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Isis Pharmaceuticals, Inc. Message Board

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  • robert.vince robert.vince Sep 20, 2012 7:48 AM Flag

    Isis Pharmaceuticals' Management Presents at UBS Global Life Sciences Conference (Transcript) Part 1

    Our apoC inhibitor is treating patients with very high cardiovascular risk as well as very high risk pancreatitis. In that study we showed nearly 80% reduction of apoC-III which is the target and nearly 50% reductions in triglycerides both fasting and postprandial triglycerides which we believe that this drug has a very attractive profile in another patients population which also has high cardiovascular risks.

    We doing in Phase 2 study right now which will finish up next year and our plans is to start the Phase 3 clinical program for this drug next year. ISIS-SMNRx our drug to treat spinal muscular atrophy treats a severe and rare but fatal childhood disease the results really in severe nerve degeneration. What happens is the two forms of this disease is that the patients don’t make a protein that is necessary for normal neuromuscular growth which is called SMN1 and I am excited about this drug because it actually works through a very normal antisense mechanism that is different from anything else that we have in our pipeline.

    Your body makes a related protein to SMN1 called SMN2 and SMN2 usually doesn’t do much many things because it’s missing an exon. So it differs it’s a truncated form of SMN1 that’s dysfunctional. This drug ISIS-SMNRx works by utilizing a splicing mechanism to cause the missing exon to be edited back into the protein. So the body makes some instead a full length protein which can perform the same function as the missing SMN1 protein. So we are utilizing the splicing mechanism to replace the SMN protein by causing the SMN2 protein to be become a functional protein.

    So this has the opportunity, we hope to make a very big difference in the neuromuscular growth of these patients. SMN is a significant disease; it’s about the same size as cystic fibrosis, its 35,000 patients worldwide and obviously if we can increase the survival of the infant who die of this disease early on and we have the opportunity to expand that market.

    We do have orphan drug designation in both the US and the EU and the Fast Track Status in the US for this program. The drugs in Phase I clinical trials right now that we will finish up this year, we plan on starting with phase II this year and then we have an accelerated path to a phase III program which would start the phase III study in infants late next year and the phase III study in childhood on set version of the disease in early 2014. As I said, with such a large pipeline, we have numerous other opportunities for Phase II data of the next 12 months to 18 months.

    Our Factor XI drug is our first anti-clotting drug and this is a drug that is initiating a large Phase II study in total knee replacement therapy that should end up in the middle of next year. ISIS- CRPRx is a broad anti-inflammatory drug, which we think has tremendous market opportunity potential.

    We are investigating that drug in three different Phase II clinical trails, one that’s endotoxin challenge study that we’ll look at the impacts down regulating CRP on a very variety of different cytokines and chemokines study in NRA and a study in atrial fibrillation. We begin to get data out of this phase II programs this year and with the remainder of it coming next year. ApoC-III for high triglycerides I’ve talked quite a bit about. Our EIF4E drug in cancer we expect to have data out of the phase II study next year. And behind that we have three different type II diabetes drugs all of which we expect to have data on in 2013 or in 2014. So quite a number of data opportunities coming out of the pipeline, along with that the opportunity to move these drugs forward in the later stages of the clinical development.

    Our satellite company strategy really is providing a tremendous amount of benefit and I think for most investors it’s probably a little bit below the radar screen. One thing that many of you might have been exposed to recently is progress by a company that we formed with our partners Alnylam and Regulus which is in the process to go in public.

    Regulus has recently done transactions with not only Sanofi and GSK but more recently with AstraZeneca and Biogen Idec. It's a company with a different RNA target then the messenger RNAs that we typically target. So this is a company targeting micro RNAs and we earn 46% of that company.

    Some other highlights of the satellite company strategy. I’ve talked about our partners at Excaliard being brought by Pfizer who is moving anti-scarring drug forward in to aggressive Phase 3 program and our partners are OncoGenex who are working on a Phase 3 program for their prostate cancer drug. In addition to that, we have continuous news events coming out of this robust set of collaborations. And as I said, we’re well funded. We ended last quarter with that $336 million and our guidance for the year is to end the year with more than $300 million and we’re right on track for that.

    And we have a lot of things coming up for the remainder of the year. We have commercial revenue from KYNAMRO on the horizon. We're looking forward to plan European approval this year and approval in the US early next year. Our business strategy continues to be successful. We have done numerous collaborations already this year and have a lot of opportunities for additional partnerships with a very large pipeline, the majority of which is unpartnered. We continue to broaden, mature and expand the pipeline. Our goal is to add three to five new drugs to our pipeline every year. We already added one this year and we’re on track to hit our goal.

    And in addition, we have numerous data events including the ones that I laid out for you earlier, a number of data events that have already happened, including data events from our first generation 2.5 Chemistry drug, or step 3 drug which is in our oncology pipeline.

    So with that, we got a couple of minutes left to ask me to save for questions in this room and then we can go to the breakout room.

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