CATALYST PHARMACEUTICALS, INC. — Call Transcript 2025

Ladies and gentlemen, the program is about to begin at this time, and it's my pleasure to turn the program over to your host, Jason Gerberry. Good day, everybody, and thanks for joining us at the BofA Virtual CNS Conference. My name is Jason Gerberry. I'm one of the biotech analysts here, and I'm joined by Pavan Patel, who's one of my colleagues who helps with Catalyst Pharma. Catalyst Pharma is our next company presenter, and Rich Daly, CEO. Rich, thanks so much for joining us and giving us the latest update on Catalyst. Jason, thanks for having us. We really appreciate it, and looking forward to the conversation. Thank you. So maybe just starting with kind of the overarching BD and licensing, that's obviously kind of core to what you do and building out the business. And you had sort of a stopgap product with Fycompa, but your two main assets for kind of the longer-term Agamree and Firdapse. And so, yeah, how important is it for you to get a resolution with Firdapse's IP before you deploy capital in a more significant way? Maybe just talk about the importance of doing a deal in 2026 as a priority. Maybe we can start there and then delve into additional questions. Sure, so we feel like with the acquisition or the arrangement with Agamree and Santhera, Santhera is the originator of the product. We feel like we've had ample time to incorporate the product and digest the product and move the product forward. We're always on the look for opportunities. As you mentioned, we're buying those company, and so we're looking for those products that are immediately accretive or nearly immediately accretive. They're in the rare space, differentiated, unmet clinical need, and a ceiling of about $500 million in sales, so what we're trying to do in that scenario is eliminate the clinical risk, minimize the regulatory risk. We did take some regulatory risk with Agamree, and then absorb the commercial risk because we think that's a hallmark and the strength of the company, so we want to get a deal done. We want the right deal, however. I think you saw in our press release for Q3 earnings, since the beginning of the year, we assessed more than 100 opportunities. One of the things that's fundamentally changing in the marketplace is the capital markets. Typically, an opportunity for us would be a company that has enough money to launch but not sustain. With our infrastructure already built, we can come in and really take over. An ex-U.S. company, Asia, Europe, that is looking for an opportunity to plant a flag in the U.S. but doesn't want to take the commercial risk. But with the capital markets opening up, again, we may be in a situation where we have to broaden the scope of what we're looking at and perhaps go back just a little bit into the pipeline, into the Phase III, which is proof of concept is assessed and is positive and looking forward, and we've been talking about this for two years, so that's the nature of what we're doing. We've been looking at those opportunities and assessing them as well as the immediately accretive or nearly immediately accretive opportunities. You mentioned, and maybe I haven't heard this part before, but a ceiling of $500 million, that would be like a peak revenue. Is that sort of like what you're screening for? Yeah, so when we think about what can move the needle for us and what can move the needle for competitors who might be larger, you stay around the $500 million, and that doesn't really necessarily move the needle for some of these companies, so it's a different set of companies that show up at the auction, so when we're there and we have about $689 million in cash and no funded debt, we're a powerful bidder. We don't want to get into a war of attrition with a much larger player, and as good as our bank is and our balance sheet is and our ability to take on debt, we're not going to compete where we can't win, and some of those larger opportunities just aren't for us right now. That will change over time. Yeah, well, I know there's been analogies to your business model to Horizon Therapeutics who had a model like this, and there was probably a time when people thought Tepezza would be a $500 million peak sale drug, right? Right. There is an opportunity for Firdapse to eclipse $500 million in revenue, right? Absolutely. So is it that maybe $500 million is sort of a conservative base case when you look at assets, but they have the upside potential to exceed that revenue threshold? Yeah. Great question and great commentary. Yes, so we look at it and we think about how we might be able to accelerate that opportunity. So when you look at the opportunity to grow products, really, you're looking at your company, rather, and the lifecycle management. Take the products that are inline commercial. How do you get patients on earlier, keep them on longer, or optimize their dose? Then there's expansion, and we do that for both the Agamree and Firdapse. And then there's expansion within the label. So you look at Firdapse as an example. That expansion for LEMS applies both to idiopathic and cancer-associated LEMS. And we have a real focus on maintaining our idiopathic business, but also growing. And then obviously, there's label expansion too. And we're going to look at that for Agamree as well. We think there's opportunity. This is a steroid that works. There are data that have just come out of Europe from Santhera, out of the that look really, really promising and support our work on the SUMMIT trial, which we can talk about. But if the steroid works and it has a favorable profile for long-term steroid use versus standard of care, we're going to look for other rare disease opportunities to apply that molecule. And so that's one of the key initiatives we've got in growing the company. So you put the whole thing together, the last one for the company is business development and looking to actually accelerate growth of the company through incremental products. When do you think you'll have a handle on that aspect of other rare opportunities for Agamree? I imagine with patent and the data exclusivity and factors like that, you can't wait forever to identify those and more visibly kind of outline those. Right. So the orphan status for Agamree will come to an end in 2029. But because of patent restoration, we believe we go out to 2034. So we have time. However, that doesn't mean we're going slow. So we're right now doing a Phase I study, and the Phase I study is looking at two elements. The first element is what's the dose equivalence between prednisone and Emflaza and Agamree so we can give better guidance to practitioners should they ask. And the other one is what's the immunosuppressive dose? And when you think about the immunosuppressive dose, we look at that from a gene therapy standpoint because steroids are used on board with gene therapy, and you need to immunosuppress those patients prior to giving them gene therapy. But we can't actually lose. So, if we find the immunosuppressive dose, there's an opportunity to apply it to gene therapy and then ratchet it down to those doses that are effective and don't cause immunosuppression for those other non-DMD opportunities. If we can't find the immunosuppressive dose, it's an ideal product for long-term use in rare conditions. And so we're really excited. We'll be seeing those data after the first of the year. And once we see the data, we'll be making the decision about where we go. So I would say first half of the year, we'll have a decision. Okay. And then going back to my initial question, what I didn't hear was that Firdapse's IP resolution was like a swing factor at all on the BD prioritization and timing of making a decision? Coming to a positive resolution on the Firdapse's IP is obviously a high priority for us. We're interested in doing it. There were three first filers. We've settled with two of the three. There was a fourth filer, not a first filer. We've settled with that company as well. And there's one defendant left. And when you look at the situation with Teva settling, Teva is obviously a sophisticated player in this space, in the generic space. And so with them settling, it gives us further confidence, even though our confidence was very, very high in our IP. To your question about whether we're waiting for that, the answer is no. We're looking for that opportunity. We have great cash flow right now. We want to continue to build the company, and we want to look at those opportunities that can really deliver for patients and for the company. I haven't checked recently, but do you have a trial date teed up for that one outstanding? We do. So the 30-month stay, and the trial date is scheduled for March. And the 30-month exclusivity or the 30-month pause actually ends in May. So we believe that gives the judge enough time if we get the trial. But again, we're going to do the right thing for the company and for the patients and for the shareholders. Okay. In terms of deal type, size, structure, is the priority still sort of an exclusive license, maybe something that's manageable on the upfront size of dollars out such that you can not have to take on leverage, or is the aperture just, "Hey, we're going to go with the best opportunity in front of us, and if that requires us to take on leverage, we'll do it"? So if there's an opportunity to take on leverage, we would want to be sure that it's a slam dunk. So when you look at we have almost $700 million. So what could we do on the upfront, plus the cash generation we have right now on a quarterly basis? So we would want to really be sure that if we were to take on leverage, that this is more than likely an on-market product with a long horizon. You mentioned the Fycompa opportunity, which was a great opportunity for us because it really diversified our income statement, gave us additional cash, and enabled us to do the Santhera or Agamree deal. If we're going to do something where we take on leverage, it has to be really spot on and pretty much a PTS of 100% on the commercial side. So on market with long legs. And then I think you guys had made some comments about looking a little bit more broadly in orphan space beyond CNS. I guess I would have looked at your business model and said, "When you're looking at all these different rare orphan indications, these deals are probably not predicated on SG&A synergies, right?" Because these sales forces are pretty small. Yeah. They're tight. So I guess the only relevance to me of that statement is just like, "Hey, maybe we have competencies in-house for looking at stuff in the CNS space, but now we feel like perhaps we have greater competency in-house to look, I don't know, in another therapeutic area specifically that's rare orphan. Sure. So from a competency standpoint, so as an example, Firdapse and Agamree are the fourth and fifth products I've worked on in the rare space in my career. So we feel like we have great competency across the board, not just with me, obviously, but with everybody, and you look at the diverse backgrounds from a therapeutic area standpoint, we've got great skills in-house. We can do that. I think when it comes to what's going on and how you support the business and support the patient, it gets back to that first statement I made about lifecycle management for a product. Get the patient on earlier, keep the patient on longer, and optimize their dose. That infrastructure sits behind the sales organization. Obviously, it's the specialty pharmacy, the patient hub, and the third-party logistics provider or the shipper of the drug. So when we look at that, we believe we're really set up. And that part of the business is applicable to any orphan or rare condition. So we believe that with the expertise we have in-house, plus the expertise in supporting the patient and the provider and the payer, we think we can move well beyond CNS. In your BD team, can you remind me how many people you have looking at transactions, and do you have the, I guess, the scope internally to look at assets coming out of China, or is the China biotech engine really less focused on some of these nichier orphan drug applications that kind of fall within your sweet spot? Because it does seem to me like there's a lot of focus on me-too sort of treatments in big large established categories, so I just wonder how that maybe fits within your scope of transactions that are interesting to you. I've worked at a number of companies that are structured a lot like Catalyst is structured. Very focused, not a huge organization. One of the things we do is we take our internal organization of five or six people who are totally committed to the business development end of it, and we leverage external providers who can bring more to the table. Because as you know, it's episodic. Even though we've evaluated more than 100 since the beginning of the year, the peaks and troughs are there as well. We want to bring those folks in when we get really far along and get expertise, supplement the expertise that we have. China is not out of our bailiwick. We like the opportunity. Any company that's not based here in the U.S. and looking to plant a flag, as I said earlier, we're interested in talking to those folks. So Asia, Europe, etc. So we're continuing to it, and we do have the bandwidth to do it. Okay. Well, maybe we'll shift gears to Agamree, which has been an important new launch for you guys. And maybe just the latest in terms of how you're sourcing patients between Emflaza and prednisone. Has it changed or evolved? Has it exceeded expectation? Where are the patient sources coming from? Sure. When we initially launched, or prior to the product, and initially launched the product, we expected that we would source mostly from Emflaza. What we're finding out is there's an incredible dissatisfaction with the standard of care out there. So we continue to source. I'm just going to give you the rough numbers, and it vacillates a percentage or two, depending on the month you're in, but 45% from Emflaza, 45% from prednisone, and about 10% are naive. So that continues from the launch up till now, which we think is an incredible sign that the product has great potential going forward. And there is a generic Emflaza on the market, and we're not affected by that. We have approvals in the almost 90% range. So the opportunity is really to look for those new patients or keep patients on if you have a superior product profile where patients will benefit. So the sourcing continues to be exactly what it was at launch, which speaks well to the product. In just rough terms, this market, right, because it is capped by size factors, right? But I think Emflaza achieved peak penetration of around a third of the market, if I recall. It's hard to tell because nobody reports data here because of our proprietary pharmacies. That's what we believe. It was about a third of the market, yeah. Okay. So call it $250-ish sales at getting a third of the market. So said a different way, it could be a $750 million market for steroids, assuming gene therapy adoption didn't somehow diminish the utilization of steroids. Is that a way to think about the ceiling of this marketplace? I think there's a couple of ways to think about it. Number one, 95% of patients have been on a steroid, and only 70% are on today. And that speaks to the dissatisfaction with the therapy. Even for patients who are not ambulatory, the ability to more successfully operate their chair because of improved dexterity or maintained dexterity, or the ability to operate a mouse or a keyboard, both of these are the window on the world. So we think there's an opportunity with a better steroid to actually grow the market, albeit it's capped at 11,000-13,000, some estimates as high as 15,000 in the U.S. patients. So when you take the conservative estimates at our price, which is roughly equivalent to the price of branded Emflaza, we believe that the addressable market is actually above $1 billion. So if we can prove, and you look back at the GUARDIAN trial, which I referenced earlier, and the SUMMIT trial, which we're doing in the U.S., we can prove that those benefits do exist, we think we can get more than our fair share of the market. How important really is SUMMIT to getting to some of these more bullish market outlooks in your mind versus ex-SUMMIT? I don't know. Do you feel like you still would be on a trajectory to having an Emflaza kind of peak sales profile? It's a great question. And when we look at the GUARDIAN trial, it validates our faith in the SUMMIT trial. So here you have 110 patients. So we're five years looking at versus standard of care. And you see less vertebral fractures in the Agamree patients. You see stature, normal stature. So you know long-term steroids in adolescents can inhibit growth. And you see fewer cataracts. So if you have an older patient population, clearly they're not going to face growth issues, but you have cataract issues, bone health, etc. So I think SUMMIT's pretty important, and the GUARDIAN results give us a lot of faith that this is the right path to take. So to answer your question, yeah, it's pretty important. And so those updates that we get from SUMMIT are going to be periodic, right, over time, and you do more follow-up. And so the first update will be sometime, can you remind me of that next year? We haven't said yet, so we're still enrolling. We're looking for 250 patients across about 30 or 35 sites, and we need to have those patients on for a significant enough period of time to see the benefit of the bone or see the benefit of the cataracts, so we believe there might be other benefits as well, such as behavior that we'll see pretty quickly if it's there, so we're looking for that, so we'll give updates as we continue to successfully enroll patients, and I want to focus that these are, in fact, patients, not subjects. These are patients who are on commercial drug, so they get a significant number of enhanced testing, and they get a significant benefit from participating, so we think it's a pretty important trial and focus a lot of effort there. Okay. And then how has the payer coverage sort of evolved? Do you feel like going into 2026 that this single generic step edit was going to be the more broadly applied kind of coverage policy type? I'm sorry. Could you repeat the question, Jason? I'm sorry. I had a hard time hearing you. Sorry. Just thinking about sort of the payer coverage, right, going into 2026, do you think that that's going to be primarily this single generic step edit that's been, I guess, in my mind, the predominant type of policy is step through one generic only, not two? Oh, yeah. So right now, the majority of patients are on a generic. They're on a generic prednisone. And those that are on Emflaza are not going to go back. It doesn't make sense for an insurance company to say, "Go back to generic Emflaza if it didn't work." So we think there are two elements to this. Number one is that we're getting, almost, as I said, 85%, almost 90% of the prescriptions approved. And when we first launched, it was probably about 30 days from the time we submitted the prescription until it was approved. So we give those patients bridge drug, right, to make sure that they get on therapy as fast as possible. Now it's down to less than two weeks. So I would see that continuing to evolve as more payers get more comfortable with the profile of the product and the use of the product. Okay. And then is there anything you can speak to more real-time in terms of interplay with gene therapy rollout, how that's impacting steroid utilization, if at all? Yeah, so I would say that the market itself, so remember that steroids are foundational therapy. It's the drug you get on first before you get on anything else. Patients that are on steroid therapy walk two, two and a half, sometimes three years longer than patients who don't go on to steroids. So we're not competing with gene therapy, but the choppiness of this water because of all the things that have happened, the performance of the product in the face of that choppiness, I think, is outstanding. So we're looking for it to settle out. We would love for, as everybody would, for there to be no more challenges with gene therapy and sort of get on a steady state, and so we've had what we call a queuing effect, so patients that are on a steroid are less likely to change if they're waiting for gene therapy. So it almost creates a line, if you will. The challenge with this scenario in the marketplace is that we saw the label expand for gene therapy, then we saw it contract. And so there's just a lot of confusion. And as that settles down, it gives us a stronger share of voice in the market because we can actually talk about the benefits of therapy with Agamree. So we're hoping it stays steady state. How are you guys thinking about pre-announcing around the time of J.P. Morgan, start of the new year for your business? Do you feel like you have enough kind of visibility into these components of the business that this would make sense for you guys to sort of pre-announce revenue for your business lines? So we may do it at a high level. I think what we want to do is we want to take a look at the markets and be sure that we have a really good understanding. Obviously, at this point in the year, your forecasting is complete, but you just want to be as appropriately cautious as possible. So last year, we announced that our earnings call for the end-of-year earnings call. And that's our plan for right now. So we would want to make sure that we have a really good understanding of how the markets are trending when we do, in fact, give guidance. Okay. Anything else that we haven't hit on with respect to this topic? I don't know if you've actually even sort of shared any of the orphan indication opportunities that are potentially of interest to you or potentially any sort of directional sizing indicators for what those expansionary opportunities could be as a Agamree follow-on indications if you can't be that specific at the moment on maybe what you're looking at. Yeah. Just at a high level, we would look for those rare conditions that require long-term steroid use and have the largest patient populations where a change in steroids is meaningful to the patient or to the provider, hopefully to both. But we're not in a position right now. We'd really like to see those data from the Phase I to have a better understanding. And that way, I think we can go forward with impunity. I mean, with the pricing paradigm that you have with this, I imagine that does the utilization need to be in sensitive populations like pediatric or I would imagine there are very large market opportunities where steroids are used, right? But trying to narrow it down, is some of this population sensitivity? You probably want to stay in rare orphan, right? You want a sensitive population so that you can maintain the pricing structure. So I'm just kind of curious. Am I thinking about that the right way? You are so rare orphan is where we want to focus and where the patient can gain the best benefit. So as we discussed earlier in this call, older patients won't have the benefit. They don't have the benefit for stature. So if there's an opportunity to provide a pediatric patient with that opportunity to grow like their peers, that's a great opportunity for us. Older patients are going to be worried about bone strength. You just reduced vertebral fractures for long-term steroid patients. That's critically important. Cataracts, that's critically important too. So the ancillary cost that you would experience or maybe avoid if the drug actually does have the profile we believe it has, I think could create tremendous value in these rare and orphan markets. Okay. Makes sense. Maybe shifting gears to Firdapse, and I actually have just a couple of mechanical questions. As we think about just the different scenarios around IP litigation, I just want to make sure I've got this right. So you have a sort of tail terminal patent that covers certain aspects of the composition, whether they do or do not have sort of this impurity that you've identified in your patent. And I believe, from what I understand historically, it's very difficult to not have that impurity in the product, right? And that is something that's patented. So is that sort of the anchor patent as you kind of look at this and what underpins the confidence kind of going into whatever patent litigation could entail? Right. So we're not getting into too many specifics. So we have two patent families here. And for somebody to actually win, they would have to win on every single patent. So any patent they violate or if the defendant is found to be in violation of it, that's a win for us. So our patent estate is robust. And so we believe our patents are robust, and we're going to defend them to the very end. Obviously, we want to do what's right for the business and get back to the business of the business, but not at the price of sacrificing future value. And we believe that the Teva and the Lupin agreements are very fine agreements for all parties. And so we like that structure. Understood, and you have a BioMarin royalty. That rolls off this year, if I understand correctly? Yeah. So as we talked about in our Q3 10-Q, third quarter 10-Q, the BioMarin royalty on Firdapse ends in January 2026. And based on the midpoint guidance, the vast majority of which is U.S., our royalty obligation is about $30 million-$35 million. And all of this is in our Q, I believe, footnote 13. And so that reduction in royalty goes straight to pre-tax income and will contribute to operating leverage. So that's a big win for the company. Okay. Pavan got some questions. Yeah. I guess just in terms of the uptake in Firdapse LEMS patients, I think you've previously estimated that there are upwards of 900 tumor LEMS patients, and only 3%-4% of those are ever diagnosed. So this is the growth segment of the LEMS patient population. Maybe if you can update us on the traction that you're seeing in those thoracic oncology centers and whether the diagnostic code implementation has improved materially to aid in the identification of those patients? Sure, so when we think about the cancer-associated LEMS part of it, remember there are two parts of the business, and it's split about 50/50 patients, there's the idiopathic or non-cancer LEMS and the cancer LEMS, and so the traction we're experiencing right now really goes back to, goes back actually a couple of years when we were making the change on the NCCN guidelines and recommending change, but the success we've had in this year of streamlining the VGCC test, so there's a test that's very indicative of whether or not a patient would actually have LEMS, so we got that testing centralized, and now there's blood pickup in the office where the patient is. Previously, the patient had to go out, come in, so the traction we're getting is building on these first steps. Then we got the NCCN guidelines in July, which is a huge win for patients and a huge win for the company. And now we're working with these large, high-control oncology practices to embed the change in NCCN guideline into the care pathway. This is a really critical step. So looking again, high volumes, high control. And then from there, working with these accounts to then help train the physician that when they see a small cell lung cancer patient, they should, in fact, do a VGCC test first. They may partner with a neurologist for a definitive diagnosis, or they may feel comfortable doing it themselves. The recommendation is to work with a neurologist. The NCCN guideline recommendation is to work with a neurologist. So the traction we're getting is the receptivity right now from the oncology practices that are saying, "Yeah, this looks very interesting." We don't anticipate significant impact for the cancer LEMS in the first half of next year because there are many of these practices. It takes a significant amount of effort to get in there and actually help them understand the change in NCCN guidelines and then what they have to do for the change in their care pathways. We have dedicated resources in the field that are looking at this and working with these accounts. And I think the traction right now is quite positive. And can you remind us what's the pool of patients that you've identified for treatment with Firdapse? And how has the composition of that pool evolved over time in terms of the split of idiopathic versus oncology LEMS patients? So the pool we keep is about 500, and we use data and machine learning to look for those patients who've gone through a certain series of events in their diagnostic journey. And then we hyper-prioritize to those who are furthest along. And so we're seeing about 50% of our patients in any quarter come from that pool, either they're idiopathic or they're cancer. Your question is really difficult to answer because on the split, it's typically about, at any point in time, it's typically about 80/20, with 80% being idiopathic. That's why we think there's such a nice upside here. The reason why it's difficult to answer when they come in is because LEMS is sometimes the first indication that a patient has small cell lung cancer. So they come in with idiopathic LEMS, but over time, they end up with the diagnosis of small cell lung cancer. So it's not as clean as we would really like. But we are seeing, as an example, through the work that we've done, an increase in VGCC testing of 9% per quarter, quarter on quarter, which is really phenomenal. We think that's really exciting. And we're not really that embedded into the care pathways yet. So I think there's a lot of upside here. Yeah. And then I guess another layer of this is the missed diagnosis of LEMS with myasthenia gravis. And I think you've noted that about half of your patients have come from this pipeline of pool of patient opportunity. So maybe if you can talk about how the rate of converting these misdiagnosed MG patients to LEMS diagnosis has accelerated over the last 12 months and how much work is there left to be done here? So this side of the business, the idiopathic side of the Firdapse business, is what we would call a prospecting business. You have to find these patients. They're hard to find. The average physician sees one in their entire career. So it's challenging. So we're following a similar path that we talked about on the oncology side. So obviously, we have this pool of patients we track, and through machine learning, we hyper-prioritize. And then our RAMs, our regional account managers, go in and prioritize again. Looking at the opportunity for growth, we think myasthenia gravis and all the great therapies that are out there for myasthenia gravis are excellent. And actually, this is a great investment that we actually don't have to make because there's a lot of DTC out there. And many of these patients will end up with what is called a seronegative myasthenia gravis test. And so we're doing the same thing with the national labs that we did on the cancer side, which is trying to get them to embed reflex testing. So if a patient comes up with seronegative for myasthenia gravis, so there's no antibodies there, reflex test them for VGCC. And we're getting a lot of traction there as well. So we believe that this will become potentially, if successful, less of a prospecting market and more concentrated in those high myasthenia gravis accounts. So that's what we're looking for. And then obviously, to grow the awareness through education, targeted education with those accounts. And that's our plan on both sides of the business. We think both have great growth potential. And so we're going to work both ends of the market. Yeah. I guess hitting on Fycompa really quickly, that's sort of an asset that's undergoing generic competition. So maybe if you can talk about the slope of that erosion. I know it's been a little less than initially expected. So is brand stickiness holding up in 4Q as well? And how do you expect that through 2026 onward? Sure. So it's a little early to tell about the fourth quarter, and let's just go back over and talk about the timeline here, so on the solid dose, there's two formulations of Fycompa. One is solid dose, and the other is suspension, oral suspension. What we've seen with the solid dose is there were two first filers in this market, and they could have come in in late May. Only one came in and came in in late June, so we got a nice maintenance of our business there, and the erosion, because only one is there, and the player, again, it goes back to Teva, is the most sophisticated player in this market. Teva came in with a really good price, market minus, so they looked at the market and said we're coming in with a 17% discount. There were other products that went generic in the epilepsy space around the same time that we did, but they had multiple players coming in. We did not experience that. So there's a lift from that. There is the stickiness that you always see with epilepsy patients. They want to have a drug that they can be sure is going to protect them from seizure. But we know that last week, two additional generics, solid dose, were approved. They are not yet on the market as far as we can tell. So they're not taking away from our Fycompa sales. And here we are in the 8th of December. So we think we can finish the year strong. And we took up guidance on this product. However, the oral suspension, we expect a single first filer to come in or to be approved in the middle of December. And prior to the incursion of generics back in June, oral suspension was 15% of our business. So we're seeing a shift, and we expect to see a shift. So product has done really, really well so far. But we all know success of brands is inversely proportional to the number of generic players. And now we're going to be experiencing more generic players. What we're looking for is to be able to cover that loss of sales with growth on our other products. And that's what we're really pointing toward. And then in terms of a higher-level question with capital allocation, so the board recently authorized a $200 million share repurchase program. So should investors be thinking about this as a signal that maybe M&A valuations are too high, or is this just a signal that the balance sheet is really strong and then you have flexibility to do both buybacks and business development deals as you see fit at a given time? And how should we think about how you're thinking about the balance of capital towards buybacks versus BD in a given year? Yeah. So excellent question. So we don't like our share price. And my joke is, if you ever met a CEO who did, you got the wrong CEO. And we think we have the opportunity to contribute value back to shareholders without hurting our opportunities and business development. So on a non-GAAP basis, we did $52 million and change in the third quarter. And so we believe we can continue to do that. And this share repurchase is over 15 months. So we are loaded. We are ready to go on business development. This share repurchase will not affect our ability. And again, we have no funded debt. So if there was an opportunity, as Jason and I were talking about earlier, if there's an opportunity to leverage on a great deal, we would take that opportunity. We think this is a good way to return some value to shareholders while executing our business. Then if I can go back to Agamree with the label expansion opportunities, I think this is something that's come up a bunch of times because it has an upside lever to a product right now in terms of the Street's forecast. Just trying to narrow down what you think are the most appealing or attractive opportunities, can you help us think through the framework that you're using for that? Sure. So when we think about the opportunity, we want those rare conditions. So it's got to be rare. We want it to be long-term and where there's a high need for a steroid. If it's episodic, it's less interesting to us. If it's continuous, and we have that profile that you see in the GUARDIAN trial where bone strength is there or growth or stature, avoidance of cataracts, etc., better behavior, we like those opportunities. So again, just focusing on the opportunity to create the greatest value for patients as quickly as possible. And we'll have a better handle on that after the new year to be able to pick the right target. Then in terms of the IP outcome that could come post a potential bench trial that's scheduled in March 2026 for Firdapse, how should we be thinking about the bull bear scenario here? Assuming worst-case scenario, again, or if Firdapse goes LOE earlier than we expected, how should investors be thinking about such a scenario and the risk profile of the company in such a scenario? So we really believe in our IP, and we're going to do everything we can to defend it. I think, again, settling with three out of the four defendants so far, I think, demonstrates a solid position for us. One of the most interesting things about the Firdapse market is we have already faced competition that was significantly price reduced in Ruzurgi. And Ruzurgi was out there at half the price of Firdapse. And the interesting thing is when we talked to, we just talked about the value of what's behind the front of the house. So the value is getting the patient onto the drug, keeping them on, and optimizing their dose for their benefit. Those systems, that infrastructure is so valuable to a company like ours. It can make a huge difference. Ruzurgi coming out at half the price, we actually didn't feel any significant dent to our business. We think that we have a pretty good system that can defend us going forward. Again, we have strong belief in our IP, and we look forward to resolving that in the most appropriate way possible for the benefit of the company and patients. Hey, Rich, I have a couple of follow-ups. So first, just on your rare orphan portfolio, how should we think about pricing power going forward? I know that these are expensive medications. And is there a certain cap, 2%, 3%, given, I don't know, with plans? These can be needle-moving, or are they so small? Maybe there's a little more latitude with pricing power. And I know that there's some Medicaid exposure here, right? So there's price penalties. So just maybe help us think through the framework there. So we look at the opportunity. If there's an opportunity to increase price to cover our increased costs or anything like that, we look at that, and we measure against what the penalties, Jason, you talked about. And we're not going to do anything egregious that you can look at a company that's trying to drive the top line but ends up with less on the bottom line. And that's actually quite possible in these markets where you can actually take a price increase that's quite high and end up making less money. We don't like that, so we don't do that. So we're always going to look for that balance of what we think is the right thing to do based on the services that we provide, the value that the drug provides, and the market conditions, whether it's CPI-Urban, or whatever's going on. So we're always going to judge that in a multifactorial way. But the pricing power of old is just not there anymore, 10, 15 years ago. Yeah, and then with cancer LEMS, my understanding in past discussions with you guys is that this is not like a buy-and-bill opportunity with these community oncs, such that this could be a money-making endeavor for community oncologists, which could spur faster utilization. I know that some people have asked me about that, but I feel like in the past, you guys have said you don't expect this to be a buy-and-bill product with these oncologists. So it could be, actually. So medical oncologists cover the cost of their practice through the reimbursement around medication, whether it's injected, infused, or oral. Many of these larger practices do, in fact, have in-house pharmacies that can distribute drug. And quite frankly, if these patients are coming in on an every three-week or four-week basis for their cancer therapy, having that care team centralized in one spot becomes critically important for the outcome. One of the things we didn't talk about is we think there's about 900, initially 900 new patients on the cancer LEMS side. Those cancer LEMS patients, they have typically small cell lung cancer. A small cell lung cancer patient lives about eight or nine months. Nobody knows why, but a patient with small cell lung cancer LEMS lives 17 months. In our discussions with oncologists, they've said, "Well, I assume the patient would only live eight months or nine months. Now that I'm seeing them live a year and a half, I think about them differently. And I want their quality of life to be higher." With LEMS, Lambert-Eaton myasthenic syndrome, as the disease progresses, the patients lose the ability to walk, ambulate, or certain control of extremities. So if they can improve, the physician can improve the quality of life of the patient, that's something they're interested in. We do believe that some of the accounts will actually use the opportunity to distribute Firdapse in-house as a way to offset costs of their practice, overhead, malpractice insurance, etc. Yeah. Okay. That's helpful to know. Appreciate your time, Rich, as always. And this was really helpful. And thanks for the pitch today. Thank you so much. I really appreciate the opportunity. Yeah, anytime. Thanks very much. Okay. Take care.

Speaker 4: Ladies and gentlemen, the program is about to begin at this time, and it's my pleasure to turn the program over to your host, Jason Gerberry. Ladies and gentlemen, the program is about to begin at this time, and it's my pleasure to turn the program over to your host, Jason Gerberry. ladies and gentlemen the program is about to begin at this time and it's my pleasure to turn the program over to your host jason gerberry

Speaker 5: Good day, everybody, and thanks for joining us at the BofA Virtual CNS Conference. My name is Jason Gerberry. I'm one of the biotech analysts here, and I'm joined by Pavan Patel, who's one of my colleagues who helps with Catalyst Pharma. Catalyst Pharma is our next company presenter, and Rich Daly, CEO. Rich, thanks so much for joining us and giving us the latest update on Catalyst. Good day, everybody, and thanks for joining us at the BofA Virtual CNS Conference. good day everybody and thanks for joining us at the bofa virtual cns conference My name is Jason Gerberry. my name is jason gerberry I'm one of the biotech analysts here, and I'm joined by Pavan Patel, who's one of my colleagues who helps with Catalyst Pharma. i'm one of the biotech analysts here and i'm joined by pavan patel who's one of my colleagues who helps with catalyst pharma Catalyst Pharma is our next company presenter, and Rich Daly, CEO. catalyst pharma is our next company presenter and rich daly ceo Rich, thanks so much for joining us and giving us the latest update on Catalyst. rich thanks so much for joining us and giving us the latest update on catalyst

Speaker 2: Jason, thanks for having us. We really appreciate it, and looking forward to the conversation. Thank you. Jason, thanks for having us. jason thanks for having us We really appreciate it, and looking forward to the conversation. we really appreciate it and looking forward to the conversation Thank you. thank you

Speaker 5: So maybe just starting with kind of the overarching BD and licensing, that's obviously kind of core to what you do and building out the business. And you had sort of a stopgap product with Fycompa, but your two main assets for kind of the longer-term Agamree and Firdapse. And so, yeah, how important is it for you to get a resolution with Firdapse's IP before you deploy capital in a more significant way? Maybe just talk about the importance of doing a deal in 2026 as a priority. Maybe we can start there and then delve into additional questions. So maybe just starting with kind of the overarching BD and licensing, that's obviously kind of core to what you do and building out the business. so maybe just starting with kind of the overarching bd and licensing that's obviously kind of core to what you do and building out the business And you had sort of a stopgap product with Fycompa, but your two main assets for kind of the longer-term Agamree and Firdapse. and you had sort of a stopgap product with fycompa but your two main assets for kind of the longer-term agamree and firdapse And so, yeah, how important is it for you to get a resolution with Firdapse's IP before you deploy capital in a more significant way? and so yeah how important is it for you to get a resolution with firdapse's ip before you deploy capital in a more significant way Maybe just talk about the importance of doing a deal in 2026 as a priority. maybe just talk about the importance of doing a deal in 2026 as a priority Maybe we can start there and then delve into additional questions. maybe we can start there and then delve into additional questions

Speaker 2: Sure, so we feel like with the acquisition or the arrangement with Agamree and Santhera, Santhera is the originator of the product. We feel like we've had ample time to incorporate the product and digest the product and move the product forward. We're always on the look for opportunities. As you mentioned, we're buying those company, and so we're looking for those products that are immediately accretive or nearly immediately accretive. They're in the rare space, differentiated, unmet clinical need, and a ceiling of about $500 million in sales, so what we're trying to do in that scenario is eliminate the clinical risk, minimize the regulatory risk. We did take some regulatory risk with Agamree, and then absorb the commercial risk because we think that's a hallmark and the strength of the company, so we want to get a deal done. We want the right deal, however. Sure, so we feel like with the acquisition or the arrangement with Agamree and Santhera, Santhera is the originator of the product. sure so we feel like with the acquisition or the arrangement with agamree and santhera santhera is the originator of the product We feel like we've had ample time to incorporate the product and digest the product and move the product forward. we feel like we've had ample time to incorporate the product and digest the product and move the product forward We're always on the look for opportunities. we're always on the look for opportunities As you mentioned, we're buying those company, and so we're looking for those products that are immediately accretive or nearly immediately accretive. as you mentioned we're buying those company and so we're looking for those products that are immediately accretive or nearly immediately accretive They're in the rare space, differentiated, unmet clinical need, and a ceiling of about $500 million in sales, so what we're trying to do in that scenario is eliminate the clinical risk, minimize the regulatory risk. they're in the rare space differentiated unmet clinical need and a ceiling of about $500 million in sales so what we're trying to do in that scenario is eliminate the clinical risk minimize the regulatory risk We did take some regulatory risk with Agamree, and then absorb the commercial risk because we think that's a hallmark and the strength of the company, so we want to get a deal done. we did take some regulatory risk with agamree and then absorb the commercial risk because we think that's a hallmark and the strength of the company so we want to get a deal done We want the right deal, however. we want the right deal however I think you saw in our press release for Q3 earnings, since the beginning of the year, we assessed more than 100 opportunities. One of the things that's fundamentally changing in the marketplace is the capital markets. Typically, an opportunity for us would be a company that has enough money to launch but not sustain. With our infrastructure already built, we can come in and really take over. An ex-U.S. company, Asia, Europe, that is looking for an opportunity to plant a flag in the U.S. but doesn't want to take the commercial risk. I think you saw in our press release for Q3 earnings, since the beginning of the year, we assessed more than 100 opportunities. i think you saw in our press release for q3 earnings since the beginning of the year we assessed more than 100 opportunities One of the things that's fundamentally changing in the marketplace is the capital markets. one of the things that's fundamentally changing in the marketplace is the capital markets Typically, an opportunity for us would be a company that has enough money to launch but not sustain. typically an opportunity for us would be a company that has enough money to launch but not sustain With our infrastructure already built, we can come in and really take over. with our infrastructure already built we can come in and really take over An ex-U.S. company, Asia, Europe, that is looking for an opportunity to plant a flag in the U.S. but doesn't want to take the commercial risk. an ex-u.s company asia europe that is looking for an opportunity to plant a flag in the u.s but doesn't want to take the commercial risk But with the capital markets opening up, again, we may be in a situation where we have to broaden the scope of what we're looking at and perhaps go back just a little bit into the pipeline, into the Phase III, which is proof of concept is assessed and is positive and looking forward, and we've been talking about this for two years, so that's the nature of what we're doing. We've been looking at those opportunities and assessing them as well as the immediately accretive or nearly immediately accretive opportunities. But with the capital markets opening up, again, we may be in a situation where we have to broaden the scope of what we're looking at and perhaps go back just a little bit into the pipeline, into the Phase III, which is proof of concept is assessed and is positive and looking forward, and we've been talking about this for two years, so that's the nature of what we're doing. but with the capital markets opening up again we may be in a situation where we have to broaden the scope of what we're looking at and perhaps go back just a little bit into the pipeline into the phase iii which is proof of concept is assessed and is positive and looking forward and we've been talking about this for two years so that's the nature of what we're doing We've been looking at those opportunities and assessing them as well as the immediately accretive or nearly immediately accretive opportunities. we've been looking at those opportunities and assessing them as well as the immediately accretive or nearly immediately accretive opportunities

Speaker 5: You mentioned, and maybe I haven't heard this part before, but a ceiling of $500 million, that would be like a peak revenue. Is that sort of like what you're screening for? You mentioned, and maybe I haven't heard this part before, but a ceiling of $500 million, that would be like a peak revenue. you mentioned and maybe i haven't heard this part before but a ceiling of $500 million that would be like a peak revenue Is that sort of like what you're screening for? is that sort of like what you're screening for

Speaker 2: Yeah, so when we think about what can move the needle for us and what can move the needle for competitors who might be larger, you stay around the $500 million, and that doesn't really necessarily move the needle for some of these companies, so it's a different set of companies that show up at the auction, so when we're there and we have about $689 million in cash and no funded debt, we're a powerful bidder. We don't want to get into a war of attrition with a much larger player, and as good as our bank is and our balance sheet is and our ability to take on debt, we're not going to compete where we can't win, and some of those larger opportunities just aren't for us right now. That will change over time. Yeah, so when we think about what can move the needle for us and what can move the needle for competitors who might be larger, you stay around the $500 million, and that doesn't really necessarily move the needle for some of these companies, so it's a different set of companies that show up at the auction, so when we're there and we have about $689 million in cash and no funded debt, we're a powerful bidder. yeah so when we think about what can move the needle for us and what can move the needle for competitors who might be larger you stay around the $500 million and that doesn't really necessarily move the needle for some of these companies so it's a different set of companies that show up at the auction so when we're there and we have about $689 million in cash and no funded debt we're a powerful bidder We don't want to get into a war of attrition with a much larger player, and as good as our bank is and our balance sheet is and our ability to take on debt, we're not going to compete where we can't win, and some of those larger opportunities just aren't for us right now. we don't want to get into a war of attrition with a much larger player and as good as our bank is and our balance sheet is and our ability to take on debt we're not going to compete where we can't win and some of those larger opportunities just aren't for us right now That will change over time. that will change over time

Speaker 5: Yeah, well, I know there's been analogies to your business model to Horizon Therapeutics who had a model like this, and there was probably a time when people thought Tepezza would be a $500 million peak sale drug, right? Yeah, well, I know there's been analogies to your business model to Horizon Therapeutics who had a model like this, and there was probably a time when people thought Tepezza would be a $500 million peak sale drug, right? yeah well i know there's been analogies to your business model to horizon therapeutics who had a model like this and there was probably a time when people thought tepezza would be a $500 million peak sale drug right

Speaker 2: Right. Right. right

Speaker 5: There is an opportunity for Firdapse to eclipse $500 million in revenue, right? There is an opportunity for Firdapse to eclipse $500 million in revenue, right? there is an opportunity for firdapse to eclipse $500 million in revenue right

Speaker 2: Absolutely. Absolutely. absolutely

Speaker 5: So is it that maybe $500 million is sort of a conservative base case when you look at assets, but they have the upside potential to exceed that revenue threshold? So is it that maybe $500 million is sort of a conservative base case when you look at assets, but they have the upside potential to exceed that revenue threshold? so is it that maybe $500 million is sort of a conservative base case when you look at assets but they have the upside potential to exceed that revenue threshold

Speaker 2: Yeah. Great question and great commentary. Yes, so we look at it and we think about how we might be able to accelerate that opportunity. So when you look at the opportunity to grow products, really, you're looking at your company, rather, and the lifecycle management. Take the products that are inline commercial. How do you get patients on earlier, keep them on longer, or optimize their dose? Then there's expansion, and we do that for both the Agamree and Firdapse. And then there's expansion within the label. So you look at Firdapse as an example. That expansion for LEMS applies both to idiopathic and cancer-associated LEMS. And we have a real focus on maintaining our idiopathic business, but also growing. And then obviously, there's label expansion too. And we're going to look at that for Agamree as well. We think there's opportunity. Yeah. yeah Great question and great commentary. great question and great commentary Yes, so we look at it and we think about how we might be able to accelerate that opportunity. yes so we look at it and we think about how we might be able to accelerate that opportunity So when you look at the opportunity to grow products, really, you're looking at your company, rather, and the lifecycle management. so when you look at the opportunity to grow products really you're looking at your company rather and the lifecycle management Take the products that are inline commercial. take the products that are inline commercial How do you get patients on earlier, keep them on longer, or optimize their dose? how do you get patients on earlier keep them on longer or optimize their dose Then there's expansion, and we do that for both the Agamree and Firdapse. then there's expansion and we do that for both the agamree and firdapse And then there's expansion within the label. and then there's expansion within the label So you look at Firdapse as an example. so you look at firdapse as an example That expansion for LEMS applies both to idiopathic and cancer-associated LEMS. that expansion for lems applies both to idiopathic and cancer-associated lems And we have a real focus on maintaining our idiopathic business, but also growing. and we have a real focus on maintaining our idiopathic business but also growing And then obviously, there's label expansion too. and then obviously there's label expansion too And we're going to look at that for Agamree as well. and we're going to look at that for agamree as well We think there's opportunity. we think there's opportunity This is a steroid that works. There are data that have just come out of Europe from Santhera, out of the that look really, really promising and support our work on the SUMMIT trial, which we can talk about. But if the steroid works and it has a favorable profile for long-term steroid use versus standard of care, we're going to look for other rare disease opportunities to apply that molecule. And so that's one of the key initiatives we've got in growing the company. So you put the whole thing together, the last one for the company is business development and looking to actually accelerate growth of the company through incremental products. This is a steroid that works. this is a steroid that works There are data that have just come out of Europe from Santhera, out of the that look really, really promising and support our work on the SUMMIT trial, which we can talk about. there are data that have just come out of europe from santhera out of the that look really really promising and support our work on the summit trial which we can talk about But if the steroid works and it has a favorable profile for long-term steroid use versus standard of care, we're going to look for other rare disease opportunities to apply that molecule. but if the steroid works and it has a favorable profile for long-term steroid use versus standard of care we're going to look for other rare disease opportunities to apply that molecule And so that's one of the key initiatives we've got in growing the company. and so that's one of the key initiatives we've got in growing the company So you put the whole thing together, the last one for the company is business development and looking to actually accelerate growth of the company through incremental products. so you put the whole thing together the last one for the company is business development and looking to actually accelerate growth of the company through incremental products

Speaker 5: When do you think you'll have a handle on that aspect of other rare opportunities for Agamree? I imagine with patent and the data exclusivity and factors like that, you can't wait forever to identify those and more visibly kind of outline those. When do you think you'll have a handle on that aspect of other rare opportunities for Agamree? when do you think you'll have a handle on that aspect of other rare opportunities for agamree I imagine with patent and the data exclusivity and factors like that, you can't wait forever to identify those and more visibly kind of outline those. i imagine with patent and the data exclusivity and factors like that you can't wait forever to identify those and more visibly kind of outline those

Speaker 2: Right. So the orphan status for Agamree will come to an end in 2029. But because of patent restoration, we believe we go out to 2034. So we have time. However, that doesn't mean we're going slow. So we're right now doing a Phase I study, and the Phase I study is looking at two elements. The first element is what's the dose equivalence between prednisone and Emflaza and Agamree so we can give better guidance to practitioners should they ask. And the other one is what's the immunosuppressive dose? And when you think about the immunosuppressive dose, we look at that from a gene therapy standpoint because steroids are used on board with gene therapy, and you need to immunosuppress those patients prior to giving them gene therapy. But we can't actually lose. Right. right So the orphan status for Agamree will come to an end in 2029. so the orphan status for agamree will come to an end in 2029 But because of patent restoration, we believe we go out to 2034. but because of patent restoration we believe we go out to 2034 So we have time. so we have time However, that doesn't mean we're going slow. however that doesn't mean we're going slow So we're right now doing a Phase I study, and the Phase I study is looking at two elements. so we're right now doing a phase i study and the phase i study is looking at two elements The first element is what's the dose equivalence between prednisone and Emflaza and Agamree so we can give better guidance to practitioners should they ask. the first element is what's the dose equivalence between prednisone and emflaza and agamree so we can give better guidance to practitioners should they ask And the other one is what's the immunosuppressive dose? and the other one is what's the immunosuppressive dose And when you think about the immunosuppressive dose, we look at that from a gene therapy standpoint because steroids are used on board with gene therapy, and you need to immunosuppress those patients prior to giving them gene therapy. and when you think about the immunosuppressive dose we look at that from a gene therapy standpoint because steroids are used on board with gene therapy and you need to immunosuppress those patients prior to giving them gene therapy But we can't actually lose. but we can't actually lose So, if we find the immunosuppressive dose, there's an opportunity to apply it to gene therapy and then ratchet it down to those doses that are effective and don't cause immunosuppression for those other non-DMD opportunities. If we can't find the immunosuppressive dose, it's an ideal product for long-term use in rare conditions. And so we're really excited. We'll be seeing those data after the first of the year. And once we see the data, we'll be making the decision about where we go. So I would say first half of the year, we'll have a decision. So, if we find the immunosuppressive dose, there's an opportunity to apply it to gene therapy and then ratchet it down to those doses that are effective and don't cause immunosuppression for those other non-DMD opportunities. so if we find the immunosuppressive dose there's an opportunity to apply it to gene therapy and then ratchet it down to those doses that are effective and don't cause immunosuppression for those other non-dmd opportunities If we can't find the immunosuppressive dose, it's an ideal product for long-term use in rare conditions. if we can't find the immunosuppressive dose it's an ideal product for long-term use in rare conditions And so we're really excited. and so we're really excited We'll be seeing those data after the first of the year. we'll be seeing those data after the first of the year And once we see the data, we'll be making the decision about where we go. and once we see the data we'll be making the decision about where we go So I would say first half of the year, we'll have a decision. so i would say first half of the year we'll have a decision

Speaker 5: Okay. And then going back to my initial question, what I didn't hear was that Firdapse's IP resolution was like a swing factor at all on the BD prioritization and timing of making a decision? Okay. okay And then going back to my initial question, what I didn't hear was that Firdapse's IP resolution was like a swing factor at all on the BD prioritization and timing of making a decision? and then going back to my initial question what i didn't hear was that firdapse's ip resolution was like a swing factor at all on the bd prioritization and timing of making a decision

Speaker 2: Coming to a positive resolution on the Firdapse's IP is obviously a high priority for us. We're interested in doing it. There were three first filers. We've settled with two of the three. There was a fourth filer, not a first filer. We've settled with that company as well. And there's one defendant left. And when you look at the situation with Teva settling, Teva is obviously a sophisticated player in this space, in the generic space. And so with them settling, it gives us further confidence, even though our confidence was very, very high in our IP. To your question about whether we're waiting for that, the answer is no. We're looking for that opportunity. We have great cash flow right now. We want to continue to build the company, and we want to look at those opportunities that can really deliver for patients and for the company. Coming to a positive resolution on the Firdapse's IP is obviously a high priority for us. coming to a positive resolution on the firdapse's ip is obviously a high priority for us We're interested in doing it. we're interested in doing it There were three first filers. there were three first filers We've settled with two of the three. we've settled with two of the three There was a fourth filer, not a first filer. there was a fourth filer not a first filer We've settled with that company as well. we've settled with that company as well And there's one defendant left. and there's one defendant left And when you look at the situation with Teva settling, Teva is obviously a sophisticated player in this space, in the generic space. and when you look at the situation with teva settling teva is obviously a sophisticated player in this space in the generic space And so with them settling, it gives us further confidence, even though our confidence was very, very high in our IP. and so with them settling it gives us further confidence even though our confidence was very very high in our ip To your question about whether we're waiting for that, the answer is no. to your question about whether we're waiting for that the answer is no We're looking for that opportunity. we're looking for that opportunity We have great cash flow right now. we have great cash flow right now We want to continue to build the company, and we want to look at those opportunities that can really deliver for patients and for the company. we want to continue to build the company and we want to look at those opportunities that can really deliver for patients and for the company

Speaker 5: I haven't checked recently, but do you have a trial date teed up for that one outstanding? I haven't checked recently, but do you have a trial date teed up for that one outstanding? i haven't checked recently but do you have a trial date teed up for that one outstanding

Speaker 2: We do. So the 30-month stay, and the trial date is scheduled for March. And the 30-month exclusivity or the 30-month pause actually ends in May. So we believe that gives the judge enough time if we get the trial. But again, we're going to do the right thing for the company and for the patients and for the shareholders. We do. we do So the 30-month stay , and the trial date is scheduled for March. so the 30-month stay and the trial date is scheduled for march And the 30-month exclusivity or the 30-month pause actually ends in May. and the 30-month exclusivity or the 30-month pause actually ends in may So we believe that gives the judge enough time if we get the trial. so we believe that gives the judge enough time if we get the trial But again, we're going to do the right thing for the company and for the patients and for the shareholders. but again we're going to do the right thing for the company and for the patients and for the shareholders

Speaker 5: Okay. In terms of deal type, size, structure, is the priority still sort of an exclusive license, maybe something that's manageable on the upfront size of dollars out such that you can not have to take on leverage, or is the aperture just, "Hey, we're going to go with the best opportunity in front of us, and if that requires us to take on leverage, we'll do it"? Okay. okay In terms of deal type, size, structure, is the priority still sort of an exclusive license, maybe something that's manageable on the upfront size of dollars out such that you can not have to take on leverage, or is the aperture just, "Hey, we're going to go with the best opportunity in front of us, and if that requires us to take on leverage, we'll do it"? in terms of deal type size structure is the priority still sort of an exclusive license maybe something that's manageable on the upfront size of dollars out such that you can not have to take on leverage or is the aperture just "hey we're going to go with the best opportunity in front of us and if that requires us to take on leverage we'll do it"

Speaker 2: So if there's an opportunity to take on leverage, we would want to be sure that it's a slam dunk. So when you look at we have almost $700 million. So what could we do on the upfront, plus the cash generation we have right now on a quarterly basis? So we would want to really be sure that if we were to take on leverage, that this is more than likely an on-market product with a long horizon. You mentioned the Fycompa opportunity, which was a great opportunity for us because it really diversified our income statement, gave us additional cash, and enabled us to do the Santhera or Agamree deal. If we're going to do something where we take on leverage, it has to be really spot on and pretty much a PTS of 100% on the commercial side. So on market with long legs. So if there's an opportunity to take on leverage, we would want to be sure that it's a slam dunk. so if there's an opportunity to take on leverage we would want to be sure that it's a slam dunk So when you look at we have almost $700 million. so when you look at we have almost $700 million So what could we do on the upfront, plus the cash generation we have right now on a quarterly basis? so what could we do on the upfront plus the cash generation we have right now on a quarterly basis So we would want to really be sure that if we were to take on leverage, that this is more than likely an on-market product with a long horizon. so we would want to really be sure that if we were to take on leverage that this is more than likely an on-market product with a long horizon You mentioned the Fycompa opportunity, which was a great opportunity for us because it really diversified our income statement, gave us additional cash, and enabled us to do the Santhera or Agamree deal. you mentioned the fycompa opportunity which was a great opportunity for us because it really diversified our income statement gave us additional cash and enabled us to do the santhera or agamree deal If we're going to do something where we take on leverage, it has to be really spot on and pretty much a PTS of 100% on the commercial side. if we're going to do something where we take on leverage it has to be really spot on and pretty much a pts of 100% on the commercial side So on market with long legs. so on market with long legs

Speaker 5: And then I think you guys had made some comments about looking a little bit more broadly in orphan space beyond CNS. I guess I would have looked at your business model and said, "When you're looking at all these different rare orphan indications, these deals are probably not predicated on SG&A synergies, right?" Because these sales forces are pretty small. And then I think you guys had made some comments about looking a little bit more broadly in orphan space beyond CNS. and then i think you guys had made some comments about looking a little bit more broadly in orphan space beyond cns I guess I would have looked at your business model and said, "When you're looking at all these different rare orphan indications, these deals are probably not predicated on SG&A synergies, right?" Because these sales forces are pretty small. i guess i would have looked at your business model and said "when you're looking at all these different rare orphan indications these deals are probably not predicated on sg&a synergies right?" because these sales forces are pretty small

Speaker 2: Yeah. They're tight. Yeah. yeah They're tight. they're tight

Speaker 5: So I guess the only relevance to me of that statement is just like, "Hey, maybe we have competencies in-house for looking at stuff in the CNS space, but now we feel like perhaps we have greater competency in-house to look, I don't know, in another therapeutic area specifically that's rare orphan. So I guess the only relevance to me of that statement is just like, "Hey, maybe we have competencies in-house for looking at stuff in the CNS space, but now we feel like perhaps we have greater competency in-house to look, I don't know, in another therapeutic area specifically that's rare orphan. so i guess the only relevance to me of that statement is just like "hey maybe we have competencies in-house for looking at stuff in the cns space but now we feel like perhaps we have greater competency in-house to look i don't know in another therapeutic area specifically that's rare orphan

Speaker 2: Sure. So from a competency standpoint, so as an example, Firdapse and Agamree are the fourth and fifth products I've worked on in the rare space in my career. So we feel like we have great competency across the board, not just with me, obviously, but with everybody, and you look at the diverse backgrounds from a therapeutic area standpoint, we've got great skills in-house. We can do that. I think when it comes to what's going on and how you support the business and support the patient, it gets back to that first statement I made about lifecycle management for a product. Get the patient on earlier, keep the patient on longer, and optimize their dose. That infrastructure sits behind the sales organization. Obviously, it's the specialty pharmacy, the patient hub, and the third-party logistics provider or the shipper of the drug. Sure. sure So from a competency standpoint, so as an example, Firdapse and Agamree are the fourth and fifth products I've worked on in the rare space in my career. so from a competency standpoint so as an example firdapse and agamree are the fourth and fifth products i've worked on in the rare space in my career So we feel like we have great competency across the board, not just with me, obviously, but with everybody, and you look at the diverse backgrounds from a therapeutic area standpoint, we've got great skills in-house. so we feel like we have great competency across the board not just with me obviously but with everybody and you look at the diverse backgrounds from a therapeutic area standpoint we've got great skills in-house We can do that. we can do that I think when it comes to what's going on and how you support the business and support the patient, it gets back to that first statement I made about lifecycle management for a product. i think when it comes to what's going on and how you support the business and support the patient it gets back to that first statement i made about lifecycle management for a product Get the patient on earlier, keep the patient on longer, and optimize their dose. get the patient on earlier keep the patient on longer and optimize their dose That infrastructure sits behind the sales organization. that infrastructure sits behind the sales organization Obviously, it's the specialty pharmacy, the patient hub, and the third-party logistics provider or the shipper of the drug. obviously it's the specialty pharmacy the patient hub and the third-party logistics provider or the shipper of the drug So when we look at that, we believe we're really set up. And that part of the business is applicable to any orphan or rare condition. So we believe that with the expertise we have in-house, plus the expertise in supporting the patient and the provider and the payer, we think we can move well beyond CNS. So when we look at that, we believe we're really set up. so when we look at that we believe we're really set up And that part of the business is applicable to any orphan or rare condition. and that part of the business is applicable to any orphan or rare condition So we believe that with the expertise we have in-house, plus the expertise in supporting the patient and the provider and the payer, we think we can move well beyond CNS. so we believe that with the expertise we have in-house plus the expertise in supporting the patient and the provider and the payer we think we can move well beyond cns

Speaker 5: In your BD team, can you remind me how many people you have looking at transactions, and do you have the, I guess, the scope internally to look at assets coming out of China, or is the China biotech engine really less focused on some of these nichier orphan drug applications that kind of fall within your sweet spot? Because it does seem to me like there's a lot of focus on me-too sort of treatments in big large established categories, so I just wonder how that maybe fits within your scope of transactions that are interesting to you. In your BD team, can you remind me how many people you have looking at transactions, and do you have the, I guess, the scope internally to look at assets coming out of China, or is the China biotech engine really less focused on some of these nichier orphan drug applications that kind of fall within your sweet spot? in your bd team can you remind me how many people you have looking at transactions and do you have the i guess the scope internally to look at assets coming out of china or is the china biotech engine really less focused on some of these nichier orphan drug applications that kind of fall within your sweet spot Because it does seem to me like there's a lot of focus on me-too sort of treatments in big large established categories, so I just wonder how that maybe fits within your scope of transactions that are interesting to you. because it does seem to me like there's a lot of focus on me-too sort of treatments in big large established categories so i just wonder how that maybe fits within your scope of transactions that are interesting to you

Speaker 2: I've worked at a number of companies that are structured a lot like Catalyst is structured. Very focused, not a huge organization. One of the things we do is we take our internal organization of five or six people who are totally committed to the business development end of it, and we leverage external providers who can bring more to the table. Because as you know, it's episodic. Even though we've evaluated more than 100 since the beginning of the year, the peaks and troughs are there as well. We want to bring those folks in when we get really far along and get expertise, supplement the expertise that we have. China is not out of our bailiwick. We like the opportunity. I've worked at a number of companies that are structured a lot like Catalyst is structured. i've worked at a number of companies that are structured a lot like catalyst is structured Very focused, not a huge organization. very focused not a huge organization One of the things we do is we take our internal organization of five or six people who are totally committed to the business development end of it, and we leverage external providers who can bring more to the table. one of the things we do is we take our internal organization of five or six people who are totally committed to the business development end of it and we leverage external providers who can bring more to the table Because as you know, it's episodic. because as you know it's episodic Even though we've evaluated more than 100 since the beginning of the year, the peaks and troughs are there as well. even though we've evaluated more than 100 since the beginning of the year the peaks and troughs are there as well We want to bring those folks in when we get really far along and get expertise, supplement the expertise that we have. we want to bring those folks in when we get really far along and get expertise supplement the expertise that we have China is not out of our bailiwick. china is not out of our bailiwick We like the opportunity. we like the opportunity Any company that's not based here in the U.S. and looking to plant a flag, as I said earlier, we're interested in talking to those folks. So Asia, Europe, etc. So we're continuing to it, and we do have the bandwidth to do it. Any company that's not based here in the U.S. and looking to plant a flag, as I said earlier, we're interested in talking to those folks. any company that's not based here in the u.s and looking to plant a flag as i said earlier we're interested in talking to those folks So Asia, Europe, etc. So we're continuing to it, and we do have the bandwidth to do it. so asia europe etc so we're continuing to it and we do have the bandwidth to do it

Speaker 5: Okay. Well, maybe we'll shift gears to Agamree, which has been an important new launch for you guys. And maybe just the latest in terms of how you're sourcing patients between Emflaza and prednisone. Has it changed or evolved? Has it exceeded expectation? Where are the patient sources coming from? Okay. okay Well, maybe we'll shift gears to Agamree, which has been an important new launch for you guys. well maybe we'll shift gears to agamree which has been an important new launch for you guys And maybe just the latest in terms of how you're sourcing patients between Emflaza and prednisone. and maybe just the latest in terms of how you're sourcing patients between emflaza and prednisone Has it changed or evolved? has it changed or evolved Has it exceeded expectation? has it exceeded expectation Where are the patient sources coming from? where are the patient sources coming from

Speaker 2: Sure. When we initially launched, or prior to the product, and initially launched the product, we expected that we would source mostly from Emflaza. What we're finding out is there's an incredible dissatisfaction with the standard of care out there. So we continue to source. I'm just going to give you the rough numbers, and it vacillates a percentage or two, depending on the month you're in, but 45% from Emflaza, 45% from prednisone, and about 10% are naive. So that continues from the launch up till now, which we think is an incredible sign that the product has great potential going forward. And there is a generic Emflaza on the market, and we're not affected by that. We have approvals in the almost 90% range. Sure. sure When we initially launched, or prior to the product, and initially launched the product, we expected that we would source mostly from Emflaza. when we initially launched or prior to the product and initially launched the product we expected that we would source mostly from emflaza What we're finding out is there's an incredible dissatisfaction with the standard of care out there. what we're finding out is there's an incredible dissatisfaction with the standard of care out there So we continue to source. so we continue to source I'm just going to give you the rough numbers, and it vacillates a percentage or two, depending on the month you're in, but 45% from Emflaza, 45% from prednisone, and about 10% are naive. i'm just going to give you the rough numbers and it vacillates a percentage or two depending on the month you're in but 45% from emflaza 45% from prednisone and about 10% are naive So that continues from the launch up till now, which we think is an incredible sign that the product has great potential going forward. so that continues from the launch up till now which we think is an incredible sign that the product has great potential going forward And there is a generic Emflaza on the market, and we're not affected by that. and there is a generic emflaza on the market and we're not affected by that We have approvals in the almost 90% range. we have approvals in the almost 90% range So the opportunity is really to look for those new patients or keep patients on if you have a superior product profile where patients will benefit. So the sourcing continues to be exactly what it was at launch, which speaks well to the product. So the opportunity is really to look for those new patients or keep patients on if you have a superior product profile where patients will benefit. so the opportunity is really to look for those new patients or keep patients on if you have a superior product profile where patients will benefit So the sourcing continues to be exactly what it was at launch, which speaks well to the product. so the sourcing continues to be exactly what it was at launch which speaks well to the product

Speaker 5: In just rough terms, this market, right, because it is capped by size factors, right? But I think Emflaza achieved peak penetration of around a third of the market, if I recall. In just rough terms, this market, right, because it is capped by size factors, right? in just rough terms this market right because it is capped by size factors right But I think Emflaza achieved peak penetration of around a third of the market, if I recall. but i think emflaza achieved peak penetration of around a third of the market if i recall

Speaker 2: It's hard to tell because nobody reports data here because of our proprietary pharmacies. That's what we believe. It was about a third of the market, yeah. It's hard to tell because nobody reports data here because of our proprietary pharmacies. it's hard to tell because nobody reports data here because of our proprietary pharmacies That's what we believe. that's what we believe It was about a third of the market, yeah. it was about a third of the market yeah

Speaker 5: Okay. So call it $250-ish sales at getting a third of the market. So said a different way, it could be a $750 million market for steroids, assuming gene therapy adoption didn't somehow diminish the utilization of steroids. Is that a way to think about the ceiling of this marketplace? Okay. okay So call it $250-ish sales at getting a third of the market. so call it $250-ish sales at getting a third of the market So said a different way, it could be a $750 million market for steroids, assuming gene therapy adoption didn't somehow diminish the utilization of steroids. so said a different way it could be a $750 million market for steroids assuming gene therapy adoption didn't somehow diminish the utilization of steroids Is that a way to think about the ceiling of this marketplace? is that a way to think about the ceiling of this marketplace

Speaker 2: I think there's a couple of ways to think about it. Number one, 95% of patients have been on a steroid, and only 70% are on today. And that speaks to the dissatisfaction with the therapy. Even for patients who are not ambulatory, the ability to more successfully operate their chair because of improved dexterity or maintained dexterity, or the ability to operate a mouse or a keyboard, both of these are the window on the world. So we think there's an opportunity with a better steroid to actually grow the market, albeit it's capped at 11,000-13,000, some estimates as high as 15,000 in the U.S. patients. So when you take the conservative estimates at our price, which is roughly equivalent to the price of branded Emflaza, we believe that the addressable market is actually above $1 billion. I think there's a couple of ways to think about it. i think there's a couple of ways to think about it Number one, 95% of patients have been on a steroid, and only 70% are on today. number one 95% of patients have been on a steroid and only 70% are on today And that speaks to the dissatisfaction with the therapy. and that speaks to the dissatisfaction with the therapy Even for patients who are not ambulatory, the ability to more successfully operate their chair because of improved dexterity or maintained dexterity, or the ability to operate a mouse or a keyboard, both of these are the window on the world. even for patients who are not ambulatory the ability to more successfully operate their chair because of improved dexterity or maintained dexterity or the ability to operate a mouse or a keyboard both of these are the window on the world So we think there's an opportunity with a better steroid to actually grow the market, albeit it's capped at 11,000-13,000, some estimates as high as 15,000 in the U.S. patients. so we think there's an opportunity with a better steroid to actually grow the market albeit it's capped at 11,000-13,000 some estimates as high as 15,000 in the u.s patients So when you take the conservative estimates at our price, which is roughly equivalent to the price of branded Emflaza, we believe that the addressable market is actually above $1 billion. so when you take the conservative estimates at our price which is roughly equivalent to the price of branded emflaza we believe that the addressable market is actually above $1 billion So if we can prove, and you look back at the GUARDIAN trial, which I referenced earlier, and the SUMMIT trial, which we're doing in the U.S., we can prove that those benefits do exist, we think we can get more than our fair share of the market. So if we can prove, and you look back at the GUARDIAN trial, which I referenced earlier, and the SUMMIT trial, which we're doing in the U.S., we can prove that those benefits do exist, we think we can get more than our fair share of the market. so if we can prove and you look back at the guardian trial which i referenced earlier and the summit trial which we're doing in the u.s we can prove that those benefits do exist we think we can get more than our fair share of the market

Speaker 5: How important really is SUMMIT to getting to some of these more bullish market outlooks in your mind versus ex-SUMMIT? I don't know. Do you feel like you still would be on a trajectory to having an Emflaza kind of peak sales profile? How important really is SUMMIT to getting to some of these more bullish market outlooks in your mind versus ex-SUMMIT? how important really is summit to getting to some of these more bullish market outlooks in your mind versus ex-summit I don't know. i don't know Do you feel like you still would be on a trajectory to having an Emflaza kind of peak sales profile? do you feel like you still would be on a trajectory to having an emflaza kind of peak sales profile

Speaker 2: It's a great question. And when we look at the GUARDIAN trial, it validates our faith in the SUMMIT trial. So here you have 110 patients. So we're five years looking at versus standard of care. And you see less vertebral fractures in the Agamree patients. You see stature, normal stature. So you know long-term steroids in adolescents can inhibit growth. And you see fewer cataracts. So if you have an older patient population, clearly they're not going to face growth issues, but you have cataract issues, bone health, etc. So I think SUMMIT's pretty important, and the GUARDIAN results give us a lot of faith that this is the right path to take. So to answer your question, yeah, it's pretty important. It's a great question. it's a great question And when we look at the GUARDIAN trial, it validates our faith in the SUMMIT trial. and when we look at the guardian trial it validates our faith in the summit trial So here you have 110 patients. so here you have 110 patients So we're five years looking at versus standard of care. so we're five years looking at versus standard of care And you see less vertebral fractures in the Agamree patients. and you see less vertebral fractures in the agamree patients You see stature, normal stature. you see stature normal stature So you know long-term steroids in adolescents can inhibit growth. so you know long-term steroids in adolescents can inhibit growth And you see fewer cataracts. and you see fewer cataracts So if you have an older patient population, clearly they're not going to face growth issues, but you have cataract issues, bone health, etc. So I think SUMMIT's pretty important, and the GUARDIAN results give us a lot of faith that this is the right path to take. so if you have an older patient population clearly they're not going to face growth issues but you have cataract issues bone health etc so i think summit's pretty important and the guardian results give us a lot of faith that this is the right path to take So to answer your question, yeah, it's pretty important. so to answer your question yeah it's pretty important

Speaker 5: And so those updates that we get from SUMMIT are going to be periodic, right, over time, and you do more follow-up. And so the first update will be sometime, can you remind me of that next year? And so those updates that we get from SUMMIT are going to be periodic, right, over time, and you do more follow-up. and so those updates that we get from summit are going to be periodic right over time and you do more follow-up And so the first update will be sometime, can you remind me of that next year? and so the first update will be sometime can you remind me of that next year

Speaker 2: We haven't said yet, so we're still enrolling. We're looking for 250 patients across about 30 or 35 sites, and we need to have those patients on for a significant enough period of time to see the benefit of the bone or see the benefit of the cataracts, so we believe there might be other benefits as well, such as behavior that we'll see pretty quickly if it's there, so we're looking for that, so we'll give updates as we continue to successfully enroll patients, and I want to focus that these are, in fact, patients, not subjects. These are patients who are on commercial drug, so they get a significant number of enhanced testing, and they get a significant benefit from participating, so we think it's a pretty important trial and focus a lot of effort there. We haven't said yet, so we're still enrolling. we haven't said yet so we're still enrolling We're looking for 250 patients across about 30 or 35 sites, and we need to have those patients on for a significant enough period of time to see the benefit of the bone or see the benefit of the cataracts, so we believe there might be other benefits as well, such as behavior that we'll see pretty quickly if it's there, so we're looking for that, so we'll give updates as we continue to successfully enroll patients, and I want to focus that these are, in fact, patients, not subjects. we're looking for 250 patients across about 30 or 35 sites and we need to have those patients on for a significant enough period of time to see the benefit of the bone or see the benefit of the cataracts so we believe there might be other benefits as well such as behavior that we'll see pretty quickly if it's there so we're looking for that so we'll give updates as we continue to successfully enroll patients and i want to focus that these are in fact patients not subjects These are patients who are on commercial drug, so they get a significant number of enhanced testing, and they get a significant benefit from participating, so we think it's a pretty important trial and focus a lot of effort there. these are patients who are on commercial drug so they get a significant number of enhanced testing and they get a significant benefit from participating so we think it's a pretty important trial and focus a lot of effort there

Speaker 5: Okay. And then how has the payer coverage sort of evolved? Do you feel like going into 2026 that this single generic step edit was going to be the more broadly applied kind of coverage policy type? Okay. okay And then how has the payer coverage sort of evolved? and then how has the payer coverage sort of evolved Do you feel like going into 2026 that this single generic step edit was going to be the more broadly applied kind of coverage policy type? do you feel like going into 2026 that this single generic step edit was going to be the more broadly applied kind of coverage policy type

Speaker 2: I'm sorry. Could you repeat the question, Jason? I'm sorry. I had a hard time hearing you. I'm sorry. i'm sorry Could you repeat the question, Jason? could you repeat the question jason I'm sorry. i'm sorry I had a hard time hearing you. i had a hard time hearing you

Speaker 5: Sorry. Just thinking about sort of the payer coverage, right, going into 2026, do you think that that's going to be primarily this single generic step edit that's been, I guess, in my mind, the predominant type of policy is step through one generic only, not two? Sorry. sorry Just thinking about sort of the payer coverage, right, going into 2026, do you think that that's going to be primarily this single generic step edit that's been, I guess, in my mind, the predominant type of policy is step through one generic only, not two? just thinking about sort of the payer coverage right going into 2026 do you think that that's going to be primarily this single generic step edit that's been i guess in my mind the predominant type of policy is step through one generic only not two

Speaker 2: Oh, yeah. So right now, the majority of patients are on a generic. They're on a generic prednisone. And those that are on Emflaza are not going to go back. It doesn't make sense for an insurance company to say, "Go back to generic Emflaza if it didn't work." So we think there are two elements to this. Number one is that we're getting, almost, as I said, 85%, almost 90% of the prescriptions approved. And when we first launched, it was probably about 30 days from the time we submitted the prescription until it was approved. So we give those patients bridge drug, right, to make sure that they get on therapy as fast as possible. Now it's down to less than two weeks. Oh, yeah. oh yeah So right now, the majority of patients are on a generic. so right now the majority of patients are on a generic They're on a generic prednisone. they're on a generic prednisone And those that are on Emflaza are not going to go back. and those that are on emflaza are not going to go back It doesn't make sense for an insurance company to say, "Go back to generic Emflaza if it didn't work." So we think there are two elements to this. it doesn't make sense for an insurance company to say "go back to generic emflaza if it didn't work." so we think there are two elements to this Number one is that we're getting, almost, as I said, 85%, almost 90% of the prescriptions approved. number one is that we're getting almost as i said 85% almost 90% of the prescriptions approved And when we first launched, it was probably about 30 days from the time we submitted the prescription until it was approved. and when we first launched it was probably about 30 days from the time we submitted the prescription until it was approved So we give those patients bridge drug, right, to make sure that they get on therapy as fast as possible. so we give those patients bridge drug right to make sure that they get on therapy as fast as possible Now it's down to less than two weeks. now it's down to less than two weeks So I would see that continuing to evolve as more payers get more comfortable with the profile of the product and the use of the product. So I would see that continuing to evolve as more payers get more comfortable with the profile of the product and the use of the product. so i would see that continuing to evolve as more payers get more comfortable with the profile of the product and the use of the product

Speaker 5: Okay. And then is there anything you can speak to more real-time in terms of interplay with gene therapy rollout, how that's impacting steroid utilization, if at all? Okay. okay And then is there anything you can speak to more real-time in terms of interplay with gene therapy rollout, how that's impacting steroid utilization, if at all? and then is there anything you can speak to more real-time in terms of interplay with gene therapy rollout how that's impacting steroid utilization if at all

Speaker 2: Yeah, so I would say that the market itself, so remember that steroids are foundational therapy. It's the drug you get on first before you get on anything else. Patients that are on steroid therapy walk two, two and a half, sometimes three years longer than patients who don't go on to steroids. So we're not competing with gene therapy, but the choppiness of this water because of all the things that have happened, the performance of the product in the face of that choppiness, I think, is outstanding. So we're looking for it to settle out. We would love for, as everybody would, for there to be no more challenges with gene therapy and sort of get on a steady state, and so we've had what we call a queuing effect, so patients that are on a steroid are less likely to change if they're waiting for gene therapy. Yeah, so I would say that the market itself, so remember that steroids are foundational therapy. yeah so i would say that the market itself so remember that steroids are foundational therapy It's the drug you get on first before you get on anything else. it's the drug you get on first before you get on anything else Patients that are on steroid therapy walk two, two and a half, sometimes three years longer than patients who don't go on to steroids. patients that are on steroid therapy walk two two and a half sometimes three years longer than patients who don't go on to steroids So we're not competing with gene therapy, but the choppiness of this water because of all the things that have happened, the performance of the product in the face of that choppiness, I think, is outstanding. so we're not competing with gene therapy but the choppiness of this water because of all the things that have happened the performance of the product in the face of that choppiness i think is outstanding So we're looking for it to settle out. so we're looking for it to settle out We would love for, as everybody would, for there to be no more challenges with gene therapy and sort of get on a steady state, and so we've had what we call a queuing effect, so patients that are on a steroid are less likely to change if they're waiting for gene therapy. we would love for as everybody would for there to be no more challenges with gene therapy and sort of get on a steady state and so we've had what we call a queuing effect so patients that are on a steroid are less likely to change if they're waiting for gene therapy So it almost creates a line, if you will. The challenge with this scenario in the marketplace is that we saw the label expand for gene therapy, then we saw it contract. And so there's just a lot of confusion. And as that settles down, it gives us a stronger share of voice in the market because we can actually talk about the benefits of therapy with Agamree. So we're hoping it stays steady state. So it almost creates a line, if you will. so it almost creates a line if you will The challenge with this scenario in the marketplace is that we saw the label expand for gene therapy, then we saw it contract. the challenge with this scenario in the marketplace is that we saw the label expand for gene therapy then we saw it contract And so there's just a lot of confusion. and so there's just a lot of confusion And as that settles down, it gives us a stronger share of voice in the market because we can actually talk about the benefits of therapy with Agamree. and as that settles down it gives us a stronger share of voice in the market because we can actually talk about the benefits of therapy with agamree So we're hoping it stays steady state. so we're hoping it stays steady state

Speaker 5: How are you guys thinking about pre-announcing around the time of J.P. Morgan, start of the new year for your business? Do you feel like you have enough kind of visibility into these components of the business that this would make sense for you guys to sort of pre-announce revenue for your business lines? How are you guys thinking about pre-announcing around the time of J.P. Morgan, start of the new year for your business? how are you guys thinking about pre-announcing around the time of j.p. morgan start of the new year for your business Do you feel like you have enough kind of visibility into these components of the business that this would make sense for you guys to sort of pre-announce revenue for your business lines? do you feel like you have enough kind of visibility into these components of the business that this would make sense for you guys to sort of pre-announce revenue for your business lines

Speaker 2: So we may do it at a high level. I think what we want to do is we want to take a look at the markets and be sure that we have a really good understanding. Obviously, at this point in the year, your forecasting is complete, but you just want to be as appropriately cautious as possible. So last year, we announced that our earnings call for the end-of-year earnings call. And that's our plan for right now. So we would want to make sure that we have a really good understanding of how the markets are trending when we do, in fact, give guidance. So we may do it at a high level. so we may do it at a high level I think what we want to do is we want to take a look at the markets and be sure that we have a really good understanding. i think what we want to do is we want to take a look at the markets and be sure that we have a really good understanding Obviously, at this point in the year, your forecasting is complete, but you just want to be as appropriately cautious as possible. obviously at this point in the year your forecasting is complete but you just want to be as appropriately cautious as possible So last year, we announced that our earnings call for the end-of-year earnings call. so last year we announced that our earnings call for the end-of-year earnings call And that's our plan for right now. and that's our plan for right now So we would want to make sure that we have a really good understanding of how the markets are trending when we do, in fact, give guidance. so we would want to make sure that we have a really good understanding of how the markets are trending when we do in fact give guidance

Speaker 5: Okay. Anything else that we haven't hit on with respect to this topic? I don't know if you've actually even sort of shared any of the orphan indication opportunities that are potentially of interest to you or potentially any sort of directional sizing indicators for what those expansionary opportunities could be as a Agamree follow-on indications if you can't be that specific at the moment on maybe what you're looking at. Okay. okay Anything else that we haven't hit on with respect to this topic? anything else that we haven't hit on with respect to this topic I don't know if you've actually even sort of shared any of the orphan indication opportunities that are potentially of interest to you or potentially any sort of directional sizing indicators for what those expansionary opportunities could be as a Agamree follow-on indications if you can't be that specific at the moment on maybe what you're looking at. i don't know if you've actually even sort of shared any of the orphan indication opportunities that are potentially of interest to you or potentially any sort of directional sizing indicators for what those expansionary opportunities could be as a agamree follow-on indications if you can't be that specific at the moment on maybe what you're looking at

Speaker 2: Yeah. Just at a high level, we would look for those rare conditions that require long-term steroid use and have the largest patient populations where a change in steroids is meaningful to the patient or to the provider, hopefully to both. But we're not in a position right now. We'd really like to see those data from the Phase I to have a better understanding. And that way, I think we can go forward with impunity. Yeah. yeah Just at a high level, we would look for those rare conditions that require long-term steroid use and have the largest patient populations where a change in steroids is meaningful to the patient or to the provider, hopefully to both. just at a high level we would look for those rare conditions that require long-term steroid use and have the largest patient populations where a change in steroids is meaningful to the patient or to the provider hopefully to both But we're not in a position right now. but we're not in a position right now We'd really like to see those data from the Phase I to have a better understanding. we'd really like to see those data from the phase i to have a better understanding And that way, I think we can go forward with impunity. and that way i think we can go forward with impunity

Speaker 5: I mean, with the pricing paradigm that you have with this, I imagine that does the utilization need to be in sensitive populations like pediatric or I would imagine there are very large market opportunities where steroids are used, right? But trying to narrow it down, is some of this population sensitivity? You probably want to stay in rare orphan, right? You want a sensitive population so that you can maintain the pricing structure. So I'm just kind of curious. Am I thinking about that the right way? I mean, with the pricing paradigm that you have with this, I imagine that does the utilization need to be in sensitive populations like pediatric or I would imagine there are very large market opportunities where steroids are used, right? i mean with the pricing paradigm that you have with this i imagine that does the utilization need to be in sensitive populations like pediatric or i would imagine there are very large market opportunities where steroids are used right But trying to narrow it down, is some of this population sensitivity? but trying to narrow it down is some of this population sensitivity You probably want to stay in rare orphan, right? you probably want to stay in rare orphan right You want a sensitive population so that you can maintain the pricing structure. you want a sensitive population so that you can maintain the pricing structure So I'm just kind of curious. so i'm just kind of curious Am I thinking about that the right way? am i thinking about that the right way

Speaker 2: You are so rare orphan is where we want to focus and where the patient can gain the best benefit. So as we discussed earlier in this call, older patients won't have the benefit. They don't have the benefit for stature. So if there's an opportunity to provide a pediatric patient with that opportunity to grow like their peers, that's a great opportunity for us. Older patients are going to be worried about bone strength. You just reduced vertebral fractures for long-term steroid patients. That's critically important. Cataracts, that's critically important too. So the ancillary cost that you would experience or maybe avoid if the drug actually does have the profile we believe it has, I think could create tremendous value in these rare and orphan markets. You are so rare orphan is where we want to focus and where the patient can gain the best benefit. you are so rare orphan is where we want to focus and where the patient can gain the best benefit So as we discussed earlier in this call, older patients won't have the benefit. so as we discussed earlier in this call older patients won't have the benefit They don't have the benefit for stature. they don't have the benefit for stature So if there's an opportunity to provide a pediatric patient with that opportunity to grow like their peers, that's a great opportunity for us. so if there's an opportunity to provide a pediatric patient with that opportunity to grow like their peers that's a great opportunity for us Older patients are going to be worried about bone strength. older patients are going to be worried about bone strength You just reduced vertebral fractures for long-term steroid patients. you just reduced vertebral fractures for long-term steroid patients That's critically important. that's critically important Cataracts, that's critically important too. cataracts that's critically important too So the ancillary cost that you would experience or maybe avoid if the drug actually does have the profile we believe it has, I think could create tremendous value in these rare and orphan markets. so the ancillary cost that you would experience or maybe avoid if the drug actually does have the profile we believe it has i think could create tremendous value in these rare and orphan markets

Speaker 5: Okay. Makes sense. Maybe shifting gears to Firdapse, and I actually have just a couple of mechanical questions. As we think about just the different scenarios around IP litigation, I just want to make sure I've got this right. So you have a sort of tail terminal patent that covers certain aspects of the composition, whether they do or do not have sort of this impurity that you've identified in your patent. And I believe, from what I understand historically, it's very difficult to not have that impurity in the product, right? And that is something that's patented. So is that sort of the anchor patent as you kind of look at this and what underpins the confidence kind of going into whatever patent litigation could entail? Okay. okay Makes sense. makes sense Maybe shifting gears to Firdapse, and I actually have just a couple of mechanical questions. maybe shifting gears to firdapse and i actually have just a couple of mechanical questions As we think about just the different scenarios around IP litigation, I just want to make sure I've got this right. as we think about just the different scenarios around ip litigation i just want to make sure i've got this right So you have a sort of tail terminal patent that covers certain aspects of the composition, whether they do or do not have sort of this impurity that you've identified in your patent. so you have a sort of tail terminal patent that covers certain aspects of the composition whether they do or do not have sort of this impurity that you've identified in your patent And I believe, from what I understand historically, it's very difficult to not have that impurity in the product, right? and i believe from what i understand historically it's very difficult to not have that impurity in the product right And that is something that's patented. and that is something that's patented So is that sort of the anchor patent as you kind of look at this and what underpins the confidence kind of going into whatever patent litigation could entail? so is that sort of the anchor patent as you kind of look at this and what underpins the confidence kind of going into whatever patent litigation could entail

Speaker 2: Right. So we're not getting into too many specifics. So we have two patent families here. And for somebody to actually win, they would have to win on every single patent. So any patent they violate or if the defendant is found to be in violation of it, that's a win for us. So our patent estate is robust. And so we believe our patents are robust, and we're going to defend them to the very end. Obviously, we want to do what's right for the business and get back to the business of the business, but not at the price of sacrificing future value. And we believe that the Teva and the Lupin agreements are very fine agreements for all parties. And so we like that structure. Right. right So we're not getting into too many specifics. so we're not getting into too many specifics So we have two patent families here. so we have two patent families here And for somebody to actually win, they would have to win on every single patent. and for somebody to actually win they would have to win on every single patent So any patent they violate or if the defendant is found to be in violation of it, that's a win for us. so any patent they violate or if the defendant is found to be in violation of it that's a win for us So our patent estate is robust. so our patent estate is robust And so we believe our patents are robust, and we're going to defend them to the very end. and so we believe our patents are robust and we're going to defend them to the very end Obviously, we want to do what's right for the business and get back to the business of the business, but not at the price of sacrificing future value. obviously we want to do what's right for the business and get back to the business of the business but not at the price of sacrificing future value And we believe that the Teva and the Lupin agreements are very fine agreements for all parties. and we believe that the teva and the lupin agreements are very fine agreements for all parties And so we like that structure. and so we like that structure

Speaker 5: Understood, and you have a BioMarin royalty. That rolls off this year, if I understand correctly? Understood, and you have a BioMarin royalty. understood and you have a biomarin royalty That rolls off this year, if I understand correctly? that rolls off this year if i understand correctly

Speaker 2: Yeah. So as we talked about in our Q3 10-Q, third quarter 10-Q, the BioMarin royalty on Firdapse ends in January 2026. And based on the midpoint guidance, the vast majority of which is U.S., our royalty obligation is about $30 million-$35 million. And all of this is in our Q, I believe, footnote 13. And so that reduction in royalty goes straight to pre-tax income and will contribute to operating leverage. So that's a big win for the company. Yeah. yeah So as we talked about in our Q3 10-Q, third quarter 10-Q, the BioMarin royalty on Firdapse ends in January 2026. so as we talked about in our q3 10-q third quarter 10-q the biomarin royalty on firdapse ends in january 2026 And based on the midpoint guidance, the vast majority of which is U.S., our royalty obligation is about $30 million-$35 million. and based on the midpoint guidance the vast majority of which is u.s our royalty obligation is about $30 million-$35 million And all of this is in our Q, I believe, footnote 13. and all of this is in our q i believe footnote 13 And so that reduction in royalty goes straight to pre-tax income and will contribute to operating leverage. and so that reduction in royalty goes straight to pre-tax income and will contribute to operating leverage So that's a big win for the company. so that's a big win for the company

Speaker 5: Okay. Pavan got some questions. Okay. okay Pavan got some questions. pavan got some questions Yeah. I guess just in terms of the uptake in Firdapse LEMS patients, I think you've previously estimated that there are upwards of 900 tumor LEMS patients, and only 3%-4% of those are ever diagnosed. So this is the growth segment of the LEMS patient population. Maybe if you can update us on the traction that you're seeing in those thoracic oncology centers and whether the diagnostic code implementation has improved materially to aid in the identification of those patients? Yeah. yeah I guess just in terms of the uptake in Firdapse LEMS patients, I think you've previously estimated that there are upwards of 900 tumor LEMS patients, and only 3%- 4% of those are ever diagnosed. i guess just in terms of the uptake in firdapse lems patients i think you've previously estimated that there are upwards of 900 tumor lems patients and only 3%- 4% of those are ever diagnosed So this is the growth segment of the LEMS patient population. so this is the growth segment of the lems patient population Maybe if you can update us on the traction that you're seeing in those thoracic oncology centers and whether the diagnostic code implementation has improved materially to aid in the identification of those patients? maybe if you can update us on the traction that you're seeing in those thoracic oncology centers and whether the diagnostic code implementation has improved materially to aid in the identification of those patients

Speaker 2: Sure, so when we think about the cancer-associated LEMS part of it, remember there are two parts of the business, and it's split about 50/50 patients, there's the idiopathic or non-cancer LEMS and the cancer LEMS, and so the traction we're experiencing right now really goes back to, goes back actually a couple of years when we were making the change on the NCCN guidelines and recommending change, but the success we've had in this year of streamlining the VGCC test, so there's a test that's very indicative of whether or not a patient would actually have LEMS, so we got that testing centralized, and now there's blood pickup in the office where the patient is. Previously, the patient had to go out, come in, so the traction we're getting is building on these first steps. Sure, so when we think about the cancer-associated LEMS part of it, remember there are two parts of the business, and it's split about 50/50 patients, there's the idiopathic or non-cancer LEMS and the cancer LEMS, and so the traction we're experiencing right now really goes back to, goes back actually a couple of years when we were making the change on the NCCN guidelines and recommending change, but the success we've had in this year of streamlining the VGCC test, so there's a test that's very indicative of whether or not a patient would actually have LEMS, so we got that testing centralized, and now there's blood pickup in the office where the patient is. sure so when we think about the cancer-associated lems part of it remember there are two parts of the business and it's split about 50/50 patients there's the idiopathic or non-cancer lems and the cancer lems and so the traction we're experiencing right now really goes back to goes back actually a couple of years when we were making the change on the nccn guidelines and recommending change but the success we've had in this year of streamlining the vgcc test so there's a test that's very indicative of whether or not a patient would actually have lems so we got that testing centralized and now there's blood pickup in the office where the patient is Previously, the patient had to go out, come in, so the traction we're getting is building on these first steps. previously the patient had to go out come in so the traction we're getting is building on these first steps Then we got the NCCN guidelines in July, which is a huge win for patients and a huge win for the company. And now we're working with these large, high-control oncology practices to embed the change in NCCN guideline into the care pathway. This is a really critical step. So looking again, high volumes, high control. And then from there, working with these accounts to then help train the physician that when they see a small cell lung cancer patient, they should, in fact, do a VGCC test first. They may partner with a neurologist for a definitive diagnosis, or they may feel comfortable doing it themselves. The recommendation is to work with a neurologist. The NCCN guideline recommendation is to work with a neurologist. Then we got the NCCN guidelines in July, which is a huge win for patients and a huge win for the company. then we got the nccn guidelines in july which is a huge win for patients and a huge win for the company And now we're working with these large, high-control oncology practices to embed the change in NCCN guideline into the care pathway. and now we're working with these large high-control oncology practices to embed the change in nccn guideline into the care pathway This is a really critical step. this is a really critical step So looking again, high volumes, high control. so looking again high volumes high control And then from there, working with these accounts to then help train the physician that when they see a small cell lung cancer patient, they should, in fact, do a VGCC test first. and then from there working with these accounts to then help train the physician that when they see a small cell lung cancer patient they should in fact do a vgcc test first They may partner with a neurologist for a definitive diagnosis, or they may feel comfortable doing it themselves. they may partner with a neurologist for a definitive diagnosis or they may feel comfortable doing it themselves The recommendation is to work with a neurologist. the recommendation is to work with a neurologist The NCCN guideline recommendation is to work with a neurologist. the nccn guideline recommendation is to work with a neurologist So the traction we're getting is the receptivity right now from the oncology practices that are saying, "Yeah, this looks very interesting." We don't anticipate significant impact for the cancer LEMS in the first half of next year because there are many of these practices. It takes a significant amount of effort to get in there and actually help them understand the change in NCCN guidelines and then what they have to do for the change in their care pathways. We have dedicated resources in the field that are looking at this and working with these accounts. And I think the traction right now is quite positive. So the traction we're getting is the receptivity right now from the oncology practices that are saying, "Yeah, this looks very interesting." We don't anticipate significant impact for the cancer LEMS in the first half of next year because there are many of these practices. so the traction we're getting is the receptivity right now from the oncology practices that are saying "yeah this looks very interesting." we don't anticipate significant impact for the cancer lems in the first half of next year because there are many of these practices It takes a significant amount of effort to get in there and actually help them understand the change in NCCN guidelines and then what they have to do for the change in their care pathways. it takes a significant amount of effort to get in there and actually help them understand the change in nccn guidelines and then what they have to do for the change in their care pathways We have dedicated resources in the field that are looking at this and working with these accounts. we have dedicated resources in the field that are looking at this and working with these accounts And I think the traction right now is quite positive. and i think the traction right now is quite positive And can you remind us what's the pool of patients that you've identified for treatment with Firdapse? And how has the composition of that pool evolved over time in terms of the split of idiopathic versus oncology LEMS patients? And can you remind us what's the pool of patients that you've identified for treatment with Firdapse? and can you remind us what's the pool of patients that you've identified for treatment with firdapse And how has the composition of that pool evolved over time in terms of the split of idiopathic versus oncology LEMS patients? and how has the composition of that pool evolved over time in terms of the split of idiopathic versus oncology lems patients So the pool we keep is about 500, and we use data and machine learning to look for those patients who've gone through a certain series of events in their diagnostic journey. And then we hyper-prioritize to those who are furthest along. And so we're seeing about 50% of our patients in any quarter come from that pool, either they're idiopathic or they're cancer. Your question is really difficult to answer because on the split, it's typically about, at any point in time, it's typically about 80/20, with 80% being idiopathic. That's why we think there's such a nice upside here. The reason why it's difficult to answer when they come in is because LEMS is sometimes the first indication that a patient has small cell lung cancer. So they come in with idiopathic LEMS, but over time, they end up with the diagnosis of small cell lung cancer. So the pool we keep is about 500, and we use data and machine learning to look for those patients who've gone through a certain series of events in their diagnostic journey. so the pool we keep is about 500 and we use data and machine learning to look for those patients who've gone through a certain series of events in their diagnostic journey And then we hyper-prioritize to those who are furthest along. and then we hyper-prioritize to those who are furthest along And so we're seeing about 50% of our patients in any quarter come from that pool, either they're idiopathic or they're cancer. and so we're seeing about 50% of our patients in any quarter come from that pool either they're idiopathic or they're cancer Your question is really difficult to answer because on the split, it's typically about, at any point in time, it's typically about 80/20, with 80% being idiopathic. your question is really difficult to answer because on the split it's typically about at any point in time it's typically about 80/20 with 80% being idiopathic That's why we think there's such a nice upside here. that's why we think there's such a nice upside here The reason why it's difficult to answer when they come in is because LEMS is sometimes the first indication that a patient has small cell lung cancer. the reason why it's difficult to answer when they come in is because lems is sometimes the first indication that a patient has small cell lung cancer So they come in with idiopathic LEMS, but over time, they end up with the diagnosis of small cell lung cancer. so they come in with idiopathic lems but over time they end up with the diagnosis of small cell lung cancer So it's not as clean as we would really like. But we are seeing, as an example, through the work that we've done, an increase in VGCC testing of 9% per quarter, quarter on quarter, which is really phenomenal. We think that's really exciting. And we're not really that embedded into the care pathways yet. So I think there's a lot of upside here. So it's not as clean as we would really like. so it's not as clean as we would really like But we are seeing, as an example, through the work that we've done, an increase in VGCC testing of 9% per quarter, quarter on quarter, which is really phenomenal. but we are seeing as an example through the work that we've done an increase in vgcc testing of 9% per quarter quarter on quarter which is really phenomenal We think that's really exciting. we think that's really exciting And we're not really that embedded into the care pathways yet. and we're not really that embedded into the care pathways yet So I think there's a lot of upside here. so i think there's a lot of upside here Yeah. And then I guess another layer of this is the missed diagnosis of LEMS with myasthenia gravis. And I think you've noted that about half of your patients have come from this pipeline of pool of patient opportunity. So maybe if you can talk about how the rate of converting these misdiagnosed MG patients to LEMS diagnosis has accelerated over the last 12 months and how much work is there left to be done here? Yeah. yeah And then I guess another layer of this is the missed diagnosis of LEMS with myasthenia gravis. and then i guess another layer of this is the missed diagnosis of lems with myasthenia gravis And I think you've noted that about half of your patients have come from this pipeline of pool of patient opportunity. and i think you've noted that about half of your patients have come from this pipeline of pool of patient opportunity So maybe if you can talk about how the rate of converting these misdiagnosed MG patients to LEMS diagnosis has accelerated over the last 12 months and how much work is there left to be done here? so maybe if you can talk about how the rate of converting these misdiagnosed mg patients to lems diagnosis has accelerated over the last 12 months and how much work is there left to be done here So this side of the business, the idiopathic side of the Firdapse business, is what we would call a prospecting business. You have to find these patients. They're hard to find. The average physician sees one in their entire career. So it's challenging. So we're following a similar path that we talked about on the oncology side. So obviously, we have this pool of patients we track, and through machine learning, we hyper-prioritize. And then our RAMs, our regional account managers, go in and prioritize again. Looking at the opportunity for growth, we think myasthenia gravis and all the great therapies that are out there for myasthenia gravis are excellent. And actually, this is a great investment that we actually don't have to make because there's a lot of DTC out there. And many of these patients will end up with what is called a seronegative myasthenia gravis test. So this side of the business, the idiopathic side of the Firdapse business, is what we would call a prospecting business. so this side of the business the idiopathic side of the firdapse business is what we would call a prospecting business You have to find these patients. you have to find these patients They're hard to find. they're hard to find The average physician sees one in their entire career. the average physician sees one in their entire career So it's challenging. so it's challenging So we're following a similar path that we talked about on the oncology side. so we're following a similar path that we talked about on the oncology side So obviously, we have this pool of patients we track, and through machine learning, we hyper-prioritize. so obviously we have this pool of patients we track and through machine learning we hyper-prioritize And then our RAMs, our regional account managers, go in and prioritize again. and then our rams our regional account managers go in and prioritize again Looking at the opportunity for growth, we think myasthenia gravis and all the great therapies that are out there for myasthenia gravis are excellent. looking at the opportunity for growth we think myasthenia gravis and all the great therapies that are out there for myasthenia gravis are excellent And actually, this is a great investment that we actually don't have to make because there's a lot of DTC out there. and actually this is a great investment that we actually don't have to make because there's a lot of dtc out there And many of these patients will end up with what is called a seronegative myasthenia gravis test. and many of these patients will end up with what is called a seronegative myasthenia gravis test And so we're doing the same thing with the national labs that we did on the cancer side, which is trying to get them to embed reflex testing. So if a patient comes up with seronegative for myasthenia gravis, so there's no antibodies there, reflex test them for VGCC. And we're getting a lot of traction there as well. So we believe that this will become potentially, if successful, less of a prospecting market and more concentrated in those high myasthenia gravis accounts. So that's what we're looking for. And then obviously, to grow the awareness through education, targeted education with those accounts. And that's our plan on both sides of the business. We think both have great growth potential. And so we're going to work both ends of the market. And so we're doing the same thing with the national labs that we did on the cancer side, which is trying to get them to embed reflex testing. and so we're doing the same thing with the national labs that we did on the cancer side which is trying to get them to embed reflex testing So if a patient comes up with seronegative for myasthenia gravis, so there's no antibodies there, reflex test them for VGCC. so if a patient comes up with seronegative for myasthenia gravis so there's no antibodies there reflex test them for vgcc And we're getting a lot of traction there as well. and we're getting a lot of traction there as well So we believe that this will become potentially, if successful, less of a prospecting market and more concentrated in those high myasthenia gravis accounts. so we believe that this will become potentially if successful less of a prospecting market and more concentrated in those high myasthenia gravis accounts So that's what we're looking for. so that's what we're looking for And then obviously, to grow the awareness through education, targeted education with those accounts. and then obviously to grow the awareness through education targeted education with those accounts And that's our plan on both sides of the business. and that's our plan on both sides of the business We think both have great growth potential. we think both have great growth potential And so we're going to work both ends of the market. and so we're going to work both ends of the market Yeah. I guess hitting on Fycompa really quickly, that's sort of an asset that's undergoing generic competition. So maybe if you can talk about the slope of that erosion. I know it's been a little less than initially expected. So is brand stickiness holding up in 4Q as well? And how do you expect that through 2026 onward? Yeah. yeah I guess hitting on Fycompa really quickly, that's sort of an asset that's undergoing generic competition. i guess hitting on fycompa really quickly that's sort of an asset that's undergoing generic competition So maybe if you can talk about the slope of that erosion. so maybe if you can talk about the slope of that erosion I know it's been a little less than initially expected. i know it's been a little less than initially expected So is brand stickiness holding up in 4Q as well? so is brand stickiness holding up in 4q as well And how do you expect that through 2026 onward? and how do you expect that through 2026 onward Sure. So it's a little early to tell about the fourth quarter, and let's just go back over and talk about the timeline here, so on the solid dose, there's two formulations of Fycompa. One is solid dose, and the other is suspension, oral suspension. What we've seen with the solid dose is there were two first filers in this market, and they could have come in in late May. Only one came in and came in in late June, so we got a nice maintenance of our business there, and the erosion, because only one is there, and the player, again, it goes back to Teva, is the most sophisticated player in this market. Teva came in with a really good price, market minus, so they looked at the market and said we're coming in with a 17% discount. Sure. sure So it's a little early to tell about the fourth quarter, and let's just go back over and talk about the timeline here, so on the solid dose, there's two formulations of Fycompa. so it's a little early to tell about the fourth quarter and let's just go back over and talk about the timeline here so on the solid dose there's two formulations of fycompa One is solid dose, and the other is suspension, oral suspension. one is solid dose and the other is suspension oral suspension What we've seen with the solid dose is there were two first filers in this market, and they could have come in in late May. what we've seen with the solid dose is there were two first filers in this market and they could have come in in late may Only one came in and came in in late June, so we got a nice maintenance of our business there, and the erosion, because only one is there, and the player, again, it goes back to Teva, is the most sophisticated player in this market. only one came in and came in in late june so we got a nice maintenance of our business there and the erosion because only one is there and the player again it goes back to teva is the most sophisticated player in this market Teva came in with a really good price, market minus, so they looked at the market and said we're coming in with a 17% discount. teva came in with a really good price market minus so they looked at the market and said we're coming in with a 17% discount There were other products that went generic in the epilepsy space around the same time that we did, but they had multiple players coming in. We did not experience that. So there's a lift from that. There is the stickiness that you always see with epilepsy patients. They want to have a drug that they can be sure is going to protect them from seizure. But we know that last week, two additional generics, solid dose, were approved. They are not yet on the market as far as we can tell. So they're not taking away from our Fycompa sales. And here we are in the 8th of December. So we think we can finish the year strong. And we took up guidance on this product. However, the oral suspension, we expect a single first filer to come in or to be approved in the middle of December. There were other products that went generic in the epilepsy space around the same time that we did, but they had multiple players coming in. there were other products that went generic in the epilepsy space around the same time that we did but they had multiple players coming in We did not experience that. we did not experience that So there's a lift from that. so there's a lift from that There is the stickiness that you always see with epilepsy patients. there is the stickiness that you always see with epilepsy patients They want to have a drug that they can be sure is going to protect them from seizure. they want to have a drug that they can be sure is going to protect them from seizure But we know that last week, two additional generics, solid dose, were approved. but we know that last week two additional generics solid dose were approved They are not yet on the market as far as we can tell. they are not yet on the market as far as we can tell So they're not taking away from our Fycompa sales. so they're not taking away from our fycompa sales And here we are in the 8th of December. and here we are in the 8th of december So we think we can finish the year strong. so we think we can finish the year strong And we took up guidance on this product. and we took up guidance on this product However, the oral suspension, we expect a single first filer to come in or to be approved in the middle of December. however the oral suspension we expect a single first filer to come in or to be approved in the middle of december And prior to the incursion of generics back in June, oral suspension was 15% of our business. So we're seeing a shift, and we expect to see a shift. So product has done really, really well so far. But we all know success of brands is inversely proportional to the number of generic players. And now we're going to be experiencing more generic players. What we're looking for is to be able to cover that loss of sales with growth on our other products. And that's what we're really pointing toward. And prior to the incursion of generics back in June, oral suspension was 15% of our business. and prior to the incursion of generics back in june oral suspension was 15% of our business So we're seeing a shift, and we expect to see a shift. so we're seeing a shift and we expect to see a shift So product has done really, really well so far. so product has done really really well so far But we all know success of brands is inversely proportional to the number of generic players. but we all know success of brands is inversely proportional to the number of generic players And now we're going to be experiencing more generic players. and now we're going to be experiencing more generic players What we're looking for is to be able to cover that loss of sales with growth on our other products. what we're looking for is to be able to cover that loss of sales with growth on our other products And that's what we're really pointing toward. and that's what we're really pointing toward And then in terms of a higher-level question with capital allocation, so the board recently authorized a $200 million share repurchase program. So should investors be thinking about this as a signal that maybe M&A valuations are too high, or is this just a signal that the balance sheet is really strong and then you have flexibility to do both buybacks and business development deals as you see fit at a given time? And how should we think about how you're thinking about the balance of capital towards buybacks versus BD in a given year? And then in terms of a higher-level question with capital allocation, so the board recently authorized a $200 million share repurchase program. and then in terms of a higher-level question with capital allocation so the board recently authorized a $200 million share repurchase program So should investors be thinking about this as a signal that maybe M&A valuations are too high, or is this just a signal that the balance sheet is really strong and then you have flexibility to do both buybacks and business development deals as you see fit at a given time? so should investors be thinking about this as a signal that maybe m&a valuations are too high or is this just a signal that the balance sheet is really strong and then you have flexibility to do both buybacks and business development deals as you see fit at a given time And how should we think about how you're thinking about the balance of capital towards buybacks versus BD in a given year? and how should we think about how you're thinking about the balance of capital towards buybacks versus bd in a given year Yeah. So excellent question. So we don't like our share price. And my joke is, if you ever met a CEO who did, you got the wrong CEO. And we think we have the opportunity to contribute value back to shareholders without hurting our opportunities and business development. So on a non-GAAP basis, we did $52 million and change in the third quarter. And so we believe we can continue to do that. And this share repurchase is over 15 months. So we are loaded. We are ready to go on business development. This share repurchase will not affect our ability. And again, we have no funded debt. So if there was an opportunity, as Jason and I were talking about earlier, if there's an opportunity to leverage on a great deal, we would take that opportunity. Yeah. yeah So excellent question. so excellent question So we don't like our share price. so we don't like our share price And my joke is, if you ever met a CEO who did, you got the wrong CEO. and my joke is if you ever met a ceo who did you got the wrong ceo And we think we have the opportunity to contribute value back to shareholders without hurting our opportunities and business development. and we think we have the opportunity to contribute value back to shareholders without hurting our opportunities and business development So on a non-GAAP basis, we did $52 million and change in the third quarter. so on a non-gaap basis we did $52 million and change in the third quarter And so we believe we can continue to do that. and so we believe we can continue to do that And this share repurchase is over 15 months. and this share repurchase is over 15 months So we are loaded. so we are loaded We are ready to go on business development. we are ready to go on business development This share repurchase will not affect our ability. this share repurchase will not affect our ability And again, we have no funded debt. and again we have no funded debt So if there was an opportunity, as Jason and I were talking about earlier, if there's an opportunity to leverage on a great deal, we would take that opportunity. so if there was an opportunity as jason and i were talking about earlier if there's an opportunity to leverage on a great deal we would take that opportunity We think this is a good way to return some value to shareholders while executing our business. We think this is a good way to return some value to shareholders while executing our business. we think this is a good way to return some value to shareholders while executing our business Then if I can go back to Agamree with the label expansion opportunities, I think this is something that's come up a bunch of times because it has an upside lever to a product right now in terms of the Street's forecast. Just trying to narrow down what you think are the most appealing or attractive opportunities, can you help us think through the framework that you're using for that? Then if I can go back to Agamree with the label expansion opportunities, I think this is something that's come up a bunch of times because it has an upside lever to a product right now in terms of the Street's forecast. then if i can go back to agamree with the label expansion opportunities i think this is something that's come up a bunch of times because it has an upside lever to a product right now in terms of the street's forecast Just trying to narrow down what you think are the most appealing or attractive opportunities, can you help us think through the framework that you're using for that? just trying to narrow down what you think are the most appealing or attractive opportunities can you help us think through the framework that you're using for that Sure. So when we think about the opportunity, we want those rare conditions. So it's got to be rare. We want it to be long-term and where there's a high need for a steroid. If it's episodic, it's less interesting to us. If it's continuous, and we have that profile that you see in the GUARDIAN trial where bone strength is there or growth or stature, avoidance of cataracts, etc., better behavior, we like those opportunities. So again, just focusing on the opportunity to create the greatest value for patients as quickly as possible. And we'll have a better handle on that after the new year to be able to pick the right target. Sure. sure So when we think about the opportunity, we want those rare conditions. so when we think about the opportunity we want those rare conditions So it's got to be rare. so it's got to be rare We want it to be long-term and where there's a high need for a steroid. we want it to be long-term and where there's a high need for a steroid If it's episodic, it's less interesting to us. if it's episodic it's less interesting to us If it's continuous, and we have that profile that you see in the GUARDIAN trial where bone strength is there or growth or stature, avoidance of cataracts, etc., better behavior, we like those opportunities. if it's continuous and we have that profile that you see in the guardian trial where bone strength is there or growth or stature avoidance of cataracts etc better behavior we like those opportunities So again, just focusing on the opportunity to create the greatest value for patients as quickly as possible. so again just focusing on the opportunity to create the greatest value for patients as quickly as possible And we'll have a better handle on that after the new year to be able to pick the right target. and we'll have a better handle on that after the new year to be able to pick the right target Then in terms of the IP outcome that could come post a potential bench trial that's scheduled in March 2026 for Firdapse, how should we be thinking about the bull bear scenario here? Assuming worst-case scenario, again, or if Firdapse goes LOE earlier than we expected, how should investors be thinking about such a scenario and the risk profile of the company in such a scenario? Then in terms of the IP outcome that could come post a potential bench trial that's scheduled in March 2026 for Firdapse, how should we be thinking about the bull bear scenario here? then in terms of the ip outcome that could come post a potential bench trial that's scheduled in march 2026 for firdapse how should we be thinking about the bull bear scenario here Assuming worst-case scenario, again, or if Firdapse goes LOE earlier than we expected, how should investors be thinking about such a scenario and the risk profile of the company in such a scenario? assuming worst-case scenario again or if firdapse goes loe earlier than we expected how should investors be thinking about such a scenario and the risk profile of the company in such a scenario So we really believe in our IP, and we're going to do everything we can to defend it. I think, again, settling with three out of the four defendants so far, I think, demonstrates a solid position for us. One of the most interesting things about the Firdapse market is we have already faced competition that was significantly price reduced in Ruzurgi. And Ruzurgi was out there at half the price of Firdapse. And the interesting thing is when we talked to, we just talked about the value of what's behind the front of the house. So the value is getting the patient onto the drug, keeping them on, and optimizing their dose for their benefit. Those systems, that infrastructure is so valuable to a company like ours. It can make a huge difference. So we really believe in our IP, and we're going to do everything we can to defend it. so we really believe in our ip and we're going to do everything we can to defend it I think, again, settling with three out of the four defendants so far, I think, demonstrates a solid position for us. i think again settling with three out of the four defendants so far i think demonstrates a solid position for us One of the most interesting things about the Firdapse market is we have already faced competition that was significantly price reduced in Ruzurgi. one of the most interesting things about the firdapse market is we have already faced competition that was significantly price reduced in ruzurgi And Ruzurgi was out there at half the price of Firdapse. and ruzurgi was out there at half the price of firdapse And the interesting thing is when we talked to, we just talked about the value of what's behind the front of the house. and the interesting thing is when we talked to we just talked about the value of what's behind the front of the house So the value is getting the patient onto the drug, keeping them on, and optimizing their dose for their benefit. so the value is getting the patient onto the drug keeping them on and optimizing their dose for their benefit Those systems, that infrastructure is so valuable to a company like ours. those systems that infrastructure is so valuable to a company like ours It can make a huge difference. it can make a huge difference Ruzurgi coming out at half the price, we actually didn't feel any significant dent to our business. We think that we have a pretty good system that can defend us going forward. Again, we have strong belief in our IP, and we look forward to resolving that in the most appropriate way possible for the benefit of the company and patients. Ruzurgi coming out at half the price, we actually didn't feel any significant dent to our business. ruzurgi coming out at half the price we actually didn't feel any significant dent to our business We think that we have a pretty good system that can defend us going forward. we think that we have a pretty good system that can defend us going forward Again, we have strong belief in our IP, and we look forward to resolving that in the most appropriate way possible for the benefit of the company and patients. again we have strong belief in our ip and we look forward to resolving that in the most appropriate way possible for the benefit of the company and patients Hey, Rich, I have a couple of follow-ups. So first, just on your rare orphan portfolio, how should we think about pricing power going forward? I know that these are expensive medications. And is there a certain cap, 2%, 3%, given, I don't know, with plans? These can be needle-moving, or are they so small? Maybe there's a little more latitude with pricing power. And I know that there's some Medicaid exposure here, right? So there's price penalties. So just maybe help us think through the framework there. Hey, Rich, I have a couple of follow-ups. hey rich i have a couple of follow-ups So first, just on your rare orphan portfolio, how should we think about pricing power going forward? so first just on your rare orphan portfolio how should we think about pricing power going forward I know that these are expensive medications. i know that these are expensive medications And is there a certain cap, 2%, 3%, given, I don't know, with plans? and is there a certain cap 2% 3% given i don't know with plans These can be needle-moving, or are they so small? these can be needle-moving or are they so small Maybe there's a little more latitude with pricing power. maybe there's a little more latitude with pricing power And I know that there's some Medicaid exposure here, right? and i know that there's some medicaid exposure here right So there's price penalties. so there's price penalties So just maybe help us think through the framework there. so just maybe help us think through the framework there So we look at the opportunity. If there's an opportunity to increase price to cover our increased costs or anything like that, we look at that, and we measure against what the penalties, Jason, you talked about. And we're not going to do anything egregious that you can look at a company that's trying to drive the top line but ends up with less on the bottom line. And that's actually quite possible in these markets where you can actually take a price increase that's quite high and end up making less money. We don't like that, so we don't do that. So we're always going to look for that balance of what we think is the right thing to do based on the services that we provide, the value that the drug provides, and the market conditions, whether it's CPI-Urban, or whatever's going on. So we look at the opportunity. so we look at the opportunity If there's an opportunity to increase price to cover our increased costs or anything like that, we look at that, and we measure against what the penalties, Jason, you talked about. if there's an opportunity to increase price to cover our increased costs or anything like that we look at that and we measure against what the penalties jason you talked about And we're not going to do anything egregious that you can look at a company that's trying to drive the top line but ends up with less on the bottom line. and we're not going to do anything egregious that you can look at a company that's trying to drive the top line but ends up with less on the bottom line And that's actually quite possible in these markets where you can actually take a price increase that's quite high and end up making less money. and that's actually quite possible in these markets where you can actually take a price increase that's quite high and end up making less money We don't like that, so we don't do that. we don't like that so we don't do that So we're always going to look for that balance of what we think is the right thing to do based on the services that we provide, the value that the drug provides, and the market conditions, whether it's CPI-U rban, or whatever's going on. so we're always going to look for that balance of what we think is the right thing to do based on the services that we provide the value that the drug provides and the market conditions whether it's cpi-u rban or whatever's going on So we're always going to judge that in a multifactorial way. But the pricing power of old is just not there anymore, 10, 15 years ago. So we're always going to judge that in a multifactorial way. so we're always going to judge that in a multifactorial way But the pricing power of old is just not there anymore, 10, 15 years ago. but the pricing power of old is just not there anymore 10 15 years ago Yeah, and then with cancer LEMS, my understanding in past discussions with you guys is that this is not like a buy-and-bill opportunity with these community oncs, such that this could be a money-making endeavor for community oncologists, which could spur faster utilization. I know that some people have asked me about that, but I feel like in the past, you guys have said you don't expect this to be a buy-and-bill product with these oncologists. Yeah, and then with cancer LEMS, my understanding in past discussions with you guys is that this is not like a buy-and-bill opportunity with these community oncs, such that this could be a money-making endeavor for community oncologists, which could spur faster utilization. yeah and then with cancer lems my understanding in past discussions with you guys is that this is not like a buy-and-bill opportunity with these community oncs such that this could be a money-making endeavor for community oncologists which could spur faster utilization I know that some people have asked me about that, but I feel like in the past, you guys have said you don't expect this to be a buy-and-bill product with these oncologists. i know that some people have asked me about that but i feel like in the past you guys have said you don't expect this to be a buy-and-bill product with these oncologists So it could be, actually. So medical oncologists cover the cost of their practice through the reimbursement around medication, whether it's injected, infused, or oral. Many of these larger practices do, in fact, have in-house pharmacies that can distribute drug. And quite frankly, if these patients are coming in on an every three-week or four-week basis for their cancer therapy, having that care team centralized in one spot becomes critically important for the outcome. One of the things we didn't talk about is we think there's about 900, initially 900 new patients on the cancer LEMS side. Those cancer LEMS patients, they have typically small cell lung cancer. A small cell lung cancer patient lives about eight or nine months. Nobody knows why, but a patient with small cell lung cancer LEMS lives 17 months. So it could be, actually. so it could be actually So medical oncologists cover the cost of their practice through the reimbursement around medication, whether it's injected, infused, or oral. so medical oncologists cover the cost of their practice through the reimbursement around medication whether it's injected infused or oral Many of these larger practices do, in fact, have in-house pharmacies that can distribute drug. many of these larger practices do in fact have in-house pharmacies that can distribute drug And quite frankly, if these patients are coming in on an every three-week or four-week basis for their cancer therapy, having that care team centralized in one spot becomes critically important for the outcome. and quite frankly if these patients are coming in on an every three-week or four-week basis for their cancer therapy having that care team centralized in one spot becomes critically important for the outcome One of the things we didn't talk about is we think there's about 900, initially 900 new patients on the cancer LEMS side. one of the things we didn't talk about is we think there's about 900 initially 900 new patients on the cancer lems side Those cancer LEMS patients, they have typically small cell lung cancer. those cancer lems patients they have typically small cell lung cancer A small cell lung cancer patient lives about eight or nine months. a small cell lung cancer patient lives about eight or nine months Nobody knows why, but a patient with small cell lung cancer LEMS lives 17 months. nobody knows why but a patient with small cell lung cancer lems lives 17 months In our discussions with oncologists, they've said, "Well, I assume the patient would only live eight months or nine months. Now that I'm seeing them live a year and a half, I think about them differently. And I want their quality of life to be higher." With LEMS, Lambert-Eaton myasthenic syndrome, as the disease progresses, the patients lose the ability to walk, ambulate, or certain control of extremities. So if they can improve, the physician can improve the quality of life of the patient, that's something they're interested in. We do believe that some of the accounts will actually use the opportunity to distribute Firdapse in-house as a way to offset costs of their practice, overhead, malpractice insurance, etc. In our discussions with oncologists, they've said, "Well, I assume the patient would only live eight months or nine months. in our discussions with oncologists they've said "well i assume the patient would only live eight months or nine months Now that I'm seeing them live a year and a half, I think about them differently. now that i'm seeing them live a year and a half i think about them differently And I want their quality of life to be higher." With LEMS, Lambert-Eaton myasthenic syndrome, as the disease progresses, the patients lose the ability to walk, ambulate, or certain control of extremities. and i want their quality of life to be higher." with lems lambert-eaton myasthenic syndrome as the disease progresses the patients lose the ability to walk ambulate or certain control of extremities So if they can improve, the physician can improve the quality of life of the patient, that's something they're interested in. so if they can improve the physician can improve the quality of life of the patient that's something they're interested in We do believe that some of the accounts will actually use the opportunity to distribute Firdapse in-house as a way to offset costs of their practice, overhead, malpractice insurance, etc. we do believe that some of the accounts will actually use the opportunity to distribute firdapse in-house as a way to offset costs of their practice overhead malpractice insurance etc Yeah. Okay. That's helpful to know. Appreciate your time, Rich, as always. And this was really helpful. And thanks for the pitch today. Yeah. yeah Okay. okay That's helpful to know. that's helpful to know Appreciate your time, Rich, as always. appreciate your time rich as always And this was really helpful. and this was really helpful And thanks for the pitch today. and thanks for the pitch today Thank you so much. I really appreciate the opportunity. Yeah, anytime. Thanks very much. Okay. Take care. Thank you so much. thank you so much I really appreciate the opportunity. i really appreciate the opportunity Yeah, anytime. yeah anytime Thanks very much. thanks very much Okay. okay Take care. take care