedicated to accelerating the discovery of drugs to prevent, treat, and cure Alzheimer’s disease. The ADDF is unique in being the only public charity focused exclusively on funding Alzheimer’s drug development, using a venture philanthropy model to support both academic and biotech research. The ADDF has played a crucial role in bringing the first Alzheimer's PET scan (Amyvid) and blood test (PrecivityAD) to market and has contributed significantly to the current diverse drug pipeline. With the support of its donors, the ADDF has awarded more than $290 million to fund over 750 Alzheimer’s drug discovery programs, biomarker programs, and clinical trials across 20 countries.