Biogen Idec Presentation at Piper Jaffray Investor Conference, January 26, 2005
Mark Kowalski, Piper Jaffray Senior Biotech analyst: … Speaking today for Biogen Idec is Gunther Winkler, Vice-President of Strategic Initiatives. Biogen is one of our top picks for 2005 based on our expectations for a strong Tysabri launch and quick adoption in the MS market. So, Gunther?
Gunther Winker: … [only matters related to Tysabri] Tysabri, the up and coming MS treatment, it has all the potential to become another blockbuster for us, in order to change how MS is treated in the future. It was registered, as you remember, at the end of the year, and then I have a few data for you later on about the performance. Finally, I want to mention that we are making good progress on the facilities front. The Oceanside manufacturing facility, large scale has [gap], I mean the construction has been completed ahead of schedule. And the facility will mainly supply Tysabri. . . .
All of this investment in R&D allowed us to produce two blockbuster drugs: Rituxan, which is #3 in the hit list of blockbusters in biotech and Avonex is #6. And certainly our new drug Tysabri has all the elements it needs to potentially become our third blockbuster on this list. …
We have five products on the market. Avonex, Rituxan, Zevalin, Amevive and Tysabri. We have another big product coming. It’s Rituxan in RA. We’re in Phase III trials. I listed it separate from the approved Rituxan because it is not approved in a very, very big market and an oral fumarate, which is in development for psoriasis and multiple sclerosis. …
In multiple sclerosis we have emerged in the last 8 years now, since 1996, as the clear leader in multiple sclerosis, by any measure you want to apply: market share, sales, anything you would like. And there is a reason for this. Avonex, which our first drug in this indication is a magnificent drug. It has all the qualities for a blockbuster: the efficacy, the safety, the convenience. And it is backed up by very strong customer support. We are applying now the same, of course, to Tysabri, and hopefully make this as big as even bigger than Avonex. A few more words to Avonex …
Now Tysabri. We are on the way to establishing Tysabri as the new standard of care in multiple sclerosis. And why do I say this? Well, first of all, let’s look at what’s out there. Right now, patients perceive, and the physicians perceive, there is very little difference among the current therapies in terms of efficacy. The side effects of some of these drugs, including Avonex, are problematic. We’re talking here about flu-like symptoms. Other drugs, other than Avonex, can also cause necrosis at the injection sites. And there are other problems with current therapies.
Then there is the convenience. Avonex is given once a week. Other therapies are given multiple times a week via injection which is very, very inconvenient and cumbersome for the patients. Now Tysabri was designed to overcome this. We developed Tysabri, as you know, with Elan and we are the .. both, we are the leaders in the industry. Both we understand MS. And we supplied lots of our experience at Biogen Idec to this development. So, what we got was
1) A product that is novel. It is an adhesion molecule inhibitor. It’s not another interferon.
2) Its efficacy is very powerful. I’m sure some of you read the literature, read the label. It’s outstanding.
 Side effects. Its side effects are benign. There are no flu-like symptoms with this product.
 And convenience is high. Although it is an IV infusion at this time, it is only given once every four weeks, versus, what I said before, weekly or even multiple times a week
Tysabri earned us a very broad indication. It was a huge study by the FDA very rapidly got approval at the end of November last year. And the broad indication statement says: “For patients with relapsing forms of MS to reduce frequency of clinical relapse.” And in the label you also find a comprehensive clinical data section. First of all, there were two underlying studies. And the studies were designed in a way that they complement each other. The monotherapy study where we saw a 66% reduction in relapse rate and 96% of patients were free of gadolineum-enhanced lesions.
On the other side, we have the Avonex add-on study. So it was a study where patients were given Avonex and Tysabri was added. And we saw in this patient population an additional 54% reduction in the relapse rate. And again, 96% of patients are free of gadolineum-enhanced lesions.
Safety. It’s generally a very well tolerated product. I listed here the three top safety issues that were seen in these studies. They are all in the low percentage numbers. The side effects are also occurring in the placebo group which means that they are not unique to Tysabri. And there are really very few infusion related reactions: 22% of patients complained about infusion-related side effects and 17% in the placebo group. So that’s really about the infusion more than the drug itself.
Low rate of immunogenicity. That’s surprising, because it’s a monoclonal antibody. Only 10% of patients have developed antibodies. Some of them were transient. They appeared and they went away. 6% of patients had persistent antibodies. And this is not, again, where you have to wait very long for these patients to have a persistent antibody. Because 90% of this 6% developed this persistent antibody response within the first 12 weeks of the treatment cycle.
There were a JP Morgan survey done with neurologists and the data was published in December 2004. And interestingly, when you listen to neurologists, they were asked, “What is the significant advantage of Tysabri?” They said, “Reduction in relapse rate.” 70% of neurologists answered this question with “This is very, very important.”
“Less frequent dosing” – so you can see there is the convenience factor. Then you go down the list. And again you see “efficacy”, “efficacy”, “safety”, “convenience”. These are the three things that Tysabri brings to MS patients and to neurologists above and beyond what these other drugs that are currently in use have.
Now you may ask, where do these new Tysabri patients come from? Well, to understand this, we have to look at the current market. The current MS market is much, much bigger than the segment that’s treated. So let’s look at the U.S. numbers only. In the U.S., about 450,000 patients have MS. 350,000 are indicated for treatment, but only 190,000, or about 60% of those that are indicated are on treatment. So there’s room for expansion. If you look for the rest of the world data, it is even worse, because there you have 400,000 indicated and only 160,000 treated.
Now, why is there such a big number of patients that are not treated? Of course, there are the naïve patients, that are diagnosed and indicated for treatment. And we have now a choice beyond the traditional drugs: they can now go on Tysabri. These are about 30,000–40,000 patients annually. Then are the patients that are off treatment because they quit treatment, for whatever reason, but mostly for inconvenience, or for side effects or for non-performance. And there is the group that is truly based on non-performance. These are the treated relapsing ones, where the physician says “Wait a moment. This doesn’t work for you. We take you off treatment and we have to wait until something better is available.” And this something better, this something new is now here. Another choice, let’s put it this way.
And then, there is, of course, the patient group that is stable on current therapies, patients that are satisfied with the current therapies. They are in the 200,000 range. So we have 200,000 patients that may or may not be tempted to switch to anything else because they are happy with their current treatment. But you have close to 400,000 patients that are unhappy, new or are quitters for whatever reason. And they could go on Tysabri.
And in the first few weeks, we saw already a very good reception amongst neurologists. First of all, we got a huge number of calls. The call volume right now is 1500 plus, incoming unsolicited calls [per day?] to Biogen Idec requesting information about Tysabri or Avonex. And of course this call volume has gone up dramatically since the approval of Tysabri. And in the meantime we also have 1600 unique MDs prescribing Tysabri. And this is as of 1/15. And remember, the drug is only on the market for about two months. The 1600, just to give you another idea of how this falls into place, the 1600 are a subgroup of the 6000 neurologists that we are targeting. Our marketing and sales philosophy is: we target those neurologists that prescribe MS, that write MS scripts. That 6000, or 35% of all neurologists. And this is our target. And 1600 of them have already written scripts.
So, we have all this to establish Tysabri as the new standard of care. And I think if you just joined this brief overview, all the elements are falling into place for this. We have the product that can achieve first-line status in the minds of the physicians and the patients. We have a product with which we can rapidly overcome barriers to treatment. I should mention most physicians are infusing in the office to date. And those that cannot infuse in the office can find readily an infusion site where they can refer the patients to. And we are building significant momentum by year end. We are on our way to becoming the market leader.
Tysabri, the two year data from our clinical studies will be available during the first half of this year. We also expect European approval during the second half of this year. …
And on the manufacturing side, we will start operations mid-year in Oceanside, our second large-scale manufacturing facility. And we broke ground for our Denmark facility.