The FY25 ALSRP Therapeutic Development Award supports research ranging from preclinical validation of therapeutic leads through FDA IND-enabling studies. The proposed studies are expected to be empirical in nature and product driven. Applicants with limited ALS experience are strongly encouraged to include collaborators with substantial experience in the relevant ALS model systems, endpoints, and pathophysiology. Candidate therapeutics that already have been granted an IND are not appropriate for the TDA.
Examples of activities that will be supported by this award include.
• Lead optimization including confirmation of candidate therapeutics obtained from screening or by other means, including optimization of potency and pharmacological properties and testing of derivatives and sister compounds.
• Validation of preliminary efficacy findings, building on initial discoveries from pilot studies (e.g., ALSRP Therapeutic Idea Awards) through expansion on assessed ALS model systems, extended dose-response characterization, or extended timepoints to more robustly establish therapeutic potential.
• IND-enabling studies to include: compound characterization; absorption, distribution, metabolism, and excretion studies; studies on formulation and stability leading to Good Manufacturing Practice production methods; dose/response and toxicology studies in relevant model systems.
• Confirmation of candidate therapeutics obtained from screening or by other means, including optimization of potency and pharmacological properties and testing of derivatives and sister compounds.
• Applications supported by this award must begin with lead compounds in hand and must include preliminary data relevant to the phase of development, such as:
o Proof of identity and purity.
o Selectivity for the intended target over closely related targets.
o Availability of primary and secondary in vitro bioactivity assays for optimization or structure–activity relationship studies.
o Availability of clear efficacy data in at least one appropriate preclinical ALS model, with adequate power and methods.
Deadlines:
• Required Pre-Application Deadline: June 6, 2025
• Application Submission Deadline: Aug. 27, 2025
Independent investigators at all career levels may be named by their organization as the Principal Investigator (PI) on the application.
For titles outside of academia that may not be analogous to traditional hierarchies, investigators at or above an independent scientist level may be named by their organization as the PI on the application.
Individuals affiliated with an eligible organization are eligible to be named as PI regardless of ethnicity, nationality, or citizenship status.
The Congressionally Directed Medical Research Programs (CDMRP) expects to allot approximately $9.8 million (M) to fund approximately four Therapeutic Development Award applications with direct cost caps of $1.5M. The maximum period of performance is three years. It is anticipated that awards made from this FY25 funding opportunity will be funded with FY25 funds, which will expire for use on September 30, 2031. Awards supported with FY25 funds will be made no later than September 30, 2026.