I think we're ready to get started here with the next session. For those who don't know, I am Vamil Divan, one of the Biopharma Analysts here at Guggenheim, thanks for joining us this year for the Emerging Outlook Biotech Summit. Multiple times we've done this conference. First time it's been called this, actually. I guess it's the first annual summit. Next up in this room, we have the Savara team. Next to me, we have Matt Pauls, the Chair and CEO, Braden Parker, the CCO, and then Brian Robinson, EVP for Global Medical Affairs. Maybe I'll just turn to you, Matt, especially for people maybe not as familiar with the Savara story, a little bit of an overview. Then we can talk about, obviously, a very important few months coming up here. We'll dive right into it after that. Just an overview would be nice to start. Sure. Yeah, thank you, Vamil and thanks to Guggenheim for the invitation to participate. We're glad to be here. Savara is a single asset, rare disease company focused squarely on orphan rare pulmonary diseases. Our asset, our late-stage program, is MOLBREEVI. It is being studied for, was studied for, and will be applying for approval—in the process of applying for approval—for a rare lung disease called aPAP, or autoimmune PAP, autoimmune pulmonary alveolar proteinosis, which we will talk in more detail about. We recently, at the end of December, resubmitted the BLA and expect, in the next couple of weeks, to hear from the FDA. We are confident that the BLA will be accepted and filed. We are also. Our base case is, given the fact that MOLBREEVI has a Breakthrough Therapy Designation, that we will also then be. MOLBREEVI will be assigned priority review and that would put PDUFA in the August timeframe. So yes, a very exciting time for Savara, but really, most importantly, for the autoimmune PAP community here in the United States. I will also mention up front that we have guided to and will hit the filing of MAAs in both Europe and UK by the end of the first quarter. So that's on track, and that will happen and more to come there. OK, great. So thanks for the overview. So maybe just going back a little bit, obviously, you filed early last year, got a refusal to file, and now have refiled in December and hopefully, it sounds like things are on track, hopefully, to get it accepted this time. Can you just sort of talk through? I know it's more of a manufacturing-related issue there, and you've changed primary manufacturers now. So maybe just the process there and the decision to make the change would be helpful to go through. Yes. So in May of last year, an RTF surprisingly was received and at that point, it was the single reason for some in-process manufacturing data that the agency wanted prior to accepting the filing. We thought that it could be something that could be solved during the review period. But nonetheless, we then met with the agency, had a Type A meeting, and gained alignment at that point around our move to an alternate drug substance manufacturer, who we had been in process of a tech transfer about 18 months at that point, in process to Fujifilm and we got alignment on the analytical comparability protocol with Fuji, and then moved forward, produced the data, and resubmitted in December. So we have resubmitted with Fujifilm as our primary drug substance manufacturer. OK, and I guess we naturally get a lot of questions then around Fujifilm and the sort of shift to them as the primary manufacturer, especially around inspections or what the process now is to ensure no issues from that side. So maybe you can just talk through what do you expect from here, assuming acceptance and the review continues. What are the next steps on the sort of manufacturing inspection side? Yeah, so it's very difficult to speculate once an application is accepted for review on what the process may or may not be. It's just very program and division dependent. If, in fact, there's any type of PAI, pre-approval inspection, that's normally a late review cycle event and it's just, again, it depends on the program, the division, et cetera. So we're very confident in the resubmitted BLA and moving it forward with Fujifilm as our primary drug substance partner. Ok and now maybe we could talk about the data. Obviously, very strong phase III results you delivered, published in the New England Journal. But in this environment, I think we've got a lot of questions around the FDA and kind of regulatory interactions. So maybe you can just speak to the clinical package that you have for the program and your conversation with the FDA, especially around DLco as the primary, and then also some of the secondary endpoints that you looked at in the trial and what you needed to show and what you showed. Yeah, so I'll make a couple of comments up front, and then I'll have Brian, Dr. Robinson, comment as well and so I think the way to think about this is with DLco as a surrogate endpoint and as a first mover in orphan rare disease like autoimmune PAP, first application, it's also important, critically important, that in the key secondary endpoints, that you show clinical benefit as well. So to see SGRQ, total SGRQ activity, as well as exercise tolerance tests via the exercise treadmill tests produce robust results in the IMPALA-2 study was very important. To see DLco when stat sig at 24 weeks, which was the primary endpoint and then a key secondary endpoint was DLco at 48 weeks to maintain that durability of effect and statistical significance of 48 weeks, again, very important. So Brian, do you have other comments? Yeah, I mean, I think that was great. Matt outlined the primary endpoint and the key secondary endpoints. But I think what's important to think about is DLco as a primary driver from a clinical perspective and why it's important and I think it's probably important for three or four reasons. First, it's really well studied, and it's a really important clinical application. It's used clinically to not only help diagnose autoimmune PAP, but also monitor treatment and to look at sort of disease progression. So it's important for that reason. It's also an important measure of the impact of surfactant burden. Because DLco, which stands for the diffusing capacity of the lungs for carbon monoxide, it looks at how well gas permeates the alveoli and gets into the bloodstream. So if you have surfactant blocking it, this is a direct well, actually, it's an indirect measure of that. But it really gives a sense of what measures that clinical impact of surfactant. I think a third reason is that it also it's really more of a responsive marker, a pulmonary function test, than other pulmonary function tests for patients who have autoimmune PAP, who are treated with GM-CSF. So that's an important thing to look at and then I think lastly, it's also in addition to being used clinically, it's really easy to use, and it's reproducible. It takes about 10 seconds to perform the maneuver. In our clinical trials, all of our clinical trial sites were given the same machine. They learned from the same manual and so it was really perfect as an endpoint for a large clinical trial like this. OK, great and again, the question we get a lot from the investor side is on the secondary endpoints beyond DLco and you showed benefit there. I guess, was there any sort of bar that you feel in terms of what's clinically meaningful, what the FDA maybe was looking to see on these sort of key secondary endpoints? Yeah, I think the way to think about it is an SGRQ. Brian can opine on this is looking to the MCID, for example, in SGRQ for COPD. Do you want to talk about that? Yeah, sure. So while SGRQ hasn't been validated in autoimmune PAP, it has been validated in a variety of pulmonary diseases that are very closely related to autoimmune PAP, COPD being one of them, LAM being another one. So it sort of fits in that sort of sphere and particularly in COPD, the MCID, or minimal clinically important difference, like what people feel is clinically meaningful, has been found to be four points, so reduction in SGRQ of four points and we saw that in SPADES in both our secondary endpoints, when you look at SGRQ total score and when you look at SGRQ activity score. OK, all right, great. So then I do want to switch over to the commercial side. I think there's a lot of questions around that as well as we get closer to, hopefully, a launch in the August, September time frame. So you recently mentioned that you have reached your goal at the end of 2025 of this line of sight to 1,000 patients with aPAP. So maybe before we get into that detail, maybe you can just talk broadly how you're sort of seeing the market, the number of patients that are out there and then specifically on line of sight, what does that mean in terms of line of sight? And how quickly do you think those patients could get on therapy post-approval? Yeah, I'll ask Braden to comment on that. Yeah, you may recall we had done some claims database analysis work in 2025 and identified approximately 5,500 patients, diagnosed prevalent population in the U.S. So line of sight is really our way of going out and confirming those patients, if you will. So with the claims database work, you get where the claim was submitted from and by whom and so line of sight is profiling those accounts, confirming that patients are still actively being managed at that location, or are they at another location, as well as who's actively managing them. So the 1,000 number, which is roughly close to 20% of the market, the ability to identify or confirm that number of patients with just a handful of folks in the field gives us even greater confidence in the information and definitely tells us that the market is real and underappreciated. ok and then I guess in terms of I know you've not given any formal guidance, but just in terms of how to think about, especially for those 1,000 or more broadly, I think people think small company launching this product, how quickly can you activate those 1,000 patients and get them on therapy, assuming approval in August? Yeah, I mean, this market is fairly concentrated. If you look at the top 500 accounts or so, it represents about 2/3 of the market. So the line of sight work combined with this concentration gives us great confidence that we can get to those patients upon approval and really begin to have those conversations about MOLBREEVI and the appropriateness of therapy early on. I think it's important to note, though, that launches are labor intensive. So there will be some time lag, if you will, between when patients post-approval will come in and see their physician, get reevaluated, and prescribed the product, as well as what I like to call the mechanics of the U.S. health care system in terms of taking those prescriptions, getting them adjudicated, and reimbursed, and ultimately filled as well. So there will be some time lag there as well. But we are very confident there's strong demand for this product, both from the health care professional side as well as the patient side. I think just to add a little bit to that well-stated summary by Braden that our partner that we recently announced, PANTHERx, is the single specialty pharmacy. While it will be labor intensive and it will take time to get kind of the oil running through the MOLBREEVI's Savara engine, fortunately, our partner, PANTHERx, is a tried and true rare disease specialty pharmacy and whom have launched a number of products. So we are looking forward to working with them to help do whatever we can to get MOLBREEVI in the hands of physicians and to patients as fast as we possibly can. Yes, maybe building on that, obviously, if there's no approved pharmacological treatment for these patients, I'm curious if you can just kind of go a little more deeper on what you've done so far and what your plans are between now and, say, the end of the year in terms of building disease awareness. Obviously, you have a diagnostic test that's now available. What are you doing to kind of get people aware of this product coming? Because there is no options for them right now. Yeah, so we've had a disease awareness campaign to physicians now for a couple of years and we launched over the last year or so a patient-level disease awareness campaign as well, so understanding signs and symptoms, encouraging testing as well. As you mentioned, we've had the aPAP ClearPath testing program out there, first as a blood draw and then most recently as a dry blood spot test, four drops of blood on a card, mail it into the lab. Within seven days, you get a response, a yes or no, and the titer level as well. So those are the type of activities that we've been undergoing to make sure that we're continuing to expand awareness of the disease state as well as help rule in or out aPAP for patients and offices. So I know you're not giving quantitative feedback on the testing yet, but just qualitatively, can you talk about sort of how the adoption of the testing has been? And is it that we're finding patients that maybe otherwise would have been misdiagnosed or not diagnosed with anything at all getting captured now through the testing? Yeah, I'll take that. So the answer is yes, we absolutely have identified de novo newly diagnosed autoimmune PAP patients. We've been running a small pilot at the University of Florida in the ILD clinic setting there and it's very clear that, in fact, in the ILD clinic setting, there are undiagnosed patients. It makes a lot of sense given the really nonspecific symptomatology cluster for autoimmune PAP. It looks similar to other lung diseases and in fact, proof positive of that is that we're in the process right now of scaling up the ILD clinic pilot to other sites around the country. So more to come on that. Brian, I don't know if you have any other comments on that. No, I mean, I think you covered it, Matt. I think the whole notion that these patients are hiding in plain sight is clear and the idea that the information that we put forward with time, money, and effort with disease state education, making people aware of this test, and also on the horizon having potentially an approved drug in this space is going to get people really excited and we're going to find more patients. I think also, Vamil, it's important to note if you take the current testing program that we have going, the refreshed claims database work that we've done, and you overlay that on top of the EPI literature, I think this is an important point, that the EPI literature currently has a range of about six or seven per 1 million diagnosed prevalence to mid-20s. The approximate 5,500 in the U.S. puts you at about 15 or 16 per 1 million. So right in the middle of the fairway. So we believe that's now kind of the floor. The real question is, especially when you start to look at we get very enthused about the potential for newly diagnosed patients based on the dry blood spot test. The question is, is the ceiling really 25 or 26 per 1 million, or is it higher than that? Is it x? We think it probably has potential to be higher than that. Ok and as you've, I guess, we sort of touched on this before, but we get a lot of questions on the launch and how to think about the uptake. Is there, with this disease awareness and people getting diagnosed, do you sense that there's a buildup of patients that want to get on already, kind of lining up to get on therapy? Or is it more once the product's approved, then it's a matter of sort of when they come in for their next visit to the doctor, then they'll talk about it? And maybe it's a six-month, nine-month process for them to actually start therapy. How are you sensing what's happening out there? Yeah, and we get the exact same questions probably from many of the same people. Yeah, which it's the question or questions and we appreciate the line of questioning. So I think the way to think about it is there will be this notion of orphan and rare, a bolus of patients. We're not going to get more specific on defining the size of bolus. But there will be a bolus of patients. Then the second question is, what's the shape of the curve for the first 12-24 months post-approval? And then the third question we get is, how big can it be at peak? And so a long answer of yes, there will be patients that will be ready to go at or around or within a reasonable proximity of launch. Again, I think not only about the line of sight work that has already been done to date, but we are bringing on the balance of the field-based team in the second quarter. So they'll be out a few months before potential approval and their job is to raise awareness and you can assume that there will be patients ready to go for therapy upon potential approval. OK, and maybe we can talk a little bit on the pricing side. I know you've it's obviously rare disease, orphan condition. I think you've talked before, sort of $400,000-$500,000 sort of range. Just curious, any additional insights you're willing to give there or just kind of how you got to that sort of range for the pricing? Yeah, we had done some previous work. We had identified a pricing corridor between $300,000 and $500,000 per patient per year, which, as you noted, has since been narrowed to $400,000-$500,000 per patient per year. Within that corridor, we see payers not terribly concerned about budget impact, also willing to cover with typical prior auth criteria. So we feel good about that pricing corridor that we see right now and feel that it will lend itself to a reimbursement environment that will allow us to get patients, a broad scope of patients, product as quickly as possible. Then you mentioned you're on track for filing outside the U.S. this quarter. Maybe you could talk what are your latest thoughts around commercialization, assuming approval in Europe and Japan? Yeah, so again, to reiterate, MAAs will be filed in both Europe and U.K. by the end of the first quarter of this year and our plan is to go it alone in EU4 and U.K. We have a team that's been there and done it. The autoimmune PAP market in Europe and the U.K. is well articulated. We know stocks and flows, reference centers, the size of the market opportunity, just based on diagnosed prevalence, is actually similar to that of the U.S. with regard to patient population. From a capitalization perspective, we're in great shape. We're funded for a long time, for years and that contemplates going it alone, on our own, in Europe and in the U.K. I think it's important to note that given some of the policy-related issues or overhang or lack of clarity, it's really important for us to maintain full control over molbreevi and we are and we can and that's the plan. OK, what's the latest on Japan? Still a wait and see. Again, I think the key there, it's similar to Europe and U.K., is that with looking for more clarity around MFN and those types of issues, that it remains to be seen. But Asia- Pacific for us, that might be a different strategic approach with regard to how we go to market versus Europe and U.K., but still to be determined. OK, and maybe just last couple of minutes here. So, near-term events, so one question we get also, I think it was December 22nd is when you put out the press release announcing the submission. So, I think it's fair to assume by the end of this month, we should hear something one way or the other and that'll be press released once you get that back from the yes and yes. Ok and then other catalysts we should think about sort of post this month, kind of how should we think about the rest of the year? I think we also get questions on, will there be a commercial day or other things to kind of get people prepped ahead of the potential approval? Yeah, still to be determined on commercial day. So stay tuned on that. We haven't made a decision that I mean, clearly, assuming priority review, the catalyst is PDUFA, which would be in the August time frame and we'll continue to provide as much clarity and updates as possible through the process. OK, and you touched on the capital position. You said well funded. Maybe you can just talk through you did the recent financing. Is this kind of fill of a memory? Yeah, latest report pro forma, $264 million on the balance sheet. We also have a royalty agreement with RTW based on FDA approval that there would be a $75 million royalty payment. We also just recently announced that we have restructured our debt facility with Hercules and upon FDA approval, have unilateral option to draw down from obviously nothing to $0 to up to $75 million. So upon FDA approval, at least $75 million and we could choose to have up to $150 million in incremental non-dilutive capital. OK, all right. I think let's leave it there. Obviously, important couple of weeks and important several months ahead. We'll look forward to.