To see TIPS and FAQS for each slide, please look at this template in Notes view.
Two Year Goal Statement
Your two year goal will frame the following assessment.
Brief background for team members and their roles on this project.Include relevant past experience, if any.
Briefly, introduce background science.What is the problem worth solving?What is your solution? Why is it novel?
Scientific Background and Opportunity
Describe additional research data needed but which is outside the scope and capability of the investigator’s laboratory.
Describe your target and biological pathwayDoes biochemical assay exist?Doesa cell-based readout exist?What additional assays (screening cascade) will you use to hone in on the best molecules?Whatis your level of experience with the assays?Areall critical reagents for the assays currently available?
Assay Feasibility Assessment
Target location(s): extracellular, intracellular, CNS, etc. and tissue type.Canthe protein be reliably expressed and purified? How?Doescrystal structure of target exist, resolution and RCSB code. If no, what are the closest homologs with crystal structure coordinates?Predictedbinding pocket affinity of target (YCMD determined); i.e., is the target druggable?Aresmall molecule inhibitors known?Syntheticaccessibility (conducted after HTS analysis)
Med. Chem.Feasibility Assessment
Is the biology (i.e.pathway) well characterized?Has modulation of the pathway been shown to correlate with changes in disease burden (e.g.phenotype of KO mice, relevant animal studies, GWAS)Is there precedent for the MOA for similar disease targets?Is there a reliable and predictive animal model that has a history in translating compounds to the clinic?Does a clinically relevant biomarker exist?Competition: Examine approved claims (efficacy and safety) of competitors. Examine the competitive environment for compounds currently in development.
Clinical Correlation Assessment
1-2 slidesaddressing:Number people affected by disease (target population)Current standard of care and market size (commercial potential)How efficient are the current approaches? (unmet clinical need)How does proposed treatment compare to standard of care? Whatis the specific value of your drug/approach to patients? What advantages does it offer over existing or competing approaches?Clinical trial feasibility. How can treatment efficiency beevaluated? If known biomarker or imaging results can predict treatment efficacy in Phase II clinical trials, include them here.
1 slide describing the desired characteristics of the proposed therapeutic for the intendedindication.Note – this slide may be more relevant to projects entering PITCH at later stages, and can be left blank for projects entering at assay development/screening stage.
Targeted Product Attributes
1 slide addressingOffice of Cooperative Research/Technology Commercialization Servicesdisclosure and patentability status
Intellectual Property Position