Ingredients for Success in Biotech

10 years ago, on October 30, 2000, on behalf of a U.K. client, we announced a partnership between two biotechnology companies promising to develop a monoclonal antibody for an exciting new target that emerged from research into the human genome.  At the time, just prior to the hype and promise of a fully sequenced human genome, we called this new target a “validated target.”  Of course all pursued targets were “validated,” in our minds (and in our press releases).  I once asked a science writer to define what a validated target meant to him.  His answer was that it is validated when the drug that impacts the target is approved by FDA.  Seemed a steep climb at the time.

Well this week one of those not-really-validated targets came very close to being fully validated.  An advisory panel of the U.S. Food and Drug Administration has recommended approving Benlysta for the treatment of Lupus.  Benlysta marks the beginning of a much needed new era in Lupus treatment, and the end of a long journey in drug development.  Benlysta, or anti-BLyS antibody, as we called it back in the day, inhibits the biological activity of B-lymphocyte stimulator, a naturally occurring protein important to immune function but highly problematic at high levels.  It is one of the few drugs ever to come out of the effort to discover every human gene.  Our client was Cambridge Antibody Technology (CAT), now part of Astra Zeneca, and the collaboration was with, of course, Human Genome Sciences (HGSI). 

So, having been part of the long journey from initial idea to (almost) approved product, we thought that it would be really smart, once and for all, to record each step along the way and report here the perfect “recipe” for developing a successful, innovative biotechnology product.  This recipe isn’t for everyday drug development, but for when you really want to prepare something special for patients and your investors. 
Time required: Minimum of 10 years

Ingredients:

• Capital:  Yes.  All of it.  And every penny you can raise.  Repeat as needed.
• Disease target:  Fresh, novel targets are best, although they often require more prep work.  But whatever you can get rights to can work.
• Antibody or small molecule:  (This is not the place to skimp:  Get the best one you can afford!)

Steps:

• Conduct extensive pre-clinical, then clinical trials.  Remember to follow directions carefully. Do-overs are very expensive.
• Fold data from studies into regulatory filings.  Take your time here; sloppy assembly leads to unpleasant surprises at the end of the process.
• Allow to cool.
• Prepare yourself to be astonished, one way or the other, especially if this is your first time.
 
• Serves millions.