Piper Sandler Healthcare Conference. My name is Yasmeen Rahimi. I'm a Senior Biotech Analyst at Piper. This is our five-year anniversary, Matt, as we're both at this healthcare conference, and I get to host you for a fireside chat. What can I say? An incredible journey over the five years, from enrolling the IMPALA- 2 study, to reading out the IMPALA study, to now getting ready to file BLA, getting commercially ready. Next year, when you and I are going to be sitting here, we can talk about commercial numbers and how launch is going. So these are the type of things that makes me really excited about the job that I have. Like, I feel like a baby growing, sort of the journey. So lots to cover. Matt, maybe a great place to start off is, obviously, MOLBREEVI's BLA refiling is on track here for December.
Yes.
I assume, sort of just to remind us, December is today. So what will be the disclosures around it? And everything seems to be on track. So just give us a quick recap.
Yeah, so thanks, Yaz, for the invitation to participate. We're glad to be here. So the BLA resubmission is on track for this month, and we will hit that for sure. Then we anticipate there's a 60-day review. And at that point, again, we're assuming that the agency will then file the BLA. We have said that we assume that we will, MOLBREEVI will receive priority review, given breakthrough designation. But we'll see. But that's the base case. And assuming that, that would put PDUFA in the August time frame or so. And also, in parallel, in the first quarter, by the end of the first quarter, we will submit the MAAs in both Europe, as well as in the U.K. So by the end of the first quarter.
Okay. And team, so fast forward, it gets accepted. You get your PDUFA date. Do you think at that point you will hear from the agency in regards to whether they would plan on hosting an ADCOM or not? And how are you preparing for it?
So while there continues to be uncertainty regarding ADCOMs in general, we have consistently said and are maintaining the fact that we are planning that there will be an ADCOM if and unless we're told that there's not. That's we just think the smartest, most pragmatic thing to do. And so we're planning for it. But we'll see soon enough.
Okay. I mean, what could be potential questions and topics, if there is one?
Yeah, I think in the past, again, it's not been unusual for an orphan rare diseases where you have a first application for a therapeutic to have an ADCOM. Oftentimes, you have clinical trial designs that are new, novel, and different. And often, again, this is in past practice. It can often be helpful for all involved around an ADCOM. But again, remains to be seen. And we will, of course, do whatever is necessary to continue to move MOLBREEVI through the process.
Great. Now let's maybe talk a little bit about the aPAP market opportunity. I think that you guys reviewed the claims database, recent data sets that basically show that there are about 5,500 diagnosed patients here in the U.S. And I think you guys also said that you have 1,000 at line of sight by the time of the U.S. launch. Help me understand what does line of sight mean?
Yeah, so I'll ask Braden Parker, our Chief Commercial Officer, to provide some color there.
Yeah, as you perfectly point out, we identified 5,500 patients through the claims database work, known diagnosed today. What we're doing now is we have a handful of folks in the field, and they're out there profiling accounts, knocking on doors, and really taking those data and confirming that patients are being managed at the location that the claims data would suggest, and by the healthcare professional that the claims data suggests as well. Oftentimes, someone else may submit the claim than who's actively managing that patient. Or the patient may be managed at a different location, so that's the work that we're doing now, and when we say we have line of sight, those are confirmed patients that are being managed at the location by the physician that we know about.
Okay. That's very, very helpful. And when we look, when we think about out of these thousands of patients that you have line of sight, are they at concentrated centers, or are they distributed sort of throughout the U.S.?
I'd say there's a fair amount of concentration in this marketplace between the major centers of excellence, secondary centers, ILD clinics, large group practices. There is a fair amount of concentration out there. And then as you kind of go further out into the community, you'll find practices that might have one or two patients out there. But certainly early on, there's a concentration enough where we can support a robust launch trajectory.
Team, I know that by the time of launch, you will have a good understanding where these patients are. What are sort of the operational aspects that you need to manoeuver through now that you can work on so that you can seamlessly get these patients on MOLBREEVI once approved?
Yeah, well, I think first and foremost, it's incredibly important to highlight that having folks in the field prior to approval is key. We have the leadership team on board now with the market development team. We're going to add the rest of the customer-facing group prior to launch, probably sometime after file acceptance, which gives them ample time to be in the field, to learn their territories, to meet their physicians, and profile their accounts, and have a sense for where the patients are. Oftentimes, with rare disease launches, you're doing all of that, hiring the field force, and then exploring kind of the countryside, if you will, post-approval. So we'll be well ahead of the game in that respect.
And that really enables the team to then, at the time of approval, to make those phone calls, to educate those physicians, and begin the process of having patients come in for evaluation from MOLBREEVI, and then start the process of prescriptions and adjudication from there.
And how large is the commercial team going to be, and maybe how many individuals at what level have been hired? And then the rest will be hired upon the approval.
Sure. All in, we're going to have probably roughly 30 customer-facing folks. That's inclusive of the leadership team, which is already in place, three regional directors, and then a handful of or a few reimbursement folks in the field as well to help with adjudicating claims and fulfilling requirements by payers.
I think it should also be noted, too, that what's important, and sometimes I think lost in the planning phase, is not only the size of the team, but who. And fortunately, we, Braden and I and others, have been there and done it with regard to commercializing launching rare disease drugs. And it's really important that you recruit and hire people who also have been there and done it, especially in a category like aPAP that, as the first mover with MOLBREEVI, they have to be able to navigate, go out, find patients, and get them started and accelerate that process. Really important that you hire the right people.
In terms of the distribution, I think you guys talk about the white glove service at the pharmacy settings. Help me understand, how do you envision? So script is written, it goes to the specialty pharmacy. How many would you have? And what is that white glove service that will be provided?
Yeah, so in this regard, it's somewhat of a classic orphan drug model. We'll have a single exclusive specialty pharmacy. They will be responsible for adjudicating the claim. Coupled with that pharmacy is the patient services that will support that in terms of helping find financial support if necessary. But it's really a case management type of model where you have a single point of contact that will help the office, as well as the patient, kind of navigate the mechanics, if you will, of the healthcare system.
Okay. And team, what may help us understand the payer mix that exists in aPAP? Like, sort of how much is commercial versus Medicare and Medicaid?
Yeah, diagnosis typically occurs between the ages of 30 and 50. So for that reason, we're anticipating the majority to be commercial payers, probably 60% or so, maybe 1/3 around Medicare and the rest Medicaid. So we'll, of course, continue to refine those numbers as we learn more as we get closer to launch, but that's the going in assumption.
Okay. And then maybe help us also understand, since we're on market access and payers, what work have you done to understand sort of the flexibilities in orphan indication, high-end med need? We've seen quite high prices on the orphan space, like Skyclarys, ViCAT, as good examples. So maybe help us understand, what has been the feedback from payers when they look at a product such as MOLBREEVI?
Sure. Not surprisingly, like other orphan indications, payers don't have a good understanding of autoimmune PAP. When you educate them on the burden of illness, they understand that. They get it. When you educate them on the clinical data associated with MOLBREEVI, they clearly see the benefit there. And they've indicated to us that between $300,000 and $500,000 per patient per year, you're looking at typical prior auth criteria to gain access. No real concern about budget impact given the size of the marketplace. And in fact, we've narrowed that range down in pricing to $400,000-$500,000 per patient per year because we do believe that there is pricing power here.
Recent New England Journal of Medicine article, no treatment alternatives, and really being the first and only therapeutic that treats the underlying pathophysiology of the disease gives us an opportunity to have a strong degree of confidence of that pricing range, as well as our ability to get access for patients.
And what would that prior authorization be? Is it just a confirmed diagnosis of aPAP, I assume? Like, what is?
It could be. There's a range they could maybe look for additional criteria. But I think typically speaking, it's to the indication.
Yeah, and I think if you look at other analogs in orphan rare, you'll see placement in specialty tier. To your point, it's not very onerous in that it's proof of diagnosis or physician attestation. But like most orphan rare disease therapeutics, there will be some amount of work that has to be done through the adjudication process.
Yeah, they're going to want to make sure that it's medically necessary and the right patients are getting the drug perfect.
Is there a reason to assume that any patient who has aPAP should be treated? It's not based on severity, right? Obviously, the longer they live with aPAP, the severer it gets. But do you think there is a need to specify sort of whether they have a certain severity score? I mean, I feel like that was good for the clinical study, but impractical in the real world. Yeah.
Yeah, I think practically, when you think about autoimmune PAP, if a patient is diagnosed with autoimmune PAP, this isn't our opinion. We hear from KOLs and physicians that we talk to in research that if you're diagnosed with autoimmune PAP, you have been sick enough to seek out some form of diagnosis and treatment. And it's often a long, arduous journey, right? Even if your symptomatology is deemed at that moment in time mild, you are dealing with a disease that's probably going to be lifelong chronic given the autoimmune nature of it. And what you're grappling with is surfactant burden. It's very clear that if you don't turn the signal back on to help the macrophages help reduce the amount of surfactant that's building up, bad things happen, right? And for some patients, that happens rapidly. Others, it happens slowly.
But whether you deem mild, moderate, or severe, MOLBREEVI, from what physicians have told us, should be offered to any and all patients.
And then, team, you guys have been working really diligently on antibody testing and then dried blood spot testing that became implemented earlier in the year. Just maybe how is that progressing? What was the advantage of implementing the dried blood spot test versus?
Yeah, so the dry blood spot test that you're referring to is a no-cost autoimmune PAP diagnostic test. Just to be very specific, it's 4 drops of blood on a card that is then sent to a private single laboratory. And within seven days, the physician receives both a yes or no, positive or negative, and a titer level tied to that patient. So 100% sensitive, 100% specific, and being utilized as we speak. This is what we call the long game, right? Meaning the currently identified approximately 5,500 patients, that's the go-to-market to make sure that we have those patients well characterized. And in parallel to that, it's very clear based on the epi literature. At one end, you have 6 per 1 million. At the low end, the high end is about 26 per 1 million. The 5,500 is right down the middle of the fairway.
That equates to about 15 or 16 per 1 million. The dry blood spot test over time is going to allow us to see what the ceiling really is. Is it really 26 per 1 million, or is it 26 per 1 million plus X, right? And we're way out ahead of it. And the dry blood spot test is user-friendly, reliable, and being utilized. Now, with regard to numbers, we're holding back on talking numbers because it's just one of those things that the minute we start talking about those, it's never enough, and etc. But stay tuned.
Okay. And who is recommended to do a dry blood spot testing? Which pulmonologists or which centers are encouraged? What symptomology do patients need to present to then just say, "Go ahead and do that"? What is the protocol by which one then is recommended to order this test?
First of all, the test is available. Any physician can go online right now and order a dry blood spot test.
Okay.
We are hopeful that over time, that's going to continue to grow where you have spontaneous community-based pulmonologists who are suspecting aPAP, autoimmune PAP. I will say there is one very distinct kind of brick-and-mortar location, the ILD clinics, that we believe strongly that there are undiagnosed autoimmune PAP patients that are in the clinic. For example, at University of Florida, there's a small pilot ongoing. We're going to scale up the ILD clinic pilot. It's very clear that our belief that there were undiagnosed autoimmune PAP patients there is true. That's, we think, very specifically pulmonologists, physicians working in ILD clinics. We're keen to make sure that they are contemplating ruling out autoimmune PAP.
Is there a strong support group or support groups for aPAP that exist? Patient advocacy groups?
Yeah, the PAP Foundation is a U.S.-based advocacy group, and they do good work and are committed to the autoimmune PAP community, and there's also a new advocacy group, the PAP Alliance, so small grassroots, but growing. I think what we're seeing on the advocacy side is that as general awareness of autoimmune PAP is growing, you're seeing the efforts that they're putting in really paying good dividends. ex-U.S., same thing.
Okay. And team, maybe help us with the European filings. So you filed in one queue. You're on track to do so. Maybe timelines of when you could potentially get approved in Europe and then sort of what the size of the market is.
Sure. Yeah. So in Europe and the U.K., we will file the respective MAAs by the end of the first quarter. On the other side of the recent financing that we did and then with the royalty financing agreement that we have based on FDA approval for MOLBREEVI, it puts us in a position where the base case for Europe and U.K., EU4 and U.K., is go it alone. So we are planning to do just that. The market opportunity in Europe and U.K. is real. The size of the patient population, again, EU4 and U.K., is similar to that in the U.S. We know we have a very good handle on the stock and flows of patients, the reference centers, the physicians. In Europe and in U.K., they were really strong enrollers in the IMPALA- 2 study. So the opportunity is real.
Our stance is that we're going to go it alone. We also are very well aware of some of the policy-related things that are still overhanging kind of the industry. So we're aware of that. So we will just make sure that we're really thoughtful to ensure that we're maximizing, for example, the U.S. launch while also prepping to have a strong launch in E.U. and U.K.
Okay. That's very, very helpful, and team, with the current financing, what does sort of the cash take you to?
Yeah. So we've not provided revised runway guidance, but suffice it to say that with pro forma over $260 million in cash on the balance sheet plus the contemplated $75 million royalty financing, it is surely beyond the previous guidance of into 2027, but more to come on that.
Great. Team, it's been wonderful to have you excited for a 2026 for MOLBREEVI to get approved and really an incredible journey to be able to witness and see the launch. So let's give a big applause to the team for a great year.
Thank you.