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1/ Biotech is a tough business.
There are 10,000+ human diseases out there. How do startups w/ promising platforms choose where to focus?
Choose wisely. @andy23tran @a16z & I offer a framework for finding your Platform-Disease Fit (PDF)
https://a16z.com/2021/01/08/platform-disease-fit/
There are 10,000+ human diseases out there. How do startups w/ promising platforms choose where to focus?
Choose wisely. @andy23tran @a16z & I offer a framework for finding your Platform-Disease Fit (PDF)
https://a16z.com/2021/01/08/platform-disease-fit/
2/ Finding PDF early in life is essential to success.
Your first disease tends to become your adjective: âAcme Co, a cancer companyâŠâ.
So what funnel criteria do you use?
Your first disease tends to become your adjective: âAcme Co, a cancer companyâŠâ.
So what funnel criteria do you use?
3/ First step: find your superpower. Whatâs your platform edge?
Itâs not enough to solve a problem; what problem can you uniquely solve?
Can you tackle an unknown or unreachable area of disease biology?
Can you dramatically improve on or leapfrog competitors?
Itâs not enough to solve a problem; what problem can you uniquely solve?
Can you tackle an unknown or unreachable area of disease biology?
Can you dramatically improve on or leapfrog competitors?
4/ Next, think through two core axes:
In which diseases do you have high potential to successfully intervene?
For which diseases can you create + capture meaningful value?
Hereâs a handy table to help you weigh some key factors
In which diseases do you have high potential to successfully intervene?
For which diseases can you create + capture meaningful value?
Hereâs a handy table to help you weigh some key factors
5/ Do (or can) you know what drives a disease?
Is it possible to get early signals that youâre on the right track?
Do you have good assays, cell lines, screens?
Are there established animal models that faithfully reproduce the human disease?
Is it possible to get early signals that youâre on the right track?
Do you have good assays, cell lines, screens?
Are there established animal models that faithfully reproduce the human disease?
6/ What does a successful clinical trial need to show for a disease?
Can you identify + recruit patients quickly?
Investors/partners look for demonstrated risk reduction: time to IND, proof of concept (PoC).
Can you identify + recruit patients quickly?
Investors/partners look for demonstrated risk reduction: time to IND, proof of concept (PoC).
7/ When it comes to market size, patient benefit can be more important than patient population.
Transformative, curative potential commands lofty price tags (like Novartisâ $2.1m gene therapy).
Transformative, curative potential commands lofty price tags (like Novartisâ $2.1m gene therapy).
8/ Some indications have smaller market sizes but early/fast PoC (like rare diseases); others have blockbuster potential but a history of late-stage failures (like autoimmune diseases).
Itâs a varied menu â determine your risk appetite and season to taste.
Itâs a varied menu â determine your risk appetite and season to taste.
9/ What does the competitive landscape look like? You need 360-degree vision.
Itâs not only commercialized products, but also pipeline (and under-the-radar) programs. And donât forget about substitutes like surgery.
Itâs not only commercialized products, but also pipeline (and under-the-radar) programs. And donât forget about substitutes like surgery.
10/ Sometimes the best market signal is heatâlook for evidence of active BD or M&A interest from biopharma companies.
Deal size + structure is a good industry barometer for promise and potential.
Deal size + structure is a good industry barometer for promise and potential.
11/ The road to making a medicine is bumpy and long; maintain a high hurdle:
Do you have the potential to be first-in-class or best-in-class?
Can you change or become the standard of care?
Do you have the potential to be first-in-class or best-in-class?
Can you change or become the standard of care?
12/ There are only so many programs a single company can work on.
Once you have your PDF picks, you need to assess strategic alignment.
PDF tells you what you CAN do; this tells you what you SHOULD do.
Once you have your PDF picks, you need to assess strategic alignment.
PDF tells you what you CAN do; this tells you what you SHOULD do.
13/ If a disease doesnât match your vision for the company, out-license it.
If you need a well-equipped + experienced guide, find a partner.
Focus on your âgoldilocksâ opportunities, where you have go-it-alone potential to build a strong foundation for your company.
If you need a well-equipped + experienced guide, find a partner.
Focus on your âgoldilocksâ opportunities, where you have go-it-alone potential to build a strong foundation for your company.
14/ Finding your PDF is an art, not a science.
As an entrepreneur I really struggled with this process, knowing these decisions would shape my companyâs destiny.
I hope we have given you a canvas to help you sketch out your journey.
As an entrepreneur I really struggled with this process, knowing these decisions would shape my companyâs destiny.
I hope we have given you a canvas to help you sketch out your journey.
15/ A huge shoutout and thank you to our friends @Camp4tx for sharing their thoughts on indication selection, many of which have been adapted for this piece.
