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Biogen IDEC Inc (BIIB)

October 26, 2011 8:00 am ET

Executives

Alfred Sandrock -

Douglas Edward Williams - Executive Vice President of Research and Development

Unknown Executive -

Kate Dawson -

Kia Khaleghpour - Associate Director of Investor Relations

Analysts

Sapna Srivastava - Goldman Sachs Group Inc., Research Division

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Matthew Roden - UBS Investment Bank, Research Division

John S. Sonnier - William Blair & Company L.L.C., Research Division

Mark J. Schoenebaum - ISI Group Inc., Research Division

Rachel L. McMinn - BofA Merrill Lynch, Research Division

Robyn Karnauskas - Deutsche Bank AG, Research Division

Geoffrey C. Porges - Sanford C. Bernstein & Co., LLC., Research Division

Yaron Werber - Citigroup Inc, Research Division

Eric Schmidt - Cowen and Company, LLC, Research Division

Joshua Schimmer - Leerink Swann LLC, Research Division

Ravi Mehrotra - Crédit Suisse AG, Research Division

Geoffrey C Meacham - JP Morgan Chase & Co, Research Division

Maged S. Shenouda - Stifel, Nicolaus & Co., Inc., Research Division

Thomas Wei - Jefferies & Company, Inc., Research Division

M. Ian Somaiya - Piper Jaffray Companies, Research Division

Gene Mack - Mizuho Securities USA Inc., Research Division

Operator

Good morning. My name is Champagne, and I will be your conference operator today. At this time, I would like to welcome everyone to the Biogen Idec BG-12 Conference Call. [Operator Instructions] I would now like to turn the call over to Ms. Kia Khaleghpour. Ma'am you may begin your conference.

Kia Khaleghpour

Good morning, and thank you for joining us on today's call. Before we begin I encourage everyone to go to the Investors section of biogenidec.com to find the press release that follows the discussions related to this call. As usual we'll start with the Safe Harbor Statement. Comments made in this conference call include forward-looking statements, including statements about the development and commercialization of BG-12 that are subject to risks and uncertainties. Words such as believe, expects, may, plan, will or similar expressions are intended to identify such statements. Actual results could differ materially from our expectations and you should carefully review the risks and uncertainties that are described in the Risk Factors section of the most recent annual and quarterly reports filed with the SEC. We do not undertake any obligation to publicly update any forward-looking statements. As a reminder, this call is only 30 minutes in duration.

Today on the call I'm joined by Dr. Doug Williams, Executive Vice President of Research and Development; and for the Q&A portion of the call we are joined by Dr. Al Sandrock, Senior Vice President of Development; and Dr. Kate Dawson, Senior Director of Medical Research. Dr. George Scangos and Paul Clancy are also joining us for this call. With that I'll now pass the call over to Doug.

Douglas Edward Williams

Thank you, Kia. And thank you, all, for joining us this morning. Earlier this morning, we announced positive top line results from CONFIRM, the second of 2 pivotal Phase III clinical trials designed to evaluate the investigational oral compound BG-12 in people with relapsing-remitting MS. We're excited about the results and believe that the results from CONFIRM do confirm the strong efficacy and safety seen in the DEFINE study, recently reported at ACTRIMS.

In CONFIRM, BG-12 dosed either twice a day or 3x a day met the study's primary end point, and showed significant efficacy in secondary end points with a favorable safety and tolerability profile. Before we recap CONFIRM's top line results, I'd like to take this opportunity to remind everyone of the design of the trial. CONFIRM was a global double-blind placebo controlled study in more than 1,400 relapsing-remitting MS patients.

The study evaluated 2 doses of BG-12, 240 milligrams twice and 3x daily in comparison with placebo. It also included a reference comparator of glatiramer acetate at a dose of 20 milligrams daily subcutaneously versus placebo. The primary end point was to determine whether BG-12 was effective in reducing the annualized relapse rate at 2 years.

Secondary end points at 2 years included measurements of MRI lesions, the proportion of patients who relapsed, disability progression of 12 weeks confirmed as determined by the Expanded Disability Status Scale as well as safety and tolerability of BG-12. CONFIRM top line results showed that 240 milligrams of BG-12 administered either twice daily or 3x daily met the study's primary end point by significantly reducing annualized relapse rate at 2 years versus placebo by 44% and by 51% with a P value of less than 0.0001.

We think these results are robust and validate results from the DEFINE study. The reference comparator group of glatiramer acetate reduced ARR by 29% versus placebo with a P value less than 0.02. In addition to significantly reducing ARR, the BID and TID doses of BG-12 also provided a 71% and 73% reduction in the number of new or newly enlarging T2-hyperintense lesions versus placebo.

The P value for both doses was highly statistically significant. Glatiramer acetate provided a 54% reduction compared to placebo. For reduction in new T1-hypointense lesions, there was also statistically significant reductions of 57% and 65% in the BID and TID arms versus placebo, respectively. Glatiramer acetate showed a 41% reduction versus placebo. With respect to the secondary end points, of the proportion of patients who relapsed to 2 years, BG-12 showed a statistically significant reduction in the proportion of patients who relapsed to 2 years. 34% for the BID arm and 45% for the TID arm versus placebo.

While glatiramer acetate showed a 29% reduction versus placebo. Initial results showed that BG-12 reduced 12 week confirmed disability progression at 2 years by 21% for BID and 24% for TID and 7% for glatiramer acetate versus placebo. While these results were not statistically significant, they may be explained by an unexpectedly low rate of disease progression in the placebo group, which was roughly half of what has been seen in MS trials of approved and experimental therapies, including the results seen in DEFINE, further analysis of this end point is underway.

With respect to safety, both dose regimens of BG-12 showed favorable safety and tolerability profiles. Overall incidence of adverse events, serious adverse events, including serious infections and discontinuations due to AEs, were similar between placebo and BG-12 treated arms. The most common AEs in the BG-12 groups were flushing and GI events. The overall incidence of hepatic and renal events was comparable on an all study groups, including placebo. There were no malignancies in the BG-12 group.

We're gratified by the strong efficacy and safety results, which when combined with BG-12's oral route of administration, position it as an important potential therapy for MS patients. We now have strong positive results for BG-12 in 2 robust pivotal clinical trials with more than 2,600 patients. We believe that BG-12 has the potential to become an effective treatment option for people living with MS.

We're committed to working with regulatory officials to expeditiously bring BG-12 to patients who need a therapy that combines strong efficacy and a solid safety profile with the ease of oral administration. We anticipate presenting detailed data from CONFIRM at a future medical meeting and a filing is on track for the first half of next year. I'll now turn it back to Kia.

Kia Khaleghpour

[Operator Instructions] Operator we're ready to open up the call for Q&A. [Operator Instructions] Operator we're ready for the first question.

Question-and-Answer Session

Operator

[Operator Instructions] Your first question is from the line of Eric Schmidt from Cowen and Company.

Eric Schmidt - Cowen and Company, LLC, Research Division

A question on the EDSS end point and the miss in statistical significance there, is there any precedent for filing on a single pivotal trial that hit EDSS and do you expect therefore to get a claim for disability?

Douglas Edward Williams

Well, in the past, certainly for the AVCRs there was only 1 pivotal trial that led to approval. In the case of TYSABRI both the combination trial and the monotherapy trial were positive on all end points, including EDSS. And for Gilenya, there was only 1 positive trial with EDSS being positive and the other trial showed no difference compared to Avonex on EDSS. So that's the precedence for approved drugs as far as I'm aware and leave it at that.

Unknown Executive

And I think the follow up to your question is that we will certainly look to get labeling for EDSS based on the data we've seen in the DEFINE study and now even though we missed on statistical significance, we were directionally moving in the right direction with the CONFIRM study as well. So we'll certainly seek that in the labeling.

Eric Schmidt - Cowen and Company, LLC, Research Division

But was it a close miss in terms of p value?

Douglas Edward Williams

Yes, I think we'll release additional data on that at future medical meetings.

Operator

Your next question is from the line of Robyn Karnauskas.

Robyn Karnauskas - Deutsche Bank AG, Research Division

If only if you maybe could discuss a little bit about the TID versus BID arms, there were, it does look like there was some differences between DEFINE and CONFIRM, and what you might expect in the label in the real world, for real world use.

Douglas Edward Williams

Yes, I think there were some differences. I think our conclusion, looking across both studies, is that both TID and BID provide approximately comparable results. We don't really see that there's a huge or meaningful difference between either dosing regimen based on the aggregate data we've seen. So we think that BID dosing for this drug will be appropriate.

Operator

Your next question is from the line of Rachel from Bank of America.

Rachel L. McMinn - BofA Merrill Lynch, Research Division

Yes. Can you just talk about the placebo rates being different than historical on disability? Can you talk about what the placebo rate, how we should think about that on relapse rate is that similar to other studies?

Douglas Edward Williams

Relapse rates is similar to other studies. I think it's only in the case of EDSS, where we're seeing what would be characterized as something different than what's been seen before. It's a lower rate than what's been seen in other studies and we're still doing analysis to try to understand what's behind that.

Alfred Sandrock

Yes. The relapse rate in the placebo group in CONFIRM was similar to DEFINE and similar to all sort of all the other contemporary MS trials. Of course, the contemporary MS trials were lower than the trials that were done 15 to 20 years ago but it's very similar to the contemporary MS trials.

Operator

Your next question is from the line of Geoff Meacham from JPMorgan.

Geoffrey C Meacham - JP Morgan Chase & Co, Research Division

Can you speak to -- just a follow up, can you speak to the baseline differences between DEFINE and CONFIRM with respect to disability? It sounds like there were differences but maybe not differences with regards to the number of lesions or years since diagnosis?

Douglas Edward Williams

We're still in the early stages of sort of evaluating all these results. I can tell you that on the first pass the populations look very, very similar between DEFINE and CONFIRM in terms of baseline characteristics. So we're looking into that some more now.

Operator

Your next question is from the line of Mark Schoenebaum.

Mark J. Schoenebaum - ISI Group Inc., Research Division

I was just wondering, I've got a bunch of questions, but I know you only want to limit to one, so I'll try and do that. There's been this controversy I guess on, at least on Wall Street, that there are design elements in the trial that may have influenced the outcome such as the re-consenting of patients. I'd be curious to get Al's feedback on this. It looks like it's pretty typical amount of MS trials, but I'd be curious just to get your feedback on that whether you think that had any influence on any of the arms in the trial, et cetera. And then if I can slip in a second question, you don't have to answer, but I'd just be curious right now as you look at all this data how you sort of see BG-12's profile stacking up versus Gilenya in the general sense, I'd be curious as to your thoughts. If you want to answer that, thanks.

Alfred Sandrock

For the first question, the re-consenting of patients is pretty typical of modern MS trials. We filed the guidance, the published guidance by Paul Mann [ph] colleagues on how to contact placebo trials, placebo controlled trials ethically. And so if you look at the modern MS trials, I think all of them actually re-consent patients, if not the vast majority because that's the guideline. Of note, even though we re-consent patients and do these things today, if you look at the Copaxone group compared to placebo in CONFIRM and also look at the Avonex group in the recent laquinimod trial compared to placebo, those 2 relapse rate effects look pretty comparable to the results that we saw 15 years ago. So even though we do re-consent patients based on these new guidelines, the old drugs still look kind of like what they did in the old trials. So I think in the second question, I think I'm probably not going to answer that question because I think we're still in the early stages. I'm very -- I think that the benefit risk profile of BG-12 looks quite attractive and it's an oral and I would hazard to guess that it looks like a first line therapy to me.

Operator

Your next question is from the line of Michael Yee.

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Actually do you have any information on brain atrophy whether in DEFINE, which represented or in the CONFIRM?

Alfred Sandrock

We're still -- we just filed the results a few days ago for the first time. We haven't gotten to the tertiary end points yet. We're still looking at primary and secondary end points and being sure we understand those results. So I'd like to defer those discussions for later.

Operator

Your next question is from the line of Gene Mack.

Gene Mack - Mizuho Securities USA Inc., Research Division

I'm wondering if you could just talk a little bit about the flush in the trial. Is that pretty much the same quality, characteristics, as DEFINE? Most patients [indiscernible] in the first month and then dissipating or is there anything else that significantly differs than what you have seen in the DEFINE trials?

Kate Dawson

Yes, I think I can speak to that. With respect to the CONFIRM study, all we have now for the first look is the incidents and the incidence rate of the flushing is very similar to what we saw in DEFINE. So we know from DEFINE and that data that we did see the incidence decreased dramatically over the first month. So we're very encouraged by that and we will look at this further when we get more additional information from CONFIRM.

Operator

Your next question is from the line of John Sonnier from William Blair.

John S. Sonnier - William Blair & Company L.L.C., Research Division

You talked a bit in the past about the potential neuroprotective effects of BG-12. Can you comment on whether or not you look to the molecular markers of neuroprotection such as Nrf2 expression and if so any observations in the CONFIRM study?

Douglas Edward Williams

In this particular study, no, there were no sort of companion studies that would look directly at that. I think there's loads of experimental data out there that supports the notion that the mechanism is through Nrf2 activation. We presented that, actually at ECTRIMS there was quite a bit of data that really bolstered that case. But no, there's nothing specific in this trial that would allow us to directly address that.

Operator

Your next question is from the line of Thomas Wei from Jefferies.

Thomas Wei - Jefferies & Company, Inc., Research Division

Just wanted to know on the proportion of patients who relapsed, that data, it doesn't actually look that much different between Copaxone and BG-12 twice-daily. But there was a relatively wide numerical difference on an annualized relapse rate reduction. Can you just help us understand how we should reconcile those 2 data points with each other?

Alfred Sandrock

Well, we said that the numbers were 34% for BID and 45% for TID. We're still actually trying to figure out the answer to your question. It's pretty complicated I think and we're just starting to digest that information now.

Operator

Your next question is from the line of Yaron Werber from Citi.

Yaron Werber - Citigroup Inc, Research Division

Just a quick question, so we understand a little bit. In Europe, what's your best guess as to, and in the U.S. for that matter, on the review time line, is there any chance this could be an accelerated review or is this going to be a normal review?

Douglas Edward Williams

Well, I think we would certainly hope to get a priority review for this, but until such time as we know what the answer to that question is, we really can't comment any further than that.

Yaron Werber - Citigroup Inc, Research Division

Okay. And just remind us, you do not have an orphan drug status for this, right?

Douglas Edward Williams

We do not.

Operator

Your next question is from the line of Ravi Mehrotra.

Ravi Mehrotra - Crédit Suisse AG, Research Division

Perhaps I could just push you on [indiscernible] of EDSS, I'll appreciate if you can't answer this but I'll appreciate if you did. A question on EDSS and disability. Can you give us any color on, for example, the baseline EDSS goal was that around 2.5 level that we saw in DEFINE?

Douglas Edward Williams

Yes, it's about that. It's basically very similar. So it's about that same level, yes, at baseline. But if you look at the 2 years, it's 1/2 the percentage of people who progressed by the end of 2 years was about 1/2 of what we saw in DEFINE and 1/2 what people see in other trials. But the baselines, they were pretty comparable to DEFINE.

Ravi Mehrotra - Crédit Suisse AG, Research Division

Okay. And the definition of EDSS was exactly as it was in DEFINE, right?

Douglas Edward Williams

Exactly. The training was the same. Everything -- the rating of the EDSS was defined and done at the sites in exactly the same way as DEFINE.

Ravi Mehrotra - Crédit Suisse AG, Research Division

Okay. So a change of above 1 sustained for 12 weeks?

Douglas Edward Williams

That's right.

Operator

Your next question is from the line of Matt Roden from UBS.

Matthew Roden - UBS Investment Bank, Research Division

The question I have is on direct comparisons between BG-12 and Copaxone. As a proxy, if you look at the BRAVO trial, which I think the full data was just presented at ECTRIMS, they did not report any statistics between the comparator arm and the active. So I'm wondering are we ever going to get that direct comparison and I guess the follow on for that is the perception of neurologists, the feedback we got at ECTRIMS was that neurologists, from the basis of DEFINE, have perceived BG-12 to be more efficacious than the AVCRs -- so can you speak to how you expect this to play out as you transition from clinical to the commercial arena, how this drug is going to be sort of perceived among the community?

Douglas Edward Williams

I guess it's hard to make the jump to how the community's going to perceive beyond the fact that you can take a look at the data and the numbers are the numbers. I think from our perspective, we've sort of confirmed the label number for Copaxone in terms of ARR results and as Al pointed out, that’s similar to what we've seen for Avonex and laquinimod studies. So the old drugs are behaving like the old drugs and I think what we've seen with BG-12 and both DEFINE and CONFIRM is a profile that in terms of both safety and efficacy, I think, speaks for itself.

Matthew Roden - UBS Investment Bank, Research Division

But will we get the direct comparisons statistically?

Douglas Edward Williams

Again, the intent of the study was never to do that direct comparison. So I think the numbers are the numbers.

Operator

Your next question is from the line of Josh Schimmer.

Joshua Schimmer - Leerink Swann LLC, Research Division

How do you envision that study or combination of other drugs positioning BG-12 in the market at that point?

Alfred Sandrock

Could you repeat the question? We only got like the last half of it.

Joshua Schimmer - Leerink Swann LLC, Research Division

I'm sorry. If you could give us an update on timelines for the EXPLORER study and how you think results may affect the positioning of BG-12 in the market.

Kate Dawson

So I can at least address the first part of your question, the EXPLORER study is fully ongoing and fully enrolled, and we look forward to that data next year.

Douglas Edward Williams

And I think in terms of positioning the product and doing additional combination studies, I think, based on the data we've seen, we're sort of questioning the need to really look at combinations or the desire to do that, I think as a single agent BG-12 performs very, very well in terms of both safety and efficacy. And I think we'll take a look at that data from the study and then re-evaluate what we see and whether there's a path forward with the combinations.

Operator

Your next question is from the line of Geoff Porges.

Geoffrey C. Porges - Sanford C. Bernstein & Co., LLC., Research Division

A question I had was the discontinuation rate in the arms. Was it similar to DEFINE in all of the arms or are there differences now that you had an injectable in the study?

Alfred Sandrock

The top line results look very similar in terms of the rates compared to DEFINE.

Douglas Edward Williams

For us, all arms.

Operator

Your next question is from the line of Ian Somaiya.

M. Ian Somaiya - Piper Jaffray Companies, Research Division

We're seeing fairly modest rate of active switching from the AVCRs to Gilenya. Can you just comment on BG-12, the data we're seeing on whether that could elicit a higher rate of active switching or what trials might need to be done to support that?

Douglas Edward Williams

I think based on the profile we've seen so far, we sort of look at BG-12 in the context of the overall benefit risk and we think in terms of as Al commented, we see this as potentially being front-line therapy based on the profile we've seen so far. We think this will be a drug for which patients will potentially want to switch to because the aggregate profile of the drug as we see it today from a benefit risk perspective compares really favorably to anything else that's out there.

Operator

Your next question is from the line of Maged Shenouda.

Maged S. Shenouda - Stifel, Nicolaus & Co., Inc., Research Division

So you'd previously indicated that you'd be moving to possibly position TYSABRI further as a front-line agent in JCV antibody negative patients. Can you help us with the planned positioning relative to BG-12 in these data?

Kia Khaleghpour

Maged, can we keep the questions just to the BG-12 and the CONFIRM study for today?

Operator

Your next question is from Sapna Srivastava.

Sapna Srivastava - Goldman Sachs Group Inc., Research Division

I apologize but most of my questions have been answered but I just wanted to get clarity on, is it possible to show that BG-12 was superior to Copaxone in the study, given the BID difference that we have seen on the efficacy rate, is the powering sufficient for that?

Douglas Edward Williams

The study was never powered to compare BG-12 to Copaxone. And I think trying to do that with the P value sort of overuses P value in some ways, because it was never designed or powered, it was really just as a reference comparator and the numbers speak for themselves.

Sapna Srivastava - Goldman Sachs Group Inc., Research Division

Right, but considering they have the same reference arm is it possible to do that or not?

Douglas Edward Williams

I'm sure it's possible, but it's probably not appropriate.

Kia Khaleghpour

Operator, I think that we're going to end our conference call today. Thank you for your participation in today's call. You may now disconnect.

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