Academic Drug Discovery, a CRO?

Recently, I had the pleasure of attending a symposium on academic drug discovery hosted by the Brain Science Institute at Johns Hopkins Medical.  The symposium brought together representatives from many of the top academic drug discovery centers (UCSF, Harvard, Vanderbilt, etc.) to discuss best practices. 

While the symposium was incredibly well run and informative, I left being unable to answer the very question that drew me to the symposium in the first place – that is, should academic centers be in the drug discovery game?

“Pharma Drug Discovery is Inefficient”

I heard that refrain over and over at the symposium.  While that may be the case (I, for one, do not 100% agree), it did not strike me as though universities had solved the inefficiencies that plague pharma companies. 

I found it somewhat interesting that all of the universities that I met with had hired former pharma people to run their operations.  If pharma was inefficient before, I can only imagine how “inefficient” those same people will be on an academic budget.    

In hearing that refrain, I also wondered why academics would be so much better at drug discovery?  It is safe to say that all of the pharma companies are basically working with the same scaffold libraries, and it is hard to believe that academic centers would be any different.  So, if everyone is working with the same raw materials to answer pretty similar questions, how can one say with any confidence that one group is really better than the other?

Avoiding the Inevitable

Doing drug discovery on an academic budget is no small feat and many of the institutions that I spoke with had received large alumni gifts to establish their centers.  While a $20-30M gift is quite a substantial amount of money, it is not a whole lot of money within the context of drug discovery.  Therefore, academic drug discovery centers will have to figure out ways to bring in additional capital to fund their operations outside of alumni gifts and the NIH.

There in lies the rub.  All of the academic centers I spoke with were designed to meet the needs of their researchers.  Realistically, less than 5% of the researchers at any given institution are conducting research that lends itself to small molecule drug discovery.  As time goes on, academic drug discovery centers will quickly work through those researchers and have to start looking outside of their walls to find groups that need their services.

As academic drug discovery matures, institutions will begin to compete with each other to perform drug discovery work.  Working with outside groups will then put academic drug discovery centers in direct competition with CROs.  This will lead to price competition, which puts academic centers at a significant disadvantage given the indirect costs associated with their institutions.  

Some Creative Ideas

Frankly, I believe there is definitely a place and a need for academic drug discovery.  That being said, the whole academic drug discovery concept is still in its infancy.  As time goes on, academic centers could possibly consider the following strategies and options when positioning their services: 

• Collaborate: Academic drug discovery centers could achieve efficiencies of scale by banding together.  While some institutions are building significant molecule libraries, the reality is that those libraries pale in comparison to those of large pharma companies.  Furthermore, despite massive R&D layoffs, pharma companies still have a sizable staffing advantage over academic drug discovery centers.  Lack of molecule diversity and adequate staffing puts individual universities at a disadvantage against pharma companies.  One way to drive efficiencies of scale would be for institutions to collaborate around a particular disease area to leverage shared resources (ex: Hopkins and Harvard are both focused on CNS).  Creating large collaborative agreements between institutions could help drive greater program recognition which should help generate more grants and interest from outside investors. 
• Novel chemistry: Most pharma companies are using the same fatigued scaffolds in their screens because preclinical teams are not incentivized to work with novel chemistry.  While many approved drugs (e.g. lapatinib) break or bend common drug development rules (e.g. Lipinski’s Rule of 5), pharma has become rigid in it’s thinking about Lipinski RO5-breakers and their ilk.  Unless a molecule fits nicely within the Lapinski bucket, a preclinical drug developer will not go to bat for a molecule in a committee meeting.   Academics, working outside of the beuracratic constraints of pharma, could pick up the slack and work with chemists to validate novel scaffolds that break or bend the RO5.  A novel scaffold with some SAR data provided by the drug discovery core would likely be of interest to a potential licensee.
• Novel biology:  Academics have traditionally been very good at identifying novel drug targets and drug discovery centers are already working with the “squishy” science folks to help determine the druggability of those targets.  I would love to see drug discovery centers expand their work in providing assistance for the early validation of biological targets.     
• Underserved areas:  Pharma and VCs are moving away from high-risk and costly drug development (e.g. diabetes, cardiovascular, and neurological disease), and I would love to see academic drug discovery centers move aggressively into those areas. While I appreciate why pharma companies have moved away developing drugs that require mega studies to satisfy regulatory hurdles, it is unlikely that the FDA’s strigent regulatory requirements will stand in perpetuity.  As biomarker development gets stronger, adverse effects will be better predicted – thus, alleviating the need for massive trial sizes.  When the FDA turns the corner, academic drug discovery centers will hopefully have molecules waiting for eager investors. 

The Future

It is really still the early days of academic drug discovery.  Over time, centers will better define their roles in the drug development continuum and strengthen their value add.  As this occurs, a consolidation of drug discovery centers will lead to greater efficiencies and visibility in the marketplace.  Hopefully, this will lead to better and stronger academic drug discovery to find novel therapeutics.