Tuesday, August 13, 2013

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.


Anonymous said...

Silence have had a very highly regarded CMO for some time and he has substantial resources to call upon in the area of potential candidates which as I understand it is not limited to tiny orphan areas as you suggest.

Anonymous said...

to add to this TKMR are also hiring for a director of business development to help with marketing and partnerships of their LNP technology.. this is a push in the right direction.

Anonymous said...

Dirk, your theory certainly holds water. John Nemunaitis has been CMO of Gradalis since the beginning. They are, without a doubt, the closest RNAi candidate to commercialisation. With recent success in Ewings Sarcoma patients, they have opened a forth PII trial testing their personalised bi-shRNA FANG vaccine in various advanced cancers. I understand your are not a fan, but with so many patients surviving a year or more over the historical averages, they must be doing something right.

By Dirk Haussecker. All rights reserved.

Disclaimer: This blog is not intended for distribution to or use by any person or entity who is a citizen or resident of, or located in any locality, state, country or other jurisdiction where such distribution, publication, availability or use would be contrary to law or regulation or which would subject the author or any of his collaborators and contributors to any registration or licensing requirement within such jurisdiction. This blog expresses only my opinions, they may be flawed and are for entertainment purposes only. Opinions expressed are a direct result of information which may or may not be accurate, and I do not assume any responsibility for material errors or to provide updates should circumstances change. Opinions expressed in this blog may have been disseminated before to others. This blog should not be taken as investment, legal or tax advice. The investments referred to herein may not be suitable for you. Investments particularly in the field of RNAi Therapeutics and biotechnology carry a high risk of total loss. You, the reader must make your own investment decisions in consultation with your professional advisors in light of your specific circumstances. I reserve the right to buy, sell, or short any security including those that may or may not be discussed on my blog.