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.
3 comments:
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.
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.
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.
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