DoD Amyotrophic Lateral Sclerosis, Therapeutic Development Award

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The FY21 ALSRP 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 include collaborators with substantial experience in the relevant ALS model systems, endpoints, and pathophysiology. 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 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 systems. 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 at least one preclinical model system of ALS, including whole animal and cellular model systems Development or further characterization of validated biomarkers, in parallel to the main therapeutic effort and for use in eventual clinical trials, is a critical component of the F 21 ALSRP Therapeutic Development Award. Efforts may include development/characterization of target engagement biomarkers, pharmacodynamic biomarkers to measure the biological effect of an investigational therapeutic, and/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. Applicants must clearly describe 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 .

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