The Drug Development RFP seeks to support in vivo preclinical studies that advance lead molecules developed for Alzheimer’s disease and related dementias to IND-enabling studies. The proposed studies should be structured to deliver a compound with strong potential for clinical and commercial application.
This funding opportunity prioritizes novel drug mechanisms and modes of action related to the biology of aging and other emerging therapeutic areas for dementia. For this reason, amyloid targeted approaches and cholinesterase inhibitor proposals will not be considered for this RFP.
Stage of discovery:
– Preclinical pharmacokinetics (PK) and pharmacodynamics (PD) (primarily in wild-type animals to inform dose selection for in vivo efficacy studies), as well as preliminary rodent tolerability studies.
– In vivo efficacy or proof-of-concept studies in animal models of disease or aging, with a focus on measures of direct and indirect markers of target engagement and downstream pharmacologic effects.
Please note: Applications that focus on basic research (including target discovery), assay development, and screening to identify hit compounds are not a priority for this RFP and will be withdrawn.
Please note: IND-enabling work and clinical trials are supported through the Program to Accelerate Clinical Trials (PACT).
Therapeutic modalities: Includes small molecules, biologics, gene therapies, antisense oligonucleotides, and stem cells. Development of novel devices or delivery systems will not be considered for this RFP.
Drug mechanisms or modes of action: Novel drug mechanisms and modes of action related to the biology of aging and other emerging therapeutic areas for dementia are considered high priority. These include, but are not limited to:
– Mitochondrial & metabolic function
– Synaptic activity and neurotransmitters
– Vascular function
– Other mechanisms and modes of action related to the biology of aging (e.g. senescent cells)
– Other novel mechanisms or modes of action that are supported by compelling evidence demonstrating a rational biological connection to the disease process
– Please note: Anti-amyloid approaches (e.g., anti-amyloid aggregation, beta-amyloid vaccines, beta- or gamma-secretase inhibitors) and cholinesterase inhibitor proposals will not be considered.