Alnylam Pharmaceuticals' CEO Presents At Morgan Stanley Global Healthcare Conference (Transcript)

Alnylam Pharmaceuticals, Inc. (ALNY)

Morgan Stanley Global Healthcare Conference

September 11, 2012, 01:30 pm ET

Executives

John Maraganore - CEO

Mike Mason - VP, Finance

Analysts

Dave Friedman - Morgan Stanley

Presentation

Dave Friedman - Morgan Stanley

Thanks everyone for joining us. Dave Friedman one of the biotech analyst and in terms of disclosures for myself and Morgan Stanley, you can look on morganstanley.com/researchdisclosures and we’ve team from Alnylam up here; very exciting platform, RNAi company and more. And on the far side here I’ve got Mike Mason, VP of Finance and on the near side, John Maraganore, CEO. And so maybe if you guys want to give a brief sort of statement or overview about the company at all and then we can then just dive right in.

John Maraganore

Sure, David thanks and thanks for having us here at the conference. Alnylam is a RNAi therapeutics company that has focused from the beginning on developing small interfering RNA drugs and over the last many years, we really been focused on perfecting the technology so that we can achieve the optimization of these molecules, introducing drug-like properties into these molecules and ultimately delivering these molecules into the right cell types and tissues in the body to be able to harness the indigenous RNAi pathway to create important new medicines.

And I think it's fair to say that over the last two years in particular, the work that we've done on achieving delivery of small interfering RNAs to liver cells and documenting in human trials that we can in fact knock down targeting genes man with very robust pharmacological effects and I think that largely comes out of the work that we’ve done in our transthyretin amyloidosis program, but also the work we've done on our PCSK9 Hypercholesterolemia program.

I think we've now clearly demonstrated that RNAi does work in humans; RNAi is the foundation for generating new medicines and we're excited to continue to executive on that plan and advance programs with a real focus on our transthyretin amyloidosis program and also on our Hemophilia program where we are targeting anti-thrombin as the foundation for building an exciting company going forward.

Dave Friedman - Morgan Stanley

So maybe if we can just start with one broad question and you guys have been around for a while and your progress appears to be you know accelerating every year. So if you can maybe just highlight from a technological standpoint the one or two key aspects over the last year or two in terms of making RNAi more and more drug-like and deliverable that you think people should understand the best in terms of understanding where you guys are headed with this?

John Maraganore

Yeah, I think there really are two things David that are notable; one is that, you know we've really industrialized and advanced lipid nanoparticle technology in clinical studies, these are liposomal formulation of small interfering RNAs and we've made substantial improvements in those molecules, in those delivery formulation that enable an appropriately robust knock down of target genes in animals and also in humans with a good therapeutic of index, which is important.

The second part, the second key thing has been the advancements in so called conjugate sRNA technology which are in many ways simpler molecules. These are small molecules that are handed out to the small interfering RNA and mediate receptor uptick of these molecules of the cells and those data have panned out beautifully in animals system right now and we’re about to go to human trials also in our TTR program.

So, both platforms with the nanoparticles, but also conjugate base delivery of small interfering RNAs really have been the key things that have enabled the advancement over the last couple of years. Of course before that let’s not forget the important underlying aspect of RNAi which is these double stranded RNA molecules and as triggers of this biology are incredibly potent and obviously have served us well in our overall drug discovery efforts.

Dave Friedman - Morgan Stanley

And in terms of the conjugated small interfering RNAs, is that a technology that is going to primarily optimize delivery to existing places where you can already get or is this part of what is going to help bring the technology in a more directed fashion to specific tissues throughout the body?

John Maraganore

Well, I think at least in the near-term it’s going to help and advance and expand how we can develop programs for liver specific gene targets and that’s because we are using the asialoglycoprotein receptor as a receptor for uptake of the sRNA. And we are very pleased with that approach, we think that that approach enables subcutaneous delivery of sRNAs and we get very potent effects with very wide therapeutic index with that technology. And in many ways we think that that’s going to be a technology for the future that really together with the LNP technology is what’s used going forward.

So we are excited about that at least in the near-term the focus will remain on liver and probably reason for that David is we are really going to execute on our plan that we call Alnylam 5x15 which is the target, liver expressed target genes with our sRNAs as a way of building exciting product opportunities for the future and that will start with TTR amyloidosis and follow with other programs that are in that pipeline.

Dave Friedman - Morgan Stanley

And anyone should feel free to have the session be as interactive as possible, so if there is any question at anytime please raise your hands, share out something and we will make sure that we can get your question asked.

Read the rest of this transcript for free on seekingalpha.com

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