Full proposal (MAX 7 slides - do NOT include confidential chemical structures or Ab sequences)

  • Rationale
    • Outline the target and drug mechanism in disease
    • Rationale and supportive preliminary data In vitro and In vivo(with either tool compounds or genetic). Please detail translatability of the proposed mechanism to human/limitation of your model(s)
    • Modality of the proposed drug (small molecule, Ab, ASO, …)
    • Human translational data to support your hypothesis/de-risk your mechanism
  • Indication and Target Product Profile (TPP)
    • Describe the patient population, treatment line and envisaged differentiation from standard of care in the intended treatment setting (differentiation could be in terms of efficacy, tolerability, dosing, route of administration)
    • Sketch the essential development path forward with related high-level costs. Please focus on the faster way to test your concept clinically providing PoC for the final indication.
    • Biomarker(s)
    • Describe key assay cascade to evaluate development candidates
    • Outline biomarkers to assess target engagement in preclinical and early clinical
    • Key Developments
    • Do you have tool compounds? Assays?
    • If asset not available: what proprietary data/information do you have to prosecute the target? (Link key paper if helpful)
  • Next steps:
    • What’s your request in terms of resources to prosecute your hypothesis?
    • What key data you could generate with €2-€5M investment? In what these data would strengthen your TPP?
    • What are the current limitations of your project?

Please send your pitch slides to Ann Chahroudi at ann.m.chahroudi@emory.edu

More info: http://pedsresearch.org/uploads/pages/doc/Medicxi-meets-template-EMORY-1.pptx