Why Appointing a Chief Medical Officer Early On could be a Good Thing

Platform-based biotechnology companies, especially those in the early stages, are often criticized that they are about technologies in search of a medical application.  RNAi Therapeutics has also been the subject of such criticism.  

Think for example of an RNAi Therapeutic going after run-of-the-mill hypercholesterolemia with an intravenously infused formulation because this is what the technology allows you to do.  Obviously, you might end up with a marketing approval, but no physician is going to prescribe it. Tekmira’s first clinical RNAi candidate, TKM-ApoB, may be thought of in this way, especially since it had been conceived long before homozygous FH and the like entered the public mind. 

As we are in the middle of the perfect storm for ultra-orphan drug development (genetic, regulatory and reimbursement-wise), riches can be created almost instantaneously in RNAi Therapeutics by just finding the appropriate severe ultra-orphan, genetically-defined indication to match your technology.  This should not be lost on the Tekmiras, Silences, and Arrowheads which all have promised that they would come out with their next development candidate in due course (I am waiting...).

As an investor, I worry whether they put adequate resources into this task.  I have yet to see job ads looking for full-time medical geneticists that do nothing but hunt for and evaluate potential development candidates.  In addition to genetics, such a person should understand the capabilities and limitations of the (delivery) technology of the company, be able to think of how best to navigate the regulatory maze, and ultimately have an inkling of whether physicians and patients would welcome such imaginary drug candidates.

My impression has been that RNAi Therapeutics companies, except for the cashed-up, chest-pounding 800-pound gorilla Alnylam, have done too little in that regard.  I therefore hope that the appointment of a Chief Medical Officer (CMO) by Tekmira announced yesterday was not done merely to help sketch out and execute the clinical development of its lead clinical candidate, TKM-PLK1, but also to provide critical input into the early pipeline.

Of course, given the enormity of genetics and the proliferation of consultancies catering to the orphan drug revolution, you also would want to tap into outside knowledge for added inspiration and critical feedback.  Considering, however, the importance of drug candidate selection, RNAi Therapeutics companies, especially those with around 30 employees and more, would be remiss not to have internal staff with an understanding of medical practice entirely focused on this task.