Good morning, everyone. Welcome to Oppenheimer's 36th Annual Healthcare Life Science Conference. My name is Andreas, analyst at Oppenheimer, and today I have the pleasure to be joined by the management team, Matt Pauls, Chair and Chief Executive Officer, Braden Parker, Chief Commercial Officer, Brian Robinson, Executive Vice President. Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases and is developing molgramostim inhalation solution known as Molbreevi .
It's a little bit easier to say, an investigational inhaled biologic, specifically an inhaled granulocyte-macrophage colony-stimulating factor GM-CSF, currently in FDA regulatory for autoimmune pulmonary alveolar proteinosis, known as aPAP. Again, another mouthful that we can shorten with a good abbreviation like aPAP. Molbreevi is delivered via a proprietary eFlow nebulizer system. Great to have you guys here today. Maybe let's first start with an overview of the company and the most recent progress, and then we can dive into some additional questions.
Sure. Thanks, Andreas, and on behalf of my colleagues, Braden and Brian, thanks to Oppenheimer for the invitation to participate. We're glad to be here. It's an exciting time at Savara. It's, you know, we just recently announced last week that the FDA accepted the BLA and also filed it, and also assigned priority review, and PDUFA date is projected as August 22nd, a six-month review. A very exciting time. Molbreevi, as you noted, is on the heels of the positive phase, global phase III registrational study in IMPALA-2, for Autoimmune PAP, is now under review by the FDA. Again, the proverbial home stretch, so to speak. We're very, very excited.
We'll talk in detail, more detail about Autoimmune PAP and the unmet need, which is significant. It's very serious long-term, chronic, rare pulmonary disease. Savara is a single asset company, built, focused, and fully valued around Molbreevi, and we're looking forward to driving the program forward and are confident that we have a path to approvability in the U.S. In parallel to that, by the end of March, we will file the MAAs in both EU and U.K., so we'll be driving forward in both of those geographies as well, in parallel to the U.S. I'll pause there.
Yeah. Fantastic. Thank you. Again, congrats on the filing for Molbreevi and aPAP and getting the priority review. You mentioned the August 22nd PDUFA date. Can you highlight for us the key components of the BLA, such as clinically meaningful and positive phase III data that give you confidence for approval?
Absolutely, I'm happy to do that, and I'll ask Dr. Robinson to take that question.
Yeah, sure. Thanks, Matt. Molbreevi addresses the cardinal manifestations of aPAP, which are impaired pulmonary gas transfer, poor respiratory quality of life, reduced patient function, functionality, and surfactant burden. You know, we feel that our IMPALA data show improvement from baseline in DLCO, SGRQ, exercise capacity, and surfactant burden as well. When you take all of this together, it gives us great confidence in our data package that supports approval.
Okay, fantastic. I was just checking for questions real quick if they came in as we started. Can you walk us through the unmet need in aPAP and current treatment burden, and how Molbreevi could potentially become the first approved in the U.S., and or Europe?
Yeah, happy to do that. Again, I'll ask Brian to comment, before he does, I think it's important to note, as you just highlighted, in the U.S. and Europe and U.K., there is currently no approved therapeutic whatsoever. The Autoimmune PAP community and ecosystem have nothing that's, you know, an approved therapeutic to help address the pathophysiology of the disease. Brian will highlight the fact that there is a really a barbaric mechanical procedure that's used as a rescue procedure, Whole Lung Lavage, and I'll ask again, Brian, to describe that in more detail.
That's a, you know, reserved for patients who are in really, you know, often in really bad condition. What we need to do, we're confident that based on the data with Molbreevi , is you need a chronic therapeutic that helps turn on the gas signal to help, you know, again, address, the core biology and pathophysiology of the disease. Brian, over to you.
Yeah. Thanks, Matt. Just to piggyback on what Matt said, you know, the unmet need and disease burden of aPAP is really defined by the fact that the only available therapy is this procedure that Matt was talking about, Whole Lung Lavage, where patients are taken to the operating room, they're intubated, they have while one lung is being respirated, the other lung is being washed. The thing about this procedure, though, is that it's only available in select centers. It requires hospitalization, OR, and ICU time. It's not standardized at all, and as Matt said, it doesn't address the underlying cause of disease. As he also says, it should be reserved as a rescue procedure.
I mean, if someone comes in, and they're really incapacitated, you know, it may make sense as a rescue procedure in that setting. The difference with Molbreevi is that it does address the pathophysiology of aPAP by allowing the activation of macrophages. This activation of macrophages allows the maintenance of surfactant homeostasis. This improves gas exchange.
Okay, great. Can we dive into the market potential for Molbreevi now, and based on, you know, updated U.S. claims data, how many patients are there who can benefit from Molbreevi as a treatment that addresses the.
Yeah, before Braden , our Chief Commercial officer, comments, I think it's important to note again, you know, this is a rare disease category that, prior to, you know, Savara's, you know, investment in the category, there's been nothing. There's been no, you know, no sponsor that has, you know, has the capital and the wherewithal that's been involved to help develop and shape the market. In sort of the past couple of years, you know, Savara, we have done just that and are really starting to see the market become much more structured in the U.S. and, you know, in anticipation of potential approval for Molbreevi . Turn it over to Braden Parker.
Thanks, Matt. Let's talk market size first. If you look at the published literature, the epidemiology and the published literature, you get a pretty broad range, which is not surprising for a rare disease, of six to seven patients per million, all the way up to around 26 patients per million. We wanted to obviously put a finer point on that, so we've done some claims database work and analysis. Last year, with that work, identified roughly 5,500 patients in the U.S., that are known and diagnosed, if you will, and that's about 16 patients per million, so right in the middle of that published range. We believe that that is the floor, really the new floor, for the U.S. market.
How high it goes, we'll see as we continue, as Matt said, to invest time, money, and effort in that. In terms of who can benefit, I think Dr. Robinson said it best. I mean, this is an autoimmune condition. The sooner you turn on the signal and reduce the surfactant burden, the better. We believe that all 5,500 patients could benefit from Molbreevi upon approval. You know, we've seen that both anecdotally in conversations with thought leaders, as well as the research that we've done, where physicians say that they will offer Molbreevi to all of their patients, regardless of disease severity.
I think it's important to note, too, that it's logical, right? If, you know, given the fact that aPAP, as an autoimmune disease, is a lifelong chronic disease, right? It's, it's clearly something that, you know, is a disease that needs to be addressed early on in the course of the disease. What's very important is that patients have an option around a chronic therapeutic, even in a mild state early on in the course of the disease. Hence the reason that KOLs and in the research that we've done, are suggestive that, you know, even patients that are deemed mild, you know, early in the course of the disease will be eligible, you know, candidates for Molbreevi .
Fantastic. Now thinking about launch preparations, can you walk us through the size of your commercial field force and your plans to target the key healthcare professionals? How many aPAP patients do they usually see that you plan to target?
Let's talk targeting first, right? Because one certainly relates to the other in terms of field force sizing. The aPAP market in the U.S. is pretty concentrated. If you look at the claims database work that we've done, the top 500 accounts manage roughly two-thirds of the patients in the market, the claims data would suggest. We can be very efficient in terms of field force sizing and our targeting efforts. We are looking at roughly 30 customer-facing folks in the field. That's inclusive of leadership. We'll eventually add a handful of field reimbursement folks as well.
In terms of the sales team, if you will, we're looking at roughly 30-ish or so. Because of the confidence that we have, in the filing and the application, the strength of the clinical data, we're looking to bring those folks on in the second quarter. There's some time for them to be in the field doing disease awareness and testing education prior to a potential approval.
Okay, great. You recently selected PANTHERx Rare as the U.S. exclusive specialty pharmacy. Can you discuss this milestone and other pre-launch activities, such as awareness campaigns and education and early testing that you conducted to prepare for the commercialization of Molbreevi ?
Over to you, Braden.
Sure. We're very excited about PANTHERx . They are an extremely experienced independent specialty pharmacy that has relevant experience for us. First of all, they are only focused on rare and orphan, as we are. They have relevant experience as exclusive specialty pharmacy. Many of the relationships are similar to the relationship that we have with them in terms of exclusive pharmacy. They have pulmonary relationships with pulmonary offices already. That's gonna behoove us as well as we get to approval, potential approval and launch. Then finally, they also have experience with the device with other customers that have similar technology as well.
For all of those reasons, in addition to the fact that their customer service scores are really unprecedented, they are the perfect partner for us and the launch of Molbreevi . As you might imagine, as we prepare for commercialization, we're in the midst right now of working with them on pharmacy programming. In addition, they're going to be working with us on our patient services side as well, so providing services to both patients and providers to help manage the mechanics of the U.S. healthcare system and reimbursement, obviously, as well. We're in the midst of all of that work.
As I mentioned, additionally, we're looking to bring on the field force early, so in the second quarter, so they have some time to continue profiling work, as well as, looking at line of sight for patients and, doing the disease awareness campaign and testing education. Finally, I'll mention on the testing front, we have an ILD clinic pilot program down at the University of Florida, and we would certainly be looking to expand that program to other ILD clinics around the country prior to approval as well.
Great. Fantastic. Then maybe a financial picture question here. How are you guys positioned from a cash perspective, as you plan, you know, you prepare for launch?
Yeah, thanks for that question. Last report, balance sheet of $264 million in cash. In addition to that, we have arranged upon FDA approval, a two-part non-dilutive, you know, structured capital infusion. One piece is a royalty agreement with RTW that would provide for a $75 million cash capital infusion upon FDA approval for a tiered royalty structure. In parallel to that, we also have a debt facility at our discretion with Hercules that up to $75 million upon FDA approval.
Overall, in, you know, the August time frame, around PDUFA, assuming FDA approval, we would have access to up to $150 million in non-dilutive financing above and beyond the strong balance sheet that we have. The company, while we haven't provided, you know, more specifics with regard to cash runway, it's safe to say we're funded for a very long time, in great shape.
Yeah, fantastic. It sounds like it, and you guys have made great progress, and this seems to be an exciting year for the company here. Again, congrats on a priority review. Hopefully, this is a good thing for patients. I'm sure they're all very excited as well. Okay, look, with that, you know, I really appreciate the time. It's, you know, it's good when stories are as straightforward as Savara's. We'll continue the conversation and really appreciate all the insights tonight and answering our questions, guys. Thanks for the fireside chat.
Great. Thanks very much.
Thank you.
Take care.
Thanks.