Let me just see. Are we ready to go? Great. Okay. Thank you very much for joining us for the next session at the Leerink Global Healthcare Conference. I'm Marc Goodman, one of the biopharma analysts, and we're lucky to have Biogen with us here. Alisha Alaimo, who is the President of North America. Thank you very much for joining us.
Thank you, Marc.
I think we'll go through the products and just kind of block them off, I think. I thought we'd start with Skyclarys. Let's just kind of jump right into it. I mean, in the United States, you know, back in the first quarter of 2024, we learned there were about 1,100 patients on the drug, and you did about $73 million of sales. Some of those patients were not paying patients. Yet it seems like since then, it doesn't feel like the revenue has changed much. $76, then it did an $82, you did a $71. It's jumping all around. Help us with what's going on behind the scenes, because sometimes revenues don't tell everything what's going on. Maybe we can start with Skyclarys.
Thank you. Thank you for having me here today. Thank you for everyone who decided to come to the room to watch this fireside chat. I really appreciate it. I think Skyclarys has been a very interesting journey to learn about. You learn real quick that, you know, every rare disease is different from the other one. Even though we have excellent rare disease capabilities, the Skyclarys or Friedreich's ataxia patient population is quite different. In the beginning, when we brought on Skyclarys, you know, a lot of those patients that you saw in the beginning, you know, like the 1,100 you're talking about, are all sitting at these centers of excellence around the country where these physicians know what Friedreich's ataxia is. They know these patients have them, and they were waiting.
That is also why when you saw our launch trajectory, we set the benchmark for rare disease launches. Even up till today, we still have the benchmark for best penetration of a drug launch. Once we got through the centers of excellence, we then have the rest of the patients. The rest of the patients are now sitting in the community. If I give you a little color around that, there are over 50 ataxias. The patients we are looking for right now do not know they have Friedreich's ataxia. They might know they have an ataxia. They also might think they have MS, which is one of the number one misdiagnoses, or ALS. They are not out there searching Friedreich's ataxia information to go in and talk to their doctors. Their doctors might think that they have something else.
Like you mentioned, the 1,100 patients, we have over 750 prescribers. That tells you how long this tail is of what we're looking for to find a patient. Behind the scenes, what we have to do is, and I've mentioned before our AI model that we have, which is a very sophisticated AI model. We get 100 leads, over 100 leads a day. This model goes through claims records, EHR records. I mean, you name it, they will then spit out leads. I have over three field forces now that work on this. I have a multi-layer complex system of how we go out and find a lead. We will have an inside team that will call these physicians' offices to say, "Have you heard of this patient? Have you heard of what they're going through?
We see that there's a claims record. If we think it's a go, we then shoot a field force out to meet this physician. This physician then will not know what Friedreich's ataxia is. They may know of who this patient is, but then they'll say, "I saw him five years ago." They probably sit with one of their other doctors. I have another field force that will have to go to the cardiologist, the ENT, the physical therapist. When I tell you the mechanism now to find the one patient in the one doctor's office, it's the heavy lift of the education that is something that we underestimated, because people do not know what Friedreich's ataxia is. That is how we're capturing patients now.
The numbers can be a little all over the place, because, for example, a couple of weeks ago, I had zero patients coming on board. Just a week ago, I had 10. Sometimes we can find a family. Sometimes, you know, it will be a misdiagnosis. We now also have, this is another interesting piece of information. I put my MS field force on it. My MS team, which is doing phenomenally well this year, we noticed that, you know, they do have a great relationship with all of these neurologists across the country.
In order for a doctor to really go through the effort to think through a patient, which does, you know, they have to really clear out the cobwebs or ask someone to go through an electronic health record, the MS patients have gone, and I just executed them a week ago. Within two days, I had a rep down in West Virginia find an FA patient, one of our MS neurology offices, and already a start form was submitted. It just shows you you have to go out and ask the right questions and find them. That is why it is a little lumpy. Also, you knew that for the part D for IRA redesign, we also, Skyclarys is about 30% of our hit, our net revenue loss for that as well. That is what you are kind of seeing happen right now.
Yeah. Yeah. I mean, is it 4,500 patients that we've identified kind of thing? Is that?
Yeah. It's like, and we actually know a lot about most of them, to be honest with you. And so we think that there's 5,500 of them are under the age of 16, so they don't qualify for Skyclarys. And then we believe there's around 4,500.
Right. We got 1,100 of them pretty quickly. And then.
Yes. We've got, we penetrated, you know, and we have start forms in for a good chunk of them as well. Now it's working them through the system.
Right, right, right. What's the last number that you've given us just?
I don't know if we gave you a last number. It was probably around the 1,100 mark. We have more patients than that. We keep adding.
No, you have to obviously give them the numbers. Yeah, yeah, yeah.
We keep adding patients. The other thing you have to keep in mind with Skyclarys, and another dynamic that is very hard for you to sort of plug into a model, is that if they have a side effect, doctors, what will look like to you could be a discon, to them could just be moderating their dose. For example, if they're taking three pills once a day, they may go down to one pill. You may not see the refills come through because they've had to taper down on the dose if they have a GI side effect, if something's happening with their liver, or they'll be pulled off the product. They'll see if their liver enzymes stabilize, and then they'll be put on at a lower dose.
You're seeing a lot of puts and takes that happen with refills, adherence, how they manage a side effect profile. That's what becomes also a little lumpy.
There was also inventory that moved around. Explain that. What happened there?
Yeah. We had inventory. You know, depending on when they order, it depends on what week it falls in. Sometimes you could have the start of the month on a Friday, and inventory is built on Thursday. It goes into that last quarter. There is nothing we can do about that. You do see, and now because of where we are with our patient population, even inventory builds will become a little sensitive.
Yeah. Yeah. I know that you don't, you're not responsible for outside of North America, but can you just give us a sense of where we are the drugs in, what key regions, what are some key regions it's not in yet?
Yeah. So it's in, I think, about 15 markets about now, mostly in Europe. Europe's actually doing a really great job with getting patients onto product. And you know, that's either through a commercial, you know, commercial pay or through an early access program. Some are paid, some aren't. And so they're actually growing at a nice clip right now, especially in patients, but you may not see it in revenue yet because depending on when we get reimbursement in that country. So we're going country by country. The ones that I think, you know, that you're seeing now are like Germany, Spain, Italy, France. And then where we're not is really in most of the regions outside of Europe and the U.S. However, we had approval recently for Chile.
If you look at, we call it the Intercontinental Region, Brazil will actually hold half of the Friedreich's ataxia patients out of all of those countries outside of the U.S. and Europe. We do expect Brazil later this year.
Later this year. Okay. That'll be the, that's probably the most consequential new region to come online this year.
Yes. Yeah.
Got it.
I believe Turkey also has quite a few patients. They are not really, I think they are on their EAP program, but you are seeing quite a few patients go on product in Turkey.
Anything else before we move on from Skyclarys? What else? Anything that you want to kind of get across? I mean, pricing, Medicare, Part D, we talked a little bit about that. I mean, these patients are 30% are Medicare. Is that what it is? What percent do you think?
They're about, I think it depends. It also depends on our, you know, the thing that also moves around a lot is discounts and allowances for us depending on, you know, what happens in that quarter. You know, about 30%, I would say.
Right. Okay. Okay. Good. So Leqembi, I mean, look, you smile. I mean, I live through Aduh elm as well. So, you know, I can smile.
We were in that together. We were in that one together.
Maybe the first question is, you know, what have you learned over the past year and what have you changed over the past year to help adoption better?
Okay. There.
I know. Loaded question.
Yeah. That's a loaded question. There were many lessons learned so far with Leqembi. I think we have said many times about infrastructure challenge. I am not going to go into that because that is a heavy lift for doctors. Every doctor is going to have to do it, and they're working through that. The bigger lesson is if you can get the one doc, if you can get one clinical champion, they will push all of it through. That is how you saw a lot of these IDNs start moving, is that you needed that clinical champion. I think that's first and foremost. The second lesson learned, I would say, is really around, again, the education needed. I mean, they did not really understand the clinical trials or how meaningful that would be for patients.
The whole reason why a lot of offices haven't moved yet is because they're not yet that champion. That champion can be moved if they are emotionally moved by either a peer or something that's happening in the community or if a patient asks. We have learned recently, we call it a grant rate. If a patient comes in and asks for a prescription, it's called a grant rate. Leqembi has one of the highest grant rates I've ever seen, and yet we don't do a lot of patient-to-consumer education. They've had to find out on their own, right? This has not been through a big broad campaign. Because the grant rates are so high, what you'll also see in our prescriber base is I have a lot of doctors who only have one or two patients on the product.
We're going, why is that when you have, we know you have many more? They go, well, the patients asked for it. They felt compelled enough to do it for the patients that asked, but yet for the rest of them, they're waiting and seeing. They're going to wait eight months. They're going to see what happens, and then they'll think about it for the rest of their patients. With that at play, the things that we've done is, one, I think, you know, we expanded the field force. What we did is we started putting two faces versus one face or two field forces versus one field force in a lot of these doctors' offices. What I can tell you, the other lesson learned is relationships matter.
Where we deployed Biogen representatives as an overlay to the Eisai team, we see faster acceleration and more depth in prescribing than what you do in the rest of the nation. These doctors were open, obviously, because it is one of their reps that they have seen before. Secondly, there was a trust and already the relationship was there. They were more apt to do the work than what they would prior. That has actually helped quite a bit. I know it is early days, but that is something that is growing. The second thing that you are going to see happen, we also redid the detail aid, redid some of the key messaging now that we understand the landscape better.
More importantly, now that we know the grant rate is so high and we know that physicians are apt to say yes when they ask for it, you will be seeing us do a much broader DTC campaign this year.
Really? Even on television?
Maybe even on television.
I mean, it does make sense, especially if these people are coming in and asking for it.
They are. They are.
Do you think that some of these physicians who are, you know, writing for two patients who are asking for it, they're waiting to, what are they waiting on? Like, are they waiting for the drug to see help? Are they waiting to make sure that it's safe?
You know what? It's both, actually. It's one, do they know how to manage if anything happens? Even though it's only one or two patients, they're very cautious because a lot of them have never dealt with ARIA or even PML, right? They're not MS docs. The second thing is like, what's the feedback? You know, what do they say? The stories that they give back are not the things that you see in a clinical trial. Like, you know, a physician told us the other day that a couple came in and the husband had said, it's the first time she remembered my name in the morning. You know, it's little things like that. Or there was a gentleman that came into the office where he's an avid, like he loves cooking, and he hadn't cooked for over a year for his family.
He had come in and the family had told the doctor, well, he just opened up the most complicated cookbook and cooked us our first dinner, right? They are wondering, what is the feedback that I get and what is it that I hear? Like, do I know if the drug's working?
Yeah. How many unique prescribers have we had so far? Have you given us that number? I don't remember.
We have not given you the number, but I will tell you it grows every quarter, but it is still not nearly enough. We still have a small percentage of the total universe that we call on that have actually written the product.
Yeah. What is going on with Lilly now that they're in the market as well? How has that whole changed the dynamic? I have always thought of big market, two players making noise, it doesn't get any better than that. I don't want three, but I love two.
Yeah. I like two too. I think two's a sweet spot. I feel the same way as you. Our hope is that the market will get bigger. I think it's too soon to tell right now. A lot of who we see that writes the competitor, the same doctors that obviously write our product, because even if you go to a new office or a new physician, you face the same challenges that we've been facing for 18 months. That does not change just because you're a new product. Coming to a product decision, you still have to do all of the workup. So far, it's everything that we sort of expected. There has not been anything unexpected to date. We have not necessarily seen the market rapidly expand, you know.
I think if anything, there is some confusion that came out because of their recommendation of, you know, an 18-month stopping rule. Doctors didn't really.
I'm glad you brought that up because has that created a lot of confusion?
It has because they're like, are we supposed to stop? Are we not supposed to stop? I think, you know, with Leqembi, we have an open label extension that shows you should not be stopping the product, that you still receive benefit even after plaque is removed because your plaque can come back. No matter how fast or slow the process is, staying on product, you will perform better than not being on product.
I mean, from a patient's perspective, you can understand this is a drug to, you know, to get rid of amyloid. Okay. I just had a scan. I have no amyloid. Why am I on drug?
Correct.
I mean, you can understand why they would say that, right? I mean, so.
Yeah. Yeah. It's the same reason why you don't go off your antihypertensive or your cholesterol medication, right? They have to understand that it is a chronic disease with a chronic condition.
It's a little bit of a mixed message given that Lilly's saying and what you're saying right now. That is causing some confusion.
It's confusion. I think now with also the positive is with our IV maintenance also being at the 18-month mark. I think physicians even, like right here in the city, a physician came to present to our executive team, and it was the day we had the IV maintenance approval. He said that, you know, he had called several of his patients and they were very excited to go to the once-a-month dosing, but they did not want to stop product. I also feel there's a safety/fear that's built into if you try and pull the product away.
Yeah. Yeah. Yeah. How do you think about blood-based biomarkers and subQ and just, I mean, to me, I think they're just so, I mean, isn't there going to be a massive change? I mean, right?
Yeah. I mean, I feel like.
How massive? Like, how do we quantify that?
Yeah. We were, so I'd love to give you a number. We were just talking about this with Tim and the team is that everything is kind of coming together at the same time right now, really for, and I think for Alzheimer's in general, it's really great because there's a lot of awareness now and much more awareness about Alzheimer's disease than ever before. Many things coming out for Alzheimer's disease, it gives people a lot of hope and interest. When you think about the blood-based biomarker, which I think will be so wonderful for the patient community and just so easy for them to confirm whether they have it or not, I think that that is going to be a game changer when it launches, when we get an FDA-approved one.
Now, if you looked or talked to a blood-based biomarker company, quarter over quarter, I mean, their numbers just keep increasing. So many more doctors are using the commercially available ones now, but they always followed up with a CSF test or a PET scan. They do not trust it.
Every one of these Alzheimer's meetings that we're going to, CTAD or, and it doesn't matter, what we're seeing is the correlation is just pretty high. I don't know how much higher we can get. I mean, we're practically at 90%, aren't we?
Yeah. They want you at 90%. It just needs to get FDA approved so you can start getting it reimbursed. The two big issues that we see in market research with blood-based biomarkers today is, one, they do not have confidence in it, and two, it does not get reimbursed. With an FDA-approved one, we think that it is going to become much more easy.
Do you think that happens? Is that a this year event? Is that a next year event?
We think it could be this year, early next year.
Right. So it's later this year, early next year.
Yes. Now, remember, blood-based biomarkers, however, they're not built like biopharma where you have reps that cover the entire country. Getting them educated and entrenched will be the hard, you know, the difficult, challenging part. We do need to figure out a way on could we support, could we help in any way? Because they're not going to be able to have that widespread reach like we would.
I mean, the pushback on subQ versus every two weeks or four weeks, I mean, like that's also going to be a huge game changer, right? I mean.
It will definitely help with physicians not having to make sure IV infusion beds are open and, you know, getting their MRI scheduled at the exact right time. It will alleviate the work on the physician and the patient for subQ. Now, oddly enough, there are patients who do like coming to their IV maintenance appointments or IV appointments because for some of them, that's their only community. It will be interesting to see, I think subQ maintenance will be a good option. It's a subQ induction that will be the game changer. To be able to offer them the different options, I think, is what also becomes really important for this patient population, depending on what stage they're in, how old they are. Are they in a nursing home? Are they at home alone? Do they have caretakers?
At least now they have options.
The physician community knows this is coming, right?
Yes.
I mean, you can't really market it. Obviously, not approved, but I'm just saying, but they know it's coming. The data is there at the meetings.
They know it's coming.
Biogen's view is we're committed here to this. We know this is coming. This is going to be a major change.
Yes. You know, it comes, subQ will come as well, induction next year along with blood-based biomarkers. We just think it's all happening at the right time.
Yeah. Yeah. I mean, it's just, I don't know, this has been one of those markets where you kind of feel like there's so many patients, but at the same time, there's a lot of friction, as we talked about, and you just think that there'd be some offsets and it would be ramping a little bit better. Outside of the U.S., anything that's happening different than in the U.S.? Like.
You know, Japan.
We're not in Europe yet, I know, but you know, Japan and China, I suppose.
Japan, China and Japan had a really amazing launch. I mean, first they had good pricing on the product, which was great. You know, their health system is a little bit different. They had a couple of centers of excellence that made it very easy for the patients to go through. We think that that launch has been absolutely excellent. It just shows you that once you get your infrastructure set up, they also do DTC there. They also use some of their primary care reps there. They really like front-loaded the launch and have done a tremendous job.
Right. Right. Right. Interesting. Anything else on Leqembi before we I want to talk about Spinraza a little bit.
Oh, I'm happy to talk about Spin. Not many people ask about Spin.
I know. I know. But you know, once, I mean, first of all, I look at it as like, first of all, it's kind of unique that we have this small market. We have three drugs approved for it. I think that's.
Shocking.
Shocking.
For rare disease.
Yeah. For rare disease.
It's like how many people are after these pieces?
I kind of feel like, like when you really look at the data, you guys have the best data. And your high dose now is reason to get kind of rejuvenated even more. I don't know, give us a sense of what's going on. Let's talk to the U.S. What's been happening in the U.S.? Because no one talks about Spinraza.
Yes. Thank you for the question. I think first, you know, we were talking about Skyclarys. And I get that question about Skyclarys. Like, oh, you know, how long are you going to grow for? I'm in year eight of Spinraza. And the last three years we've grown Spinraza, right? So it just tells you, and that was with two other competitors. If you wind back to, you know, basically 2019, 2020, that's when the competitors started launching. And when the oral option launched, it was during COVID at, you know, stay-at-home orders where our patients for Spinraza couldn't go in and get their intrathecal injection, right? And my field force was also on lockdown. So there was almost nothing. We did our best, but we lost a lot of patients during that time. Now, everyone thought, oh, it's just because it's convenience. Actually, it was a perfect storm.
Fast forward to today, a lot of the patients that are coming on product are switchbacks. What we've learned about the patient population is it's not that they actually want the convenience. They are just searching constantly for more efficacy. Once they've switched off and they've seen a decline, they switch back on again. A lot of this are the moving between products. We also have a lot that have come from Zolgensma. They've had Zolgensma. They'll see wearing off and it's like, I want to go on Spinraza. Spinraza HD. High-dose Spinraza really was born out of the fact that we were hearing from physicians' offices many years ago, like we have this waning off effect. You know, we feel so great after we get that injection. Right before the next dose, it wanes.
Like, can I get the dose sooner? We said, well, what if we gave them a bigger dose? Sure enough, we have higher dose. We're looking at getting an approval in fourth quarter this year. Patients are already asking about it. We believe it will provide another great option for them, especially if they're looking for something that is perceived to be.
I think it's interesting you said that about the perfect storm of COVID because I would think in this market, it's all about efficacy. When we talk to people, they talk about efficacy. That's all they want to really talk about. It's not like the adverse events or anything to scare everybody away from using these products. It's interesting. Your view is this is a growth product in the U.S.
Yeah. The U.S., I mean, the last three years we've grown and we have fierce competitors. We're not going up against small little companies. I mean, they have a lot of money to spend and they have good products. The team's done really well. I think, you know, the switchbacks have been important. We still get new patients every year as well on Spinraza.
Where are we on patients? I don't even know when the last time you gave us a patient number.
We do not do patient numbers anymore because at the end of the day, we are all switching, you know, back and forth. We do believe over 70% of the population have been penetrated. We do believe that where we are going to get the rest of our business, you know, for new patients is really, it is the adult business that becomes important. We have a very large adult business and they do a lot more switching.
On U.S., anything you can help us with there?
On Spinraza, you know, they're a little bit behind us in the sense of the competitors, right? We were U.S. experienced the competition first and they're now dealing with the competition. They've had a lot of lessons learned. I think Europe is holding good ground. I think you see some weird things outside of U.S. and Europe when it comes to, you know, the shipments or tenders and things like that cause some lumpiness. Other than that, they're doing well.
Yeah. You view this product as we're fully behind it. We're supporting it. We think it's a growth product. The high dose is going to be a changer.
I think the high dose is going to be very good for patients.
Yeah. So Zurzuvae. We're going to hit them all.
I can't believe it. I love a good Zurzuvae.
I guess the same question I asked before. What have we learned? What have you changed since the launch?
Okay. Zurzuvae, I'd say we've had the most learnings and probably where we've had the most change. Zurzuvae was a product which, if I can remind everyone, it was supposed to be for MDD and PPD. MDD, obviously, multi-billion dollar opportunity, didn't get approval and ended up launching PPD. Launched PPD, but we prepared for MDD. We went at this. We had very little investment. I think we told you we didn't invest. We were very modest with our investment last year, had small field force footprint and thought through the data that you purchase, prescription data that psychiatrists would be the ones that write majority of it because that's what it showed in prescription data. We were wrong. There's a lot of miscoding in this area. There's a lot of things coded as PPD, but it was really MDD.
What we ended up learning is that OB-GYNs were by far going to be the biggest champion for women and for PPD. The OB-GYNs, you also, we learned that you can't just target one doctor because most OB-GYNs sit in an actual group practice now. There aren't a lot of individuals. Even though one physician will show as a high writer, they may just be putting the products under that one doctor. It doesn't mean that that doctor's writing it. We had to change our entire approach to one, targeting OB-GYNs, two, going to group practices, not individual writers. Now they have to do an entire group practice call because every single physician in there may be prescribing a product or seeing a patient with PPD. Third, we learned that basically you will be diagnosed and treated within the first 90 days postpartum.
After that, the numbers dropped off dramatically. Within those first 90 days, there is only one appointment that a woman has for 15 minutes. In that 15 minutes is the window where they have to get diagnosed and treated with Zurzuvae. It comes down to a split second to get a prescription for this product. For the physicians who understand PPD, they will make sure in that 15 minutes it's brought up. However, if you have a patient who knows what PPD is and they say, no, I know I have postpartum depression, the physician will also write even though they don't know.
Interesting.
We have had to change our entire model over the year. We think that this year is going to be a very different year.
Right. As far as the focus, right? We've changed the focus. You've had to change the messaging a little bit, right? How much advertising do we do? Is this a DTC type of advertising?
I believe, yeah, I believe that this is the most perfect product ever for DTC.
I would think so too.
There are 250 prescribers for PPD in the United States, 250,000 prescribers, which is large. We are trying to go to a small slice in women's health or an OB-GYN in order to cover what we think is going to be the majority. We also know that if you do DTC, that patients will then come in and ask for it for this one specifically. We are looking into that. I saw some storyboards the other day. I think we have some more work to do, but I'm not sure when we're going to pull that trigger because we just put out also our expanded field force in this first quarter. I am waiting and seeing how the expanded field force does, what it does with prescriptions. Is it really more depth or is it new writers?
Because at the end of the day, we need many more writers to come on board.
Yeah. So you've watched the pipeline evolve over the past few years.
Eight years.
I'm just curious like what your thoughts are about, you know, lupus or, you know, I don't know.
Yeah.
Any of these drugs.
Yeah. I have a lot of thoughts. I think first of all, what I've loved about Chris coming on board is he's put all the regional presidents at the table for pipeline discussions and whether we should move forward or not, which has been really great to be a part of. I think when you look back to when he started, he did a really large overhaul and reprioritization of our R&D. As you know, both groups, you've seen all the announcements through Fit for Growth and then also overhauling the pipeline. Where he has now doubled down and where we have made decisions is lupus, Alzheimer's, and rare disease.
Yeah.
I was just asked in a meeting earlier this morning, you know, what are you most excited about? You know, I blurted very quickly lupus because of the unmet need. I just happened to have met four patients not that long ago. When you hear the unmet need and you hear how there is just really nothing out there for them, and this disease is devastating for SLE or CLE, you can see that if these products read out positively, it is a huge potential for the company. I also think in the pipeline, you've seen us do things like HI-B io, which I mean, Felzartamab is an amazing product. When you look at kidney and rare disease, I mean, if this works for AMR or PMN, I mean, we'll have more competition for IgAN.
This will be very good growth for the company, especially before 2030. You saw Stoke recently. Even though it is ex-U.S., I would have loved to have had that product in my portfolio. Another perfect product for a high unmet disease. When you look at Europe and intercontinental region, the growth that will happen just in those two regions will be great. I think that the pipeline is in really good shape right now. We have some really promising and, we believe, higher probability of success assets. We have doubled down, as you know, in Alzheimer's. We are kind of here to stay with Alzheimer's as well.
Thank you. Thanks for joining us.
Thank you.
Appreciate your time.
Appreciate it.
Yeah. It's great.