For biopharma co's pitching VCs, always think about how you can contextualize your data. Positive controls (“benchmarking data”) utilized in the same study as your active agent are the best way to do this in the preclinical setting. (Think carefully about how these...1/8
...comparators are dosed – aiming to achieve the equivalent concentration or exposure as used clinically is reasonable.) But even if that’s not possible (relevant agents can’t be obtained / made), think about what other preclinical or clinical data can provide meaning to...2/8
...your study results. Perhaps a competing molecule in development was studied in the same model and you can reference that data. Or perhaps there’s relevant preclinical AND clinical data on an abandoned compound with the same MoA (e.g., had some clinical efficacy but also...3/8
...had tox your molecule avoids in some way; or it wasn’t potent or selective enough and your molecule is; etc.) There’s also the commercial context; what are (or were, for off-patent drugs) yearly sales of other branded drugs approved in the indication? Always need to...4/8
...justify the question of why your MoA / molecule / clinical approach (e.g., perhaps you’re using genetic stratification) will be better than what’s available. This may be obvious, but each time you undertake a new study, think critically about animal model / cell line,...5/8
...dose groups, controls, timing of dosing (prevention vs. treatment model and how does this relate to the likely clinical paradigm), duration of the study, etc., and ask, “does this particular study move the needle for us?” Sometimes a deeper data set in only one or two...6/8
...models is better than a broad but shallow data set. Boxes to check include establishing your molecule’s MoA, tying the MoA to the disease pathogenesis, establishing a good dose response curve, and showing convincing efficacy and safety in relevant models. And always...7/8
...take a step back and ask, before finalizing your pitch, “have we demonstrated both that this is on its way to becoming a drug AND that this drug will matter?” 8/8
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