Friday, December 18, 2009

RNAi Therapeutics Highlighted in Roche Media Day Presentation

Roche can be considered the bellwether of how Big Pharma/Biotech is looking at RNAi Therapeutics for rejuvenating their pipeline, and their moves will be closely watched by the investor community. I therefore found it extremely interesting what a Media Day presentation in 2009 revealed about their RNAi Therapeutics intentions. This presentation was given by Lee Babiss (President & Global Head of Roche Pharma Research) who has been a champion of RNAi Therapeutics at Roche. It is worth noting though that according to musings on the cafepharma discussion board, he has unfortunately left the company.

Roche sees RNAi Therapeutics as an integral part of their personalized medicine efforts conducted in concert with their diagnostics unit and which offers fast development timelines. Notably, doing the math, there were about 5 RNAi Therapeutics research programs in 2008 (0 in 2007) at what appear to be Lead Identification and Optimization stages. Relevant to RNAi Therapeutics, this portfolio of about 100 programs is jammed full with solid cancer programs which is THE poster child of personalized medicine (almost 20% of programs). As has been made public in a previous RNAiNews interview with, Roche intends to work with Tekmira to bring two RNAi Therapeutics programs into the clinic near-term, including one IND in 2010. According to slide 26 this is likely to be a program (‘MVD’) in metabolic disease as this has been the most advanced program (‘phase 0’) at the time of the presentation. ‘MVD’ is categorized under ‘metabolic disease’ and therefore could mean ‘macrovascular disease’. Type II diabetes, respiratory, and solid cancer RNAi therapeutics programs follow not far behind. It would not surprise me that the second program would be one for solid cancer, paralleling efforts at Tekmira (phase I SNALP-ApoB ongoing; solid cancer 2010 IND).

In delivery, it is, however, not all about liposomal delivery. Roche’s dynamic polyconjugates were highlighted in rhesus monkey studies which showed quite significant (>90%) and very prolonged (almost maximal at 3 weeks although this could be repeat administration data) knockdown, albeit at the relatively high doses of 20mg/kg. siRNAs coupled to antibodies were also highlighted in cancer applications which together with SNALP and DPCs may form Roche’s 3-legged stool of siRNA delivery.

Alright, this is it for me in 2009. I would like to thank everybody for reading some of the highly speculative stuff and who have made writing the blog particularly interesting to me either through your comments or by personal contact. I look forward to what promises to be a very exciting year in RNAi Therapeutics.


Anonymous said...


Do you see the year 2009 as the year reality set in for the RNAi space? I remember how the buzz of monoclonal antibodies fell off for a few years after initial excitement and high related share prices.

Is RNAi that far off that institutional investors would rather wait for progress before committing to the space? Obvious to ne the leader ALNY has seen the glow come off its stock price as it has been with others. TKM
is like a lottery ticket to me.

Care to comment?

Dirk Haussecker said...

I believe you are right in that RNAi, after some of the buzz it rightly generated, is going through a phase in its development where investors have been increasingly reminded of the realities of the lengthy drug development process in the absence of new blockbuster deals and disappointment around some of the first RNAi Therapeutics drug candidates. All this is probably normal for a new exciting class of drugs, and your analogy to MAbs is probably right on. But like MAbs, there is no way that such a technology is going to ever disappear from the drug development landscape and will eventually succeed big. Of course, it does not immediately follow from that that NOW is the right time to invest.

I agree that TKM is a 'speculative' investment, but at the same time IMO all stock investments are 'speculative' and there is nothing wrong with that categorization as long as one knows a company fairly well. Much better than an investment in many banks 4-5 years ago which were deemed to be 'safe', 'quality' investments- a justification for lowering expected returns. I just believe that in this part of the its development, e.g. with the delivery and innate immunity challenge, TKM presents the best risk:reward at the moment. I'd rather have 5 lottery tickets such as TKM in my portfolio than an equal number of 'safe' stocks.

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.