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Isis Pharmaceuticals, Inc. (



Citi Global Health Care Conference

February 28, 2012 2:00 PM ET


Lynne Parshall – COO and CFO


Lynne Parshall

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Thank you and thank you for joining us this afternoon. Our strategy from the beginning in pioneering the development of antisense technology has been to create an environment for innovation, to be innovative, to structure that innovation and moving forward, what we think is a unique technology platform for drug discovery and one that can be very productive. To utilize that productivity to create a large and growing pipeline of novel drug. We're right now 26 drugs in development and to build around that you need business strategy to allow us to really leverage the technology platform and create as many opportunities for the technology as we can. Our goal has been to create as many drugs as we can for as many different diseases so that we can help as many patients as possible, lead healthier and more hopeful lives.

So ISIS has created, the antisense technology platform and antisense technology works, it works and is proven by data and numerous different animal models, numerous different clinical models and with numerous partners. We control this technology platform. We're one of the most innovative companies in United States with over 1,500 issued patents I think on a per capita basis that would make us far and away the most productive and innovative company.

We’ve used the technology platform as I said to create a pipeline that's large, diverse, growing and currently includes 26 different drugs and we do this with a small and focused R&D organization. We have 300 people in not only R&D but also our SG&A organization; we've had another 60 in our manufacturing organization. So what is a relatively small company with a very manageable cost structure has been able to utilize this very efficient and productive technology platform to create a large and growing pipeline.

Our business strategy that has supported this is also unique. Our goal is to do what we do best, to stay small, to stay innovative, stay focused, to make as many drugs as we can and move them forward to clinical proof of concepts and then work together with pharmaceutical company or biotechnology company partners to do the latter stages of development and commercialize the drug. This has allowed us to create this kind of a pipeline with variable means of financing and be a financially strong company as we enter into the prospects this year of our very first systemic antisense drugs being launched in the market.

Antisense technology is the only direct route from genes to drugs. We make uniquely specific drugs but knock out a single disease causing protein without affecting other related proteins. The early development and discovery and development is extremely efficient because improvements that we make in one part of the platform are directly applicable across the board through all of our research and development programs. So if we improve manufacturing, we applied to all of our programs. If we improve in analytical method, we apply to all of our programs. If we find a chemical medication that improves potency, we can apply across all of our research programs, so the efficiency and effectiveness of technology platform improvement gets amplified throughout the programs that we have. That's allowed us to generate a continuing pipeline again with a very small R&D organization.

This is the pipeline, it's hard to read. I am not going to spend too much time on it but you can see our principal area to focus are cardiovascular disease led by our lead drug Kynamro metabolic disease, so we have drugs not only for type 2 diabetes but also our first peripherally acting anti-obesity drugs. A large cancer franchise, a much newer but very exciting severe disease franchise and a variety of other programs that we are working on with companies who want to exploit antisense technology in their own particular therapeutic area.

Kynamro is our most advanced product. We have filed in Europe last summer for homozygous familial hypercholesterolemia as well as a severe heterozygous FH patients and we plan to submit the US NDA this quarter. Our partners at Genzyme and Sanofi are very actively preparing for the commercial launch of this drug this fall and as well Genzyme and Sanofi are investing in the future of this drug with our focus FH study that's underway. This is the study that intended to create data that will allow us to broaden the indications both in the US and Europe as well as to include alternative dosing regimen so that we can provide patients with multiple different choices of how they get to take Kynamro.

As we said, our first filing in the United States is for homozygous FH, that's about 3,000 patients in the US and the focus FH study is intended to support expansion of that indication to that sever heterozygous FH patients which will add another 15,000 patients to our US market.

Our initial filing in Europe encompasses both of these markets, both the homozygous FH and the severe heterozygous FH for a total of approximately 18,000 patients. And the next potential filing in Europe would be a filing that would look at the next layer of severity of LDL cholesterol, patients who are 160 and above and we hope that data from the focus FH study will be part of the data to support a potential expansion of the indication in Europe.

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