Isis Pharmaceuticals, Inc. (ISIS)
Goldman Sachs 33rd Annual Global Healthcare Conference
June 03, 2012 4:20 PM EST
Executives
Richard S. Geary – Senior Vice President, Development
Presentation
Moderator
Moderator
Thanks for joining us everyone. I’m Terence Flynn, one of Biotech Analysts here at Goldman, and we are very pleased this afternoon to have with us Isis. And today from the company we have Richard Geary, the Senior VP of Development. Thanks a lot for being with us. Definitely appreciate the time.
Maybe you can just start off with some of the kind of current Antisense Technology. Some of the hurdles that are left, and maybe just give us kind of a brief background and history. So where it’s been and what you guys have done since then and may be what’s left to improve upon. May be some to give it a fair amount of experience on the Antisense front?
Richard S. Geary
Okay, thanks Terence. It’s good to be here. Thank you for the opportunity to talk a little bit about the company and where we’ve been and particularly the technology. I’ve been with the company almost 17 years. So I’ve been through a number of generations and the chemistries and I was reflecting with my colleague earlier today that it mirrors what was happening back in the ‘80s and early ‘90s with the antibodies and how much technology had to happen and how much change in the technology and just the basic science had to advance to a point where that could be a success.
We strongly believe at Isis that RNA-based therapeutics has a great future in the pharmaceutical industry as a third arm if you will, if you count small molecules as an arm and antibodies and proteins as an arm and then Oligonucleotides based strategies as a third.
Back in the 90’s we were working with first generation chemistry and it was clear that the half-lives of these molecules, the biological stability, the potency of the molecules was such that they were not going to be suitable for chronic use. And it was at probably 2000, 2001 where we kind of turned the corner with second generation chemistries and the medicinal chemists had worked on literally hundreds of different modifications to these Oligonucleotides and it was clear that the two prime substituent, what we call 2'MOE second generation chemistry was going to be the future that would take us to a more stable product biologically and also a more potent product that would allow us to dose of course lower doses less frequently.
And so was then that we started the program, our second generations. In 2003 mipomersen was discovered in our laboratories and we began to bring that forward. It was kind of a novel idea in cholesterol lowering if you will. Thinking about the importance of apoB as a carrier of all Afrogenic Lipids. Thinking about how we might be able to bring value to an unmet area and that would be the familial hypercholesterolemia patients. And it was from there that as we progressed in the early dosing, it was very clear that we had a potent product that was working on production obviously and instead of clearance. So all the other medications are either involved in blocking absorption or taking out the cholesterol through clearance.
This is the first time we were actually targeting something that would stop synthesis of atherogenic particles and in doing so we found in the early going that every atherogenic particle actually was taken out. That’s not a terrible surprise. I think the biggest surprise was that LPa, which is another atherogenic particle was also being impacted. And so the rest of the story, I think many of you know we moved into Phase 3 in 2007 and have now put forward our filings in both the US at the FDA and in Europe last summer, the EMA.
Moderator
Great. Maybe just going back to the technology for one second. In terms of I guess if you look forward, in terms of the next iteration antisense technology. Can you maybe walk us through some of the steps you guys are working on, how you see that evolving over the next several years and then we’ll get to the pipeline?
Richard S. Geary
Yes, thank you. And I think it’s important to note and you allude to it, we continue to work on next generation and continue to work on improving potency and therapeutic index and more reliable dosing. And as we’ve done that, we’ve began to recognize a new chemical space, we call it version 2.5, but it’s essentially another 2'MOE modification that increases the potency of these molecules yet again tenfold. And it’s that generation that we are beginning to – while we have a very good modification with the 2'MOE second generations and really a pipeline built on that, we now have a new generation, we’re starting to bring that now into cancer, and some of the other rare disease areas where perhaps even lower doses or less frequent doses could be managed and would be very beneficial for that patient population.
Moderator
Okay, great. So maybe – and then in terms of just thinking about Isis as a whole. So you have a number of drugs that are partnered, but then as maybe some of those drugs come to the market and you start to have more revenue coming in, how do you guys think about retaining rights to maybe some of these products that are further in the pipeline? Is that something that the company would like to do or do you prefer to kind of out-license these products especially if you get into some of these orphan areas as you touched on?
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