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” (http://www.economist.com/printedition/). 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.