Matthew Calistri - Biogen, Inc. Michel Vounatsos - Biogen, Inc. Michael D. Ehlers, M.D., Ph.D. - Biogen, Inc. Alfred W. Sandrock, Jr., M.D., Ph.D. - Biogen, Inc. Paul McKenzie - Biogen, Inc..

Umer Raffat - Evercore Group LLC Geoffrey Meacham - Barclays Capital, Inc. Eric Schmidt - Cowen and Co. LLC Ying Huang - Bank of America Merrill Lynch Cory W. Kasimov - JPMorgan Securities LLC Geoffrey C. Porges - Leerink Partners LLC Carter Gould - UBS Securities LLC Matthew K. Harrison - Morgan Stanley & Co. LLC Terence Flynn - Goldman Sachs & Co.

LLC Aharon Gal - Sanford C. Bernstein & Co. LLC Michael J. Yee - Jefferies LLC.

Good morning. My name is Dan, and I will be your conference operator today. At this time, I would like to welcome everyone to the Biogen third quarter 2017 financial results and business update. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. Thank you.

I would now like to turn the conference over to Mr. Matt Calistri, Vice President, Investor Relations. You may begin your conference..

Thanks, Dan. Thank you and welcome to Biogen's third quarter 2017 earnings conference call. Before we begin, I encourage everyone to go to the Investors section of biogen.com to find the press release and related financial tables, including a reconciliation of the GAAP to non-GAAP financial measures that we'll discuss today.

Our GAAP financials are provided in Tables 1 and 2. Table 3 includes a reconciliation of our GAAP to non-GAAP financial results. We believe non-GAAP financial results better represent the ongoing economics of our business and reflect how we manage the business internally.

We have also posted slides on our website that follow the discussions related to this call. I would like to point out that we will be making forward-looking statements, which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our actual results may differ materially.

I encourage you to consult the risk factors discussed in our SEC filings for additional detail. On today's call, I'm joined by our Chief Executive Officer, Michel Vounatsos, and Dr. Michael Ehlers, EVP of Research and Development. We will also be joined for the Q&A portion of the call by our Chief Medical Officer, Dr. Al Sandrock, and Dr.

Paul McKenzie, EVP of Pharmaceutical Operations and Technology. Before I conclude, I would also like to note that, starting with the Q1 2018 earnings call, Biogen intends to cease publishing press releases related to future earnings calls, earnings releases, and investor events via newswire services.

We will post these materials on the Investors section of Biogen's website, www.biogen.com, and issue a statement on Twitter when they become available. Our Twitter handle is @biogen. Now, I will turn the call over to Michel..

optimizing partnerships to better position us for new growth; advancing R&D candidates; actively looking for pipeline assets; enhancing our BD capabilities; and further building our senior management team. Before I turn it over to Mike, a few highlights; first, we renegotiated our agreement with our partner, Eisai.

Biogen will now receive 55% of profits in the U.S., 68.5% of the profits in Europe, and 20% of the profits in Japan. This leverages Biogen's strong presence in the U.S. and Europe and Eisai's distinct advantage in Asian markets. Eisai also exercised its option to jointly develop and commercialize adalimumab.

We are pleased by this demonstration of Eisai's confidence for our lead Phase 3 Alzheimer's assets. We also agreed to decrease our royalty obligations to Neurimmune. We believe both of these new agreements will improve the long-term economics of adalimumab.

Second, we announced new data for the long-term extension of our ongoing Phase 1b study on aducanumab. We are encouraged by these results, as they are consistent with our previous reported data and support to the design of our ongoing Phase 3 studies.

We will present detailed data from this analysis at the CTAD [Clinical Trials on Alzheimer's Disease] conference next week, and Mike will provide more details shortly. Third, we advanced multiple assets in our pipeline. We completed enrollment for natalizumab in stroke and BIIB054 in Parkinson's disease.

We initiated trials for natalizumab in epilepsy for our tau ASO in Alzheimer's disease in collaboration with Ionis and for opicinumab in MS. And we had a positive readout of STX-100 in idiopathic pulmonary fibrosis. Finally, we continued to build our new senior management team, which we believe is critical to maintaining this momentum.

We could not become the leader in neuroscience without our world-class talents. Camille Lee has joined Biogen as Senior Vice President, Alzheimer's Therapeutic Area. Dr. Sanjay Jariwala has joined as Senior Vice President, Worldwide Medical. Anabella Villalobos has joined as Senior Vice President of Biotherapeutic Medicinal Science.

In addition, we are progressing well in our search for a new CFO and hope to make an announcement in the near future. I will now turn the call over to Mike for an update on our recent progress in R&D..

continued leadership in MS, including remyelination; a growing body of clinical data within SMA; an industry-leading Alzheimer's disease portfolio; a compelling program in neuropathic pain; a unique clinical portfolio in stroke; defining new mechanisms of biology in IPF; advancing assets in Parkinson's disease, epilepsy, and ALS; and a growing number of high-quality drug candidates reaching the clinic.

I will now pass the call back to Michel..

BAN2401 in Alzheimer's disease; natalizumab in stroke; BIIB067 for ALS; BIIB074 for painful lumbosacral radiculopathy; BIIB054 in Parkinson's; and our gene therapy program for XLRS.

Before we go into Q&A, I would like to thank our employees around the world who are dedicated to making a positive impact on patients' lives and all of the physicians, caregivers, and participants in our clinical development programs. The past and future achievements could not be realized without their passion and commitment.

With that, we will open the call for questions..

Your first question comes from the line of Umer Raffat with Evercore. Please go ahead..

Hi, guys. Thank you so much for taking my question. I'll leave out SPINRAZA patient count stuff for the rest of the call but, Michel, I actually wanted to focus on aducanumab for a minute, and two parts to that.

First, I just wanted to think through what the $150 million payment to Neurimmune on the royalty reduction implies for the valuation of aducanumab. And the reason why I ask is because the implied valuation for aducanumab based on this $150 million payment is very different than what the market implies.

So I was just curious and just wanted to understand the context of this negotiation a bit better. And then also on aducanumab, I was just curious.

How much of your R&D spend is going towards aducanumab right now? And I ask because of the amount of payments Eisai is going to have to make starting 2019, whether that's appropriately reflected in Street expectations for your R&D spend in 2019 onwards or not. Thank you..

So thank you for the good questions. We are very pleased with these new agreements. We believe in the potential opportunity with aducanumab, and therefore, we act in alignment with this belief. And this took a lot of work, and it's probably the most important alliance will be the agreement we reached in 2017 so far, so I think this is very important.

For the $150 million, we are working on our long-term – long-range operating plan, and we remain, with the right assumptions, very bullish on the opportunity that we have with this product. With Neurimmune, we have a long-term collaboration and there are many things in play, in the past, in the present, and in the future.

And I will say it's logical that working together we achieved this agreement between the two companies. So I would not go much further in terms of the speculation of the potential link, but I think that it's very positive for the overall future profitability of aducanumab if we're able to succeed. So we are pleased with this outcome.

Concerning the clinical spend, the only thing I can tell you is that as for the time Eisai starts to contribute meaningfully to the clinical spend in 2019, a large portion of the overall spend will need to occur in 2019 and beyond. Does somebody want to add something, okay..

Your next question comes from the line of Geoff Meacham with Barclays. Please go ahead..

Good morning, guys. Thanks for the question. I want to get some context for SPINRAZA just for the U.S. Obviously, it's flattish in Q3 and you're also saying that, Michel, for Q4, are you guys seeing discontinuations or difficulties in getting reimbursement, or is this just wave two in the U.S.

is more difficult than putting on wave one, which would be the worst patients, the most severe patients? Thank you..

Thanks for the question. We are very pleased with the momentum in the U.S. And having 75% patient growth is not a simple achievement, so I compliment the team.

As expected and also as difficult to model as it can be, based on the dosing regimen, the impact of the patients we got in Q1 and on the early access program was fully reflected in the Q3 because of the dosing regimen.

Over time this will normalize, and that's why we are prudent the way we have been prudent since the beginning of the year, if you'll recall well, so we expect that the dynamic will normalize as the maintenance dose becomes the majority of the revenue, so this is basically the momentum we see. We enlarged the reach.

We see a larger proportion of the patients above the age of 15.

Meeting with scientific leaders, they are getting more experience on complicated spine in order to dose the patients, and they are investigating potential routes of administration and procedure for patients more complicated, other one with the fusion, and those ones are the more complicated.

And it is anticipated, just to come back because I know that everybody has that in their mind, so yes, we said 60% of the patients have a complicated spine or mostly the patients with the Type 2 and Type 3, mostly the Type 2. But out of this proportion, it's actually between 10% and 20% of those patients who have fusion that are more complex.

The others are easier. And apparently, according to scientific leaders, the proportion of fusion ex-U.S. is much lower, just to give some context. So we remain optimistic on the potential of SPINRAZA in the U.S. and ex-U.S., and our peak sales objective did not change..

Your next question comes from the line of Eric Schmidt with Cowen and Company. Please go ahead..

Thanks for taking the question. I guess I'm just having the same problem with squaring the circle here on SPINRAZA. It sounds like you did very well with new patient adds, a 75% increase in number of patients on therapy from end of Q2 to end of Q3, and a variety – a large majority of those patients had to receive multiple doses.

So I guess maybe not to ask the same question a different way, Michel, but how is it that you can't get growth with that many new patients being added here? There must be some change to gross-to-net or free drug or something of that sort..

The free drug, unless I make a mistake, was 20%. This is correct, so there was a slight increase but not a major impact. Again, it's the 75% patient growth that was dosed, 75% of patient growth that were dosed with the initial dosing regimen and very little of patients with the maintenance dose. This is the dynamic that we see.

It is clear that for the later onset of disease and more complicated patients, it takes a bit more time to get those. That's why we have hundreds of start forms that are not yet for patients who are not yet dosed. So this is another dynamic that is not new. But again, as I said, the infrastructure and the system is adjusting..

Do you expect growth in Q1?.

I will not comment yet on Q1, and the last element that we saw was an inventory play between Q2 and Q3. Thanks, Eric..

Your next question comes from the line of Ying Huang with Bank of America Merill Lynch. Please go ahead..

Hi, good morning. Thanks for taking my question. Maybe, Michel, you talked about the formulary access for MS products.

Can you talk on a high level what your expectation is for 2018 net pricing for TECFIDERA and also the other products? And then maybe another question is with Eisai just opting in for aducanumab, and we know that the BAN2401 program is coming to an end, did Eisai have any clarity on the analysis of the results from the 12-month follow-up before they opted in? Thank you..

So I will get started on the market access for 2018, and that is now almost under contract. And as communicated, we are very pleased with the progress for the U.S. And the gross-to-net impact is similar to prior years and the typical contracting arrangements for which our leadership position is helping, as you can imagine.

So we didn't see a dramatic erosion in order to maintain or enhance. We saw a very limited one actually. So we are pleased with the momentum, including open formularies and including the win with some major payers like Aetna. So this is an important achievement..

Ying, this is Al Sandrock. In terms of what Eisai saw, they saw all of the same things we've seen so far, which include data from the Phase 1b trial that Mike talked about that we'll be presenting next week, which is the 36-month follow-up in the fixed-dose cohorts and the 24-month follow-up in the titration cohorts..

Your next question comes from the line of Cory Kasimov with JPMorgan. Please go ahead..

Hey, good morning, guys, and thank you for taking my question. I'll keep it to one. Are you surprised by the relative strength of SPINRAZA overseas? I guess is there anything noticeably different relative to the U.S. launch that you've seen over there in these early days? Thanks..

Thanks for the question. We are very pleased. And as communicated previously, we did anticipate that, unlike MS, we see that for SPINRAZA, mostly driven by the prevalence of the disease. We see a higher proportion of opportunities in terms of top line revenue ex-U.S. and U.S.

So we are pleased with the early launch countries, Germany, a couple of Nordics countries, Japan since a few weeks. But this was mostly driven by Germany and some named patient sales in the Middle East, in Turkey, and in Latin America. Germany is going very strong.

We got the journal official in Italy, and you'll recall that we had more than 100 – close to 120 patients on the early access program.

And overall, maybe one element that is helping also the momentum in Europe, or ex-U.S., was the very large number of early access program patients that were already identified just waiting to gain reimbursement in order to be shifted to commercial goods.

So obviously now, the question is, are we anticipating the same trends that basically many of you have anticipated as opposed – not just linear growth on a country basis ex-U.S.? We believe that based on the sequence of access, reimbursement, and unlocking opportunities in different countries that is not at the same time that this trend will not be the same as in the U.S.

I think that this will be a smoother trend in the right direction. And again, overall, it's very nice momentum, and we are trending above what we thought were our internal targets..

Cory, just to put some numbers on it, we had more than 500 patients in the early access program across 18 countries in Europe. That compares to about 60 patients we had in the U.S., probably due to timing a little between when we saw the data and when the drug was approved, so a lot more interest in the EAP in Europe..

Your next question comes from the line of Geoffrey Porges with Leerink. Please go ahead..

Thank you very much, a quick clarification for Al, if I could, and then a question, Michel, for you.

Al, on the intrathecal ASO for Alzheimer's, could you just talk about the dosing frequency and the access there in terms of being able to deliver the treatment? And then, Michel, could you talk about your policy on pricing? Of course, pricing has been a significant driver to the MS category overall in prior years, and then the company changed the policy this year, and you've taken single-digit price increases on a one-time basis this year.

Is that what we should be modeling going forward? Thanks. And I apologize for the double question..

Geoff, this is Mike. I'll take a stab at the first one. I think it was about intrathecal access for this. It turns out that we don't anticipate any more significant problems in terms of intrathecal delivery with this program than in the SPINRAZA program at all.

And the initial studies are really that they're safety dose-ranging findings in Alzheimer's subjects. So we're trying to get an idea of exactly the right dose range. And then from a technical standpoint, we don't see or anticipate any more significant challenge than what we've seen with SPINRAZA..

So concerning the second question, let me start by saying that we are very pleased with the resilience of our MS portfolio. That is so important in terms of cash flow generation, and we intend actually to continue this momentum.

We organized a meeting in a few days with all the Biogen leaders, where we are going to recommit so that we can defend as much as we can. The market share where we speak is holding at 38%. It was 38.3% before. I don't know when exactly. And as expected, an OCREVUS launch will erode the share.

And if I look at the performance over the last two quarters, including OCREVUS, that was still a minimum contribution, it is the last best two quarters ever for Biogen, so it's not a cliff. And we'll continue to drive the activity to generate demand and volume, and we will not comment on the price policy..

Your next question comes from the line of Carter Gould with UBS. Please go ahead..

Good morning, guys, congrats on your results and all the updates.

Just to come back to aducanumab, on the updates with Eisai and Neurimmune, how should we think about these? Were these proactive moves on your end or more opportunistic moves after overtures from those companies?.

So I will not intend to the details of the first move. These are contracts that were signed a few years back. We see data coming in the Phase 1/1b, and we are relatively pleased with this data.

We'll hear more at CTAD, and I thought it would be an opportunity to see how we could improve the operation or give the best chance for the operation and the core promotion the day we have potentially the product, and therefore playing out who's the best where.

And obviously, I have the agenda to increase the opportunity for Biogen, and I think that with the two deals, this is what we are achieving. But it's a win-win, and the partner at Eisai gets also the opportunity to co-promote the MS portfolio, which is important. So we are very pleased with these two upgraded, I would say, arenas..

Your next question comes from the line of Matthew Harrison with Morgan Stanley. Please go ahead..

Great, good morning. Thanks for taking the question. I'd like to just go back to SPINRAZA one more time. I'm sorry for starting to beat this too much. Can you just help us because the numbers don't square? And I'm just trying to figure out what piece we may be missing.

So if you back out the inventory from the second quarter, you had 32% sequential revenue growth, but you had 75% growth in new patients. And I thought at the beginning, you said that 10% of patients had a maintenance dose.

So it seems like you almost doubled – I mean a little less, but you had substantial – hundreds of new patients add on and some patients taking maintenance dose. So it seems like the price per patient has come down by 40%.

So is that right, or what else are we missing in those numbers?.

Hey, Matthew, this is Matt. Let me try to help clarify. So first, when we say 75% patient growth, what we're looking at is at the end of second quarter to the end of third quarter. If you look at the number of patients on therapy at the end of the second quarter versus at the end of the third quarter, we had 75% more patients.

Now how many of those patients at the end of the third quarter received one, two, or three doses is unclear. We haven't revealed that. So that's probably part of the reason it's a little more difficult for you to square that math.

And the other thing we talked about was 10% of the revenues came from patients who had been on a loading dose in prior quarters, so there's a lot of dynamics here. And the nature of loading doses versus maintenance doses I know makes it a little bit complicated, but that's how the math plays out.

So I don't think it's a function of gross-to-net impacting. As you saw, we believe it's 20% of patients are on free. It's more a function of just the timing of doses. And as we said, we expect this is something that normalizes over time. I think the other thing you're all trying to solve for is the commentary we said about Q4.

We said the majority of growth is expected to come outside of the U.S. I don't think the message is that there's no growth in the U.S. I think the message is that there would be growth in the U.S., but the majority of growth would come from outside of the U.S.

So I don't think we're trying to send a message that you're not seeing growth in SPINRAZA in the U.S. We're just comparing it to the dynamics in the rest of the world.

Does that help?.

Yes, I think it does. And I think you've commented on ex-U.S. trajectory. But should we consider – I guess maybe you could just give us some guideposts on what's going to grow ex-U.S. trajectory.

Is it new country launches or additional patients in existing countries?.

You can assume new country launches would make – we believe new country launches will be one of the drivers there that could be. As you just look at the comments we've made, Germany and Turkey made up a majority of the rest of the world sales.

But as you look at the countries that have already approved and some of the traction we're making, you're going to see more diversity across other countries. That's our belief..

But Germany still has a long way to go, so you can actually expect both. But in the first phase, obviously the new product launches in new countries like Italy, while we speak, will add momentum, should add momentum..

Your next question comes from the line of Terence Flynn with Goldman Sachs. Please go ahead..

Hi, good morning thanks for taking the question, maybe one for Michel or Mike. You both referred to business development looking externally. Neuro focus, maybe just give us your latest thoughts on early stage, later stage, how you're thinking about business development and maybe the cadence of the deal or deals. Thanks..

So I will get started, and Mike can add more color. So first, we have strong cash flow generation, and we expect that this cash flow will grow mostly after 2019, when we have less royalties to pay on TEC [TECFIDERA]. So we will make sure that the allocation of this capital will secure the maximization of the shareholder return.

And we have communicated that, unlike in the past where we did the majority to the dollar back to shareholders in terms of share repurchase, we will also do the share and prioritize, as for now, the capital allocation efforts towards building the neuroscience pipeline.

And in addition, we may continue to repurchase shares on an opportunistic manner based on the price of the share and other elements. So we are looking at different opportunities. Eisai and Neurimmune took a lot of time. And there are opportunities, and the sweet spot of Biogen remains at the early stage.

And we'll be very careful if we go beyond the early stage of the valuation of those assets or companies.

Mike?.

Yes, I'd add just a little bit to that. We're quite committed to a couple of things within the R&D portfolio. Number one was increasing productivity of our existing pipeline, and number two was to be able to add important new assets to the pipeline. So we're quite committed to portfolio growth.

Michel mentioned – our sweet spot, we believe, is in early-phase clinical assets because of our ability to really derisk those and advance those through proof-of-concept well. But of course, we look within that sweet spot and beyond. We've got a number of active discussions ongoing. We're augmenting our external innovation capabilities.

We've been bringing online more search and evaluation, and we're really retuning a lot of the entire R&D organization and beyond to be more externally oriented. So I think that you will see over time both an increase in the productivity of the existing portfolio and a number of assets coming into the portfolio..

Your next question comes from the line of Ronny Gal with Bernstein. Please go ahead..

Good morning and thanks for fitting me in, a question about the gene therapy platform. Good to hear that you think you'll be starting now your trials in mid-2018. I guess my question is on the manufacturing here.

Is there an opportunity here to leapfrog in terms of your both capacity and cost? Folks would be talking about how difficult it is to get enough material to be able to treat an entire patient population as opposed to just the Type 1 SMA. Cost per therapy suggests in the hundreds of thousands of dollars per patient.

Have you been able to move away from triple (57:20) transactions to something more stable? Will you be able to address the cost structure? If you could, just give us some landmarks around that..

Sure, Ronny, this is Paul McKenzie. Thanks for the question and thanks for your great recent gene therapy seminar that you gave. A lot of people really benefited from that in the industry. You bring up a great question around our ability and beliefs around our development and manufacturing capability.

As I shared with you before that we have doubled down on our development capability. We believe that will allow us to drive to a gene therapy platform that's industry-leading. We also have invested in manufacturing partnerships and platforms that allow us to do these activities, both development and manufacturing excellence, in parallel.

We're very excited around our progress around our clinical supplies for the second half of 2018 dosing. And we continue to make great strides on our PCL development for the future commercial process.

So more to come in the upcoming months, but we believe we're well positioned to continue to drive our agenda in gene therapy across the portfolio as a real platform play..

Ronny, this is Mike. Just following up on Paul's comments, manufacturing is certainly one area where we believe there's an opportunity for real competitive advantage in a platform approach that could be attractive for SMA and beyond.

Within SMA in particular, there are a number of things that we are quite focused on, which is the right patient subtypes. We certainly have gained a lot of experience with patient subtypes within SMA with our SPINRAZA experience.

And I think along with that are going to be important things that we're looking at, which are delivery route timing of dosing administration, potential combinations with SPINRAZA. And of course, front and center in our mind is how all that ties in with clinical safety, and I think that a lot of this still has to play out.

So between manufacturing, patient sub-type, delivery route, safety profile, those are key elements of our gene therapy strategy..

And your final question comes on the line of Michael Yee with Jefferies. Please go ahead..

Hey, thanks for the question.

Given the update on the Eisai opt-in, can you just remind us with the BAN2401 data coming, how to interpret that from the standpoint of a high hurdle given aducanumab's advancement in Phase 3 and so far ahead, how you would think about whether there would be two Phase 3 programs or just the fact that there's a high hurdle for this program given that they've now opted in? Thanks..

Mike, this is Al. First of all, it's prespecified in the agreement what the hurdles actually would be, and so there's that. The way I look at that data is first I want to see. Is there plaque reduction with BAN2401? If there isn't, then I don't know how to interpret the rest of the data.

If there is plaque reduction, then the next thing we want to see, is there at least a trend or a favorable effect on the composite measure, which includes things like CDR sum-of-boxes, which would allow us to compare the effect of aducanumab with BAN2401.

And then finally, of course, we need to look at the safety, what are the rates of the key adverse events, such as ARIA. And then with that, we'll make a decision. But as I said, the contract prespecifies what the efficacy hurdles are..

So thank you all and have a great day, bye for now..

Thank you to everyone for attending today. This will conclude today's call, and you may now disconnect..