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Biogen Inc. Presentation at Morgan Stanley Healthcare Conference
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2024-09-04
2025-07-30
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Biogen Inc. # Biogen Inc. presents at Morgan Stanley 22nd Annual Global Healthcare Conference Wednesday, September 4, 2024 10:00 AM #### Event Participants Analysts 1 Terence Flynn Executives 2 Michael McDonnell, Priya Singhal Attendees 1 Timothy Miller #### Terence Flynn Analyst I think we're going to get started. But thanks for joining us, everyone. I'm Terence Flynn, the U.S. biopharma analyst at Morgan Stanley. We're very pleased to be hosting Biogen today. From the company, we have Mike McDonnell, the CFO; and Priya Singhal, who is Head of Development. Thanks so much both for being here. And again, for research disclosures, please see the Morgan Stanley website at www.\researchdisclosures.com. But again, thanks both for being here. I think you wanted to kick o! with some prepared remarks before we go into questions. #### Michael McDonnell Executive Yes. Thank you very much, Terence, and thank you all for being with us this morning. I'm very happy to be here in Times Squ...
Biogen Inc. # Biogen Inc. presents at Morgan Stanley 22nd Annual Global Healthcare Conference Wednesday, September 4, 2024 10:00 AM #### Event Participants Analysts 1 Terence Flynn Executives 2 Michael McDonnell, Priya Singhal Attendees 1 Timothy Miller #### Terence Flynn Analyst I think we're going to get started. But thanks for joining us, everyone. I'm Terence Flynn, the U.S. biopharma analyst at Morgan Stanley. We're very pleased to be hosting Biogen today. From the company, we have Mike McDonnell, the CFO; and Priya Singhal, who is Head of Development. Thanks so much both for being here. And again, for research disclosures, please see the Morgan Stanley website at www.\researchdisclosures.com. But again, thanks both for being here. I think you wanted to kick o! with some prepared remarks before we go into questions. #### Michael McDonnell Executive Yes. Thank you very much, Terence, and thank you all for being with us this morning. I'm very happy to be here in Times Square today. And just a little bit of background on Biogen and what we've been up to. We are continuing to be very focused on four new product launches, which we're excited about. We have LEQEMBI in Alzheimer''s, we have SKYCLARYS in Friedreich's ataxia. We have ZURZUVAE in postpartum depression, and we have QALSODY in ALS. All of those launches are progressing in line with expectations or in some cases, a bit ahead of our expectations. We are very focused on returning to sustainable growth. That continues to be our #1 priority and in the interest of growing on both the top line and on the bottom line. We have initiated a very large program, which we call Fit for Growth. And that program, we remain committed to garnering about \$1 billion of cost savings by 2025, and we kicked o! that initiative at the very end of 2023. And so over a couple of years, we'll take \$1 billion out. We expect to reinvest about \$200 million of that, so netting about \$800 million of savings and that will help our bottom line. And we are starting to see some results from that. Our most recently reported quarter was on August 1, we reported our second quarter, and we did have top line growth of 1% at constant currency. Our core pharmaceutical revenue grew at about 6% at constant currency and that was a function of the new launches slightly more than o!setting some of the mature products that we have in our MS franchise, which is declining. But I think more notably, we also had good bottom line results. We had a priority review voucher that we sold during the quarter that produced about \$0.52 of earnings. So if you normalize for that, we had about 30% improvement on our operating income line. and about 18% improvement on our EPS line. So we're pleased that we're back to growth on the bottom. We remain committed to moving towards growth on the top -- and in the interest of that, we continue to have a very strong balance sheet. We've done quite a bit of business development. We had a large acquisition of Reata back in the -- we closed that in September of last year. We closed on the HI-Bio acquisition in the early part of the third quarter of this year, which we're very pleased about. I'm sure we'll talk more about those transactions. And we continue to have a very strong balance sheet that we can continue to deploy in the interest of "lling our pipeline further and producing more growth. I think Priya, maybe you had a few comments that she wanted to add about what she's been up to in the research and development world. #### Priya Singhal Executive Yes. Thank you. So I think from a research and development perspective, we are in a very good place right now. We have really completed essentially our reprioritization, termination of programs where we felt that from an operational or scienti"c perspective or commercial perspective, it didn't make a lot of sense. And we are now in the rebuild phase and invest-towin phase. And with that, what I mean is we're investing in areas like Alzheimer's. We continue to provide end-to-end implementation for LEQEMBI, where we've got formulations we're addressing the presymptomatic population and more "ling worldwide. And then with other products like SKYCLARYS, we're focusing on pediatric development where we believe there's a very important unmet need. And then moving on to the preapproval portfolio, very excited about BIIB080, where we were recently able to almost halve the study size and actually accelerate our proof-of-concept time line. And so we're very excited about that. We think this will be the next generation of anti-tau therapies, and we did this based on data. Same with litu"limab, dapi, where we have -- we are in Phase III already, we're waiting for a dapi readout in the next few weeks. And we are preparing potentially for a second Phase III here. So that's very exciting. And then we are doing this also for what we're looking for from the outside and the external rebuild of the Biogen pipeline. And I think it's really a testament where we broaden felza which is an anti-CD38 antibody, and this is really based on biomarker and clinical data. So we remain focused on neurology, rare and immunology, but always applying consistent criteria internally and externally to really build the pipeline that can provide the sustained growth. #### Terence Flynn Analyst Great. Well, I appreciate the opening remarks and it's a great way to frame a lot of the topics that we'll dig into now. #### Terence Flynn Analyst As we think about heading into '25, Mike, I mean, the question we get is, when you look at consensus, generally #at revenue growth, you talked about the Fit for Growth program and the spend. But then obviously, you have the felzartamab, the HI-Bio asset, that Priya just mentioned and some incremental spends. So under this scenario where there's still this net cost savings, are you going to be able to deliver earnings over \$17 in 2025? Or is there anything else we should consider as we think about kind of that schematic for next year? #### Michael McDonnell Executive Sure. So we won't guide for 2025 until we report the fourth quarter of 2024, and that would likely be in the February of '25 time frame. So we will have more to say about 2025 guidance at that point in time. But there's a few things that I could say. One is, as it relates to the top line, and I'll kind of address the top line and then the cost structure separately. On the top line, our trajectory is pretty heavily tied to the pace of our MS franchise and the pace at which that's declining. As I mentioned, we've got some very mature products there. and how well that gets o!set with the new launches. I mean, those really are the dynamics. We do have site dynamics in the MS franchise that we're keeping a close eye on. There's a potential for further biosimilar competition with one of our products, which is called TYSABRI. We also have -- we're entitled to regulatory protection on TECFIDERA in Europe through February of 2025. We also have a patent through 2028 that we're working to enforce, but that's something that we're monitoring closely. So the pace of -- or the trajectory of MS versus the progression of the launches is what will really dictate the top line trajectory. And then on the cost side, we remain committed to the \$1 billion of savings. And on that \$800 million net, where we reinvest \$200 million of the \$1 billion we'll get about \$400 million of that in 2024 and another \$400 million in 2025. So certainly, that will provide a boost to the bottom line. And as I mentioned before, we'll continue to stay very active looking at other forms of business development and making the right investments. So that's probably all I can say at this point about next year. ### Terence Flynn Analyst Okay. Got it. Maybe just again, high level, I touched on this a little bit, but maybe "rst, Mike, you could kind of start in terms of the kind of outlook for M&A and BD right now. Obviously, you're delevering from some of the prior deals. But how do you think about kind of the forward cadence? And then Priya from your seat, just what are the areas that you're most focused on right now? I think you touched on these, but maybe you could just delve in a little deeper on some of those? # Michael McDonnell Executive Sure. So I can start with the kind of the wherewithal and some of the things we're looking at and Priya can comment on some of the areas that we like, in particular, perhaps. But we'll continue to stay active. It is a fair comment that we did empty the treasury a bit with the Reata transaction and then doing HI-Bio. But we continue to have a very strong balance sheet. We ended the second quarter with over \$1 billion of cash, and we generate about \$2 billion of free cash #ow per year. And we also have a pretty modest amount of leverage. We could certainly add a turn or more of leverage for something that we were really committed to and that we really like. So when you roll that all together, there's probably \$8 billion to \$10 billion of wherewithal that we would have over the next couple of years. And whether we deploy that in a series of smaller transactions or something larger is something to be determined? And maybe Priya can comment a little bit on some of the areas that we like. ## Priya Singhal Executive Sure. so overall, I think the most important thing from our perspective is that we remain focused in areas of strength and expertise and capability. That is de"nitely neurology, rare and immunology because we've been in these spaces for many years, both from a research development as well as a commercial footprint. So we're looking at all those. . But when you double-click on that, we are always looking for data that can give us the opportunity to derisk and bring to an in#ection point earlier with con"dence. So we will be looking to use our own tools or tools that we see with the company that have these products to be able to derisk at early time points. The other thing I think that's important to remember is we're looking across R&D. So we are looking at very early stage programs. We're looking at mid-stage programs, and we're looking at programs that could generate revenue in the next 3 to 5 years. So they're all in play and eventually, it's being driven by clear criteria. So by that, what I mean is if it's a rare disease, we'd be looking to assess how con"dent we are about the diagnostic pathway. Where the screening is available, what is the status of regulatory endpoints? What's the clinical tractability. Do we have con"dence in natural history data, do we think we can get it to the next stage. These are the types of questions. And then we really go hard at diligence. And I think HI-Bio is an example where we really looked for proof of concept not only by clinical data but also by biomarker. So we let data guide us generally. #### Timothy Miller Attendee What -- and right now, would you say, again, it's more of a buyer's market or seller's market in terms of the assets that you guys are seeing come in through the vetting process. ## Michael McDonnell Executive Yes. I mean that's a hard one. I think that -- I would say that there are I guess the way I'd answer it, there's a number of assets out there that are interesting. We certainly have a very good lens on assets that could be of interest to us, and we look at a lot of di!erent things. I would say that as you would expect, the later-stage assets where you have more certainty, certainly continue to be priced at a premium. ## Terence Flynn Analyst Yes. Okay. Okay. Great. I want to touch on LEQEMBI and the launch, obviously, that's one of the important launches here. You touched on that a little bit, Mike, and then I had some follow-up for Priya as well. But maybe just give us the latest perspective on kind of launch progress. and your partner, Eisai has provided some guidance for kind of the next 12-month basis of about \$390 million on a worldwide basis. So maybe just help us think about kind of the framework on the guidance side that they provided. # Michael McDonnell Executive Sure. So the launch continues to progress. And as you know, there are not a lot of analogs for a product like LEQEMBI, it is unique. We did have \$40 million of revenue in the second quarter worldwide \$30 million in the U.S., \$10 million outside of the U.S. Importantly, we continue to see good progress in terms of patients, we had about -- when you looked at the patient count at the end of the second quarter, about 40% of those patients actually came on to treatment in the second quarter. And we also saw about a 50% increase in the number of prescribing physicians during the second quarter. So it continues to progress and move forward. We are dedicating some additional resources to the launch along with Eisai and working in partnership with them. In terms of the revenue information that they put out, we typically guide in the aggregate for revenue. They talk a little bit more at the product level, and we have an inherent di!erence in our "scal years where they're on a March 31 "scal year, and we're on December. So it's a little bit hard to comment on the product level on a di!erent "scal year. But I would say that the launch continues to progress. We continue to be working very well with Eisai, our partner, and we're going to continue to invest appropriately, and we continue to believe that this can be a very good and successful product over time and obviously something that's very important and helpful for patients. #### Terence Flynn Analyst Yes. What -- I know you talked about the priority 100 IBNs that you guys are focused on. But obviously, you and Eisai are kind of broadening that footprint with some additional sales representatives. So as you think about that target prescriber base, can you just remind us kind of like what that looks like here and the achieved -- when do you think that breadth would be su\$cient where you "nd kind of fully blanketed that prescriber base? ## Michael McDonnell Executive Yes. I mean I think that not a lot I can add there. I would say that the majority of prescribing physicians to date have been neurologists. Certainly, there is the potential to go deeper with primary care physicians as well, and that's something that we're focused on. Optimizing that obviously, will take some time because this is something that hasn't done before, and there's a large queue of patients that are waiting to get into see specialists and then specialists that are now actually prescribing treatment for Alzheimer's patients for the "rst time because prior to this, there really wasn't anything that they could do. So all of that will take some time. . #### Terence Flynn Analyst What as we think about kind of the U.S., the other kind of new wrinkle is Lilly's Kisunla was recently approved. And so as you think about that dynamic back half of the year, anything early on the launch you're hearing in terms of -- from the sales force in terms of how physicians are choosing between these options I mean is that a rising tide lifts all boats? Or is this a situation where new starts are now going to be split between these assets? What are you guys hearing from kind of early days there? #### Michael McDonnell Executive Sure. I can start, and then Priya may want to add to it. I mean, much too early to really comment on -- on the commercial side, I would say that we do believe that having two products in the market. It's certainly a large enough market for two. And we have a view that there could be some opening up of patient care pathways and more awareness by having a second entrant into the market, but you might want to comment a little bit more on the science side and how the products are di!erent and so forth. ## Priya Singhal Executive Yes. So I think overall, we believe that this is going to be a positive for patients and the space. The Alzheimer's disease infrastructure is quite fragmented. In the United States, we think that LEQEMBI's made a real foray there, but having Kisunla will certainly help. Having said that, it's early to comment from a competitive perspective. But what I can tell you is that we don't believe that the same formulary decisions and the same data, the same protocols by centers can be easily translated or adopted because essentially, it's a di!erent product. So both LEQEMBI and Kisunla are quite di!erent. They are both anti-amyloid therapies, but the way that they were studied in clinical trials is really di!erent. The populations were di!erent and therefore, di!erent protocols need to be developed. So that is going to be an important factor that will play out. The other thing I think that's important to remember here is the di!erence between LEQEMBI and Kisunla. LEQEMBI really has a dual action. We believe it acts way beyond the 18-month clearance of plaque, and we recently showed data again at AAIC, out to 36 months which shows that if you stay on LEQEMBI, you have a 0.95% reduction on CDR-Sum of Boxes. That is very compelling and continues to be very important because LEQEMBI acts beyond the plaque clearance. So it's a whole di!erent proposition. And to that end, we've already "led for the IV maintenance, and we've already "led for optionality on subcutaneous, and we continue to work on the induction subcutaneous formulation as well. The "nal thing is, I think from a presymptomatic perspective, the data that we've shown shows that when patients have low levels of tau, or no tau and the only Phase III trial where patients with this low and no tau were enrolled was the Clarity AD. We showed that the clinical decline is actually improved by 76% and almost 60% of patients do not progress or reverse. That is a very compelling fact, which is attributable to LEQEMBI. And we think that this could be an important sort of harbinger, we hope of what we might see in the presymptomatic trial. So we continue to be encouraged about the enrollment in the AHEAD 3-45. And so we really see LEQEMBI as an end-to-end implementation. And we think that this is going to be relevant. Finally, I think the safety is really important because LEQEMBI and the Clarity AD, while there was no head-to-head comparison, it shows the lowest rates of [indiscernible] and we know patients continue to be concerned as do physicians. So this is another compelling factor in the interest of LEQEMBI. # Terence Flynn Analyst Okay. Great. Maybe a couple of follow-ups on those points. I guess on the protocol development, when you look back at LEQEMBI, like I know there were the reimbursement dynamics and the background going on, but how long did the protocol development take for you guys to kind of roll that out fully across the sites as we think about Kisunla and how long that might take for Lilly to do. ## Priya Singhal Executive Yes, I can say that it's been variable, and it depends on how complex the center is. So what we've seen is once the center gets going, they can they can initiate, but it also depends on the results that they get from patients and more feedback, the label speci"cs monitoring, access to infusion centers. So it's several factors, several factors. ## Terence Flynn Analyst Okay. But does that like -- was that like, was it months? Is that fair? Was it weeks? #### Priya Singhal Executive Yes. I think it was generally in the months. #### Terence Flynn Analyst Okay. Okay. Got it. And the other feedback we've gotten is just that every other week dosing versus once monthly dosing. It sounds like that's been somewhat of a hurdle for some patients who maybe live further from an infusion center. And so maybe that gives Kisunla somewhat of an advantage. You talked about subcutaneous. So maybe just what are the next steps on subcutaneous from a regulatory perspective? What are the kind of the milestones that we should be focused on, both on the maintenance side and the induction side because I know those are two di!erent pieces. #### Priya Singhal Executive Yes. So on the subcutaneous maintenance, we've already initiated the rolling submission. And once we complete the submission, which is on track, as Eisai has commented for Q4 this year, we would get what kind of review it would have, which is priority or standard. But we expect that we'll get an outcome around mid 2025. That is what we've communicated. With regards to the bioequivalence data that we had, we learned that really we could remove more plaque. And so we are trying to balance the dose of the initiation of LEQEMBI and that work is ongoing. But at this point, we've communicated that we expect a regulatory outcome from FDA by Q1 of 2026. So those are already in play. And IV maintenance already has a PDUFA date of January 2025. So that's as well on the docket here. With regards to biweekly and monthly I think that the data are mixed on that because there are patients who want to come in, want to be seen and especially in the early days, physicians want to see them and monitor them. So I think that still needs to play out, and it will depend on the safety burden and other factors like that. So I think we have to wait to see how that plays out. #### Terence Flynn Analyst Okay. On the subcu induction, can you give us any sense of like how much more data like patient numbers, duration? I know you gave the kind of bookend in terms of regulatory outcome by 1Q '26. So you'd have to "le sometime kind of probably by mid-2025, but any more insight in terms of like how much data you're generating for that question? #### Priya Singhal Executive Well, what I can tell you is we are working with Eisai and we're working with FDA very closely to really agree on what the package would be. And we're sort of executing on that, but we haven't shared more data than that. We will when the time is right. #### Terence Flynn Analyst And then the "nal target pro"le would be a once-weekly, self-administered auto-injector. #### Priya Singhal Executive That's right. ### Terence Flynn Analyst And what any market feedback in terms of like what percentage of patients might prefer that versus going in to see their physician, like you said, like kind of more of the high touch like. ## Priya Singhal Executive Yes. We believe -- so that's actually been part of our strategy, which is why we went with the subcutaneous maintenance because we believe that once the plaque is cleared, which is also, again, a feature really only speci"c as can be because when we act the soluble species post-plaque clearance, we think that, that is a space where physicians will initially potentially be more comfortable. So we see that coming in. We also think that subcutaneous could potentially in the future, have a role to play in a presymptomatic setting. But that is all still to be determined. So I think there's a bigger strategic perspective with which we've been developing these options and formulations. ### Terence Flynn Analyst Okay. Great. I'll come back to the presymptomatic in a minute. But the other question is just the EU CHMP reexamination. So maybe just remind us why you're optimistic here because, again, I think when we look at some of the precedents, it looks like it's a high bar when companies go back for a reexamination. So why -- what gives you con"dence, I guess, going back to the CHMP? #### Priya Singhal Executive Yes. So overall, what I can say is we are very disappointed. We're very disappointed. As are many communities, many physicians and many patients who provided public letters in support of LEQEMBI being through the CHMP process. You're right, it is a high bar on reexamination. There's no question about that. But the reason that we remain encouraged is twofold. One, we believe the data are compelling, really compelling in terms of bene"t risk. We think that we have already shown even more recently at AIC, data out to 3 years in the open-label extension of Clarity AD. We've shown data with the early and no tau patients. So there's a reason to believe why patients should be treated now. That's important. The second piece is we have now collected a large number of patients in the real world being treated. So we have a lot of additional incremental data that we have available to us, which is actually global because it's U.S., Japan, other countries where LEQEMBI has been approved. So we think that working very closely, we're working very closely with Eisai, and this will be a relatively fast process. So we expect the result before the end of 2024. And the other piece here is that the way the process is designed is a new rapporteur and a new rapporteur are assigned to the process. So we believe that they could bring fresh eyes, we could have discussions with them to really assess on what could move this needle. And I think we have a lot of new real-world data that we could add to this package. # Terence Flynn Analyst Do you have the rapporteurs yet or they haven't been assigned a new rapporteurs?. ## Priya Singhal Executive We can't comment on that. # Terence Flynn Analyst Okay. Okay. Understood. The maybe question for Mike follow-up there is just the infrastructure, rest of world infrastructure. If you don't get the positive CHMP decision ultimately, is there -- are there investments that have already been made that would be scaled back in some of those geographies? Or is that something that you guys were waiting on the decision to pull the trigger on those investments? # Michael McDonnell Executive Probably a little bit more of the latter. But when you look at the size of the company in totality, not a material needle mover. What I would say is that in a hopeful event that we're successful on reexamination we'll be ready. That's the most important thing. ### Terence Flynn Analyst And what -- outside of Japan, what are some of the other key geographies we should think about for the kind of ramp of the ex U.S. side? Because I know, obviously, Japan was a big part of that second quarter sales result, you mentioned ex U.S., but as we think about kind of like the expansion beyond Japan, what are some of the other geographies? # Michael McDonnell Executive Yes. And I think we're very excited about how things are progressing in Japan. We're also now approved and up and running in China. That would probably be the next one that I would tick o! and then we're also approved now in South Korea. So those are the early ones. But I think, obviously, we're very focused on the U.S. We're very focused on the situation in Europe as Priya talked about and then Japan and China would probably be the next on the list. # Terence Flynn Analyst Okay. Maybe just to round out the LEQEMBI discussion. Bayou referenced the AHEAD Phase III trial in prevention setting. Maybe just what gives you con"dence here in a positive outcome. And I mean, the other debate, which, again, I know goes back to some of the early aducanumab data is just like clinical bene"t here in Alzheimer's across the board. So how are you de"ning clinical meaningfulness, clinical bene"t in the context of prevention? Like what's good enough to really drive commercial uptake? ## Priya Singhal Executive Yes. It's a very important question and one that we've thought about for many years and sort of built our programs around it. So First, I would say that I think the biggest barrier was really to be able to demonstrate that you could "nd Alzheimer's patients and clear plaque. And we were able to do that. So we were able to demonstrate that you can clear amyloid plaque, and we were able to demonstrate unequivocally, I think, with Clarity AD. speci"cally, that clearance of plaque translates to clinical bene"t. So that's number one. The other piece is sort of parallel evidence that a movement of about approximately 0.5 on CDR-Sum of Boxes in domains like memory, hobbies, community a!airs. The distinction is loss of independence and slight impairment. That is pretty huge in terms of burden of disease, in terms of caregiver burden in terms of patient being able to be operating independently. So I think that's something we need to keep in mind. And then the third piece is that we have shown now that these patients who are very early, they have low levels of tau. Because that's the second culprit sort of in the bigger picture of the pathogenesis that other protein, which only comes in couple of years prior to patients having symptoms. So what we've shown is that if you don't have tau or you have very low levels of tau, you can actually reverse disease. And this is not an incidental "nding, it was a tau sub-study in Clarity AD, where it was placebo-controlled. So it's really very controlled data that's showing us this, and that is compelling. So then you dial back to how we designed AHEAD 3-45 it's a platform of two trials. AHEAD 3 is amyloid levels below 40, so centiloids. So 20 to 40 very early, whereas AHEAD 45 is above 40 to higher levels of amyloid. In the AHEAD 3 trial, we have just a biomarker outcome because we think we've already made the case that removing amyloid is a positive. And these patients are far away from having disease in terms of symptoms. But in the patients who have a higher load of amyloid, we're looking both at biomarker and clinical outcomes. So -- and these are at 216 weeks. So you do need to treat for a certain amount of time. Right now, we are really encouraged about how enrollment is going, and we hope that we will be completed by 2026 Q1, and that's really our goal because it's a very large trial. It's a 1,400 patient trial. And as you can imagine, it's hard to "nd patients who are not symptomatic. But I think very good progress. Eisai is the lead, and they've really done a magni"cent job here. # Terence Flynn Analyst Okay. Great. # Priya Singhal Executive So very exciting. # Terence Flynn Analyst And that 1Q, '26 would be for enrollment completion and when would be the earliest that we could see data potentially? # Priya Singhal Executive We haven't commented on that. We're looking at all possibilities. We'll tell you more as it becomes clearer, but we're looking at many di!erent options. ### Terence Flynn Analyst That would include like an interim potentially? ## Priya Singhal Executive It's always possible. We just haven't communicated on whether it's going to happen. ## Terence Flynn Analyst Okay. Okay. Great. Maybe just in the interest of time, just moving on to -- the other one I wanted to cover is felzar- felzartamahab, which I can't pronounce, HI-Bio asset. ## Michael McDonnell Executive Felzartamab. ## Terence Flynn Analyst Obviously, one of the higher pro"le BD deals aside from Reata that you guys have done here. Maybe just one debate out there is the pros and cons of going after CD38 versus BAF in April, such as the Vertex, Alpine asset. So why do you have the con"dence that this approach is maybe going to be the better approach here? ### Priya Singhal Executive Yes. So I think stepping back, we believe that felza was a real compelling option for us. And the reason is that anti-CD38, what we saw in the data is that it's very speci"c and selective. So speci"cally for a disease like IgAN, what it ends up, what the data demonstrate is that after dosing for 5 months, we saw reductions in IgA right up to 24 months. So this is a patient population that can potentially have like a break from treatment. So that's one implication. The other, though, that's also equally important is that the levels of IgG and IgM spring right back up at 6 months. And that's important because we believe that the anti-CD38 mechanism of action is so selective and speci"c that it's addressing the plasma cells that are responsible for releasing these auto antibodies, but it's preserving the rest of the B-cell lineage. So you have this balance because in these patients, while you need to immunosuppress them to get e\$cacy, you always want to balance it in terms of safety and bene"t. So from our perspective, it's a very good balance on bene"t risk. So there are several advantages that we see. One is the bene"t risk, the second is the durability and the third is the selectivity and speci"city. And we believe that this kind of mechanism of action could be relevant in several other disease indications beyond the three that obviously HI-Bio has prioritized. So we're looking at getting those Phase III trials up and running, but we're also looking very carefully of what else could we do to bring to patients. ## Terence Flynn Analyst Okay. Great. Maybe just in the last minute because I know that was out this morning, the SPINRAZA high dose data. Just any comments on how to think about that relative to low dose? And then is that likely going to convert the market over to high dose or both options are going to coexist. ## Priya Singhal Executive Yes. So very excited to release that data this morning. This is the DEVOTE trial and the pivotal cohort is the Cohort B where we looked at infantile onset SMA. And what's important to understand is that this is a population now that has several options. So we didn't -- to "nd naive patients wasn't easy, but our team did a fantastic job in getting the trial recruited despite COVID and all of that, and we have some very good results, statistically signi"cant on CHOP INTEND. That is a very important outcome. Now with regards to whether we would replace and these are discussions we'll have. But right now, what we are going to be working on is really preparing this package for registration because we intend to "le and it also reduces the number of injections that a patient has to receive because it's 250-milligram loading doses, 14 days apart and then 28 milligrams every quarter. and we saw results in neuro"lament as early as day 64 over the 12 milligram, which is already very compelling as we've shown with RESPOND and other data. So very exciting news. # Terence Flynn Analyst Okay. Well, I think we're up against time. But Mike, Priya, thank you so much for joining us. Really appreciate the comments. # Michael McDonnell Executive Thanks for having us. Priya Singhal Executive Thank you.
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