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Sergey Kornilov's avatar

Hm. Interesting analysis but I would be careful with inference. Rare disease isn't a random treatment assignment, it's usually a choice made by companies that typically have clearer biological targets (often monogenic), stronger IP positions, and regulatory considerations (orphan drug designation, smaller trial sizes, priority review).

The multinomial logistic regression did control for location, modality, and lead stage, but it almost certainly did not control for quality of underlying biology, strength of genetic validation, founding team. So I think in this case "rare disease focus" may be proxying for "well-defined mechanism with strong target validation" which would predict success regardless of disease prevalence.

The acquisition signal is particularly suspect? Big pharma has been systematically acquiring rare disease companies for a specific financial reason ie to get orphan drug pricing power combined with small, manageable commercial footprints. That's a market structure thing, not really evidence that rare disease programs are scientifically better or perform better in the end.

I.e., the article may make it seem like going into rare diseases is a lower risk endeavor than some other indications, but it couldn't be farther from the truth.

The AI Architect's avatar

Nice work here. The flyover state finding is brutal but makes sense given how hard it is to recruit senior biotech talent outside hubs. Curious if the "Trading-" bucket might hide successful pivots that haven't recovered market cap yet, they'd look indistinguishable from slow failures in this data. Phase III IPO sweet spot probably reflects survivorship bias too.

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