DoD Amyotrophic Lateral Sclerosis Research, Therapeutic Development Award

Description

The Therapeutic Development Award supports research ranging from validation of therapeutic leads through U.S. Food and Drug Administration (FDA) Investigational New Drug (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 collaborate with those having substantial expertise in ALS research and/or ALS model systems. Examples of activities that will be supported by this award include: 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 early pilot studies, including the use of multiple ALS model systems and/or replicating preliminary data with more time points or additional doses Studies on formulation and stability leading to Good Manufacturing Practice (GMP) production methods IND-enabling studies, to include compound characterization, absorption, distribution, metabolism, and excretion (ADME) studies, and dose/response and toxicology studies in relevant model system Validation of treatment approaches in appropriately powered and controlled studies usinbiological correlates of disease activity and progression in pre-existing, de-identified human specimens from well-characterized patient cohorts is encouraged. Examples of acceptable cohorts for study include controlled clinical trials, observational studies, and registries (e.g., Centers for disease Control and Prevention [CDC] National ALS Registry and/or Biorepository; https://wwwn.cdc.gov/als/). Active duty military and/or Veteran patient populations or resources should be considered. All specimens must exist at the time of application submission; collection of new specimens will not be supported. 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: Proof of identity and purity Selectivity for the intended target over closely related targets Availability of primary and secondary in vitro bioactivity assays for optimization or structure-activity relationship studies Availability of proof-of-concept efficacy data in appropriate preclinical model systems of ALS, including whole animal and cellular model systems, or informative clinical data from a related human disease Investigators interested in basic research focused on ALS drug discovery are encouraged to apply for the FY19 ALSRP Therapeutic Idea Award (Funding Opportunity Number: W81XWH- 19-ALSRP-TIA), which does not require preliminary data (https://cdmrp.army.mil/funding/alsrp).Therapeutically Relevant Marker Option: The FY19 ALSRP Therapeutic Development Award Therapeutically Relevant Marker Option encourages applicants to develop a specific marker(s) in parallel with the advanced therapeutic development effort. Additional funding, as described in Section II.D.5, Funding Restrictions, is being offered for the co-development of markers that will enhance the drug development process. Efforts may include development of objective pharmacodynamic biomarkers to measure the biological effect of an investigational therapeutic or predictive/cohort-selective biomarkers that indicate whether a specific therapy will be effective in an individual patient or patient subgroup. Development of markers for the purposes of diagnosis, prognosis, or measurement of disease progression without consideration of the therapeutic development process will not be supported. To apply for this option, applicants must submit a Therapeutically Relevant Marker Statement (see Attachment 11) that clearly describes the marker and its potential to improve the efficacy of the therapeutic development process. For further description, see Section II.D.2.b.ii, Full Application Submission Components. Before preparing a Therapeutically Relevant Marker Statement, applicants are encouraged to review the draft guidance issued by the FDA, “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment,” as well as the patient-focused document developed by the ALS community, “Drug Development for Amyotrophic Lateral Sclerosis Guidance for Industry,” which provides direction beyond the FDA guidance and includes the community consensus on key therapeutic development topics. The anticipated direct costs budgeted for the entire period of performance for an FY19 ALSRP Therapeutic Development Award will not exceed $1,000,000. The anticipated direct costs budgeted for the entire period of performance for an FY19 ALSRP Therapeutic Development Award with the Therapeutically Relevant Marker Option will not exceed $1,250,000. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.

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