...important – and why there’s likely to be a therapeutic window for your molecule. This can include genetic data, correlation between expression patterns and outcomes, published data around the MoA, and your own preclinical work around efficacy & safety in animal models /...2/11

...human tissues. Any time you can point to work done by others that validates the target to any extent, you should. You may want to supply a white paper on the topic or put some additional slides in the appendix. With novel modalities, do your best to anticipate questions...3/11

...around delivery, manufacturability, tissue distribution / pK, immunogenicity, safety, etc. You won’t have all the answers but you should communicate a plan for when and how you’ll answer those questions. Likewise, if you are pursuing a novel regulatory path & as of yet...4/11

...you don’t have regulatory feedback, you may need to address the fact that there could be several regulatory outcomes. Perhaps there are comps or FDA guidance / statements that are helpful. Investors will want to know that the program is still investable even in downside...5/11

...scenarios (particularly with legislation being introduced around changes in the accelerated approval pathway). On the one hand, it implies high unmet need if nothing has been approved in a particular indication; on the other hand, investors don’t love stepping into...6/11

...unbounded downside risk. This is also the case for undefined commercial opportunities. If you’re pursuing a rare disease population, for instance, for which epidemiology, diagnosis rates, etc. are unclear, the onus is on you to try to clarify the size of the addressable...7/11

...population. Are there registries? Can you query the top centers on how many patients they see? Of course, a full literature review around epi will be helpful. Be realistic around the segment of the population you’re targeting (e.g., just moderate to severe? Those...8/11

...early or late in the disease progression? A genetic subset?), in terms of initial clinical trial and ultimate addressable population. Also think through how long patients are likely to stay on your drug. This will go a long way toward building credibility. If you are...9/11

...dependent on rare disease designation for exclusivity, look to see if others have gotten this designation for your target disease and get some consultant input early – it isn’t always as straightforward as it might seem. In summary, terms like “novel target”, “new...10/11

...modality”, and “no approved therapies yet” get investor attention – but they also carry outsized risks and therefore require more legwork and supporting materials relative to programs with less novelty. 11/11

@SaraNayeem At what point in development would you expect to see a TPP and PTRS assessments in a proposal?