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Proxy communication statement:
Biogen Idec and its directors and executive officers may be deemed to be participants in the
solicitation of proxies from the stockholders of Biogen Idec in connection with the companys 2010
annual meeting of stockholders. The names, affiliations and interests of such individuals may be
found in Biogen Idecs annual report on Form 10-K for the year ended December 31, 2009 and its
proxy statement for the 2009 annual meeting, each of which are filed with the SEC. To the extent
holdings of Biogen Idec securities have changed since such documents were filed, such changes have
been or will be reflected in statements of change in ownership on Forms 3 and 4 filed with the SEC.
Additional information regarding such individuals will be included in the companys proxy
statement in connection with the companys 2010 annual meeting of stockholders when such document
is filed with the sec. Biogen Idec files annual, quarterly and special reports with the SEC. The
proxy statements and other reports, when available, can be obtained free of charge at the SECs web
site at www.sec.gov or from Biogen Idec at www.biogenidec.com. Biogen Idec stockholders are advised
to read carefully the proxy statement relating to the companys 2010 annual meeting of stockholders
and any other relevant documents filed by the company with the SEC when they become available
before making any voting or investment decision, because they will contain important information.
The companys proxy statement will also be available for free by writing to Biogen Idec Inc., 14
Cambridge Center, Cambridge, MA 02142. In addition, copies of the proxy materials, including the
companys white proxy card, may be requested after they have been filed with the SEC from our proxy
solicitor, MacKenzie Partners, Inc., by toll-free telephone at 1-800-322-2885 or by e-mail at
proxy@mackenziepartners.com.
CORPORATE PARTICIPANTS
Paul Clancy
Biogen Idec Inc. Executive Vice President and Chief Financial Officer
Al Sandrock
Biogen Idec Inc. Senior Vice President, Neurology Research and Development
Bob Hamm
Biogen Idec Inc. Chief Operating Officer
PRESENTATION
Eric Schmidt Cowen & Company
I am here to welcome you all to day three of our 30th annual healthcare conference. We are
delighted that you were able to wake up bright and early and see our next presenting company,
Biogen Idec. Its always a pleasure to introduce whats clearly one of the leaders in the biotech
sector now for 15 or 20 years.
Today we have a full team of executives with us. We have Paul Clancy, the Companys Chief Financial
Officer. Paul will be giving a talk that will probably last about 20 minutes and we will have some
time immediately following that presentation in this room to take some of your questions.
During that time period, I think Paul will be joined by Bob Hamm, who is the Companys Chief
Operating Officer. I am told Al Sandrock, Senior VP of R&D, is somewhere in the room as well. And I
know Andrew Hirsch, VP of Strategy, is here too. So well have a full session after Q&A, a full
breakout session next door as well, and that breakout is going to be in the northeastern room.
Paul Clancy
Thanks, Eric. We are very pleased to be here. Its actually a short commute for us, but we think
this is a great conference, so pleased to speak on behalf of Biogen Idec.
Im going to hit briefly early on in the presentation on our 2009 performance and a little bit look
back on historical perspective for our financial results. And then I will spend the bulk most of
the time talking about our critical business drivers for the business as we go forward.
Given that we will be making forward-looking statements, I just want to start with this important
information. I will be making forward-looking statements in clearly I point you to the SEC
filings as well as our press releases on an ongoing basis.
So just to take a look back at 2009 in review, we entered the year knowing that we were going to
have some headwinds facing our business on the top line as well as the bottom line. We knew that we
actually had some expiry as it related to the rest of world royalties on RITUXAN®. We knew foreign
exchange volatility would create interesting dynamics, as well as the unemployment impact in the
United States actually put pressure in our AVONEX® business with respect to free goods.
Despite that, we had very, very solid performance. Revenue grew about 7.5% to just under $4.4
billion. That was largely driven by great performance as it relates to TYSABRI®. TYSABRI achieved
blockbuster status surpassing $1 billion of total in market sales, so a very, very good year for
TYSABRI. We grew the business 30% on the revenue front, 30% on the patient front, and we will talk
certainly a lot more about that in terms of our plans going forward.
Very importantly, we felt we made tremendous progress with respect to maturing and advancing our
late stage pipeline. And I will note and point out three key accomplishments in 2009 and we will
talk more about each of these programs later.
First, the PEGylated interferon program in June of 2009 had first patient in and we were granted
fast-track designation, so very, very excited about that program. Well talk a little bit more
about it.
Our play with respect to an oral entry in MS, BG-12, both of the trials were fully enrolled in
2009. We are now working hard to prosecute those trials.
And the third thing Id like to point out with respect to our late stage pipeline is we acquired
the ex-US rights to fampridine. We will talk more about that. But very important milestones and
achievements in 2009. With that said, on the product and pipeline performance, we also achieved
double-digit earnings growth on the bottom line.
Then in October 2009, our Board of Directors approved $1 billion share repurchase program. This was
in addition to our normal share stabilization program and we made significant progress on that,
which I will point out later in the presentation here.
So just to quickly put those 2009 results in a little bit of a framework, if you pan back a little
bit further, this is our revenue performance over the last seven years as well as our bottom line
non-GAAP earnings per share performance. It is quite strong financial results over this time
period. On a compound annual growth rate basis, revenues have grown 15% over this time period and
on the bottom line, weve gotten leverage to grow non-GAAP earnings per share 22%. Some years have
been faster on the top line, some years slower, as well as the bottom line, but very, very solid
results with six consecutive years of double-digit earnings per share growth over this time period.
And as a result during this time period, weve actually modestly diversified our revenue base on
really two dimensions, on a product dimension and then on a geographic dimension. On the left hand
side, you just kind of see the revenue stack of our products in 2003 and then in 2009 split between
AVONEX, RITUXAN revenue as well as TYSABRI. Then on the right-hand side, you see the percentage of
that.
So we have diversified our revenue basis with TYSABRI achieving blockbuster status in 2009 and
importantly one of the strategic imperatives that we had a number of years ago was really to expand
and have a geographic presence outside the United States, expand that international part of our
business, and importantly as we go forward we think we can leverage and capitalize on that with
respect to our pipeline.
So now moving forward lets talk about the critical business drivers for Biogen Idec. They really
are in this simple kind of cartoon, it really takes our business plan and reduces it down to kind
of four key quadrants. First, accelerating TYSABRI growth. Well talk about our plans as it relates
to TYSABRI and the initiatives that we have underway.
Looking at our core franchises in AVONEX and RITUXAN and continuing to grow in those two lifecycle
management. Third, prosecuting and advancing the pipeline. I think we are now at a point where we
are actually bearing the fruit of a lot of the efforts over the last number of years.
And then we are in an enviable position with respect to the cash flow generation of the Company and
certainly that puts important pressure on critical decisions as it relates to business development,
M&A strategy, and then returning cash to shareholders. And the way we think about that is making
sure that we have a disciplined use of that cash.
What I will do is now walk through these key business drivers for the balance of the presentation,
starting with TYSABRI. This chart leaves out the in-market sales that were shared between Biogen
Idec and Elan for TYSABRI. You recall in 2006 TYSABRI was brought back to the market in the United
States in mid-2006 and about that same time period, we got approval in Europe, so very, very
similar time period and we had a staged rollout, which is quite normal in Europe.
If you look at this, the third full year of TYSABRI in the marketplace has achieved blockbuster
status. We have had steady, strong growth in terms of an uptake curve both in the United States as
well as outside the United States. And in fact in 2009, sales outside the United States eclipsed
those sales inside the United States, a dynamic that we had always anticipated for a number of
years and had fully expected. And in fact, we always like to point out that the number of patients
on disease modifying therapies outside the United States has far eclipsed the number inside the
United States. So thats quite normal.
So very, very strong performance as it relates to TYSABRI over the last three plus time period.
Nevertheless, we still think theres tremendous opportunity, right? Tremendous opportunity really
can be framed up with even with this type of performance we are still at single-digit share of
market as it relates to TYSABRI. So we continue to press forward as it relates to TYSABRI and it
really is about driving forward the benefit risk profile of TYSABRI.
We have now as we talked about have three years of post-marketing experience since the
relaunch and we are continuing to learn more about the efficacy of TYSABRI as well as the safety.
When the FDA Advisory Committee recommended approval of TYSABRI, the drug was supported by the
standard efficacy metrics, reduction in relapses and reduction in disease progression. The 68%
reduction in relapse rate, the 54% reduction in disease progression. And these were unparalleled
efficacy measures.
In addition, what we have seen is that the product actually has demonstrated tremendous benefits to
patients above and beyond those standard failure-based efficacy metrics and in fact moving to ones
of improvement or migrating in that direction.
About a year ago in Lancet, a publication demonstrated 37% of patients were free in a retrospective
analysis of disease activity. So free of relapses, free of disease progression, and free of any
kind of radiological measures. So really strong and this is what we see anecdotally with the strong
efficacy of the product, that the quality of patients lives is fundamentally changing.
In terms of safety, TYSABRI is one of the most extensively monitored drugs for the treatment of MS
with one of the most robust risk management programs. We believe PML is likely the result of a
confluence of factors and we have been and continue to invest significant time evaluating all the
risk factors.
In terms of management of PML outcomes, we have seen an improvement in PML outcomes vis-a-vis the
clinical trials. And that is really the result of incredible strong clinical vigilance and the work
we are doing with respect to plasma exchange and drug screening for more effective ways of managing
PML risk.
With regard to prediction and prevention, we are exploring both viral and patient factors. There
appears to be factors specific to individual patients that may make him or her more vulnerable to
developing PML including prior treatment with immunosuppressants as well as prior exposure to the
JC virus. If were able to isolate these factors, we may be able to identify patients who are more
or less at risk for developing PML.
So this really kind of lays out a large set of initiatives that we have underway to drive future
TYSABRI growth. It spans a lot of what we just talked about in terms of stratifying patient risk to
basic blocking and tackling in terms of continuing our geographic expansion in 2010.
Specifically with respect to the JC virus antibody assay, we now are undertaking two clinical
trials, one we call STRATIFY-1, STRATIFY-2, and we have initiated STRATIFY-1 in terms of patients
into the clinical trial. Looking for JC virus incidents as well as the utility for this as a tool
for risk stratification.
We have plans underway and pretty far along with respect to evaluating a switching study. This
would be a study to evaluate taking patients that are already on disease-modifying therapy and
switching them either to TYSABRI, Rebif® or Copaxone® and understanding the clinical outcomes going
forward.
Plans are underway to expand into other indications including secondary progressive MS. A large
part of the MS population that was really a tremendous amount of unmet need. We are
looking hard at developing a subcutaneous formulation and expanding plans to expand this year in
2010 in Mexico, Brazil, Turkey, Columbia, Argentina, and Egypt.
Now moving to AVONEX, AVONEX is a tested, tried and true product for Biogen Idec. If you look here,
we have had double-digit compound annual growth rate over the last five years. It is now a $2.3
billion brand with $1.4 billion in sales in the US and the balance in the rest of world. Highlights
over the last couple of years I would just like to point out is that in August of 2009, we were
granted an additional patent on a method of use for AVONEX, which provides protection for the
product extending into 2026.
Very importantly, we initiated a first patient in with respect to our PEGylated interferon trial in
the middle of 2009 and plans were pushing forward on that trial in terms of accruals.
Our sales and marketing teams are leveraging the CHAMPIONS 10-year trial information, 10 year
results, and its really the only product in the MS space that has this length of data
demonstrating strong efficacy over a long period of time. And we have significant efforts underway
to reinvigorate the US business, which we feel from a unit perspective has underperformed our
expectations.
Weve changed around a lot of leadership as it relates both on the medical affairs as well as the
sales and marketing front and brought to bear a tremendous team. We are now working hard on
solidifying strong clinical messaging and really executing hard on a sustainable sales and
marketing plan.
Moving to RITUXAN, we have also enjoyed double-digit compound annual growth rate over a similar
time period, five plus years, and we think the area going forward is to continue to expand
indications as it relates to RITUXAN, making it the cornerstone of therapy in non-Hodgkins
lymphoma and expanding it into indications elsewhere.
We have gotten positive results on the PRIMA study for the first-line maintenance results and
expect and have plans to file for approval in 2010. Very recently, we have been granted in terms of
our label with respect to CLL. It also points out we have efforts are under way with respect to our
next-generation molecules, a humanized version of CD20 both for ocrelizumab as well as GA-101.
Roche and ourselves released a press release early this week notifying that we are suspending
dosing as it related to ocrelizumab in immunology. Its really too early to tell what plans are for
going forward. We will have to really understand that data going forward. Nevertheless GA-101
continues to move forward in the oncology setting and Phase III with respect to CLL.
Now lets transition to talk about our pipeline. Before we get into kind of a lot of the
nitty-gritty in terms of the programs, Id like to just frame up the two phases as it relates to
our R&D efforts at Biogen Idec over the last handful of years.
If you go back six or seven years when we brought Biogen and Idec together, I think we really
believed that we had insufficient pipelines to sustain further growth going into the next decade.
And as a result, the first phase as a result, subsequent to the merger of Biogen Idec, was very
focused on external growth, very focused on business development and efforts to bolster the
pipeline. If you look across our pipeline, we actually have over a third to close to half of our
pipeline that was externally sourced.
In the neurology pipeline, the great majority of that pipeline was actually externally sourced. In
the bottom right-hand quadrant of this slide, what we call just simply our emerging pipeline,
essentially all of that pipeline was externally sourced. In the oncology and immunology, one or two
programs each externally sourced. So that was a big phase is to really bring in a lot more
products into the pipeline through in-licensing, through collaborations, as well as tuck-in
acquisitions.
In mid-2006, I think we moved into a second phase in terms of our R&D leadership and our pipeline
management, and that was all about prosecuting the pipeline. We brought in talent from outside of
the Company that had expertise in drug development. We reorganized the Company around these
therapeutic areas and we worked really hard and have moved made significant progress. We are
actually now starting to bear fruits of that progress to the point that we actually have six
programs in registrational trials in 2010.
Now if you take a closer introspection of the pipeline and really look just like at a subset of the
neurology pipeline, really it highlights the strong MS franchise that we have in the pipeline, and
that ranges from the ex-US rights to fampridine as well as to anti-LINGO, which is in early stage,
now has moved into Phase I that actually has the potential to reverse the disease.
In between is obviously AVONEX and TYSABRI, which are on the market that offer AVONEX offering
great opportunities for patients for first line therapy and TYSABRI when patients need further
efficacy. We are studying PEGylated interferon, which is a real interesting play in terms of
interferon, BG-12 oral entry, as well as daclizumab which may offer a new, more novel mechanism of
action. So a real franchise is being built with respect or has been built and continues to be built
with respect to multiple sclerosis.
I would like to now kind of just highlight three or four programs that are further advanced in our
pipeline. And the first one being fampridine. In June 2009, we entered into agreement with Acorda
for the ex-US rights of fampridine. And since that time, we have really worked hard to prosecute
the filing of outside the United States. In December of 2009, we filed in the EU and recently
have filed in Canada, Switzerland, and Australia. We actually have plans to continue to file in
countries around the world, leveraging our global footprint.
We have a global neurology salesforce and as a result from a business point of view, this fits hand
in glove with our existing MS infrastructure. As a result, the investment in terms of operating
expenses going forward as we turn into 2011 is quite manageable and the business can be accretive
on both the top line and on the bottom line very, very quickly.
Prelaunch efforts are under way right now that range from pharmacoeconomic research to packaging
development to manufacturing inspection.
Second program I would like to point out is BG-12 and last year in 2009, I had pointed out earlier
we have completed the enrollment last patient in for both Phase III clinical studies DEFINE and
CONFIRM. The studies have over 2500 patients enrolled across 30 countries across 300 sites across
the world. This is our oral therapy entry into the MS market. Clearly oral is a high unmet need for
MS patients. We think we can actually compete in the emerging oral segment with the point of
differentiation being safety.
I think what I would like to just point out is FUMADERM, which is a version of oral FUMADERM has a
15-year track record in psoriasis in Germany, so if that track record can convey to BG-12, we
actually think we can compete on a safety differentiation.
Third program also in MS is the PEGylated Interferon beta program. This is another very novel
molecule, first in class compound that could offer patients a more convenient dosing option with
efficacy either equivalent or better to current interferons. If you think about it, it takes the
best attribute of AVONEX once a week and extends that to either twice a month or once a month and
it takes probably the least attractive attribute of AVONEX, intramuscular injection, and moves it
to subcutaneous injections.
So with the potential for a very well-known safety and efficacy profile, a subcutaneous injection
with dosing once or twice every month, PEGylated Interferon really has the potential to be quite
strong for Biogen Idec. Phase III trials are initiated in June 2009. The enrollments are
progressing largely as planned. And Id just like to continue to point out that we have had
regulatory agreements that this would be a one-year trial.
The last program Id like to point out is a new market for us, hemophilia. This is a case where our
R&D as well as our biologics manufacturing expertise came together to have the potential to create
significant value. In 2007, we acquired Syntonix, a small biopharmaceutical company developing
therapies using fusion protein technology. This also required significant manufacturing expertise.
As a result, Biogen Idec was a perfect fit.
The business case for us was also very strong. Hemophilia therapy is a multibillion-dollar market
with a clear unmet need around a more reasonable treatment regimen. Our goal is to take the
treatment regimen which really often required up to 150 injections a year down to close to 50
injections a year.
In October with our partner, Biovitrum, we announced the decision to move our Factor IX product
into registrational trials and late in 2009, we moved Factor VIII into the clinic also. We recently
announced a restructured collaboration which clarifies roles and decision-making between the two
parties. We will actually bear a higher R&D burden in the short term for better economics
downstream.
Lastly, I would just like to point out some highlights as it relates to our disciplined use of
cash. As noted, our Board of Directors announced in October 2009 that they had approved a $1
billion share repurchase with the objective of returning cash to shareholders. As we proceed on
this share repurchase program, shares are being retired. It was designed as an open market program
with an open-ended time period. Nevertheless, weve made significant progress in the fourth quarter
and then right through our earnings call in early 2010. And in fact, of the program we have taken
in $712 million over that time period and we think that it will be largely complete in a very short
period of time.
So to summary, four key business drivers around the Biogen Idec story, accelerating TYSABRI growth,
really driving forward both the efficacy as well as making progress on the risk profile of TYSABRI,
extending AVONEX and RITUXAN, continuing to generate cash flow of that business, advancing,
maturing, and bearing the fruits of what is becoming an interesting pipeline and ensuring that we
have a disciplined use of our cash.
I think we have time, Eric, for 10 minutes or so for questions. Al, if I would like you to come
up front and join us, so myself, Bob Hamm, our Chief Operating Officer; as well as Dr. Al Sandrock,
our Senior Vice President of Neurology R&D will be here to talk about questions.
Given that this is webcast, we will actually repeat the questions for those that are following
through the webcast.
QUESTION AND ANSWER
Unidentified Audience Member
You were mentioning stratification as important to grow the TYSABRI franchise. What is happening
spontaneously in the field as we speak?
Paul Clancy
Al, do you want to just help a little bit on that question? Come on up. I think the only place we
have a mic is up front.
So the question was with respect to the TYSABRI risk stratification efforts, what is actually
happening in the field as we talk about I think I will just editorialize and say cut across key
opinion leaders as well as kind of community neurologists?
Al Sandrock
Well I think people right now are making benefit/risk decisions for their patients and the
most common reason that people come to TYSABRI is that the first-line therapies have not been of
adequate benefit for them. And thats the key reason that people are coming now.
On the risk side, there arent a whole lot of things that people can use. Thats one of the reasons
why we are initiating these anti-JCV assay studies to provide some more tools on the risk side.
Unidentified Audience Member
Paul, theres a lot of M&A activity out there. I am wondering if you have an opinion on Facet
for $27 a share, whether you are going to share that with us.
Unidentified Audience Member
(inaudible question)
Paul Clancy
So a two-part question. Surprised it took it to the second question. But the first part of the
question was as it relates to Facet, any comments on that? And then more broadly as it relates to
augmenting our pipeline through business development efforts.
The first part of the question is, look, we welcome a new partner into the Biogen Idec fold. We
have tremendous respect for Abbott. I love traveling to Chicago, great people there. So we actually
just welcome into the fold and we will now continue the collaboration just with a new partner as we
go forward. Thats the plan assuming that their offer goes through.
As it relates to kind of business development and augmenting our pipeline, I had noted that weve
kind of gone through a couple of phases as it related to our R&D efforts. A big phase five, six,
four years ago in that time period was really a tremendous amount of efforts that was very fruitful
on the business development front, in-licensing, collaborations, and some tuck-in acquisitions.
And I think we have been a little bit spotty on that as it relates over the last couple of years
with certainly the notable exception of fampridine last year. I think thats a tremendous and it
continues to be proven out and we are very, very hopeful that that will be proven out to be a
tremendous in-licensing deal for Biogen Idec as well as for that matter for Acorda.
I think what we should be moving towards in terms of our next phase is doing them both at the same
time, bringing in on a continuing basis, replenishing the pipeline across all different parts of
the pipeline. The reality is its easier to replenish pipelines in a pre-proof of concept manner,
but I think we continue to do that and simultaneously prosecute the organic as well as the pipeline
that we brought in.
Unidentified Audience Member
Youve been working on kind of rejuvenating AVONEX sales for a while now, several months. Are there
any early signs, Bob, that you want to point us to? Any thing that you need to execute on before we
see any success?
Bob Hamm
I think we should see some benefit in the second part of the year frankly. The new teams
(inaudible) and its a matter of share voice and increasing our clinical presence (inaudible).
Unidentified Audience Member
Many of your PML cases have come from Europe. Is clinical practice in Europe changing as a result
of this?
Paul Clancy
Great question. Ill ask Al to answer the question but for folks on the webcast, the question is
the essence of it is that many of theres been a disproportionate number of patients that have
gotten PML from outside the United States, from Europe, and in fact even more specifically, country
of Germany. Do we have a point of view on that with respect to clinical practice as well?
Al Sandrock
Well, we know that at least part of the reason might be the use of immunosuppressant drugs. We know
that theres differences in clinical practice between Europe and US and the frequency that these
kind of drugs are used. That may relate in part to the risks that we are seeing.
Unidentified Audience Member
Do you see the clinical practices changing?
Al Sandrock
Well, we are not we dont see any major changes at this point but
Unidentified Audience Member
(Inaudible question microphone inaccessible)
Paul Clancy
Yes so the question is with respect to the disease modifying therapies in MS, have had price
increases over the last few years. What is our expectation going forward?
We dont plan for it essentially. So our business plan going forward is for very, very modest if
any price increases. I think thats probably reflective of the way we think about the marketplace
going forward.
Unidentified Audience Member
Al also mentioned starting some studies on TYSABRI in secondary progressive MS. What is the
rationale there and what is the timeline?
Al Sandrock
Well, we havent established the timeline yet. The rationale is that when we look at registries and
when we talk to patients, we hear that cognition is improved. When you look at registries of large
cohorts like in Sweden, cognitive measures such as the SDNT seem to improve in patients more than
we would expect from chance alone or from practice effects.
So its that kind of thing and as you know, cognition is worse in people with SPMS. Its a major
unmet need. Theres really nothing to address that. So thats one of the reasons why we are very
interested in that.
Unidentified Audience Member
(inaudible) regulatory authority?
Al Sandrock
Welcome that needs to be the question was is cognition is there an endpoint for cognition
with regulatory authorities? Theres been no precedents for drug approved for cognition in SPMS but
theres clearly precedents for other diseases including diseases that were not previously linked
very closely with cognition such as Parkinsons disease.
Thats a movement disorder but there are drugs now that are approved for cognition in Parkinsons
disease and I dont see why the same couldnt apply in the multiple sclerosis.
Unidentified Audience Member
Are you seeing steady or accelerating growth of TYSABRI sales compared to the fourth quarter?
Paul Clancy
Yes, so the question is are we seeing steady or accelerating growth compared to the fourth quarter?
In the middle of the quarter, we just as a matter of practice dont use and give investor
conferences generally to comment on our sales outlook. So apologize, but we will have to wait until
we turn to our quarterly results to kind of see the pace of that.
Eric Schmidt Cowen & Company
Is there one last question perhaps? Okay, we can go next door then.
Paul Clancy
Okay, so for those that want to, we are going to moving next door to the northeastern room, Eric,
and for further Q&A. Thank you very much.
BREAKOUT SESSION
Eric Schmidt Cowen & Company
Thank you to everyone for joining us. For those of you that werent in the session earlier, we are
joined here by the majority of the team at Biogen Idec. Weve got Paul Clancy, the CFO; Bob Hamm,
the COO; Al Sandrock, VP of Neurology, and Andrew Hirsch, who is the VP of Strategies. They would
be happy to take any questions that youve got.
Paul Clancy
So this is webcast, so we will repeat the questions. Al is actually Senior VP, so we dont want to
give him a demotion out of the gates here.
Unidentified Audience Member
A couple things we know. We know the CD20 mechanism works in NHL, CLL, it works in RA, it works in
MS. There havent been any problems with RITUXAN. We havent seen any problems yet with
GA-101. So is there an issue with ocrelizumab itself that is causing these infections in this
the second or third trial thats been halted or stopped?
Paul Clancy
Yes, so the question I will repeat questions just given that we are in a webcast is can we
glean any information as it relates to ocrelizumab vis-a-vis what we
know about RITUXANs
safety profile as well? This is really the backdrop being the news that came out earlier this week
with a press release with Roche as well as Biogen Idec around suspending dosing on the ocrelizumab
RA trials and lupus trials.
We are very early into this in terms of that, so I think that we are probably premature in trying
to jump to any conclusion. Al, if you can any further
Al Sandrock
I agree. We certainly are just beginning to look at the data. Its very complicated because theres
regional differences in where the use of ocrelizumab occurred versus its occurring now within
RITUXAN. There is just a lot of complicated factors. I think its too early to tell.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
The MS trial with ocrelizumab continues to go on. Theres an extension study from the Phase II.
Patients are being dosed in that.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Yes, well, the drug safety monitoring committee will meet and discuss the safety and the wisdom of
continuing dosing in the MS extension study. But thats a separate it is a separate benefit/risk
discussion. The concomitant meds are separate so I think it just needs to be a separate discussion
and we will wait for that.
Unidentified Audience Member
(Inaudible question)
Bob Hamm
Sure. The auto injector is simply a means to have people that really are averse to injections not
be reminded of it by seeing a long needle. So its immediate impact, withdraw, and therefore it
mitigates the concern about the intramuscular injection.
Titration is a way to mitigate the flu-like symptoms that emerge by bringing up the dose over time
and thats simply the benefit to the convenience of the patients.
Unidentified Audience Member
(Inaudible question)
Bob Hamm
Yes they are.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
The question was have we disclosed any data from the daclizumab trial. The only trial that data
have been disclosed is from the CHOICE study. It was an add on trial where daclizumab, two doses of
daclizumab are added to interferon placebo-controlled double-blind trial that was presented a
couple of ECTRIMS meetings ago by Javier Montauban and colleagues. Thats the only trial where data
have been disclosed.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Well, we believe that its going to have substantial efficacy superior to first-line therapies and
the safety profile is going to have its own unique safety profile. Its an immunomodulator, so
there will be some risks but it will be unique to daclizumab, we think.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
A design of Phase III? Well, we are actually in the process of discussing with investigators a
protocol. We have met with investigators. We have agreement with regulators on the actual design to
some extent and we are very close to initiating the trial.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Im sorry, could you repeat? I didnt quite hear it.
Eric Schmidt Cowen & Company
Can you talk about STRATIFY?
Al Sandrock
Oh, STRATIFY. So STRATIFY is the study that is being initiated right now to look at the anti-JCV
virus to get the assay done and to know whether or not there is validity and whether or not the
result of that assay is predictive of the risk of PML.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Well, if you look at the clinical trials, the Phase III trials of the interferons and Copaxone, the
majority of people have at least one relapse over the two-year trial period and many of them also
have progression of disability. So a substantial proportion of people will have at least one
relapse and some will have more than one and its not a small number.
But in the clinical trials, its about 75% or so will have a relapse.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Well, TYSABRI is generally recommended for people who have had an inadequate response.
There are no quantitative guidelines in terms of what that means. I think thats left up to the
clinician. In Europe, they are more specific.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Well, the question was about side effects with daclizumab and Im not aware of any reports of
leukemia. So just to answer your question specifically. The CHOICE study, the MS study that I just
referred to, the add-on to interferon, there was a slight increase in infections and in the
daclizumab group compared to placebo and there were some skin reactions. Thats what I recall from
Professor Montalbans presentation two years ago.
Unidentified Audience Member
(Inaudible question)
Paul Clancy
Yes, so the question was is the plan for pricing on AVONEX different today than it has
been in the past? Our plans going forward really kind of assess a very modest if any price
increases going forward. So thats the way we think about it from a business planning perspective
which probably is the best indication of the way we think about it from in terms of what our
expectations are going forward.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
The STRATIFY trials have already been initiated. STRATIFY-1 has been initiated. STRATIFY-2 is very
close to enrolling the first patient, but the other one has already enrolled.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
In terms of results, that depends on how rapidly we can get blood tests done and how many cases of
PML we get to know whether or not the blood test predicts PML. Its very hard to know exactly.
Unidentified Audience Member
(Inaudible question)
Paul Clancy
Yes, so the question is can we comment on the fampridine regulatory process? We have filed for
approval with the EMEA. Actually it was December 2009. I think we announced it early in one of the
investor conferences in the first or second week of January. Weve additionally filed in Canada,
Australia, as well as Switzerland, and have plans to actually file in other countries throughout
the year.
So we think its it has been the first order of business subsequent to entering into the
agreement with Acorda for us in terms of our activities is to work hard on getting filings under
way and actually weve beat our timetables.
With respect to the second part of the the active comparator .
Al Sandrock
Well, this is a first in class molecule and its a functional modifier. There is no other drug
approved for functional modification in MS. So there is no comparable drug to compare with.
Unidentified Audience Member
When is the launch?
Paul Clancy
We dont know. Its up to obviously EMEA, but generally speaking kind of its probably not
our planning contemplates kind of a 12 to 15 month time period.
Unidentified Audience Member
(Inaudible question)
Paul Clancy
So the question is do we know how many patients in are taking the compounded version dont? We
actually dont have insight into that at this point.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
So the question was whether we can use retrospective samples that we collected from other studies.
And in fact, thats what we did. We were lucky enough to have 11 blood samples one to three years
prior to the diagnosis of PML. These were collected either within our own extension studies or they
were collected in the context of registries. And all 11 were positive for the JCV antibody
according to our assay. And as you know, about 50% of people would be expected to be positive.
So for that reason we think theres interesting preliminary data and thats why were initiating
these STRATIFY-1 and STRATIFY-2 studies.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
No, because most people dont collect blood and store them in practice in clinical practice. So
these were we were just lucky that some people had collected blood in the context of registries
because for example in Sweden, they are very interested in biomarkers of MS. So they happen to
collect blood and we were able to pull it out of the bank and test.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Yes, yes, so the question is what about all the patient do you have samples from people? Yes, we
have looked thats how we got our 53.7% seroprevalence rate. We looked at about 900 850 to
900 patients from our one of our long-term MS studies. We have other studies that we are
currently also looking at to confirm what we saw and fortunately, it was from a broad geographical
distribution as well. And we had longitudinal samples. So thats where we got the data that gave us
some confidence that the assay had analytical validity.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Yes, I believe they were all prior to TYSABRI. They were clearly one to three years prior
to the onset of PML and in most cases, I think all, it was before TYSABRI.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
The current design is a superiority head-to-head trial monotherapy.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Yes.
Unidentified Audience Member
(Inaudible question)
Al Sandrock
Yes.
Paul Clancy
Any Red Sox questions? Okay, if there are no further questions, thank you all for attending.