Sunday, June 17, 2007

RNAi Therapeutics Universe Expanding and Gaining Visibility

Today, I will pause and take stock of what I think have been remarkable months for the public understanding of RNAi Therapeutics.

Indeed, the climate for RNAi Therapeutics has never been better. Only recently, a number of external factors have come together that should significantly support the progress of developing the basic science of RNAi into a whole new drug class. Following the award of the Nobel Prize for the discovery of RNAi late last year, the political willingness to support RNAi has begun to extend beyond the NIH to initiatives like the one announced last month by the Governor of Massachusetts to provide $1 billion support over the next 10 years for innovative life sciences, particularly therapeutic RNAi.

These developments have consequently reached a stage where the leading political/financial journal The Economist is featuring SMALL RNAs on this week’s title page of the print issue, under the heading of “Biology’s Big Bang- Unravelling the secrets of RNA” ( All this excitement comes at a time when the pace of Investigational New Drug applications for RNAi Therapeutics has reached one IND filed every month such that eight announced RNAi Therapeutic clinical development programs are now underway in the US and Europe. And there is every sign that this pace will pick up before it slows down.

Appropriately, the second half of this year has the potential to mark another milestone in the validation of RNAi as a human therapeutic. The leading RNAi company Alnylam intends to announce by the end of this month the detailed clinical plan for a phase II experimental infection study of ALN-RSV01, an siRNA for the treatment of RSV infection in infants, the elderly and other immune-compromised adults. In this study, volunteers are inoculated nasally with attenuated RSV and the effect of siRNA on various parameters of infection is measured, most importantly viral titers.

According to the company, the study will involve 90 volunteers in a randomised, double-blind, placebo-controlled trial. If results are as hoped, this trial therefore has the potential to show for the first time statistically significant antiviral, that is therapeutic activity of an RNAi agent in man. This would represent another major de-risking event which should not only benefit Alny’s corporate objectives, but the field as a whole.

Note: Last week Alnylam (ALNY) gained almost 10% to $16.89, possibly in anticipation of these potentially value-creating events. This comes after Alnylam lost 1/3 of its value from its all-time high of $24.46, partly pressured by short sale tactics following the issuance of new shares. It should be noted that little more than 11% is held non-institutionally and if the above events should attract new major investors, prices could easily be propelled higher. Notably, last Friday saw a number a large blocks changing hands on more than twice the average daily volume, sending the stock higher by 6.43% in one day.


Mike said...


Thanks for the blog. I enjoy it very much. I am not a scientist but have spent some years now in the VC community (although not in biotech).

Regarding Alnylam's IP, are companies allowed to do as much research they want internally in RNAi with the understanding that should they wish to file an IND that they would have to license Alnylam’s platform? More specifically, are there any limitations to pre IND research without access to Alnylam's platform? Do Roche\Takeda make the move early in RNAi because they are far enough along in their own research that we should expect them to file INDs soon? Or do they move early primarily because sooner usually means cheaper? Finally, how concerned should an Alnylam investor be about Dicerna?

I apologize for my ignorance and thanks in advance for any feedback you might have.


Dirk Haussecker said...

Mike- Good questions. With regard to the use of RNAi Therapeutics IP before commercialisation, it appears that with the Merck v. Integra ruling patented inventions can be freely applied in preclinical research and development, even if the ultimate intent was commercialization of a drug based on such technologies. But like you say, the longer you wait, the higher the price-tag will be. Also, keep in mind that you don’t want end up having heavily invested in a target that Alnylam or one of its close partners have already chosen for themselves. With regard to Dicerna, my short answer is no. For more technical details, please refer to: and Also, to be an attractive platform licensing partner, you need to be able to offer a) a broad IP estate, including preferably issued patents, for freedom-to-operate; b) provide translational know-how. Only few companies have such broad capabilities.

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.