Bio Entrepreneurs: The Importance of Selecting Disease Areas

1/ A thought for early-stage bio companies on the eve of the upcoming #JPM19 networking bonanza in SF: Bio entrepreneurs, especially first-time founders, often underestimate the importance of analyzing and selecting potential disease areas.

2/ Your resources are (probably) finite. Your platform may have broad potential but if your goal is to develop therapeutics (Tx) or diagnostics (Dx), you need a rigorous process/framework for selecting disease indications. It should be an early core competency for your company.

3/ This is particularly important given how long, risky and expensive it can be to develop a new Tx or Dx. We’ve written previously on 16 pitfalls to avoid when building a computational Tx startup, many of which touch on this issue (especially #5).

4/ Can indication selection wait? Maybe, but strategy should inform (and be informed by) target diseases. Seek early signal that your tech is relevant for solving specific, important problems. Or you risk being perceived as a solution looking for a problem, a precarious situation.

5/ As you grow, you’re growing roots. Your team’s collective expertise and experiences may not translate easily from one disease to another, limiting future flexibility. A bio startup’s first disease area tends to become its adjective: “Acme Co, a diabetes company…”.

6/ Some platforms are so broadly disruptive that they defy gravity, remaining platform-centric vs disease-limited. These tend to have unique business models or early, significant industry buy-in. But these are rare; most platforms have to prove themselves in specific diseases.

7/ There are on the order of 10,000 known human diseases. Each has its own distinct atmosphere and landscape. How do you narrow down your options? What funnel criteria do you use? Every situation is unique but it’s about establishing evaluation and decision-making capabilities.

8/ Is your platform particularly well-suited for tackling a specific area of unknown/unexplored disease biology (greenfield opportunities)? Or, in more contested indications, can you dramatically improve on existing solutions or leapfrog competitors(competitive advantages)?

9/ For a specific disease, is it possible to get early, convincing signals that you’re on the right track? Do you have good in vitro assays, cell lines, screens? Are there established in vivo animal models that faithfully reproduce the human disease? Is value created early?

10/ Do you go-it-alone or go down the BD/partner path? Go-it-alone will constrain your addressable disease universe (usually to those w/ smaller patient populations). If you’re looking for a partner, your initial disease areas will determine your dating pool (so choose wisely).

11/ What does the clinical development/commercialization plan look like for the disease? Programs need significant funding to go the distance. Investors/partners look for demonstrated risk reduction, proof of concept (PoC). What are the expected milestones? When is value created?

12/ Some indications have smaller market sizes but early/fast PoC (certain rare genetic diseases); others have blockbuster potential but a history of late-stage failures (like some autoimmune diseases). It’s a varied menu — determine your risk/reward appetite and season to taste.

13/ What does the competitive landscape look like? You need to have 360-degree vision. It’s not only commercialized products, but also pipeline programs (be paranoid: biopharma may have under-the-radar programs). And don’t forget substitutes/alternatives like surgery, monitoring.

14/ Indication selection is critical. Don’t do it in a vacuum. Lots of wisdom out there. Network. Meet KOLs. Don’t recreate the wheel: this is a core function in biopharma and seasoned talent is increasingly interested in (and available for) early-stage bio. Happy hunting!