Catalyst Pharmaceuticals, Inc. (CPRX) Earnings Call Transcript & Summary

Today, it's my pleasure to be hosting Catalyst Pharmaceuticals for a fireside chat at Citi's Global Healthcare Conference. I'm joined today by CEO, Rich Daly; CFO, Mike Kalb; and Chief Commercial Officer, Jeff Del Carmen. Thank you so much for being here today, Rich, Mike and Steve.

To start, maybe, Rich, you could just like level set for us for those that might not be familiar that are listening in, can you just give us an overview of Catalyst and each of your product candidates?

Sam, thanks for the opportunity to be here. We really appreciate it. Happy to go over the products and be joined by my colleagues here and to talk about the scope and span of the business. So Catalyst -- the focus of Catalyst is orphan and rare diseases. We have 3 products. We have FIRDAPSE, which has been on the market since 2019, and it's for Lambert-Eaton myasthenic syndrome, both idiopathic and cancer associated, and we'll talk about that in a little bit. We also have a drug -- our second drug was FYCOMPA for epilepsy. While not a rare disease epilepsy, actually, the acquisition of FYCOMPA actually served the company quite well, and we can go into some detail around that. The solid dose of FYCOMPA actually lost its patent protection back in May, late May, and we've seen some incursion into the business, but the products has overperformed our expectations, and we're really happy about that. The last product is AGAMREE for the treatment of Duchenne muscular dystrophy. And it's a steroid, but we believe it has potential to actually benefit patients in a way that other steroids or standard of care steroids can't. And some recent data that's come out from our colleagues in Europe really demonstrate the potential benefits of that, and we look forward to talking about that. We are a buy-and-build company. We don't do basic research, although we did do the research that supported the approval of FIRDAPSE. We are looking to acquire products that are significantly derisked, so products that are on the market or near the market with a decent runway of opportunity for continued commercialization. That's our focus right now, but -- and we'll talk about this, I'm sure, during the course of this discussion. We are considering our options as we move back into the pipeline, further into the pipeline for continued development. So products that might be a little bit more on the regulatory risk side, or might be in late-stage development proof of concept is well understood. And we believe our very strong cash position, balance sheet and the cash flows that we have can continue to support our strategy of buying products, but also working our way back into the portfolio and then also supporting our products and life cycle management as well.

Thank you, Rich. That was a really good overview. Lots to go off of there. Maybe just let's just talk about the overall strategy for the commercial engine first. As you think about the next 12 months, how do you expect that to evolve in terms of your priorities as you commercialize your 3 products?

I think first and foremost, one of the things we want to do is we want to make sure that we cover the gap for FYCOMPA. So as I said, the product is overperforming, and we're thrilled with that. But we do know that one product has been approved for the -- as a generic. And we know that 2 others were recently approved, but not yet on the market. So we expect to lose price and volume there, and we've said that consistently as we talk about FYCOMPA. But again, we're overperforming. So our focus has been to minimize that impact while optimizing and maximizing the impact on -- for the patients with Lambert-Eaton myasthenic syndrome with FIRDAPSE and those with Duchenne's muscular dystrophy with AGAMREE. So we really have taken, I think, an appropriately aggressive approach to the market. Let's talk about FIRDAPSE with LEMS. The market is divided into 2 parts, idiopathic or non-cancer LEMS and then cancer-associated LEMS. And with myasthenia gravis and the number of products that are -- have been approved in gMG, we're seeing on the idiopathic side, a really great opportunity to tail -- get a tailwind from that. We did -- and Jeff can talk about that more in detail. On the cancer-associated LEMS, we've -- Jeff and the team have done a great job in getting frictionless testing done and for the patients because there is a test for VGCC test. And then changing the NCCN guidelines. So this is a really important thing for us as we take our next step. So we have a 4-step process, the treatment -- I'm sorry, the testing with VGCC and the National Labs frictionless testing, changing the NCCN guidelines and then working on improving care pathways for patients and then really looking at the opportunity to educate the physicians within the high-volume, high-control oncology practices. The last part is looking at AGAMREE. AGAMREE is an incredible product. We really, really like it. We think there's potential beyond DMD in rare conditions that require steroid. So we're conducting a study right now to see if we can determine the immunosuppressive dose of the drug. Regardless of how that turns out, it's positive for us. So if we can't find the immunosuppressive drug, this is a really good thing for patients who are on long-term steroids in other rare conditions. If we do find it, then we obviously can figure out the best way to work with those patients, but also work in gene therapy as well because patients who are going to go gene therapy require immunosuppression. So we almost can't lose in this opportunity. We should see those data early in the year -- later this year or early in the year next year.

And that's from the SUMMIT study that you're.

No. So this is a Phase I study. Great question. I'm sorry I didn't make that clear. It's from a Phase I study where we're doing 2 parts. First, we're looking for the dose equivalency between -- I'm sorry, Emflaza, prednisone and AGAMREE. So when a patient moves, 45% of our patients come from prednisone, 45% of our patients in the DMD market come from Emflaza. But there's no information on the label about the appropriate dose to move them to. So we think that's really important. The second thing in this Phase I study is looking at this immunosuppression I talked about and figuring out how best the product can be used beyond DMD. And we'll be looking at that once these data come out and making a determination about the life cycle path for the product because we think it has potential given some of the profiles we've seen some work that's from work that's been done in Europe. So on the SUMMIT study, that's a 5-year 250 patient. And I mean patient, not subject because it is commercial drug, we'll be tracking and looking for the benefits of AGAMREE over standard of care steroids. And the benefits that we believe are out there are better behavior, less aggression, potentially better growth or stature, better bone health, fewer cataracts and then also potential for cardiovascular benefit. Cardiovascular benefit is the #1 cause of death for patients with DMD. Recently, our European colleagues at Santhera came out with a high-level summary of the GUARDIAN study. We can talk about that a little bit more. They haven't published much of the data yet. They're waiting for some other opportunities in professional meetings, which 111 patients over 5 years, and it showed a benefit on stature. So normal stature for patients on AGAMREE, bone health, excellent bone health and then fewer cataracts. So this, we believe, is a really good indication that our faith in SUMMIT is well placed. And so we're excited to see more of that data come out at some of these scientific meetings that are coming up.

Got it. Okay. That's helpful clarification. So for the Phase I, when you say additional indications outside of DMD, are we staying in the neuromuscular space? Or could we expand beyond that? I mean there are plenty of diseases where steroids are still make a foundation of the treatment paradigm. So how large is the spread for AGAMREE potentially in your view?

It's too early to tell the spread, but we're looking at anything in the rare space. So as long as it's rare, as you know, Sam, from the conversations we've had and the statements we've made, we're therapeutically area agnostic because we believe in the rare space, the infrastructure that we have where we support the patient, help the patient get on drug and importantly, stay on drug and then optimize their therapy, we believe that infrastructure is incredibly strong and can be -- is applicable to anything in the rare space. So if it were to go beyond CNS, we'd be comfortable with that.

Got it. Okay. And so then when you think about AGAMREE and its penetration into the market, you have some of this data that you just mentioned from Santhera. You have the SUMMIT study that you'll be able to rely on when that data starts to roll out. I guess how should we anticipate the launch to grow over time? Like what does that curve look like potentially?

Okay. I'm going to turn it to Jeff, so he can give you some background and some insight. Jeff?

Sure. So Sam, as we've discussed, we're very pleased with the launch so far. And we've seen broad adoption. There are 100 COEs, give or take a few, for DMD. And we have about 95% penetration within those 95 -- or those 100 COEs. By penetration, I mean at least one enrollment coming from each of those COEs. And then the top 45 COEs make up about 80% of the steroid business for DMD, and we have 100% penetration in those accounts. So we've seen broad adoption. Now what we're trying to do is get deeper adoption within each account. So that's where we focus. And that's what we anticipate seeing over the coming year plus down the road. And that's how we expect the launch to go is to continue to deepen that penetration.

Right. And in terms of the data that we'll see out of SUMMIT over time is the behavioral piece, I think you've said in the past is likely to be one of the first data readouts. I guess when you have that in hand, how does that help to just drive the launch further in terms of the breadth you're looking for or the depth, excuse me?

So I'll take a first crack at that. So when we see that data for the first time, it won't be in our label. So from a commercial viewpoint, we won't be able to do anything with it because we are a compliant company. But obviously, those things are -- that information gets out through medical meetings and through publications. And we're going to look to leverage our voice in the market outside of the commercial space to be sure that physicians have the best information and can make the best decisions along with their patients and the caregivers in the DMD space. But there is potential for a label change, but obviously, it's never guaranteed. And so we're going to look to make sure that there's awareness about that, but again, in a totally compliant manner.

We believe that the behavioral data will be helpful. However, that's very easily seen today by physicians that are utilizing it because you can see it within a week and how patients are -- the boys are reacting in school and how they're behaving. So those types of things. It's the other data coming from the SUMMIT that will be extremely helpful if positive.

I see. Okay. And how should we think about just the overall peak market opportunity for AGAMREE -- now that you've had almost 2 years launched coming up, I believe. I guess what have you learned? And what should we think about the peak opportunity of this drug?

So we believe this is -- at today's prices, we believe this is about a $1.4 billion opportunity in the total addressable market. And that takes into account 95% of patients have received a steroid, but only 70% are currently getting a steroid. So without any incremental growth in the treated population, the total addressable market is about $1.2 billion. All else being equal, if we're just as good as the other players, we'll get 25%. If we can show and demonstrate superiority in certain elements, whether it's behavior, stature, bone health, cataracts, cardiovascular over time, one would believe we should get an unfair share because we would have data that demonstrates that the product is better. So a 25% share, $300 million, but we haven't given any long-term forecast. We're just talking hypothetically. But if we can continue to demonstrate what we saw in GUARDIAN, we think there's significant potential here because these patients are struggling and they're looking for different options. Jeff, I don't know if you want to make any comments.

Nothing to add. Well said.

Got it. No, that's super helpful context. And then I think you mentioned this as well, Rich, about the potential immunosuppression for a gene therapy as well. I'm wondering like where does that stand? And where could we maybe see an impact from that?

So it depends if we find it, right?

Sure.

So there's a potential we won't. And if that happens, then we'll obviously not be a part of that market. If it's there and we have some of the other benefits that the physicians and patients and caregivers can see, then I think we can participate in that part of the market. But it will be a long time before that's actually in our label. So again, it's -- this is a very, very tight community, the DMD community, the patients, the caregivers, usually their parents, typically the mom and the physicians are very, very tight. So again, we can act in a compliant manner and get the word out. And then obviously, it's up to the physician in conjunction with the patient and the caregiver to make the decision on what they want to do. But again, I want to just focus -- I really believe that regardless of how that turns out, we can go in either direction. And so we're pretty excited about those data coming out.

Yes, absolutely. I mean it sounds like the base business that you have right now is quite strong, and there's a number of tailwinds that we could see materialize over the next, call it, 12 to 24 months potentially, at least data-wise, maybe if not future label change.

Agreed, yes.

Yes. No, that's a really good position for AGAMREE. Okay. So why don't we go ahead and switch over to FIRDAPSE then? I mean we started -- you gave us a really good overview of that one as well. Why don't we start with the oncology segment, if you don't mind. I'm wondering all of your efforts, I think you laid out what they are quite clearly already. But how have those been received by physicians? How is that education progressing, the efforts that you're making?

Great. Jeff, do you want to take that?

So the education is coming from multiple fronts, and we're focused obviously on thoracic oncologists, but really the community-based oncologists. So we are going to conferences. We have 2 MSLs fully dedicated to cancer-associated LEMS. We just launched an unbranded website, cancerassociatedlems.com that's providing this information. We have educational efforts within these high-volume, high-control GPOs out there so that when physicians have a newly diagnosed small cell lung cancer patient, they receive education from the company, from their GPO that kind of provides updates from the NCCN guidelines on what their approach should be. So those types of things, and it's being very, very well received. Rich, I don't know if you want to talk about one of the meetings you just had.

So changing the care pathways, it's the old saw about HMOs. If you've seen one HMO, you've seen HMO. Changing the care pathways across this continuum of accounts is on a one-off basis. So we're focused on the high volume, high control, just like you would with the managed care, where the physicians within the structure of the company, the oncologists are judged by how closely they follow the care pathway. Recently, we've been reaching out. I worked in oncology for 15 years. And so we reach out to folks we know and say, look, the NCCN guidelines have changed, have you updated? And I had a conversation with the leader of an oncology account of about 50 oncologists. And he said, okay, we'll make that change. And about 10 minutes later in the call, I said, I just want to remind you, you need to make that change. And his response was, I made it while we're on the call, move on, which was great. That's the exception, though. We don't expect that to happen because these are very large organizations with varying levels of control and size within their own ones. So McKesson is the largest. And so we plan to work top down through McKesson and bottom up through the plans as well through the -- not the plans, but the accounts themselves and get the education out there. And then some of the best investment we can make when they change that care pathway is to be working on education so that the physician knows if I have a small cell lung cancer patient and the test panel comes up, check VGCC because you want to know. And as we know from our experience, often the first sign of small cell lung cancer, not often, sometimes the first sign of small cell lung cancer is Lambert-Eaton myasthenic syndrome. So we want to be watching that as well. And the physicians have shown a great deal of comfort in being able to not only diagnose, but also actually treat the patient in the oncology setting. And that makes sense. They'll be back in on a regular basis. So again, we're seeing lots of progress. But we don't expect any impact, any significant impact before probably midyear of next year because it just takes time to get through all of these. And we're talking about 3% of 15,000 or 30,000 patients who have small cell lung cancer. And because of the life expectancy of those patients, there are at any time about 900 patients that are out there. So we think this is -- has significant potential for growth for the company.

A couple of things to add. This is a significant opportunity, like Rich mentioned. And 90% of the cancer-associated LEMS patients are undiagnosed today. So that's why all our focus right now is educating physicians on this and screening so that we can help these patients get diagnosed and help these patients out. So that's one of the reasons why we're so focused on screening.

No, that makes a lot of sense and the opportunity is there for sure. When you think about that inflection point, I think you said mid-'26, you're obviously working to change a bunch of care pathways. Does that all -- I guess, do you get enough momentum at that point? Is that when you expect the education to hit sort of like, I guess, an upward curve where the diagnosis rate should start increasing? And is that just like a critical mass of these care pathways changed in your favor?

So yes. When you think about our business, so Jeff said, and then we have to keep these numbers very clear, less than 10% of these patients have been diagnosed. Now let's talk about our patient pool, the number of the patients that are being treated with FIRDAPSE, less than -- around 20% of our patient pool. So getting behavior changes takes time and continued effort on education. So we're seeing a lot of positive traction for the care pathway change, but then we need to invest, and we've planned it out for next year in the education by making sure that physicians are aware. Once -- what we've seen, not broadly, but when we talk to physicians, they say, these patients live on average about 9 months. A patient with Lambert-Eaton myasthenic syndrome and cancer lives about 17 months. And that's a lights on moment for the oncologist because they think, wow, this patient is actually going to live about twice as long as I thought. So quality of life really does matter. What can I do to help that patient? So getting that word out, getting the education just takes time and effort, and we are well positioned to do that as we go forward.

Yes. That makes sense. And I guess just can you talk a little bit about that strategy, about how many of the accounts -- you mentioned McKesson being one of the large ones. Are there any others that are on your list? I'm curious if you have like a couple of waves of targets. And then just how are you thinking about, I guess, that as it progresses? Like how do you progress through wave 1, wave 2, wave 3, if they exist?

So I think Florida Oncology is probably the largest oncology practice in the U.S., single practice. Then you have Tennessee Oncology, Texas Oncology. And all of those are part of one of the GPOs. So again, working the top down and the bottom up with the field resources that Jeff has talked about from the MSLs to the national account directors. Again, I think to your point and to the word you used before, momentum, there will come a point where we'll hit that momentum and there'll be more tests done. So the strategy is really to approach it from both sides of the business and to try and help grow. Jeff?

Yes. Just to add to it, about the 3 largest GPOs make up about 80%, have control over a top 80% of the community oncologists. So it's to our benefit if we focus on those big GPOs. And so that's how we anticipate getting traction sooner. And as soon as we can get traction within U.S. oncology with Florida Oncology, then we go to Texas Oncology, then you can start building momentum. But that's the fastest way to be able to get share of voice with thoracic oncologists.

Is there -- or would you consider sharing sort of, I guess, testing rates and how they have increased over time as like a KPI that maybe we could track for the oncology segment?

Sure. So it's very hard to tease out oncology -- oncologists that have initiated a test or a neurologist that has initiated a test. But what we have seen is a significant increase in the number of VGCC antibody tests that have been done. over the last 1.5 years, quarter-on-quarter, there's at least a 9% growth each quarter. So we're seeing that growth come along. It coincides with the amount of education that we're putting out there, too. So we strongly believe our efforts are making a difference, and we expect that to grow continuously as we move on.

Got it. That's super helpful. And then, just talking about the idiopathic opportunity, Rich, you said that there's still a large amount of opportunity there. Maybe just talk about how you're going to further penetrate that segment over time and what dynamics we should be aware of for that?

Sure. I'll turn it to Jeff, but it's pretty similar in approach to a systematic stepwise approach to the market and leveraging the increased noise around general myasthenia gravis because that's the most frequently misdiagnosed condition for somebody who has idiopathic Lambert-Eaton myasthenic syndrome. Jeff?

We're very excited about the opportunity that still remains with the idiopathic side. When we look at it, I mentioned that we always have about 500 or so these pool of patients, we call them pipeline patients that are somewhere in their diagnostic journey to LEMS, but we know -- strongly believe they're LEMS patients. So about half of our enrollments each month come from these pool of patients, so we know them. So what we need to do is continually find new patient leads to backfill ones that are converted on therapy. And we have been doing this for years. And we always find new sources to backfill. So not only do we leverage the sources that we have, but we find new sources to also add into this pool of patients. And we are very confident that based on these new sources and the pool of patients that we have, the growth will continue. One of the reasons is because of what Rich mentioned, if we can get all these patients tested for MG and then they're negative for AChR, then we need to get on a panel so that it's on the MG panel so that VGCC antibodies are there, then you can rule out MG and then, oh, by the way, they have deposited for VGCC, they have LEMS instead of excluding other things first and prolonging that diagnostic journey. So along with that increased rate of testing for those patients. Now we get new leads coming in because we get that data from national laboratories.

So Sam, I want to build on that, too, because I think Jeff's answer is right on point. When we think about what's going on in the general myasthenia gravis market, the amount of noise direct-to-consumer is going to increase the diagnosis rate for gMG, no doubt about it. But it's also going to bring in a lot of patients who will initially get misdiagnosed with gMG. And therefore, I think our opportunity increases. One of the ways I like to describe it is the best money -- their DTC campaign is the best money we never spent because it will help raise that up. And we want to be sure they have the physician as a solution. It will take time to get both of those AChR and the VGCC done at the same time, but initially, it will probably be a reflex test. Do you have seronegative AChR, let me go down to the next logical thing. And again, that's an educational opportunity. There's not as much control in the idiopathic market as there is in the cancer-associated LEMS market. So it's still very much a prospecting business and really using the data that Jeff talked about to identify that patient who's most likely to be a LEMS patient and continuing to leverage that data set that's out there and do education and change the testing. So it looks very similar to the cancer-associated LEMS path, but it's a much more labor-intensive opportunity.

Yes. I'll add to, it's with these leads, we have the most data in the world for LEMS. And we need to better understand the leads that have become patients on therapy and how those patients are doing. So we're benefiting these patients. But the more efficient we can be with the leads we have, then the faster we can help these patients. And we've become more efficient because we've learned more. And that's why we're now pointing the RAMs, our field force in the right direction within it. We score the leads and we'll say this one is a higher priority than this lead over here. So that's why we're seeing an acceleration, and we've been able to sustain this growth.

Yes. I was going to ask how long does a patient typically stay in your patient pool? Is there an average? Or are there, to your point, a couple of different types of patients that move through at different rates?

Yes. So Sam, it's all over the spectrum. And we have seen some patients that unfortunately, they will go on some non-FDA-approved treatments or not just get there based on education, it's the first time the physician -- physicians on average, see one LEMS patient in their career.

On the idiopathic.

On the idiopathic, correct. So it's really educating them on that. But the ones that we've seen convert the fastest on to therapy are those patients that are negative AChR for generalized myasthenia gravis and positive for VGCC. So they've had both tests done, then they convert faster. And in some cases, the RAMS get the lead and within a week, that patient is on therapy. So it's very quick in some cases.

So Sam, it's so rare, right? One physician or one patient per physician. The frustration level is really, really high. And so if it takes 4 or 5 years for that patient to go from first symptom to true diagnosis and the correct diagnosis, crushing that cycle time becomes really positive for the patient. And so that's where we think the testing comes in because it looks so much like -- it's easy to do a differential diagnosis, but you just -- it's so rare. So by helping them with the test, it's not -- the test is not the end all and be all, but it's a great step forward.

Is there a dedicated MG panel that you can add VGCC testing to? Or is it just, again, individual physician-by-physician educating to remind them they should be testing for this for every potential MG patient?

It's a combination depending on the lab, too. Some labs like Quest and Mayo, you can get -- try to get on their labs. So that's what we do. And it's a long process, but we're well underway in discussions on making that happen.

Got it. Okay. Great to hear. So then maybe let's just talk about the overall opportunity. I know you've outlined it before, Rich, but I think it's helpful to maybe say it again. And I'm curious how you think about the overall LEMS and then segmenting it out between the idiopathic and the oncology opportunities.

So there's probably 3,600 to 5,400 patients out there. When we talk about the opportunity, we always talk about it in the most conservative way. We take the smallest patient population, and we take the fact that whatever our price is today, we don't -- we assume no price increases in the analysis. That's not to say we wouldn't have any, but we just assume no price increases. So the overall market opportunity is split 50-50 between idiopathic and cancer-associated LEMS. Because the idiopathic patients live longer, there are a few of them and they live longer. There's about 150, we assume diagnosable every year, but they live a long time. On the cancer-associated LEMS side, there's about 900 that are there. And so you have about 1,700 that are alive at any point in time, if they're, in fact, living those 17 months we talked about. So as we think about that with the most conservative structure and assuming just a total addressable market of 85% of those patients who are diagnosed because some are too early in their process to require treatment and some might be too late to actually gain a benefit. We assume that's about 85% based on some data we've seen in the marketplace. And again, all those conservative assumptions, the total addressable market is $1.2 billion. And the upper end is obviously going to be much higher without going into specifics. But again, we're just focused on that. And we have an ongoing patent issue. We have one left. We can talk about that in a second. If we successfully resolve that, we have more than 9 years at the current -- with the state of the current agreements that we have with other generic defendants. We would have more than 9 years to continue to build this market. And the testing, again, and getting that word out on education becomes critically important.

Right. So you've settled 3 of the 4 challengers and those 3 settlements said 2034, correct?

February of 2035.

February of 2035. That's how you get to your 9. Can you talk a little bit about expectations for when that fourth challenger might have a resolution?

So I can't give any specifics because it's ongoing litigation, but thanks for the question. We will continue to do what's right for the company and right for the patients. And so we're going to look to gain the best advantage we can. We believe in our IP. Our IP is very, very strong. And we're excited to bring that to closure, but we won't do anything that hurts the company.

Right. Okay. That's helpful. So we look forward to a resolution potentially in the future for that. And I guess -- so I think that was great on FIRDAPSE. We talked a little bit about FYCOMPA already in the beginning of the call. But maybe just overall for the business, FYCOMPA is sticky, and we're seeing that, but it is losing revenues over time. We have the strong AGAMREE launch that we talked about, continued FIRDAPSE growth. I guess how do you just think about the overall growth of the business as we move into 2026? And I'd love your thoughts on what a potential BD transaction, how that could impact growth and just in broad terms because obviously, you can't be specific about some of these.

When we think about the overall growth of the business, obviously, we knew when we bought FYCOMPA, we knew what the life expectancy was. That was a play that we did to improve the balance sheet and increase our flexibility to acquire more products. And it worked because without that, I'm not sure we would have gotten AGAMREE. So now that the time comes when you have the natural decline of the product to generic incursion. The question is, can we -- with the current things we're doing, overcome that decline. And we believe we can. And not just because of the size of the markets, but Jeff can speak to some of the programs we've instituted. We've had very strong patient adherence programs, but we've really ratcheted that up because we believe once a patient gets on, obviously, it's in their best interest and our best interest to keep that patient on. So we've done that, but we believe that we can outgrow the decline, and we are very confident in that because of the traction we see in the market. But Jeff, do you want to speak to some of the changes we've made around -- specifically around FIRDAPSE?

Sure. So like Rich mentioned, there are multiple levers that you can pull to help grow the business. And one, oncology is a huge opportunity for us, obviously, continued growth in the idiopathic. The other side is the patients that you do have. Our discontinuation rates, our annual discontinuation rate for LEMS for FIRDAPSE is 20%, 15% to 20%. So what we noticed some of the patients that discontinued, they were not reaching their maintenance dose or their optimal dose for therapy. So we've instituted programs and really ramped up efforts with the specialty pharmacy and educational efforts to educate the HCP as well as the patient on that titration process to make sure these patients are giving FIRDAPSE an adequate trial. And we've seen benefits from that because of lower discontinuations of those patients that are initiating therapy. So things like that will help us optimize FIRDAPSE without new patient acquisitions. So we feel like that's definitely going to benefit patients in FIRDAPSE.

Got it. Okay. That's helpful. So -- and is it -- would it still be 15% to 20% year-over-year growth as we move into '26? Or would you, I guess, give us an update early next year?

So we believe for the midterm, 15% to 20% is achievable on a bigger base. This is one of the challenges we get how can you keep doing this? And Jeff's team has really -- so he talked about the pool of patients, but they not only prioritize patients, then they hyperprioritize the patients. So -- and that's -- there's a constant churn there. So we just don't say, okay, it's 500 patients that are in that pool. We're always looking to who is the most appropriate one. And again, having that confidence of 50% of the patients come from that pool every quarter, every year, we can actually have a really good predictive approach to our business. So yes, we do believe that. And then AGAMREE there's a growth opportunity there. As I said earlier, 95% of patients have been on the steroid, only 70% are. Lots of reasons why it's not higher than 70%. But it's one of those reasons that the adverse events you have with the steroid are so bad that people drop off because the benefit goes away. And if you have a superior steroid, can you grow that to 85%? Can you grow it to 90%, again, of the addressable market, so...

Okay. Okay. That's helpful. And then just you touched on this as well in the beginning, Rich, but just on BD and the potential opportunities out there, can you just remind us what that ideal BD deal looks like in terms of development stage, I think you were saying you could take a little bit of risk, late-stage development or regulatory, but also just the peak sales potential, how much you have to deploy capital-wise?

Sure. So our ideal opportunity is a rare disease. We are therapeutically area agnostic because of the infrastructure we have to support peak sales, about $500 million. And the rationale for that is we would be a very strong player in that subset of the market. If we go much above that, that moves the needle for bigger companies, and then we're not as strong a buyer, if you will. So we would want to be -- or currently doing things that -- acquiring things that are either immediately accretive or nearly immediately accretive. And then we are looking to take on more regulatory risk as the strength of our balance sheet increases because we believe that we want to have future-oriented discussions. We love the execution of the company, but that's by its nature, a backward-looking metric. We want to be able to have those forward-looking ones. I think one of the best indices of our success as a commercial organization is more than 90% of the opportunities that come through our door are coming to us. We're not seeking them out. So somebody comes in and says, hey, you're a great commercial marketer. We want to talk to you about it. And I think we said in our press release around the most recent earnings call, we've done more than 100 assessments since January. And so we believe we can continue to grow the company through not only the execution on the life cycle management, on the sales and improving the retention of patients, but also through business development.

Yes, absolutely. And then, Mike, I've left you alone this whole session, so I want to make sure you have a question as well. Can you just remind us just a little bit of the financial health of the company, the cash that you have to deploy and I guess, how you're thinking about as we move into '26?

Sure. So we have, as reported at the end of September, $689.9 million of cash and cash equivalents, no funded debt. With the cash flow generation, depending on specifics of any opportunity, we're comfortable that we could pay north of $1 billion for one or a series of acquisitions. I know we announced the buyback. That does not change anything in our BD strategy or our ability to execute on that strategy.

Excellent. Okay. And then -- so Rich, I just want to turn it back to you. Any closing remarks you might have? And anything you think is really important to emphasize for investors?

The company continues to execute, which again, is a great story. There's great interest in other companies leveraging their assets through our infrastructure. And we're excited about those discussions. We will continue to be diligent and discriminatory because we believe we have a good track record of acquiring products that can perform. And we feel that pressure every day to do the right deal, not to just do a deal, but to do the right deal that will continue to grow the company. And we're really excited about the things we're seeing. It's challenging. The market is very challenging. As interest rates fall, companies have more access to capital, and so may need somebody less like us. So that's one of the reasons why we've talked for the last year about moving a little bit further back into the pipeline to take on opportunities that could accelerate the growth of the company. And we're excited about that.

Yes. As am I. Well, thank you so much. It was wonderful to us having you here today, and it was a great discussion. Thank you.

Thank you very much. We appreciate that.

Thank you.