Okay. We're going to get started with the next session. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies. Thanks for joining us, and it's my pleasure to have the Savara team with me. To my direct left is Braden Parker, Chief Commercial Officer, then to his left is Matt Pauls, Chairman and CEO, and to Matt's left, Brian Robinson, Executive Vice President, Global Medical Affairs. Is that correct? Thank you for joining me today, team. There are some people who may be less familiar with the Savara story, so can you just give us an introduction about the company, talk about MOLBREEVI, where you are at, milestones over the next 12 months, and then we can go from there. Yes. Thank you, Andrew, and thanks to Jefferies for the invitation to participate. We appreciate it. I'm Matt Pauls, Chair and CEO of Savara. Savara is a orphan rare disease company, single asset, MOLBREEVI, which is a novel inhaled biologic for being currently under review at the FDA, for a rare lung disease called autoimmune PAP. It's autoimmune pulmonary alveolar proteinosis. We will refer to it as APAP for the remainder of the presentation. It's an autoimmune disease, lifelong chronic disease, that Dr. Robinson will talk in more detail about. Suffice it to say that it is a disease at a very basic level of surfactant burden and surfactant buildup in the lungs, which causes pretty significant issues related to breathing, quality of life, and given the fact that it is autoimmune, that it is more often than not a lifelong disease. Currently, in the U.S. and in Europe and the U.K., there is no therapeutic approved. There is a rescue procedure of arguably last resort called whole lung lavage, and when patients, their dyspnea and breathing and quality of life get significantly bad, if they're able to find a physician and an institution that would be willing to effectively power wash their lungs to mechanically remove the surfactant for arguably, again, short-term relief, then they may elect to undergo that. That rescue procedure, whole lung lavage, is surely not the answer to a chronic therapeutic approach to addressing autoimmune PAP. Most recently, we reported that we had $203 million on the balance sheet, and in parallel to that, we've also, from a cap stack, cap structure, capitalization strategy perspective, have, upon FDA approval, a royalty agreement with RTW that would be a $75 million capital infusion. We've also, in parallel, arranged a $75 million debt facility at our discretion with Hercules. Up to $150 million in non-dilutive financing upon FDA approval. PDUFA date is November 22nd, so we're within the six-month window. We have a high level of confidence and conviction in path to approval in November. It's a very exciting time. Great. Thank you. Maybe to start framing the market opportunity for this indication, remind us the total addressable market, how many patients are there in the U.S. alone, and why are you convinced in that number? Yeah. Thanks for the question, Andrew. When you look at the literature, the epidemiology and the literature, you get a wide range, from six patients per million all the way up to 26 patients per million. We obviously wanted to put a finer point on that, so we've done, over the years, some claims database work, and most recently leveraging the Veeva database, which represents over 300 million lives, over 100 billion records with open and closed source data. We're fortunate in that there is a specific ICD-10 code for PAP. Autoimmune PAP represents 90%+ of all PAP. When you look through that data set, we identified 6,100 patients with a PAP code, so about 5,500 patients when you take the 10% haircut that have autoimmune PAP in the U.S. We feel very confident in that number for a number of reasons. First, every patient was tokenized within the data set, so we know there's no duplication. Secondly, they had to be active in the system within a recent period of time, so some type of claim had to come in, so we know that they're alive. Finally, to reduce any kind of miscoding error, we had a number of clinical criteria that we applied to the data set as well, leveraging a third-party company, and that gives us great confidence in the 5,500. Now, in the context of the published literature, that would represent about 16 patients per million, so right in the middle of that range that I noted earlier. That, we believe, is the floor. Now in the U.S. market, and we'll see how high it goes from there. Great. I ask you every time too, on price, just to make sure nothing's been changed. What are you thinking about the pricing bookends on a net basis? Yeah. I appreciate the question every time. I will give you a similar answer, which is we've identified a pricing range of $400,000-$500,000 per patient per year for this product. Within that range, we have a high degree of confidence of coverage and coverage to typical prior authorization criteria. No real concern about budget impact from payers. We feel that that is a nice sweet spot for this product to extract value, but also to gain access for patients. Great. Just to double down on the ultimate use case of this product. People can understand if a patient's moderate or severe, they PAP. The question is mild. Why would this be used in mild patients as well if it was approved? Yeah. I'll take that, and I'll ask my colleagues to opine as well. I think what we've heard from physicians, and most importantly from autoimmune PAP patients, is that if a patient is symptomatic enough to have been or are currently diagnosed with autoimmune PAP, even if deemed mild from a symptomatology perspective, they're impaired enough that they went on the journey to figure out what was going on. This is a disease that clearly can kind of wax and wane. What doesn't change is when you're diagnosed with autoimmune PAP, you're diagnosed with autoimmune PAP. Let's talk about the patients that maybe at this moment in time have milder symptoms, right? Maybe their dyspnea has stabilized. It's still impaired, but it's not getting worse for a short period of time. What, again, feedback we've gotten from patients as well as physicians is what they're very keen to do is to try to do everything they can to prevent patients from going from mild to moderate. Right? Any patient that has gotten worse, bad enough to have a whole lung lavage, never wants to have another whole lung lavage. I think the way to think about this is that if you can get to patients as early in the course of disease as possible and hold them there, that is a great thing. That's why, again, the feedback from physicians and from patients is that even if deemed mild, they want and should be considered for starting on MOLBREEVI as soon and as early as possible. Makes a lot of sense. Yeah. Maybe talk about the ultimate compliance discontinuation rate of this product. For instance, what did you see in the phase III studies on those rates? You also just shared open-label extension data at ATS a couple of weeks ago or so. Maybe talk about how you're thinking about those rates, because that can affect sales each year. Absolutely. Let me give a little framework, and then I'll turn it over to Dr. Robinson. When we launched IMPALA-2, first patient in was in June of 2021. We launched a rare lung disease global phase III trial right in the midst of COVID. Daring, and/or maybe fortunately, it worked out really well. At the time, it was a little nerve-wracking. We fully enrolled it on time, actually over-enrolled it on time during the pandemic. We targeted 160. We enrolled 164. A 48-week double-blind placebo-controlled trial. In the midst of the pandemic, we over-enrolled this rare lung disease trial where patients raised their hand and wanted to be included in a 48-week double-blind placebo-controlled trial where they had a 50/50 chance of getting nothing for a year. Right? With the hopes that at the end of that, they would be able to get MOLBREEVI in the open-label extension. Of the 164, 159 completed the 48-week portion of the trial, all 159 or 100% of those patients rolled over into the open-label extension. Brian, do you want to talk about the open-label extension? Yeah, sure, Matt. I just want to go back to one question that you had, Andrew, regarding mild patients. One of the things that's important to consider is that by nature, autoimmune PAP puts patients in an immune-compromised setting. Because of that, patients are susceptible to opportunistic infections. This is yet another reason that's important to treat mild patients. I just think that's an important point to be made. Understood. To answer the question about the open-label extension, as Matt said, 159 patients went into the open-label extension period. We just reported at ATS last month the first 48 weeks of the open-label extension, where those 159 patients e ntered that phase of the study. In that study, there were nine patients that dropped out of the study. It's important to note that these nine patients dropped out not because of treatment-related adverse events or lack of tolerability. There were a variety of reasons why patients dropped out, including patients moving, and things of that nature. The retention rate is significant, and I think it's because patients feel better and do better, and are able to breathe. That's a key part of what happened, what we've seen in the first 48 weeks of the OLE. That's great. That's two years for some of these patients. That's right. Okay, very good. Now, the application is being reviewed by the FDA. You recently got an extension from the FDA fairly early on post-acceptance. What exactly happened here, and why should we feel that your application is okay and will be approved ultimately? It was a very general correspondence from the agency, just stating that they were giving us a three-month PDUFA extension. There was no comment around safety, efficacy, or manufacturing. Also just very generalized direction, and that was it. Wow, okay. The positioning is, it sounds like it's a FDA bandwidth situation. It's not something related to If it's not safety, efficacy, CMC, what else could it be? Is my line of questioning. Yeah, no. Fair question. I don't know. All I know is that there was no mention of safety, efficacy, nor manufacturing, one, I can't, nor I shouldn't speculate on bandwidth, et cetera, with the agency. I do think when you look at precedent analysis, that over the last few years, especially with applications that have Priority Review, the six-month review versus a standard 10-month review, it hasn't been uncommon for there to be a three-month- Yeah PDUFA extension. Okay. No problem if you don't want to answer it. I do get asked, so I'm just very curious. Absolutely. It was triggered by an information request by the FDA. What was exactly that request that triggered this whole ordeal? There wasn't anything named specifically that was the reason for the three-month extension. It really was just a very general communication that said, "We're going to need more time to review the application, and so therefore, we're extending your PDUFA date by three months." That was it. Thanks. You're welcome. Okay. At the same time though, as it's being reviewed, we're not necessarily out of the woods just yet, even though the precedents would support an approval. Like if the FDA wanted to reject it, I think they could've waited for the original PDUFA and issue a CRL for instance. I don't know. Anyway, CMC side of things, sure, you got it. There was an RTF originally with the first supplier, but you've essentially resolved it, at the same time, there is still going to be inspections with the second supplier. How should we feel, or why should we feel confident that CMC inspections will be okay for this review? Yeah. I think the way to think about this is look again at the journey over the last year, coming out of the RTF, getting alignment with the agency around the analytical comparability protocol was step one. We did. Two was then produce the data to support the analytical comparability protocol with Fujifilm, which we did. That then supported the BLA resubmission, because but for that, we wouldn't or couldn't have resubmitted the BLA. We did. It was accepted. It's under active review. I think as a reminder on this tech transfer process, while it's a biologic, it's a pretty elementary kind of basic biologic and tech transfer process, non-glycosylated. It's the same capacity. It's not a scale-up. It's really just taking the same process and same capacity and moving it to Fuji. They're a world-renowned partner, and they have a lot of experience in tech transfer, and we're at the biologic center of excellence for them in the U.K. We have a high level of, again, confidence that we have a path to approval in November across the application, the BLA that we submitted. Yep. Then, at the time of the acceptance earlier this year, did the FDA happen to confirm no AdCom is needed explicitly? Yeah, they did, and we disclosed that previously that they did express that there was, at that time, no plans for an advisory committee, and that was when they accepted and filed the BLA. Great. Come November, let's just say it is approved, which I think will be the case, what are you hoping the front page label to read specifically? The front page label claim, is it for APAP, full stop? Not a skinny label by any means. Is that the expectation? We anticipate that it'll be for the treatment of autoimmune PAP. Yep. As I think about the precautions, safety section, is there really much to mention on that side of things too? Fortunately, for us, and most importantly for patients and physicians, is that with the large body of data now, based on IMPALA-2, which is the largest autoimmune PAP trial in the history of autoimmune PAP, and then the supportive trial, the IMPALA-2 trial, the really proof of concept phase II/III trial. If you look, there's no safety signal, and tolerability is remarkably good, and I think that is evidenced by the retention rates of patients staying on drug and in both trials. Yeah. Let's just say again, you're approved in November, when are you prepared to launch specifically? Yeah. If you think about timing, the end of November, you're into the end of the year, holiday period, et cetera. We're really orienting everyone around drug being in the channel and kind of getting the engine turned on in the first quarter. Like every other orphan rare disease launch, especially a first mover like MOLBREEVI in autoimmune PAP. That first quarter, you're adjudicating patient start forms, prescriptions, you're working really on a manual basis of getting patients through the payer and reimbursement process. The goal here is you launch in the beginning of the first quarter, and then the second quarter, you're hit with great momentum. Yep. You used to share a nice data point on how many patients are ready to go. You called it direct line of sight and it was 1,000 as of December. You've mentioned that it would be significantly higher by the time you're approved. If I did 1,000 on a $400,000 net price, that's already $400 million in year one. Understood that Q1, you're adjudicating. Should this be a bolus in Q2, Q3, Q4? Yeah. I'll have Braden comment, but let me set this up a little bit. I think the way to think about-- Line of sight was in response to the buy side suggesting that while the ICD-10 code and claims database work that we did is helpful, there was recommendation that we do confirmatory work around those patients. We set an arbitrary number of 1,000. We went out and confirmed them. They're there. Our conviction and confidence in not only a path to approval in November is high, but our conviction around the market, and the market size is high. We've already brought on our 23-person sales team. They're out every day finding patients, getting them ready, and doing market development work. Obviously, market development work until approval, so non-promotional, but just doing general market development, disease state awareness work. It's very clear in conversations with physicians that autoimmune PAP patients are being diagnosed, and they're in dire need of some approved therapeutic. Braden, do you want to comment? Yeah, I think you stated it well. We have a high degree of confidence in the market opportunity from the claims database work. The way you operationalize a launch is you take those data and you leverage it as targeting information, cutting territories. As Matt mentioned, we have 23 rare disease specialists on board already, and they're out there profiling accounts. They're knocking on doors. They're confirming that the patients and the claims that came in from a location, the patient's still being actively managed at that location by that physician, or they may be at a different location, or being actively managed by a different physician. With the time that we have now between today when the field force came on and potential approval in November, they're doing that work such that when, knock on wood, if we're fortunate enough to have approval, they'll know exactly where to go in the early stages. It's important to remember that, and Matt's mentioned this, that there's a process of patients coming in to get assessed by their physician again, by the prescription actually being written, and then adjudicating that claim. Early on in the launch, there's no policies in place. You're doing that on a very manual basis. There's going to be some time between when patients are identified and prescribed to when they actually get on drug as well. That's part of the process that you'll start to see in early 2027 and beyond. Okay. I looked at the consensus numbers for 2027. I think it's like $70 million. Maybe that's 150 patients if they started on day one, but it's not going to be the case. My question to you is, let's just say 300 patients were needed in 2027. You feel very confident about that? I think consensus, which you highlighted, we think is a manageable expectation, and I'll leave it at that. Very good. Bigger picture, are there any precedents or launch analogs you've been thinking about that could be similar here of a situation? On a non-specific basis, the answer is yes. This is a team, this group of people and many others on the Savara team. We are firm believers in precedent analysis, and keeping our eyes and ears open about what has worked and what hasn't. There have been numerous recent orphan rare disease launches that we've taken great learnings from. For example, I think the way to think about it is what will be the pre-launch metrics that we're going to talk through, talk about, what are going to be the post-launch metrics. We're being very thoughtful about what those are and when and why. We appreciate that the market is going to want and need some metrics to be able to anchor on. I also feel strongly that we need to be really choiceful about what direction we point everybody in, and pace and pulse that over time. Sure. Thank you. Interestingly, as we think about ex-U.S., I think in the U.K. side of things, the MOLBREEVI could be approved in Q4 of this year as well. I think EU, it could be approved in Q1 2027. Do you intend to launch in those regions by yourselves in 2027? The EU and U.K. strategy at a very high level is given some of the policy-related issues that our industry is dealing with, for example, MFN. The point of departure is really do no harm to the U.S. launch opportunity. In parallel to that, it's very critical that we continue to move the MOLBREEVI applications forward in both Europe and U.K., which we are, and because there are a significant number of autoimmune PAP patients in dire need of a chronic therapeutic like MOLBREEVI in both of those geographies. We'll continue to drive them forward to approval. In parallel to that, do the baseline work, especially around some of the HTA prep that we know will be valuable regardless of the MFN clarity or lack thereof that may be forthcoming. We'll sort out timing related to launch once we have additional clarity. We do intend to launch in both the EU and U.K. on our own, because we can. We have a team that's been there and done it. We can do it in a very capital and labor efficient way. Timing is still to be determined based on, again, clarity around or additional clarity around the policy related issues. Yeah. Okay. Bigger picture strategy question, life cycle management for MOLBREEVI. Any intention to expand to other indications or any intention to bring something from the pipeline and introduce that as you're launching? Life cycle management, we're keeping the market as well as the organization squarely focused on autoimmune PAP. We have a ton of opportunity, huge opportunity, and a ton of work to do related to autoimmune PAP. That doesn't mean that we're not evaluating other potential opportunities around life cycle management. It just means that right now, when you think about capital deployment as well as energy focus, it's about autoimmune PAP. The second part of the question is stay tuned because success with MOLBREEVI in autoimmune PAP is going to create great optionality for the organization. We must be successful, and we will be with MOLBREEVI. Great. Thank you very much for all the updates, and good luck on the PDUFA later this year. Thank you everyone for listening. Thank you.