Alnylam CEO gives Corporate Update at BofA Healthcare Conference

In this blog I will summarise the highlights of the corporate update given today by John Maraganore, the CEO of Alnylam Pharmaceuticals, at the Banc of America Healthcare Conference in Las Vegas. Alnylam can be considered the leader in RNAi Therapeutics, and it is their success so far that has opened up the prospect for a whole new class of drugs based on RNAi. As such, Alnylam is proud to note that their work was cited by the Karolinska Institute in the justification for the recent Nobel Prize to Fire and Mello for their discovery of RNAi.

In his presentation, JM detailed the breadth of Alnylam’s clinical and pre-clinical programs encompassing all major therapeutic areas except for bacterial infection. There are more than 20 such programs ongoing at Alnylam, Alnylam partners, or licensees. Moreover, over 50% of current clinical RNAi programs and over 75% of siRNA reagent sales are covered by Alnylam IP. Besides scientific excellence, IP is a key issue for Alnylam’s financial ability to turn RNAi into drugs, and JM made it clear that Alnylam is the gatekeeper in the commercialisation of RNAi therapeutics: Alnylam has access to all the 8 key issued patents relating to RNAi Therapeutics, 7 of them on an exclusive basis. As a consequence, the company has already taken in over $120M in milestone payments and fees, not including the potentially more than 1 billion USD in future milestone payments.

As key business goals, JM cited continued progress on the science, particularly in delivery, moving along the clinical pipeline (for 2007: phase II for RSV, INDs for PCSK9 and pandemic flu; announcement of 2 more pre-clinical programs) and one or more significant alliances by year-end. What JM seemed to value most, however, is their plan to show human proof-of-concept data for an RNAi Therapeutic within the next 12-18 months. This could either come from their phase II RSV experimental infection studies, slated to start by the end of June, or early results from the PCSK9 hypercholesterolemia program. With regard to PCSK9 and possible competition from ISIS’ antisense compound targeting the same target, JM was very diplomatic in his response saying that he is encouraged by developments in the antisense arena, and the future will show what each technology platform can do therapeutically. Unlike ISIS and in spite of considerable outside interest, Alnylam would not have any immediate plans to partner PCSK9 which Alnylam is pursuing with the leading Brown-Goldstein labs at UT Southwestern.

Overall, a solid performance by JM and everything is appears to be on track.

Note: Alnylam dropped today another 4.5% to $15.49 amid heavy trading, despite an overall positive market. At this point, investors just have to be patient. Policies that make it clear that Alnylam is not for sale and saying certain programs are to stay within the company may not be popular in the short-term, but Alnylam is about building a real business, and is not an M&A project.