Prevention

Must be received by 5:00 pm ET on the deadline date.

Funding is open to researchers and clinicians worldwide at:

• Academic medical centers and universities or nonprofits. Industry partnerships are encouraged.
• Biotechnology companies. Existing companies and new startups are both eligible.
• NOTE: Funding is provided through mission-related investments that require return on investment based upon scientific and/or business milestones (see Our Research Strategy for more information).

Award Amount
- Up to $5,000,000 for clinical trials based on stage and scope of research
- For studies requiring additional support, co-funding from other funding agencies or investors is encouraged
- Payment structure will be negotiated and based on milestone achievements and recruitment

Average Duration
Multi-year

Allowable costs
Only direct costs are allowed. Please review our Funding Policies

For clinical trial applications, the following guidance may assist you in developing a strong application that allows reviewers to better evaluate the science and merit of your proposal. The strongest proposals will contain many or all of the aspects listed below, although we recognize that the field is still in need of a better understanding of underlying biological mechanisms and validated biomarkers.

Please note: For clinical trial applications, ADDF prioritizes novel drug candidates with composition of matter intellectual property (IP) and repurposed or repositioned drugs with strategies to develop novel IP.

Mechanisms or modes of action: Applicants are encouraged to provide a clear rationale and compelling evidence for targeting the proposed mechanism or mode of action in the disease or condition and should specifically address these questions in the proposal:

• Is the mechanism or mode of action novel? How is the target biology more compelling than other related targets that have been tested for the disease?
• Is there human genetic evidence linking the target biology to the disease?
• Is the target expressed in disease-relevant regions of the brain (or where applicable, in the periphery) in humans and/or animal models?
• Are there changes in target mRNA/protein expression or activity in human disease specimens, and do they correlate with disease severity and cognitive functions?
• Does genetic and/or pharmacological manipulation of the target in disease-relevant in vitro (e.g., primary cultured neurons/glia or cells derived from patient iPSCs) or in vivo models alter disease phenotypes?
• If the molecular target is unknown, the strength of the evidence for the mode of action and its link to disease pathophysiology will be evaluated. The applicant should summarize the existing evidence in the proposal

Preliminary data: Applicants are encouraged to include data around many or all of the following:

• Supportive preclinical efficacy data in relevant animal models
• Evidence of blood-brain barrier penetration (for CNS targeted therapies)
• Preclinical and, if available, clinical PK/PD data on dose optimization for the intended route of administration and dosing regimen
• Preclinical, and if available, clinical safety data. Proposals should include plans to address remaining safety concerns if any are identified in earlier studies

Clinical population: Proposals including patients should provide justification for the selected clinical population and how enrollment criteria such as clinical subtype, stage of severity, known genetics (e.g. ApoE status), and neuropathology (e.g. amyloid positivity) relate to the proposed mode of action. Applicants must provide information about recruitment of the target population to demonstrate that a sufficient number of patients are available to meet recruitment goals.

Expectations for biomarker-based proof-of-concept studies:

• Provide justification for the dose(s) selected
• Design studies to answer specific questions about a therapy’s activity, including whether an intervention is safe, engages its target, induces expected downstream pharmacological effects, and leads to changes on disease-related measures
• Include biomarker outcomes that align with the proposed mechanism or mode of action and, where possible, are predictive of clinical efficacy
• Include clinical assessments that align with the appropriate domains of the clinical syndrome, stage of disease, and mechanism of action. Although these studies may not be powered to detect differences in clinical outcomes, the inclusion of cognitive and neuropsychological endpoints may be informative as exploratory outcomes. Expected directional changes that correlate with biomarker changes may be observed and can inform planning for subsequent studies
• Engage a biostatistician early in the development of the study design. Statisticians can help to determine the appropriate study design options and sample size calculations for these smaller biomarker-based studies, particularly when novel biomarkers without established effect sizes are used

Investigative team: Clinical trials often require resources beyond those available at a single organization and collaboration with other investigators and contract research organizations and consultants are encouraged.

Please note: All clinical trials receiving ADDF funding must register and submit results for “applicable clinical trials” on the ClinicalTrials.gov Protocol Registration and Results System Information website.