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).
It’s a varied menu — determine your risk appetite and season to taste.
9/ What does the competitive landscape look like? You need 360-degree vision.
It’s not only commercialized products, but also pipeline (and under-the-radar) programs. And don’t forget about substitutes like surgery.
10/ Sometimes the best market signal is heat—look for evidence of active BD or M&A interest from biopharma companies.
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?
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.
13/ If a disease doesn’t match your vision for the company, out-license it.
If you need a well-equipped + experienced guide, find a partner.
Focus on your ‘goldilocks’ 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 company’s 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.
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3/ Developed in Sam’s lab at Stanford, Earli’s technology can program bits of biology to force cancer cells to reveal themselves—a “synthetic biopsy” to precisely locate cancer cells inside the body so they can be treated, early.
2/ Biotech has historically had a multi-armed bandit problem.
Spend each incremental dollar feeding the goose? Or invest it in incubating a golden egg?
Companies tend to starve their platform in order to nurture a potential product.
3/ But a new wave of productive platforms changes that calculus.
The shift from bespoke craftsmanship to industrialized drug development will transform what we can make, how (and how fast) we make it, and how the industry is structured.
1/ “The price of a drug should never be the rate-limiting factor to patient access.” — Alexis Borisy, EQRx CEO. @EQRxINC raises $500 million Series B in service of their mission to bring important new medicines to society at radically lower prices.
2/ The Series B investor syndicate includes life science specialists, mutual funds and other generalist funds—as well as payers + health systems that cover more than 20% of insured lives in the US.
3/ Launched just one year ago, EQRx has built a deep pipeline; it has disclosed late-stage cancer programs targeting PD-1, PD-L1, EGFr, CDK4/6. In partnership with health systems + payers, EQRx estimates it could save the US healthcare system 50-70% of drug spend in key diseases.
2/ Within 48 hrs(!) of having a digital copy of the virus genome, Moderna designed a vaccine - the exact version now being shipped - without having access to the physical virus.
This is *the machine that made the vaccine*
We used to grow our vaccines; now we can print them.
3/ The tech works by delivering mRNA instructions for cells to make a protein. In this case, the vaccine is delivering instructions for making the SARS-CoV-2 spike protein (so our immune system learns to attack it).
@sbancel describes it as sorta like making chocolate mousse :)
1/ Recently $NVS announced a $2.1m gene therapy, the most expensive medicine in history. Gene therapies are potentially curative. How should we price them? How will we pay for them? Could a cure be considered a pre-existing condition? 🧬 💊 a16z.com/2019/05/30/cur…
2/ Rare disease therapies are expensive because: i) they treat devastating diseases, ii) patient pops are small, so biopharma recoups R&D investment w/ high price, iii) payors tolerate because limited risk of many patients in a given plan + limited # of new therapies to cover.
3/ Individually, rare diseases (by definition) affect few people; collectively, 30m Americans (1 in 10) suffer from one of 7000 rare diseases. There are now lots of gene therapies on the horizon for lots of rare diseases; many >$1m therapies in our future. How will we pay?
💰💰💰
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
(thread 👇)
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) a16z.com/2018/04/30/bui…