Alnylam Pharmaceuticals, Inc. Q4 2008 Earnings Call Transcript

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2009-02-24 20:57:17.0

Tags: Formulation, Delivery, Call Transcript, Earnings, Cell, Alnylam Pharmaceuticals Inc., Seeking Alpha

Question-and-Answer Session

Operator

(Operator's instruction) Your first question comes from the line of Sapna Srivastava - Morgan Stanley.

Analyst for Sapna Srivastava - Morgan Stanley

It is Dave calling in for Sapna. Just a question on delivery, I know that is sort of an ongoing topic for everyone in the field. My assumption is you are using some type of lipid delivery for VSP but I was wondering if you have made any progress or if there has been any developments either a key stone or otherwise about more targeted or cells specific delivery and whether you think you can incorporate that into the VSP program or the next program to target more specific cells within the liver or cells outside of the liver.

John Maraganore

Dave that is a great question. Let me handle it but Akshay may have some additional comments as well. So with the VSP program, also with our PCSK9 program and our transthyretin program, we are using systemic delivery with the liposomal formulation that has been developed together with our collaborators at Tekmira Pharmaceuticals and that formulation has performed exceptionally well in delivery of small appearing RNAis to broad cell types in the parenchyma of the liver and we believe it is a robust platform.

In addition, we have of course, also developed liposomal formulations as part of our MIT collaboration but also continue to improve these formulations together with our collaboration with Tekmira and the University Bridge Columbia amongst other efforts that we have in the Company. So that approach really works quite well for targeting any target chain in the liver that is involved in the cause or pathway of human disease and that of course is a lot to handle right there.

Now, in addition to the work we are doing on liposomes, we are extremely involved in doing work on conjugate based delivery which is focused on more targeted specific cell types. We commented at Keystone and other meetings on delivery using Foley conjugates for tumor, delivery for example. Of course we have also talked about our cholesterol conjugate approach in the past and also have used certain sugar conjugates to get delivery to hepatocytes potentially to macrophages as well and that is part of an ongoing effort where we showed data at the recent Keystone meeting.

Beyond that, we also have a broader set of work that is going on with the Company that is going to be disclosed in the future but not ready yet for disclosure but areas that we are very excited about for other approaches for systemic delivery to other tissues in the body. So, I would say Dave that in a nutshell, the progress that has been made has been phenomenal to date. We are extremely happy with it but as I have said before, we will always be working on delivery just like people are still working at antibody engineering 30 to 35 years after Koehler and Milstein discovered monoclonals and this could be a part of an ongoing area of research in the Company but the good news, of course, is that we have extremely good versions of delivery technologies that can allow us to build an exciting clinical pipeline and set of marketed products, we believe based on what we have already.

 

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