CATALYST PHARMACEUTICALS, INC. — Call Transcript 2025

All right. Well, thanks, everyone, for coming to the panel for Catalyst Pharma. I'm very pleased to have right next to me Steve Miller, CSO and COO, and then Richard Daley, CEO. So thank you guys for making it up here. Maybe if we can begin with an overview of the company and any highlights from the first half of this year. Sure. So thank you very much for having us. We really appreciate it. So we're Catalyst is a buy and build company. We look for opportunities in the market and work with partner companies to bring products in. We have no R, very little D, and we really focus on the commercial elements of the business. And we look to reduce commercial risk through execution, but also manage our regulatory risk as well. So we actually did develop our first product Firdapse for Lambert Eaton myasthenic syndrome. And there's probably about three thousand five hundred to five thousand four hundred patients in The U. S. We are a U. S.-focused organization. We did develop that and launched it in 2019. Have had great success with the product in identifying patients and bringing that forward as an opportunity to help patients live better lives. We also licensed a product from Eisai for epilepsy, and this was pretty much a financial transaction. We did this a number of years ago actually bolster the balance sheet but also get diversity of income. We consider epilepsy to be more of a retail play and not something we want to be in for the long haul unless it's rare epilepsy. And our most recent acquisition is a GammaRay for Duchenne's muscular dystrophy. And we launched that in March and have had great success in a highly competitive space. And we really look forward to continuing the growth of the company through acquisitions and also lifecycle management of the products. Great. Well, wanted to start by talking about Firdapse, and specifically for the idiopathic side of LEMS. Can you talk about Firdapse's position in the treatment paradigm, the level of penetration in this patient population, and any challenges with diagnosis or getting patients on Sure. So LEMS is a really interesting therapeutic opportunity. It seems to be split about fiftyfifty between idiopathic or noncancer Lamberti myasthenic syndrome versus cancer associated LEMS. So on the idiopathic side, it's very much a prospecting market. We have to look for data in the marketplace that tells us where the patients are. So we do this in a compliant way, and we look for opportunities to identify how a patient might be misdiagnosed. And the number of misdiagnosis is myasthenia gravis. And so patients will end up getting therapy for myasthenia gravis and not responding, and we see an opportunity to help those patients. One of the things that we find really exciting is the increase in the number of myasthenia gravis therapies that are out there. We believe that this effort, on the part of other companies, way we classify it, is the best money we never spent. It will get an elevated number of patients diagnosed and unfortunately misdiagnosed. When the myasthenia gravis therapies don't work for those patients, we expect then those patients might roll over to where we are. Because the physician, the neurologist will see that this drug is not working, they'll and see that we have an opportunity to help those patients. So the addressable market there is about seventeen hundred patients to 2,002 patients. And we our penetration there is about 30% in the market. And so we see tremendous upside, not only on the idiopathic side, but also on the cancer associated LEMS side. Okay. And then in terms of growth drivers still for the idiopathic side, so you talked about the misdiagnosis of myasthenia gravis. But I guess, yeah, like how does that flow through to getting patients on deferred apps? And then what do you see driving further growth? So one of the things we do is we look for these markers for patients who might be misdiagnosed and might have LEMS. And we are actively keeping a pool of about 500 or more patients who are in their diagnostic journey. And when we see the markers through data market data, so data we get from IQVIA, etcetera, we prospect, we send our sales representatives in, and we have a team of about 16 people who cover the entire country. And they'll go and speak to the doctor and try and bring the doctor along in their therapeutic options. The average physician on the idiopathic side will see one patient in their entire career. So truly understanding what this looks like and how this might affect the patient is really important for the sales representative to actually be in there and talk to the doctor and help them along as they move along. So we really see about eighty percent of the physicians write only one prescription. So it's really important that we have that. So we continue to keep this pool of 500 patients. And every quarter, about 50% of our new patients come from this pool. So not only are we hyper categorizing these patients, but then we have a super list of patients that are very, very close. And we watch those patients very closely. And we've been very productive in bringing that forward. All right. And then how would you say the dynamics for idiopathic lens contrast with cancer associated lens? And then can you also talk about what the penetration looks like for that cancer associated population? It sounds like, you know, recently had NCCN guidelines inclusion, and then also antibody testing as So a the antibody testing, the VGCC testing applies to both pools, both sides of the patient population. And we recently did a deal with Quest Diagnostics for what we call frictionless testing. So prior to this, a patient would have to leave the doctor's office, go to a distant site, a Quest site, have a blood draw, and then they would get back the results of that blood draw and the test within maybe a month, month and a half. What we did was we cut a deal with Quest to do in office blood draw and reporting back to the doctor and to the patient what the results are. This pays huge dividends for getting the patient further along in their journey. So it becomes really important, on the cancer side. So the difference between the cancer and the idiopathic side, to your question, the cancer side's not a prospecting side. So both these pools are about the same. Both these patient populations are about the same size, but about nine hundred patients per year could qualify as LEMS patients. So it's about three percent of the small cell lung cancer population. And every year that's about nine hundred patients. The cancer associated LEMS patients live twice as long as a patient with small cell lung cancer that doesn't have LEMS. So the opportunity is to really work with the GPOs that manage over eighty percent of the oncologists in The U. S. So we have frictionless testing was the first step. NCCN guidelines, pretty much the go to source for how to treat a patient with cancer in the oncology space. And then to look to actually get out and contract with some of these organizations and get into their care pathways. So the physicians are generally judged pretty closely on how they adhere to the NCCN guidelines or the care pathways within each practice. So whereas the idiopathic side is prospecting, you have a much more concentrated center of patients, higher diagnosis per year unfortunately, they don't live as long a patient with idiopathic LEMS lives a normal life. So there are far fewer patients diagnosed every year. But nine hundred patients and because of the survival of those patients with cancer, you see about seventeen hundred patients in this space. And we believe the addressable market for both of these both sides of this business each one is about $600,000,000 And our penetration on this side is only about ten percent, because LEMS looks like a weak patient. It looks like a patient who has cancer. Looks like a patient undergoing chemotherapy. And if you do the VGCC testing and you confirm through a physical diagnosis, you can find these patients. And they actually would live a much better life. So they generally have weakness starting at the girdle, starting at the shoulder and the hip, and going outward. Whereas a myasthenia gravis patient, it goes the other way, right? So you want to be sure that you understand which patient you're dealing with. And can you remind us when you gained NCCN guidance inclusion, and then when you started implementing the frictionless antibody testing, just to give us a sense of just like where we are in the inflection point Sure. Of So we got we finalized the contract for frictionless testing about the May, June. We got NCCN guideline updates, which were very favorable for the LEMS patients at the July. However, back in May, betting that we would actually have a really good outcome with our lab discussions and the NCCN guidelines, we put dedicated resources in the field to work with the GPOs. So we've already begun that process of actually getting in their care pathways. So we're really excited about this. So this is all, I think, a momentum builder for us in actually 2026. Okay. And I've seen you've talked before about a 20% discontinuation rate on Firdapse. Can you talk about just what goes into that and if it is possible to bring that rate down? Sure. So we've recently done an analysis. So we see patients will discontinue. The number one reason why a patient discontinues is because they pass away. It's relatively benign, Steve. Relatively benign therapy. What are the primary? Primary safety indications are tingling of the hands and feet called paresthesia, and a little bit of GI distress in some patients. So relatively benign. What we see are patients who start at less than thirty milligrams per day have a higher likelihood of discontinuing. So obviously we can't do anything about patient passing away, but we can look at the opportunity. And so we've instituted a couple of things. Number one, we instituted a titration schedule when a new patient enrolls. Because by label, patients are supposed to titrate. But because a physician only sees one patient in their entire career, they're not really aware of what should happen. So we've instituted that as they enroll into our specialty pharmacy and into our hub. But we also now are reaching out pharmacist to physician, so health care provider to health care provider, as the patient comes on board. If the patient doesn't have a titration schedule or their initial dose is below thirty milligrams, pharmacist at our specialty pharmacy calls the doctor and says, I see hundreds of these patients in my career. Let me help you. And this is something we just recently instituted in June, And we're seeing really nice pickup on this. So again, we don't think this is going to fundamentally change the milligrams per day so much as it will change the persistency of the patient staying on therapy. Got it. And you started to touch on this with just updating the care pathways. But I was wondering if you could elaborate more in terms of where you stand with the current large group oncology practices and GPOs, perhaps any kind of target base and where you are in reaching that So we see this as a four step process. They called out frictionless testing, NCCN guidelines, getting on the care pathway, and then contracting with those large GPOs that can make a difference. So we are in the process of working with them to bring them up to speed on these recently improved NCCN guidelines. And then typically, a large group practice in oncology will have its own care pathway model on NCCN. Some use ASCO guidelines, but most use NCCN. It's the predominant one. So we have our dedicated field resources calling on the top of the house in GPOs to say, hey, here are the new guidelines. You really should implement these. And then as we have more and more success, we'll look to do contracting for minimal discounts in the oncology space to help drive the success of the brand and the success of therapy for patients. I was wondering for 2025 guidance of $355,000,000 to $360,000,000 per year for Firdapse, what are the assumptions going into this? How are year to date trends tracking against those assumptions? Then what is your confidence in reaching that over $1,000,000,000 addressable market opportunity? So the assumptions that went into the business were that we would we recent last year, we got an increase in the maximum dose for Firdapse from eighty milligram to one hundred milligram. Patients would march up that appropriately. Typically neurologists like to be conservative and leave what we call headroom for their therapy. So we would see a fair number of patients at eighty milligrams, but not even a high plurality. Once we got the one hundred milligram, we started seeing patients move closer to the one hundred. And so every cohort in our group actually increased its average daily dose as you got that. So you can see more comfort with the product. Major drivers were actually reassigning the sales force, and now we have a dedicated team. Prior to this, our team of 18 sales representatives called both Firdapse and a Gamri. And you can imagine where the shine was, where excitement was. The excitement is in the new product, GammaRay, which is a great product. The opportunity then is, you know, with a dedicated force to actually be really focused. Your mission every day is to, you know, help more patients on both sides of business. So we're really excited about that. The opportunity to reach $1,000,000,000 again, this is just the addressable market. If we take out the patients who don't have very many symptoms or the patients who have so many symptoms at the top end of the range that it might not benefit them, we end up with about 80% of the total market being addressable. And both sides, about 600,000,000 So we're pretty confident that this is attainable, especially I'm sure one of your questions is going to be about the IP around the product. We settled with two of the three first filers and four of the litigants so far three of the four litigants so far. And Steve can talk about that. But we have life till patent life if we went on the last case till? Yeah. If if the last litigant exits the litigation the same way the others did, we will have exclusivity to about February 2035. So one of the challenges you give a brand team is, you know, if you think that the average product has seven years of life and we've already had six plus, what would you do how would you invest in the product from a commercial perspective, from a promotion perspective if you knew you had ten more years of life? What would you do? And so this becomes a really great opportunity for brand teams. And now we have a dedicated brand team for idiopathic, a dedicated brand team for cancer because it's one molecule but two brands. Still the same brand name, but two different markets and two different ways of approaching the market. How would you invest? What would you do? And the team is appropriately aggressive in looking at opportunities to continue to grow the business. Okay. And just to provide more clarity on the IP situation, can you tell us like what is the status or timelines with the remaining litigants and then also the upcoming Markman hearing? Sure. The only remaining litigant is hetero. I really can't comment on what's going on specifically with regard to hetero. But you're correct. There is an upcoming Markman hearing on October 7. And the Markman hearing is the hearing where all of the where the the claims will be discussed and how they apply to each of the two litigants. And so one of the things that you see historically in patent litigation is that there's usually a lot of settlement activity around the time of the Markman hearing. Okay. Wanted to talk about a gamma ray for DMD. Can you talk about the differentiation versus existing corticosteroids and what has been driving conversion thus far? Sure. Think on the differentiation, Steve as the chief scientific officer should probably address that. Well, thank you, Rich. The defining features of a gamma ray relative to the other corticosteroids that are on the market is that there's a lot of published literature that shows that a gamma ray has superior bone health, bone growth, as well as stature for the patients. There is much less exhibiting of aggression in patients. There may also be less cataracts. And our hope is that there also will be better cardiovascular safety with the gamma relative to the other corticosteroids. The physicians who were participating in the clinical trials actually observed a number of those things in the patients that they were treating for the clinical trials and have been very enthusiastic and have been early prescribers for the product. Okay. And can you talk about perhaps access for a gamma ray? I think just given, like, the assumed lower cost of steroids, how is the gamma ray positioned in terms Sure. So before we launched the product, we did market research with 17 active decision makers and payers. We said, okay, here's the profile. Steve just laid out a profile. Potentially better behavior, bone health, bone and growth stature for the patient in cardiovascular. What should we do? How should we price this? Knowing that in February, the month before we launched, knowing that a generic Emflaza was coming. There's three players in the market. There's prednisone generic Emflaza, which was branded and now has a generic component. We asked these 17 payers, decision makers, what should we do? How would we price the product? And they said, if you come in just slightly below branded Emflaza, knowing a generic is coming, we won't block you. And think about the market. The market's very well established. Patients start on prednisone. It's the foundation. Steroids are the foundation of therapy. So they start on prednisone. If they're not happy, they may move to Emflaza. And Emflaza has been on the market for four or five years. We come in the market. There's no step through therapy at this point because the patients that want a gamma ray really have already gone through prednisone, Emflaza. No one's gonna send them back to generic Emflaza, so there's an opportunity for us to actually take advantage of this. And we are sourcing patients roughly 45, 45, 10. And this has been true since the launch of the product, which is amazing. Originally, we thought we'd be participating just in the Emflaza part of the market, and it would be a cannibalization. Forty five percent it changes a percent or two in any given month. Forty five percent of our patients come from prednisone, forty five percent of our patients come from Emflaza, and ten percent are naive. What that means is instead of participating in roughly 30% or 40% of the market, which is where we think Emflaza's market share was, we're participating in 100% of the market. So it's a huge opportunity for us. And the fact that that's been going on now for sixteen months is a very, very good sign. We do anticipate it will change over time. And we do anticipate that there'll be opportunities to continue to grow the product through life cycle management. And we're working on that right now. We're working on how to go about the life cycle management for the product. Do you see faster conversion from prednisone or generic Emflaza or naive? And is there a channel that you want to grow more in? I think over time, it'll be harder to get the naive patient. Okay. Because they're gonna start on a generic prednisone. Yeah. That's gonna happen. The game for us is can we be that next choice? Right? So the generic's gonna be there. Our average patient pays less than $2 a month for the product. So it's not from a patient perspective, it's not a price play. So they're looking at and saying, Hey, if I have the support of the company, I think there's good things that can happen here for me as a patient, given the profile that Steve laid out. I think over time, though, the goal to really look for the opportunity to be that second steroid in line. I mean, every patient should be on a steroid. Ninety five percent have been on a steroid and only seventy percent are currently on a steroid, which speaks to the gap. There's something wrong with the current options for the patient. If you can address the issues that Steve talked about, and you're just as good as the other players, I think you can improve quality of life. Remember, behavior is not just an issue for the young boy. Remember, all of these patients are boys. It's a if they misbehave, it's a problem for the family. They're in school. It's a problem for the classroom. So if they have better behavior, they progress emotionally, psychologically, and so does everyone else around them. So it's just a relief. And we see that coming back to us in the market. It's not in our label, but we see that coming back to us in the market from feedback from doctors. Okay. And can you talk about some of the assumptions going into that 100,000,000 to $110,000,000.20 25 guidance? How are year to date trends tracking against those assumptions? And then also what it takes to reach that over $1,000,000,000 addressable market opportunity? So just to address the addressable market this is the fundamental difference between Firdapse and a GammaRia. The addressable market for GammaRay is a billion 2. But there are four players in the market. And if you get your fair share, you get 300,000,000. If you're better, you should get an unfair share. Right? So Firdapse in its addressable market of $1,000,000,000 is all alone. I mean, so everybody's very excited about a gamma ray. We're excited about a gamma ray. But when you look at the potential for the product and you have you know you're sitting on potentially ten more years, Firdapse is very exciting molecule. Gamma is very exciting. So the assumptions that went into the forecast, we there are 100 centers of excellence, two fifty doctors write 80 to 90% of the prescriptions, And 45 of the centers write 90% of the prescriptions. So we want to be penetrated. So we have had use in ninety three of the centers. And now so we've gone broad. Now the goal is to go deeper. And you see the early adopters obviously going to the product, which is fantastic. But we want their colleagues then to see the benefit. And so working with those 100 centers is really where we're focused in on education and making sure that we have that reach and frequency that's appropriate in this market. And this is a very difficult market because of some of the challenges that we see with gene therapy. This is a really unfortunate situation and it creates a lot of noise around the therapy. And so we actually have this queuing effect where patients have to get an immunosuppressive dose of steroid. We do not have an immunosuppressive dose in our label, we can't be used there. And then so we see patients waiting. Now that there's been these issues in gene therapy market, there's a lot of consternation, and people are trying to figure out what's next and how to do this. As this settles out, and we hope it does for the sake of patients, we should be getting a little bit more traction in the market. Okay. And in terms of this queuing effect, like, yeah, have you seen this continue so far? Because it seems like some of the uncertainty may be resolving. Or how long do you think this could benefit in terms of like the duration of patients on the gamma ray? Sure. So the queuing effect, the name comes from getting in line, right? So if a patient is waiting for therapy, gene therapy, the patient may actually not want to, and the physician may not want to change their therapy. So they could be on generic prednisone, or they could be on Emflaza. And since they're going to use one of those two for the immunosuppressive dose, they say let's not make a change to the steroid. And so that's where we see the queuing effect. So we're sort of in line. But once they come out of that, we're seeing some patients actually post gene therapy go to drug like a gamma ray. The opportunity for us is to see if we can find an immunosuppressive dose. So we're working on that. And then make sure that we can be there when the patient wants to make a change, if they want to make a change. So there's a lot going on in the market right now, and it's really unfortunate for patients. So we're hoping that we you know, everybody can get past that. And right now, gene therapy, the label was expanded to include all patients, ambulatory and non ambulatory. And now it's not inclusive of non ambulatory patients. Our average age is about 12. And that's about the point at which a patient loses ambulation. So if those patients are no longer in the line, we think that there's upside for us there. Okay. And so you have the immunosuppressive study coming out like late twenty twenty five, early twenty twenty six. Correct. Can you just talk about what you're looking to see from the data? You know, what is the significance of this in terms of the utilization of the gene? Sure. So we're looking to see if we can find an immunosuppressive dose. There's no guarantee we will find an immunosuppressive dose for the drug. And if it does, then we will have if we do find it, we will have the opportunity potentially to participate in this run up to gene therapy. So that's what we're looking for in that. But again, it's research, you know, basic clinical development, and there's no guarantee. So we await the results. Okay. And then you also have the SUMMIT study going on. I was wondering if you could talk about the significance of this, and then when we can expect more data. Sure. Steve? Sure. The SUMMIT study is a study being conducted in patients who are on commercial or gamma ray. And we are looking for all of those safety signals that I previously mentioned. And we will be categorizing all of that data and watching it as it proceeds. The comparison group is actually going to be the natural history of DMD. And that's so that we can do a comparison to the natural history and look for statistically significant improvements in that safety information and then do a submission to our drug application to update the label to include specific safety information regarding those endpoints. Now the problem with doing those kinds of comparisons is every time you do that, you have to spend a little bit of alpha, which in a nutshell means that when you do that comparison, the next time you do the comparison, it's a little harder to achieve statistical significance. You need a slightly lower p value. And so we will be judicious in our choice of when we do those studies. And we're going to want to make sure that we watch the data for a while. It's going to take about a year to recruit enough patients because we have to have a reasonable size n and a large enough period in which to observe the change in the patients before we do that comparison. So it could be at least eighteen months or a couple years before we do the first comparison. Now the good news is it is open label, which means that we can watch those safety endpoints without doing the actual statistical comparison and publicly make that information available to physicians so that they can start to learn about the safety characteristics of the drug even earlier. And so we'll present that information from time to time at professional conferences. Okay. Got it. So in terms of being able to get this data on the label, like is waiting for this still kind of a gating factor for certain physicians? It depends on the physician. As I mentioned, there were some early adopters who participated in the trials, and they recognized the advantages of the drug immediately just by observation of their own patients. There are some physicians who read the literature and are aware of it, and there are some who are strictly I'm just going to do what the answer sheet says. And so the short answer to your question is it would be helpful. Okay. Good. I wanted to talk about Fycompa and just what that cadence of generic erosion could look like this year. Since you've already achieved 70,000,000 in the '5, how do we get to 2025 guidance of 90,000,000 to $95,000,000 Sure. So in our most recent call, we announced our performance in the first half is $70,000,000 on guidance of 90,000,000 to 95,000,000 as you mentioned. So we got a significant amount of push, as you can imagine. What are you expecting? And really, we were expecting our assumptions going into this were there were two first filers, and they would be good actors in the market. And so Teva was one of the first filers, and they actually did get approval, and they launched. But they launched a month late, month later than we thought. They came to commercial. They had commercial presence. So the second first filer has yet to launch. And so we wait, and we want to be prudent in our guidance. But we know that two more will come in November. Okay. And while our prescriptions are strong, if you look at Aptium, Aptium is another product that went generic in May, another epilepsy product. We outperformed them in June. And we continue to outperform them, but our erosion of branded prescriptions is going down in cadence now with Aptivum. But it's one of the things we say about epilepsy is it's a sticky market. Patients that are on epilepsy products don't like to change from the brand to a generic. They do change eventually, but it's slower. It's a slower erosion. It's not your typical eight weeks and you're 90% down. So it's slower. So we forecasted that in. We think the prescriptions will be fine to the end of the year, but the two generic two additional generics that come in will actually be selling and loading the channel in October and into November. So while prescriptions may be fine, there's only so many prescriptions. Our share is 100% of us. But as these new come in, wholesalers will start buying less of us and more of the generics. And so while our prescription channel looks fine, we think our position is defensible because we will lose dollar share. And we all know that we can't eat prescriptions, we eat dollars. And that's what we look for. So we want to be sure that we're, again, prudent on that. And we've said consistently we expect to lose share, and we will. It's the nature of genericization of products. But we would see an acceleration of dollar share loss toward the end of the towards the middle of the fourth quarter, which we think makes sense for a number in and around our guidance. Again, we want to be prudent because the second player has not entered, and we don't know what their price play is. Teva entered, and Teva was a very, very good competitor coming in at a 17% discount to the brand, which is very healthy. And we were very pleased with that. Got it. So very sensible guidance. You are not sandbagging. We get accused of a lot of things, but we are prudent. We think we are prudent forecasters. All right. Wanted to ask about SG and A investment. So you have started to see some payoff from having dedicated sales force, preferred apps and a GammaRay. What goes into the decision to like how do you expect to evaluate the need or opportunity for future SG and A investments? One of things we said was if this ten year realization actually happens, there's a limit to what you can do as far as the number of sales people that you want to put in the field. You can overwhelm an office. Right now in the DMD space, as an example, it's just very crowded. And there are a lot of issues surrounding that. And you when I was in big pharma I worked for four big pharmas. The strategy was you hold them, I'll hit them. That's how we promoted to doctors. Right? We just kept pounding away on the docs, pounding away. And you don't do that in in orphan rare. You lose a lot of credibility. You have to be thoughtful. And so the 16 the 12 sales representatives we have on the DMD side is right. Okay. How we go about reaching the patient through nontraditional means, direct to patient, you know, social, etcetera, that's an area we see a lot of opportunity. Working with the patient advocacy groups is another area where we see a lot of opportunity to gain credibility. Our business model is one where we because we buy and build, we come late to the game. The product is well developed. Other people have done the work and maybe not done as much work as they should. And so we have to make up ground. So we find out we're getting a product. It's immediately accretive or nearly immediately accretive, which means we have to get in touch with the patient groups really fast. And we generally show up and they're like, who are you? We have to build credibility really quickly. It's very challenging. But it's the model we adopted and we like. So when we think about the opportunity on cancer LEMS and idiopathic LEMS on the Firdapse side, we see this as two markets. We don't think there's a need for more sales representatives. There may be a need for effort at the top of these GPO decision maker, but that's not significant. Okay. So the SG and A profile is beautiful for this this opportunity, for all of the opportunities we have. Yeah. And we're ratcheting down all of our investment in Fycompa because it is actually generic. We stopped we ratcheted down significantly in January, And it's mostly social media and samples through the end of the year. So on your business strategy, you have a strong balance sheet. How are you thinking about potential BD opportunities in terms of therapeutic areas of interest, alignment with your current portfolio versus adding to it, and then stage of development of assets? Sure. So our we're a CNS focused company, but we believe that the infrastructure that we have to help patients get on drug, stay on drug, and optimize their dose is applicable to any therapeutic area. We get to ask the question, if you got a new drug, would you put it on top of your current sales forces? My answer is typically no. Because the SG and A is so strong here and so light, we can afford to put 12 people, 14 people, 16 people on the field, and it would be positive payback for us. So when we think about the opportunity to continue to invest BD, we're looking at products that are we're therapeutically agnostic, immediately accretive, nearly immediately accretive. We want to stay below a $500,000,000 peak year sales for us right now. Because when we show up at the auction, if it's above 500, you have other companies that have better balance sheets bigger than us, and then Steve and I are buying them coffee. So we don't really participate. So if we stay below $500 it's a really good opportunity for us. And so we're that's what we're looking at. All right. And wanted to leave the audience with kind of a parting statement. One year from now, what could be your top one to two achievements for the year? Getting closer to closer. I want to be real clear on this. Closer to full enrollment on the SUMMIT study. Okay. All right? And beginning to look at what data we could harvest from that. And then our cancer LEMS initiative has really hit the ground and is really moving forward. Because we have we're on the care pathways. We're contracting with folks. And the patients are, in that concentrated environment, really gaining the benefit of the therapy. Very positive. So with that, thanks everyone for attending. Thanks so much Rich and Steve for being here. Thank you. Have a great rest of your afternoon. Thanks. You too.

Speaker 1: All right. Well, thanks, everyone, for coming to the panel for Catalyst Pharma. I'm very pleased to have right next to me Steve Miller, CSO and COO, and then Richard Daley, CEO. So thank you guys for making it up here. Maybe if we can begin with an overview of the company and any highlights from the first half of this year. All right. all right Well, thanks, everyone, for coming to the panel for Catalyst Pharma. well thanks everyone for coming to the panel for catalyst pharma I'm very pleased to have right next to me Steve Miller, CSO and COO, and then Richard Daley, CEO. i'm very pleased to have right next to me steve miller cso and coo and then richard daley ceo So thank you guys for making it up here. so thank you guys for making it up here Maybe if we can begin with an overview of the company and any highlights from the first half of this year. maybe if we can begin with an overview of the company and any highlights from the first half of this year

Speaker 2: Sure. So thank you very much for having us. We really appreciate it. So we're Catalyst is a buy and build company. We look for opportunities in the market and work with partner companies to bring products in. Sure. sure So thank you very much for having us. so thank you very much for having us We really appreciate it. we really appreciate it So we're Catalyst is a buy and build company. so we're catalyst is a buy and build company We look for opportunities in the market and work with partner companies to bring products in. we look for opportunities in the market and work with partner companies to bring products in We have no R, very little D, and we really focus on the commercial elements of the business. And we look to reduce commercial risk through execution, but also manage our regulatory risk as well. So we actually did develop our first product Firdapse for Lambert Eaton myasthenic syndrome. And there's probably about three thousand five hundred to five thousand four hundred patients in The U. S. We have no R, very little D, and we really focus on the commercial elements of the business. we have no r very little d and we really focus on the commercial elements of the business And we look to reduce commercial risk through execution, but also manage our regulatory risk as well. and we look to reduce commercial risk through execution but also manage our regulatory risk as well So we actually did develop our first product Firdapse for Lambert Eaton myasthenic syndrome. so we actually did develop our first product firdapse for lambert eaton myasthenic syndrome And there's probably about three thousand five hundred to five thousand four hundred patients in The U. and there's probably about three thousand five hundred to five thousand four hundred patients in the u S. s We are a U. S.-focused organization. We did develop that and launched it in 2019. Have had great success with the product in identifying patients and bringing that forward as an opportunity to help patients live better lives. We also licensed a product from Eisai for epilepsy, and this was pretty much a financial transaction. We are a U. we are a u S.-focused organization. s.-focused organization We did develop that and launched it in 2019. we did develop that and launched it in 2019 Have had great success with the product in identifying patients and bringing that forward as an opportunity to help patients live better lives. have had great success with the product in identifying patients and bringing that forward as an opportunity to help patients live better lives We also licensed a product from Eisai for epilepsy, and this was pretty much a financial transaction. we also licensed a product from eisai for epilepsy and this was pretty much a financial transaction We did this a number of years ago actually bolster the balance sheet but also get diversity of income. We consider epilepsy to be more of a retail play and not something we want to be in for the long haul unless it's rare epilepsy. And our most recent acquisition is a GammaRay for Duchenne's muscular dystrophy. And we launched that in March and have had great success in a highly competitive space. And we really look forward to continuing the growth of the company through acquisitions and also lifecycle management of the products. We did this a number of years ago actually bolster the balance sheet but also get diversity of income. we did this a number of years ago actually bolster the balance sheet but also get diversity of income We consider epilepsy to be more of a retail play and not something we want to be in for the long haul unless it's rare epilepsy. we consider epilepsy to be more of a retail play and not something we want to be in for the long haul unless it's rare epilepsy And our most recent acquisition is a GammaRay for Duchenne's muscular dystrophy. and our most recent acquisition is a gammaray for duchenne's muscular dystrophy And we launched that in March and have had great success in a highly competitive space. and we launched that in march and have had great success in a highly competitive space And we really look forward to continuing the growth of the company through acquisitions and also lifecycle management of the products. and we really look forward to continuing the growth of the company through acquisitions and also lifecycle management of the products

Speaker 1: Great. Well, wanted to start by talking about Firdapse, and specifically for the idiopathic side of LEMS. Can you talk about Firdapse's position in the treatment paradigm, the level of penetration in this patient population, and any challenges with diagnosis or getting patients on Sure. Great. great Well, wanted to start by talking about Firdapse, and specifically for the idiopathic side of LEMS. well wanted to start by talking about firdapse and specifically for the idiopathic side of lems Can you talk about Firdapse's position in the treatment paradigm, the level of penetration in this patient population, and any challenges with diagnosis or getting patients on Sure. can you talk about firdapse's position in the treatment paradigm the level of penetration in this patient population and any challenges with diagnosis or getting patients on sure

Speaker 2: So LEMS is a really interesting therapeutic opportunity. It seems to be split about fiftyfifty between idiopathic or noncancer Lamberti myasthenic syndrome versus cancer associated LEMS. So on the idiopathic side, it's very much a prospecting market. We have to look for data in the marketplace that tells us where the patients are. So we do this in a compliant way, and we look for opportunities to identify how a patient might be misdiagnosed. So LEMS is a really interesting therapeutic opportunity. so lems is a really interesting therapeutic opportunity It seems to be split about fiftyfifty between idiopathic or noncancer Lamberti myasthenic syndrome versus cancer associated LEMS. it seems to be split about fiftyfifty between idiopathic or noncancer lamberti myasthenic syndrome versus cancer associated lems So on the idiopathic side, it's very much a prospecting market. so on the idiopathic side it's very much a prospecting market We have to look for data in the marketplace that tells us where the patients are. we have to look for data in the marketplace that tells us where the patients are So we do this in a compliant way, and we look for opportunities to identify how a patient might be misdiagnosed. so we do this in a compliant way and we look for opportunities to identify how a patient might be misdiagnosed And the number of misdiagnosis is myasthenia gravis. And so patients will end up getting therapy for myasthenia gravis and not responding, and we see an opportunity to help those patients. One of the things that we find really exciting is the increase in the number of myasthenia gravis therapies that are out there. We believe that this effort, on the part of other companies, way we classify it, is the best money we never spent. It will get an elevated number of patients diagnosed and unfortunately misdiagnosed. And the number of misdiagnosis is myasthenia gravis. and the number of misdiagnosis is myasthenia gravis And so patients will end up getting therapy for myasthenia gravis and not responding, and we see an opportunity to help those patients. and so patients will end up getting therapy for myasthenia gravis and not responding and we see an opportunity to help those patients One of the things that we find really exciting is the increase in the number of myasthenia gravis therapies that are out there. one of the things that we find really exciting is the increase in the number of myasthenia gravis therapies that are out there We believe that this effort, on the part of other companies, way we classify it, is the best money we never spent. we believe that this effort on the part of other companies way we classify it is the best money we never spent It will get an elevated number of patients diagnosed and unfortunately misdiagnosed. it will get an elevated number of patients diagnosed and unfortunately misdiagnosed When the myasthenia gravis therapies don't work for those patients, we expect then those patients might roll over to where we are. Because the physician, the neurologist will see that this drug is not working, they'll and see that we have an opportunity to help those patients. So the addressable market there is about seventeen hundred patients to 2,002 patients. And we our penetration there is about 30% in the market. And so we see tremendous upside, not only on the idiopathic side, but also on the cancer associated LEMS side. When the myasthenia gravis therapies don't work for those patients, we expect then those patients might roll over to where we are. when the myasthenia gravis therapies don't work for those patients we expect then those patients might roll over to where we are Because the physician, the neurologist will see that this drug is not working, they'll and see that we have an opportunity to help those patients. because the physician the neurologist will see that this drug is not working they'll and see that we have an opportunity to help those patients So the addressable market there is about seventeen hundred patients to 2,002 patients. so the addressable market there is about seventeen hundred patients to 2,002 patients And we our penetration there is about 30% in the market. and we our penetration there is about 30% in the market And so we see tremendous upside, not only on the idiopathic side, but also on the cancer associated LEMS side. and so we see tremendous upside not only on the idiopathic side but also on the cancer associated lems side

Speaker 1: Okay. And then in terms of growth drivers still for the idiopathic side, so you talked about the misdiagnosis of myasthenia gravis. But I guess, yeah, like how does that flow through to getting patients on deferred apps? And then what do you see driving further growth? Okay. okay And then in terms of growth drivers still for the idiopathic side, so you talked about the misdiagnosis of myasthenia gravis. and then in terms of growth drivers still for the idiopathic side so you talked about the misdiagnosis of myasthenia gravis But I guess, yeah, like how does that flow through to getting patients on deferred apps? but i guess yeah like how does that flow through to getting patients on deferred apps And then what do you see driving further growth? and then what do you see driving further growth

Speaker 2: So one of the things we do is we look for these markers for patients who might be misdiagnosed and might have LEMS. And we are actively keeping a pool of about 500 or more patients who are in their diagnostic journey. And when we see the markers through data market data, so data we get from IQVIA, etcetera, we prospect, we send our sales representatives in, and we have a team of about 16 people who cover the entire country. And they'll go and speak to the doctor and try and bring the doctor along in their therapeutic options. The average physician on the idiopathic side will see one patient in their entire career. So one of the things we do is we look for these markers for patients who might be misdiagnosed and might have LEMS. so one of the things we do is we look for these markers for patients who might be misdiagnosed and might have lems And we are actively keeping a pool of about 500 or more patients who are in their diagnostic journey. and we are actively keeping a pool of about 500 or more patients who are in their diagnostic journey And when we see the markers through data market data, so data we get from IQVIA, etcetera, we prospect, we send our sales representatives in, and we have a team of about 16 people who cover the entire country. and when we see the markers through data market data so data we get from iqvia etcetera we prospect we send our sales representatives in and we have a team of about 16 people who cover the entire country And they'll go and speak to the doctor and try and bring the doctor along in their therapeutic options. and they'll go and speak to the doctor and try and bring the doctor along in their therapeutic options The average physician on the idiopathic side will see one patient in their entire career. the average physician on the idiopathic side will see one patient in their entire career So truly understanding what this looks like and how this might affect the patient is really important for the sales representative to actually be in there and talk to the doctor and help them along as they move along. So we really see about eighty percent of the physicians write only one prescription. So it's really important that we have that. So we continue to keep this pool of 500 patients. And every quarter, about 50% of our new patients come from this pool. So truly understanding what this looks like and how this might affect the patient is really important for the sales representative to actually be in there and talk to the doctor and help them along as they move along. so truly understanding what this looks like and how this might affect the patient is really important for the sales representative to actually be in there and talk to the doctor and help them along as they move along So we really see about eighty percent of the physicians write only one prescription. so we really see about eighty percent of the physicians write only one prescription So it's really important that we have that. so it's really important that we have that So we continue to keep this pool of 500 patients. so we continue to keep this pool of 500 patients And every quarter, about 50% of our new patients come from this pool. and every quarter about 50% of our new patients come from this pool So not only are we hyper categorizing these patients, but then we have a super list of patients that are very, very close. And we watch those patients very closely. And we've been very productive in bringing that forward. So not only are we hyper categorizing these patients, but then we have a super list of patients that are very, very close. so not only are we hyper categorizing these patients but then we have a super list of patients that are very very close And we watch those patients very closely. and we watch those patients very closely And we've been very productive in bringing that forward. and we've been very productive in bringing that forward

Speaker 1: All right. And then how would you say the dynamics for idiopathic lens contrast with cancer associated lens? And then can you also talk about what the penetration looks like for that cancer associated population? It sounds like, you know, recently had NCCN guidelines inclusion, and then also antibody testing All right. all right And then how would you say the dynamics for idiopathic lens contrast with cancer associated lens? and then how would you say the dynamics for idiopathic lens contrast with cancer associated lens And then can you also talk about what the penetration looks like for that cancer associated population? and then can you also talk about what the penetration looks like for that cancer associated population It sounds like, you know, recently had NCCN guidelines inclusion, and then also antibody testing it sounds like you know recently had nccn guidelines inclusion and then also antibody testing

Speaker 2: as So a the antibody testing, the VGCC testing applies to both pools, both sides of the patient population. And we recently did a deal with Quest Diagnostics for what we call frictionless testing. So prior to this, a patient would have to leave the doctor's office, go to a distant site, a Quest site, have a blood draw, and then they would get back the results of that blood draw and the test within maybe a month, month and a half. What we did was we cut a deal with Quest to do in office blood draw and reporting back to the doctor and to the patient what the results are. This pays huge dividends for getting the patient further along in their journey. as So a the antibody testing, the VGCC testing applies to both pools, both sides of the patient population. as so a the antibody testing the vgcc testing applies to both pools both sides of the patient population And we recently did a deal with Quest Diagnostics for what we call frictionless testing. and we recently did a deal with quest diagnostics for what we call frictionless testing So prior to this, a patient would have to leave the doctor's office, go to a distant site, a Quest site, have a blood draw, and then they would get back the results of that blood draw and the test within maybe a month, month and a half. so prior to this a patient would have to leave the doctor's office go to a distant site a quest site have a blood draw and then they would get back the results of that blood draw and the test within maybe a month month and a half What we did was we cut a deal with Quest to do in office blood draw and reporting back to the doctor and to the patient what the results are. what we did was we cut a deal with quest to do in office blood draw and reporting back to the doctor and to the patient what the results are This pays huge dividends for getting the patient further along in their journey. this pays huge dividends for getting the patient further along in their journey So it becomes really important, on the cancer side. So the difference between the cancer and the idiopathic side, to your question, the cancer side's not a prospecting side. So both these pools are about the same. Both these patient populations are about the same size, but about nine hundred patients per year could qualify as LEMS patients. So it's about three percent of the small cell lung cancer population. So it becomes really important, on the cancer side. so it becomes really important on the cancer side So the difference between the cancer and the idiopathic side, to your question, the cancer side's not a prospecting side. so the difference between the cancer and the idiopathic side to your question the cancer side's not a prospecting side So both these pools are about the same. so both these pools are about the same Both these patient populations are about the same size, but about nine hundred patients per year could qualify as LEMS patients. both these patient populations are about the same size but about nine hundred patients per year could qualify as lems patients So it's about three percent of the small cell lung cancer population. so it's about three percent of the small cell lung cancer population And every year that's about nine hundred patients. The cancer associated LEMS patients live twice as long as a patient with small cell lung cancer that doesn't have LEMS. So the opportunity is to really work with the GPOs that manage over eighty percent of the oncologists in The U. S. So we have frictionless testing was the first step. And every year that's about nine hundred patients. and every year that's about nine hundred patients The cancer associated LEMS patients live twice as long as a patient with small cell lung cancer that doesn't have LEMS. the cancer associated lems patients live twice as long as a patient with small cell lung cancer that doesn't have lems So the opportunity is to really work with the GPOs that manage over eighty percent of the oncologists in The U. so the opportunity is to really work with the gpos that manage over eighty percent of the oncologists in the u S. s So we have frictionless testing was the first step. so we have frictionless testing was the first step NCCN guidelines, pretty much the go to source for how to treat a patient with cancer in the oncology space. And then to look to actually get out and contract with some of these organizations and get into their care pathways. So the physicians are generally judged pretty closely on how they adhere to the NCCN guidelines or the care pathways within each practice. So whereas the idiopathic side is prospecting, you have a much more concentrated center of patients, higher diagnosis per year unfortunately, they don't live as long a patient with idiopathic LEMS lives a normal life. So there are far fewer patients diagnosed every year. NCCN guidelines, pretty much the go to source for how to treat a patient with cancer in the oncology space. nccn guidelines pretty much the go to source for how to treat a patient with cancer in the oncology space And then to look to actually get out and contract with some of these organizations and get into their care pathways. and then to look to actually get out and contract with some of these organizations and get into their care pathways So the physicians are generally judged pretty closely on how they adhere to the NCCN guidelines or the care pathways within each practice. so the physicians are generally judged pretty closely on how they adhere to the nccn guidelines or the care pathways within each practice So whereas the idiopathic side is prospecting, you have a much more concentrated center of patients, higher diagnosis per year unfortunately, they don't live as long a patient with idiopathic LEMS lives a normal life. so whereas the idiopathic side is prospecting you have a much more concentrated center of patients higher diagnosis per year unfortunately they don't live as long a patient with idiopathic lems lives a normal life So there are far fewer patients diagnosed every year. so there are far fewer patients diagnosed every year But nine hundred patients and because of the survival of those patients with cancer, you see about seventeen hundred patients in this space. And we believe the addressable market for both of these both sides of this business each one is about $600,000,000 And our penetration on this side is only about ten percent, because LEMS looks like a weak patient. It looks like a patient who has cancer. Looks like a patient undergoing chemotherapy. And if you do the VGCC testing and you confirm through a physical diagnosis, you can find these patients. But nine hundred patients and because of the survival of those patients with cancer, you see about seventeen hundred patients in this space. but nine hundred patients and because of the survival of those patients with cancer you see about seventeen hundred patients in this space And we believe the addressable market for both of these both sides of this business each one is about $600,000,000 And our penetration on this side is only about ten percent, because LEMS looks like a weak patient. and we believe the addressable market for both of these both sides of this business each one is about $600,000,000 and our penetration on this side is only about ten percent because lems looks like a weak patient It looks like a patient who has cancer. it looks like a patient who has cancer Looks like a patient undergoing chemotherapy. looks like a patient undergoing chemotherapy And if you do the VGCC testing and you confirm through a physical diagnosis, you can find these patients. and if you do the vgcc testing and you confirm through a physical diagnosis you can find these patients And they actually would live a much better life. So they generally have weakness starting at the girdle, starting at the shoulder and the hip, and going outward. Whereas a myasthenia gravis patient, it goes the other way, right? So you want to be sure that you understand which patient you're dealing with. And they actually would live a much better life. and they actually would live a much better life So they generally have weakness starting at the girdle, starting at the shoulder and the hip, and going outward. so they generally have weakness starting at the girdle starting at the shoulder and the hip and going outward Whereas a myasthenia gravis patient, it goes the other way, right? whereas a myasthenia gravis patient it goes the other way right So you want to be sure that you understand which patient you're dealing with. so you want to be sure that you understand which patient you're dealing with

Speaker 1: And can you remind us when you gained NCCN guidance inclusion, and then when you started implementing the frictionless antibody testing, just to give us a sense of just like where we are in the inflection point And can you remind us when you gained NCCN guidance inclusion, and then when you started implementing the frictionless antibody testing, just to give us a sense of just like where we are in the inflection point and can you remind us when you gained nccn guidance inclusion and then when you started implementing the frictionless antibody testing just to give us a sense of just like where we are in the inflection point

Speaker 2: Sure. Of So we got we finalized the contract for frictionless testing about the May, June. We got NCCN guideline updates, which were very favorable for the LEMS patients at the July. However, back in May, betting that we would actually have a really good outcome with our lab discussions and the NCCN guidelines, we put dedicated resources in the field to work with the GPOs. So we've already begun that process of actually getting in their care pathways. Sure. Of sure of So we got we finalized the contract for frictionless testing about the May, June. so we got we finalized the contract for frictionless testing about the may june We got NCCN guideline updates, which were very favorable for the LEMS patients at the July. we got nccn guideline updates which were very favorable for the lems patients at the july However, back in May, betting that we would actually have a really good outcome with our lab discussions and the NCCN guidelines, we put dedicated resources in the field to work with the GPOs. however back in may betting that we would actually have a really good outcome with our lab discussions and the nccn guidelines we put dedicated resources in the field to work with the gpos So we've already begun that process of actually getting in their care pathways. so we've already begun that process of actually getting in their care pathways So we're really excited about this. So this is all, I think, a momentum builder for us in actually 2026. So we're really excited about this. so we're really excited about this So this is all, I think, a momentum builder for us in actually 2026. so this is all i think a momentum builder for us in actually 2026

Speaker 1: Okay. And I've seen you've talked before about a 20% discontinuation rate on Firdapse. Can you talk about just what goes into that and if it is possible to bring that rate down? Okay. okay And I've seen you've talked before about a 20% discontinuation rate on Firdapse. and i've seen you've talked before about a 20% discontinuation rate on firdapse Can you talk about just what goes into that and if it is possible to bring that rate down? can you talk about just what goes into that and if it is possible to bring that rate down

Speaker 2: Sure. So we've recently done an analysis. So we see patients will discontinue. The number one reason why a patient discontinues is because they pass away. It's relatively benign, Steve. Relatively benign therapy. What are the primary? Sure. sure So we've recently done an analysis. so we've recently done an analysis So we see patients will discontinue. so we see patients will discontinue The number one reason why a patient discontinues is because they pass away. the number one reason why a patient discontinues is because they pass away It's relatively benign, Steve. it's relatively benign steve Relatively benign therapy. relatively benign therapy What are the primary? what are the primary

Speaker 3: Primary safety indications are tingling of the hands and feet called paresthesia, and a little bit of GI distress in some patients. Primary safety indications are tingling of the hands and feet called paresthesia, and a little bit of GI distress in some patients. primary safety indications are tingling of the hands and feet called paresthesia and a little bit of gi distress in some patients

Speaker 2: So relatively benign. What we see are patients who start at less than thirty milligrams per day have a higher likelihood of discontinuing. So obviously we can't do anything about patient passing away, but we can look at the opportunity. And so we've instituted a couple of things. Number one, we instituted a titration schedule when a new patient enrolls. So relatively benign. so relatively benign What we see are patients who start at less than thirty milligrams per day have a higher likelihood of discontinuing. what we see are patients who start at less than thirty milligrams per day have a higher likelihood of discontinuing So obviously we can't do anything about patient passing away, but we can look at the opportunity. so obviously we can't do anything about patient passing away but we can look at the opportunity And so we've instituted a couple of things. and so we've instituted a couple of things Number one, we instituted a titration schedule when a new patient enrolls. number one we instituted a titration schedule when a new patient enrolls Because by label, patients are supposed to titrate. But because a physician only sees one patient in their entire career, they're not really aware of what should happen. So we've instituted that as they enroll into our specialty pharmacy and into our hub. But we also now are reaching out pharmacist to physician, so health care provider to health care provider, as the patient comes on board. If the patient doesn't have a titration schedule or their initial dose is below thirty milligrams, pharmacist at our specialty pharmacy calls the doctor and says, I see hundreds of these patients in my career. Because by label, patients are supposed to titrate. because by label patients are supposed to titrate But because a physician only sees one patient in their entire career, they're not really aware of what should happen. but because a physician only sees one patient in their entire career they're not really aware of what should happen So we've instituted that as they enroll into our specialty pharmacy and into our hub. so we've instituted that as they enroll into our specialty pharmacy and into our hub But we also now are reaching out pharmacist to physician, so health care provider to health care provider, as the patient comes on board. but we also now are reaching out pharmacist to physician so health care provider to health care provider as the patient comes on board If the patient doesn't have a titration schedule or their initial dose is below thirty milligrams, pharmacist at our specialty pharmacy calls the doctor and says, I see hundreds of these patients in my career. if the patient doesn't have a titration schedule or their initial dose is below thirty milligrams pharmacist at our specialty pharmacy calls the doctor and says i see hundreds of these patients in my career Let me help you. And this is something we just recently instituted in June, And we're seeing really nice pickup on this. So again, we don't think this is going to fundamentally change the milligrams per day so much as it will change the persistency of the patient staying on therapy. Let me help you. let me help you And this is something we just recently instituted in June, And we're seeing really nice pickup on this. and this is something we just recently instituted in june and we're seeing really nice pickup on this So again, we don't think this is going to fundamentally change the milligrams per day so much as it will change the persistency of the patient staying on therapy. so again we don't think this is going to fundamentally change the milligrams per day so much as it will change the persistency of the patient staying on therapy

Speaker 1: Got it. And you started to touch on this with just updating the care pathways. But I was wondering if you could elaborate more in terms of where you stand with the current large group oncology practices and GPOs, perhaps any kind of target base and where you are in reaching that So Got it. got it And you started to touch on this with just updating the care pathways. and you started to touch on this with just updating the care pathways But I was wondering if you could elaborate more in terms of where you stand with the current large group oncology practices and GPOs, perhaps any kind of target base and where you are in reaching that So but i was wondering if you could elaborate more in terms of where you stand with the current large group oncology practices and gpos perhaps any kind of target base and where you are in reaching that so

Speaker 2: we see this as a four step process. They called out frictionless testing, NCCN guidelines, getting on the care pathway, and then contracting with those large GPOs that can make a difference. So we are in the process of working with them to bring them up to speed on these recently improved NCCN guidelines. And then typically, a large group practice in oncology will have its own care pathway model on NCCN. Some use ASCO guidelines, but most use NCCN. we see this as a four step process. we see this as a four step process They called out frictionless testing, NCCN guidelines, getting on the care pathway, and then contracting with those large GPOs that can make a difference. they called out frictionless testing nccn guidelines getting on the care pathway and then contracting with those large gpos that can make a difference So we are in the process of working with them to bring them up to speed on these recently improved NCCN guidelines. so we are in the process of working with them to bring them up to speed on these recently improved nccn guidelines And then typically, a large group practice in oncology will have its own care pathway model on NCCN. and then typically a large group practice in oncology will have its own care pathway model on nccn Some use ASCO guidelines, but most use NCCN. some use asco guidelines but most use nccn It's the predominant one. So we have our dedicated field resources calling on the top of the house in GPOs to say, hey, here are the new guidelines. You really should implement these. And then as we have more and more success, we'll look to do contracting for minimal discounts in the oncology space to help drive the success of the brand and the success of therapy for patients. It's the predominant one. it's the predominant one So we have our dedicated field resources calling on the top of the house in GPOs to say, hey, here are the new guidelines. so we have our dedicated field resources calling on the top of the house in gpos to say hey here are the new guidelines You really should implement these. you really should implement these And then as we have more and more success, we'll look to do contracting for minimal discounts in the oncology space to help drive the success of the brand and the success of therapy for patients. and then as we have more and more success we'll look to do contracting for minimal discounts in the oncology space to help drive the success of the brand and the success of therapy for patients

Speaker 1: I was wondering for 2025 guidance of $355,000,000 to $360,000,000 per year for Firdapse, what are the assumptions going into this? How are year to date trends tracking against those assumptions? Then what is your confidence in reaching that over $1,000,000,000 addressable market opportunity? I was wondering for 2025 guidance of $355,000,000 to $360,000,000 per year for Firdapse, what are the assumptions going into this? i was wondering for 2025 guidance of $355,000,000 to $360,000,000 per year for firdapse what are the assumptions going into this How are year to date trends tracking against those assumptions? how are year to date trends tracking against those assumptions Then what is your confidence in reaching that over $1,000,000,000 addressable market opportunity? then what is your confidence in reaching that over $1,000,000,000 addressable market opportunity

Speaker 2: So the assumptions that went into the business were that we would we recent last year, we got an increase in the maximum dose for Firdapse from eighty milligram to one hundred milligram. Patients would march up that appropriately. Typically neurologists like to be conservative and leave what we call headroom for their therapy. So we would see a fair number of patients at eighty milligrams, but not even a high plurality. Once we got the one hundred milligram, we started seeing patients move closer to the one hundred. So the assumptions that went into the business were that we would we recent last year, we got an increase in the maximum dose for Firdapse from eighty milligram to one hundred milligram. so the assumptions that went into the business were that we would we recent last year we got an increase in the maximum dose for firdapse from eighty milligram to one hundred milligram Patients would march up that appropriately. patients would march up that appropriately Typically neurologists like to be conservative and leave what we call headroom for their therapy. typically neurologists like to be conservative and leave what we call headroom for their therapy So we would see a fair number of patients at eighty milligrams, but not even a high plurality. so we would see a fair number of patients at eighty milligrams but not even a high plurality Once we got the one hundred milligram, we started seeing patients move closer to the one hundred. once we got the one hundred milligram we started seeing patients move closer to the one hundred And so every cohort in our group actually increased its average daily dose as you got that. So you can see more comfort with the product. Major drivers were actually reassigning the sales force, and now we have a dedicated team. Prior to this, our team of 18 sales representatives called both Firdapse and a Gamri. And you can imagine where the shine was, where excitement was. And so every cohort in our group actually increased its average daily dose as you got that. and so every cohort in our group actually increased its average daily dose as you got that So you can see more comfort with the product. so you can see more comfort with the product Major drivers were actually reassigning the sales force, and now we have a dedicated team. major drivers were actually reassigning the sales force and now we have a dedicated team Prior to this, our team of 18 sales representatives called both Firdapse and a Gamri. prior to this our team of 18 sales representatives called both firdapse and a gamri And you can imagine where the shine was, where excitement was. and you can imagine where the shine was where excitement was The excitement is in the new product, GammaRay, which is a great product. The opportunity then is, you know, with a dedicated force to actually be really focused. Your mission every day is to, you know, help more patients on both sides of business. So we're really excited about that. The opportunity to reach $1,000,000,000 again, this is just the addressable market. The excitement is in the new product, GammaRay, which is a great product. the excitement is in the new product gammaray which is a great product The opportunity then is, you know, with a dedicated force to actually be really focused. the opportunity then is you know with a dedicated force to actually be really focused Your mission every day is to, you know, help more patients on both sides of business. your mission every day is to you know help more patients on both sides of business So we're really excited about that. so we're really excited about that The opportunity to reach $1,000,000,000 again, this is just the addressable market. the opportunity to reach $1,000,000,000 again this is just the addressable market If we take out the patients who don't have very many symptoms or the patients who have so many symptoms at the top end of the range that it might not benefit them, we end up with about 80% of the total market being addressable. And both sides, about 600,000,000 So we're pretty confident that this is attainable, especially I'm sure one of your questions is going to be about the IP around the product. We settled with two of the three first filers and four of the litigants so far three of the four litigants so far. And Steve can talk about that. But we have life till patent life if we went on the last case till? If we take out the patients who don't have very many symptoms or the patients who have so many symptoms at the top end of the range that it might not benefit them, we end up with about 80% of the total market being addressable. if we take out the patients who don't have very many symptoms or the patients who have so many symptoms at the top end of the range that it might not benefit them we end up with about 80% of the total market being addressable And both sides, about 600,000,000 So we're pretty confident that this is attainable, especially I'm sure one of your questions is going to be about the IP around the product. and both sides about 600,000,000 so we're pretty confident that this is attainable especially i'm sure one of your questions is going to be about the ip around the product We settled with two of the three first filers and four of the litigants so far three of the four litigants so far. we settled with two of the three first filers and four of the litigants so far three of the four litigants so far And Steve can talk about that. and steve can talk about that But we have life till patent life if we went on the last case till? but we have life till patent life if we went on the last case till

Speaker 3: Yeah. If if the last litigant exits the litigation the same way the others did, we will have exclusivity to about February 2035. Yeah. yeah If if the last litigant exits the litigation the same way the others did, we will have exclusivity to about February 2035. if if the last litigant exits the litigation the same way the others did we will have exclusivity to about february 2035

Speaker 2: So one of the challenges you give a brand team is, you know, if you think that the average product has seven years of life and we've already had six plus, what would you do how would you invest in the product from a commercial perspective, from a promotion perspective if you knew you had ten more years of life? What would you do? And so this becomes a really great opportunity for brand teams. And now we have a dedicated brand team for idiopathic, a dedicated brand team for cancer because it's one molecule but two brands. Still the same brand name, but two different markets and two different ways of approaching the market. So one of the challenges you give a brand team is, you know, if you think that the average product has seven years of life and we've already had six plus, what would you do how would you invest in the product from a commercial perspective, from a promotion perspective if you knew you had ten more years of life? so one of the challenges you give a brand team is you know if you think that the average product has seven years of life and we've already had six plus what would you do how would you invest in the product from a commercial perspective from a promotion perspective if you knew you had ten more years of life What would you do? what would you do And so this becomes a really great opportunity for brand teams. and so this becomes a really great opportunity for brand teams And now we have a dedicated brand team for idiopathic, a dedicated brand team for cancer because it's one molecule but two brands. and now we have a dedicated brand team for idiopathic a dedicated brand team for cancer because it's one molecule but two brands Still the same brand name, but two different markets and two different ways of approaching the market. still the same brand name but two different markets and two different ways of approaching the market How would you invest? What would you do? And the team is appropriately aggressive in looking at opportunities to continue to grow the business. How would you invest? how would you invest What would you do? what would you do And the team is appropriately aggressive in looking at opportunities to continue to grow the business. and the team is appropriately aggressive in looking at opportunities to continue to grow the business

Speaker 1: Okay. And just to provide more clarity on the IP situation, can you tell us like what is the status or timelines with the remaining litigants and then also the upcoming Markman hearing? Okay. okay And just to provide more clarity on the IP situation, can you tell us like what is the status or timelines with the remaining litigants and then also the upcoming Markman hearing? and just to provide more clarity on the ip situation can you tell us like what is the status or timelines with the remaining litigants and then also the upcoming markman hearing

Speaker 3: Sure. The only remaining litigant is hetero. I really can't comment on what's going on specifically with regard to hetero. But you're correct. There is an upcoming Markman hearing on October 7. Sure. sure The only remaining litigant is hetero. the only remaining litigant is hetero I really can't comment on what's going on specifically with regard to hetero. i really can't comment on what's going on specifically with regard to hetero But you're correct. but you're correct There is an upcoming Markman hearing on October 7. there is an upcoming markman hearing on october 7 And the Markman hearing is the hearing where all of the where the the claims will be discussed and how they apply to each of the two litigants. And so one of the things that you see historically in patent litigation is that there's usually a lot of settlement activity around the time of the Markman hearing. And the Markman hearing is the hearing where all of the where the the claims will be discussed and how they apply to each of the two litigants. and the markman hearing is the hearing where all of the where the the claims will be discussed and how they apply to each of the two litigants And so one of the things that you see historically in patent litigation is that there's usually a lot of settlement activity around the time of the Markman hearing. and so one of the things that you see historically in patent litigation is that there's usually a lot of settlement activity around the time of the markman hearing

Speaker 2: Okay. Okay. okay

Speaker 1: Wanted to talk about a gamma ray for DMD. Can you talk about the differentiation versus existing corticosteroids and what has been driving conversion thus far? Wanted to talk about a gamma ray for DMD. wanted to talk about a gamma ray for dmd Can you talk about the differentiation versus existing corticosteroids and what has been driving conversion thus far? can you talk about the differentiation versus existing corticosteroids and what has been driving conversion thus far

Speaker 2: Sure. Think on the differentiation, Steve as the chief scientific officer should probably address that. Sure. sure Think on the differentiation, Steve as the chief scientific officer should probably address that. think on the differentiation steve as the chief scientific officer should probably address that

Speaker 3: Well, thank you, Rich. The defining features of a gamma ray relative to the other corticosteroids that are on the market is that there's a lot of published literature that shows that a gamma ray has superior bone health, bone growth, as well as stature for the patients. There is much less exhibiting of aggression in patients. There may also be less cataracts. And our hope is that there also will be better cardiovascular safety with the gamma relative to the other corticosteroids. Well, thank you, Rich. well thank you rich The defining features of a gamma ray relative to the other corticosteroids that are on the market is that there's a lot of published literature that shows that a gamma ray has superior bone health, bone growth, as well as stature for the patients. the defining features of a gamma ray relative to the other corticosteroids that are on the market is that there's a lot of published literature that shows that a gamma ray has superior bone health bone growth as well as stature for the patients There is much less exhibiting of aggression in patients. there is much less exhibiting of aggression in patients There may also be less cataracts. there may also be less cataracts And our hope is that there also will be better cardiovascular safety with the gamma relative to the other corticosteroids. and our hope is that there also will be better cardiovascular safety with the gamma relative to the other corticosteroids The physicians who were participating in the clinical trials actually observed a number of those things in the patients that they were treating for the clinical trials and have been very enthusiastic and have been early prescribers for the product. The physicians who were participating in the clinical trials actually observed a number of those things in the patients that they were treating for the clinical trials and have been very enthusiastic and have been early prescribers for the product. the physicians who were participating in the clinical trials actually observed a number of those things in the patients that they were treating for the clinical trials and have been very enthusiastic and have been early prescribers for the product

Speaker 1: Okay. And can you talk about perhaps access for a gamma ray? I think just given, like, the assumed lower cost of steroids, how is the gamma ray positioned in terms Okay. okay And can you talk about perhaps access for a gamma ray? and can you talk about perhaps access for a gamma ray I think just given, like, the assumed lower cost of steroids, how is the gamma ray positioned in terms i think just given like the assumed lower cost of steroids how is the gamma ray positioned in terms

Speaker 2: Sure. So before we launched the product, we did market research with 17 active decision makers and payers. We said, okay, here's the profile. Steve just laid out a profile. Potentially better behavior, bone health, bone and growth stature for the patient in cardiovascular. Sure. sure So before we launched the product, we did market research with 17 active decision makers and payers. so before we launched the product we did market research with 17 active decision makers and payers We said, okay, here's the profile. we said okay here's the profile Steve just laid out a profile. steve just laid out a profile Potentially better behavior, bone health, bone and growth stature for the patient in cardiovascular. potentially better behavior bone health bone and growth stature for the patient in cardiovascular What should we do? How should we price this? Knowing that in February, the month before we launched, knowing that a generic Emflaza was coming. There's three players in the market. There's prednisone generic Emflaza, which was branded and now has a generic component. What should we do? what should we do How should we price this? how should we price this Knowing that in February, the month before we launched, knowing that a generic Emflaza was coming. knowing that in february the month before we launched knowing that a generic emflaza was coming There's three players in the market. there's three players in the market There's prednisone generic Emflaza, which was branded and now has a generic component. there's prednisone generic emflaza which was branded and now has a generic component We asked these 17 payers, decision makers, what should we do? How would we price the product? And they said, if you come in just slightly below branded Emflaza, knowing a generic is coming, we won't block you. And think about the market. The market's very well established. We asked these 17 payers, decision makers, what should we do? we asked these 17 payers decision makers what should we do How would we price the product? how would we price the product And they said, if you come in just slightly below branded Emflaza, knowing a generic is coming, we won't block you. and they said if you come in just slightly below branded emflaza knowing a generic is coming we won't block you And think about the market. and think about the market The market's very well established. the market's very well established Patients start on prednisone. It's the foundation. Steroids are the foundation of therapy. So they start on prednisone. If they're not happy, they may move to Emflaza. Patients start on prednisone. patients start on prednisone It's the foundation. it's the foundation Steroids are the foundation of therapy. steroids are the foundation of therapy So they start on prednisone. so they start on prednisone If they're not happy, they may move to Emflaza. if they're not happy they may move to emflaza And Emflaza has been on the market for four or five years. We come in the market. There's no step through therapy at this point because the patients that want a gamma ray really have already gone through prednisone, Emflaza. No one's gonna send them back to generic Emflaza, so there's an opportunity for us to actually take advantage of this. And we are sourcing patients roughly 45, 45, 10. And Emflaza has been on the market for four or five years. and emflaza has been on the market for four or five years We come in the market. we come in the market There's no step through therapy at this point because the patients that want a gamma ray really have already gone through prednisone, Emflaza. there's no step through therapy at this point because the patients that want a gamma ray really have already gone through prednisone emflaza No one's gonna send them back to generic Emflaza, so there's an opportunity for us to actually take advantage of this. no one's gonna send them back to generic emflaza so there's an opportunity for us to actually take advantage of this And we are sourcing patients roughly 45, 45, 10. and we are sourcing patients roughly 45 45 10 And this has been true since the launch of the product, which is amazing. Originally, we thought we'd be participating just in the Emflaza part of the market, and it would be a cannibalization. Forty five percent it changes a percent or two in any given month. Forty five percent of our patients come from prednisone, forty five percent of our patients come from Emflaza, and ten percent are naive. What that means is instead of participating in roughly 30% or 40% of the market, which is where we think Emflaza's market share was, we're participating in 100% of the market. And this has been true since the launch of the product, which is amazing. and this has been true since the launch of the product which is amazing Originally, we thought we'd be participating just in the Emflaza part of the market, and it would be a cannibalization. originally we thought we'd be participating just in the emflaza part of the market and it would be a cannibalization Forty five percent it changes a percent or two in any given month. forty five percent it changes a percent or two in any given month Forty five percent of our patients come from prednisone, forty five percent of our patients come from Emflaza, and ten percent are naive. forty five percent of our patients come from prednisone forty five percent of our patients come from emflaza and ten percent are naive What that means is instead of participating in roughly 30% or 40% of the market, which is where we think Emflaza's market share was, we're participating in 100% of the market. what that means is instead of participating in roughly 30% or 40% of the market which is where we think emflaza's market share was we're participating in 100% of the market So it's a huge opportunity for us. And the fact that that's been going on now for sixteen months is a very, very good sign. We do anticipate it will change over time. And we do anticipate that there'll be opportunities to continue to grow the product through life cycle management. And we're working on that right now. So it's a huge opportunity for us. so it's a huge opportunity for us And the fact that that's been going on now for sixteen months is a very, very good sign. and the fact that that's been going on now for sixteen months is a very very good sign We do anticipate it will change over time. we do anticipate it will change over time And we do anticipate that there'll be opportunities to continue to grow the product through life cycle management. and we do anticipate that there'll be opportunities to continue to grow the product through life cycle management And we're working on that right now. and we're working on that right now We're working on how to go about the life cycle management for the product. We're working on how to go about the life cycle management for the product. we're working on how to go about the life cycle management for the product

Speaker 1: Do you see faster conversion from prednisone or generic Emflaza or naive? And is there a channel that you want to grow more in? Do you see faster conversion from prednisone or generic Emflaza or naive? do you see faster conversion from prednisone or generic emflaza or naive And is there a channel that you want to grow more in? and is there a channel that you want to grow more in

Speaker 2: I think over time, it'll be harder to get the naive patient. I think over time, it'll be harder to get the naive patient. i think over time it'll be harder to get the naive patient

Speaker 1: Okay. Okay. okay

Speaker 2: Because they're gonna start on a generic prednisone. Yeah. That's gonna happen. The game for us is can we be that next choice? Right? Because they're gonna start on a generic prednisone. because they're gonna start on a generic prednisone Yeah. yeah That's gonna happen. that's gonna happen The game for us is can we be that next choice? the game for us is can we be that next choice Right? right So the generic's gonna be there. Our average patient pays less than $2 a month for the product. So it's not from a patient perspective, it's not a price play. So they're looking at and saying, Hey, if I have the support of the company, I think there's good things that can happen here for me as a patient, given the profile that Steve laid out. I think over time, though, the goal to really look for the opportunity to be that second steroid in line. So the generic's gonna be there. so the generic's gonna be there Our average patient pays less than $2 a month for the product. our average patient pays less than $2 a month for the product So it's not from a patient perspective, it's not a price play. so it's not from a patient perspective it's not a price play So they're looking at and saying, Hey, if I have the support of the company, I think there's good things that can happen here for me as a patient, given the profile that Steve laid out. so they're looking at and saying hey if i have the support of the company i think there's good things that can happen here for me as a patient given the profile that steve laid out I think over time, though, the goal to really look for the opportunity to be that second steroid in line. i think over time though the goal to really look for the opportunity to be that second steroid in line I mean, every patient should be on a steroid. Ninety five percent have been on a steroid and only seventy percent are currently on a steroid, which speaks to the gap. There's something wrong with the current options for the patient. If you can address the issues that Steve talked about, and you're just as good as the other players, I think you can improve quality of life. Remember, behavior is not just an issue for the young boy. I mean, every patient should be on a steroid. i mean every patient should be on a steroid Ninety five percent have been on a steroid and only seventy percent are currently on a steroid, which speaks to the gap. ninety five percent have been on a steroid and only seventy percent are currently on a steroid which speaks to the gap There's something wrong with the current options for the patient. there's something wrong with the current options for the patient If you can address the issues that Steve talked about, and you're just as good as the other players, I think you can improve quality of life. if you can address the issues that steve talked about and you're just as good as the other players i think you can improve quality of life Remember, behavior is not just an issue for the young boy. remember behavior is not just an issue for the young boy Remember, all of these patients are boys. It's a if they misbehave, it's a problem for the family. They're in school. It's a problem for the classroom. So if they have better behavior, they progress emotionally, psychologically, and so does everyone else around them. Remember, all of these patients are boys. remember all of these patients are boys It's a if they misbehave, it's a problem for the family. it's a if they misbehave it's a problem for the family They're in school. they're in school It's a problem for the classroom. it's a problem for the classroom So if they have better behavior, they progress emotionally, psychologically, and so does everyone else around them. so if they have better behavior they progress emotionally psychologically and so does everyone else around them So it's just a relief. And we see that coming back to us in the market. It's not in our label, but we see that coming back to us in the market from feedback from doctors. So it's just a relief. so it's just a relief And we see that coming back to us in the market. and we see that coming back to us in the market It's not in our label, but we see that coming back to us in the market from feedback from doctors. it's not in our label but we see that coming back to us in the market from feedback from doctors

Speaker 1: Okay. And can you talk about some of the assumptions going into that 100,000,000 to $110,000,000.20 25 guidance? How are year to date trends tracking against those assumptions? And then also what it takes to reach that over $1,000,000,000 addressable market opportunity? Okay. okay And can you talk about some of the assumptions going into that 100,000,000 to $110,000,000.20 25 guidance? and can you talk about some of the assumptions going into that 100,000,000 to $110,000,000.20 25 guidance How are year to date trends tracking against those assumptions? how are year to date trends tracking against those assumptions And then also what it takes to reach that over $1,000,000,000 addressable market opportunity? and then also what it takes to reach that over $1,000,000,000 addressable market opportunity

Speaker 2: So just to address the addressable market this is the fundamental difference between Firdapse and a GammaRia. The addressable market for GammaRay is a billion 2. But there are four players in the market. And if you get your fair share, you get 300,000,000. If you're better, you should get an unfair share. So just to address the addressable market this is the fundamental difference between Firdapse and a GammaRia. so just to address the addressable market this is the fundamental difference between firdapse and a gammaria The addressable market for GammaRay is a billion 2. the addressable market for gammaray is a billion 2 But there are four players in the market. but there are four players in the market And if you get your fair share, you get 300,000,000. and if you get your fair share you get 300,000,000 If you're better, you should get an unfair share. if you're better you should get an unfair share Right? So Firdapse in its addressable market of $1,000,000,000 is all alone. I mean, so everybody's very excited about a gamma ray. We're excited about a gamma ray. But when you look at the potential for the product and you have you know you're sitting on potentially ten more years, Firdapse is very exciting molecule. Right? right So Firdapse in its addressable market of $1,000,000,000 is all alone. so firdapse in its addressable market of $1,000,000,000 is all alone I mean, so everybody's very excited about a gamma ray. i mean so everybody's very excited about a gamma ray We're excited about a gamma ray. we're excited about a gamma ray But when you look at the potential for the product and you have you know you're sitting on potentially ten more years, Firdapse is very exciting molecule. but when you look at the potential for the product and you have you know you're sitting on potentially ten more years firdapse is very exciting molecule Gamma is very exciting. So the assumptions that went into the forecast, we there are 100 centers of excellence, two fifty doctors write 80 to 90% of the prescriptions, And 45 of the centers write 90% of the prescriptions. So we want to be penetrated. So we have had use in ninety three of the centers. And now so we've gone broad. Gamma is very exciting. gamma is very exciting So the assumptions that went into the forecast, we there are 100 centers of excellence, two fifty doctors write 80 to 90% of the prescriptions, And 45 of the centers write 90% of the prescriptions. so the assumptions that went into the forecast we there are 100 centers of excellence two fifty doctors write 80 to 90% of the prescriptions and 45 of the centers write 90% of the prescriptions So we want to be penetrated. so we want to be penetrated So we have had use in ninety three of the centers. so we have had use in ninety three of the centers And now so we've gone broad. and now so we've gone broad Now the goal is to go deeper. And you see the early adopters obviously going to the product, which is fantastic. But we want their colleagues then to see the benefit. And so working with those 100 centers is really where we're focused in on education and making sure that we have that reach and frequency that's appropriate in this market. And this is a very difficult market because of some of the challenges that we see with gene therapy. Now the goal is to go deeper. now the goal is to go deeper And you see the early adopters obviously going to the product, which is fantastic. and you see the early adopters obviously going to the product which is fantastic But we want their colleagues then to see the benefit. but we want their colleagues then to see the benefit And so working with those 100 centers is really where we're focused in on education and making sure that we have that reach and frequency that's appropriate in this market. and so working with those 100 centers is really where we're focused in on education and making sure that we have that reach and frequency that's appropriate in this market And this is a very difficult market because of some of the challenges that we see with gene therapy. and this is a very difficult market because of some of the challenges that we see with gene therapy This is a really unfortunate situation and it creates a lot of noise around the therapy. And so we actually have this queuing effect where patients have to get an immunosuppressive dose of steroid. We do not have an immunosuppressive dose in our label, we can't be used there. And then so we see patients waiting. Now that there's been these issues in gene therapy market, there's a lot of consternation, and people are trying to figure out what's next and how to do this. This is a really unfortunate situation and it creates a lot of noise around the therapy. this is a really unfortunate situation and it creates a lot of noise around the therapy And so we actually have this queuing effect where patients have to get an immunosuppressive dose of steroid. and so we actually have this queuing effect where patients have to get an immunosuppressive dose of steroid We do not have an immunosuppressive dose in our label, we can't be used there. we do not have an immunosuppressive dose in our label we can't be used there And then so we see patients waiting. and then so we see patients waiting Now that there's been these issues in gene therapy market, there's a lot of consternation, and people are trying to figure out what's next and how to do this. now that there's been these issues in gene therapy market there's a lot of consternation and people are trying to figure out what's next and how to do this As this settles out, and we hope it does for the sake of patients, we should be getting a little bit more traction in the market. As this settles out, and we hope it does for the sake of patients, we should be getting a little bit more traction in the market. as this settles out and we hope it does for the sake of patients we should be getting a little bit more traction in the market

Speaker 1: Okay. And in terms of this queuing effect, like, yeah, have you seen this continue so far? Because it seems like some of the uncertainty may be resolving. Or how long do you think this could benefit in terms of like the duration of patients on the gamma ray? Okay. okay And in terms of this queuing effect, like, yeah, have you seen this continue so far? and in terms of this queuing effect like yeah have you seen this continue so far Because it seems like some of the uncertainty may be resolving. because it seems like some of the uncertainty may be resolving Or how long do you think this could benefit in terms of like the duration of patients on the gamma ray? or how long do you think this could benefit in terms of like the duration of patients on the gamma ray

Speaker 2: Sure. So the queuing effect, the name comes from getting in line, right? So if a patient is waiting for therapy, gene therapy, the patient may actually not want to, and the physician may not want to change their therapy. So they could be on generic prednisone, or they could be on Emflaza. And since they're going to use one of those two for the immunosuppressive dose, they say let's not make a change to the steroid. Sure. sure So the queuing effect, the name comes from getting in line, right? so the queuing effect the name comes from getting in line right So if a patient is waiting for therapy, gene therapy, the patient may actually not want to, and the physician may not want to change their therapy. so if a patient is waiting for therapy gene therapy the patient may actually not want to and the physician may not want to change their therapy So they could be on generic prednisone, or they could be on Emflaza. so they could be on generic prednisone or they could be on emflaza And since they're going to use one of those two for the immunosuppressive dose, they say let's not make a change to the steroid. and since they're going to use one of those two for the immunosuppressive dose they say let's not make a change to the steroid And so that's where we see the queuing effect. So we're sort of in line. But once they come out of that, we're seeing some patients actually post gene therapy go to drug like a gamma ray. The opportunity for us is to see if we can find an immunosuppressive dose. So we're working on that. And so that's where we see the queuing effect. and so that's where we see the queuing effect So we're sort of in line. so we're sort of in line But once they come out of that, we're seeing some patients actually post gene therapy go to drug like a gamma ray. but once they come out of that we're seeing some patients actually post gene therapy go to drug like a gamma ray The opportunity for us is to see if we can find an immunosuppressive dose. the opportunity for us is to see if we can find an immunosuppressive dose So we're working on that. so we're working on that And then make sure that we can be there when the patient wants to make a change, if they want to make a change. So there's a lot going on in the market right now, and it's really unfortunate for patients. So we're hoping that we you know, everybody can get past that. And right now, gene therapy, the label was expanded to include all patients, ambulatory and non ambulatory. And now it's not inclusive of non ambulatory patients. And then make sure that we can be there when the patient wants to make a change, if they want to make a change. and then make sure that we can be there when the patient wants to make a change if they want to make a change So there's a lot going on in the market right now, and it's really unfortunate for patients. so there's a lot going on in the market right now and it's really unfortunate for patients So we're hoping that we you know, everybody can get past that. so we're hoping that we you know everybody can get past that And right now, gene therapy, the label was expanded to include all patients, ambulatory and non ambulatory. and right now gene therapy the label was expanded to include all patients ambulatory and non ambulatory And now it's not inclusive of non ambulatory patients. and now it's not inclusive of non ambulatory patients Our average age is about 12. And that's about the point at which a patient loses ambulation. So if those patients are no longer in the line, we think that there's upside for us there. Our average age is about 12. our average age is about 12 And that's about the point at which a patient loses ambulation. and that's about the point at which a patient loses ambulation So if those patients are no longer in the line, we think that there's upside for us there. so if those patients are no longer in the line we think that there's upside for us there

Speaker 1: Okay. And so you have the immunosuppressive study coming out like late twenty twenty five, early twenty twenty six. Okay. okay And so you have the immunosuppressive study coming out like late twenty twenty five, early twenty twenty six. and so you have the immunosuppressive study coming out like late twenty twenty five early twenty twenty six

Speaker 2: Correct. Correct. correct

Speaker 1: Can you just talk about what you're looking to see from the data? You know, what is the significance of this in terms of the utilization of the gene? Can you just talk about what you're looking to see from the data? can you just talk about what you're looking to see from the data You know, what is the significance of this in terms of the utilization of the gene? you know what is the significance of this in terms of the utilization of the gene

Speaker 2: Sure. So we're looking to see if we can find an immunosuppressive dose. There's no guarantee we will find an immunosuppressive dose for the drug. And if it does, then we will have if we do find it, we will have the opportunity potentially to participate in this run up to gene therapy. Sure. sure So we're looking to see if we can find an immunosuppressive dose. so we're looking to see if we can find an immunosuppressive dose There's no guarantee we will find an immunosuppressive dose for the drug. there's no guarantee we will find an immunosuppressive dose for the drug And if it does, then we will have if we do find it, we will have the opportunity potentially to participate in this run up to gene therapy. and if it does then we will have if we do find it we will have the opportunity potentially to participate in this run up to gene therapy So that's what we're looking for in that. But again, it's research, you know, basic clinical development, and there's no guarantee. So we await the results. So that's what we're looking for in that. so that's what we're looking for in that But again, it's research, you know, basic clinical development, and there's no guarantee. but again it's research you know basic clinical development and there's no guarantee So we await the results. so we await the results

Speaker 1: Okay. And then you also have the SUMMIT study going on. I was wondering if you could talk about the significance of this, and then when we can expect more data. Okay. okay And then you also have the SUMMIT study going on. and then you also have the summit study going on I was wondering if you could talk about the significance of this, and then when we can expect more data. i was wondering if you could talk about the significance of this and then when we can expect more data

Speaker 2: Sure. Steve? Sure. sure Steve? steve

Speaker 3: Sure. The SUMMIT study is a study being conducted in patients who are on commercial or gamma ray. And we are looking for all of those safety signals that I previously mentioned. Sure. sure The SUMMIT study is a study being conducted in patients who are on commercial or gamma ray. the summit study is a study being conducted in patients who are on commercial or gamma ray And we are looking for all of those safety signals that I previously mentioned. and we are looking for all of those safety signals that i previously mentioned And we will be categorizing all of that data and watching it as it proceeds. The comparison group is actually going to be the natural history of DMD. And that's so that we can do a comparison to the natural history and look for statistically significant improvements in that safety information and then do a submission to our drug application to update the label to include specific safety information regarding those endpoints. Now the problem with doing those kinds of comparisons is every time you do that, you have to spend a little bit of alpha, which in a nutshell means that when you do that comparison, the next time you do the comparison, it's a little harder to achieve statistical significance. You need a slightly lower p value. And we will be categorizing all of that data and watching it as it proceeds. and we will be categorizing all of that data and watching it as it proceeds The comparison group is actually going to be the natural history of DMD. the comparison group is actually going to be the natural history of dmd And that's so that we can do a comparison to the natural history and look for statistically significant improvements in that safety information and then do a submission to our drug application to update the label to include specific safety information regarding those endpoints. and that's so that we can do a comparison to the natural history and look for statistically significant improvements in that safety information and then do a submission to our drug application to update the label to include specific safety information regarding those endpoints Now the problem with doing those kinds of comparisons is every time you do that, you have to spend a little bit of alpha, which in a nutshell means that when you do that comparison, the next time you do the comparison, it's a little harder to achieve statistical significance. now the problem with doing those kinds of comparisons is every time you do that you have to spend a little bit of alpha which in a nutshell means that when you do that comparison the next time you do the comparison it's a little harder to achieve statistical significance You need a slightly lower p value. you need a slightly lower p value And so we will be judicious in our choice of when we do those studies. And we're going to want to make sure that we watch the data for a while. It's going to take about a year to recruit enough patients because we have to have a reasonable size n and a large enough period in which to observe the change in the patients before we do that comparison. So it could be at least eighteen months or a couple years before we do the first comparison. Now the good news is it is open label, which means that we can watch those safety endpoints without doing the actual statistical comparison and publicly make that information available to physicians so that they can start to learn about the safety characteristics of the drug even earlier. And so we will be judicious in our choice of when we do those studies. and so we will be judicious in our choice of when we do those studies And we're going to want to make sure that we watch the data for a while. and we're going to want to make sure that we watch the data for a while It's going to take about a year to recruit enough patients because we have to have a reasonable size n and a large enough period in which to observe the change in the patients before we do that comparison. it's going to take about a year to recruit enough patients because we have to have a reasonable size n and a large enough period in which to observe the change in the patients before we do that comparison So it could be at least eighteen months or a couple years before we do the first comparison. so it could be at least eighteen months or a couple years before we do the first comparison Now the good news is it is open label, which means that we can watch those safety endpoints without doing the actual statistical comparison and publicly make that information available to physicians so that they can start to learn about the safety characteristics of the drug even earlier. now the good news is it is open label which means that we can watch those safety endpoints without doing the actual statistical comparison and publicly make that information available to physicians so that they can start to learn about the safety characteristics of the drug even earlier And so we'll present that information from time to time at professional conferences. And so we'll present that information from time to time at professional conferences. and so we'll present that information from time to time at professional conferences

Speaker 1: Okay. Got it. So in terms of being able to get this data on the label, like is waiting for this still kind of a gating factor for certain physicians? Okay. okay Got it. got it So in terms of being able to get this data on the label, like is waiting for this still kind of a gating factor for certain physicians? so in terms of being able to get this data on the label like is waiting for this still kind of a gating factor for certain physicians

Speaker 3: It depends on the physician. As I mentioned, there were some early adopters who participated in the trials, and they recognized the advantages of the drug immediately just by observation of their own patients. There are some physicians who read the literature and are aware of it, and there are some who are strictly I'm just going to do what the answer sheet says. And so the short answer to your question is it would be helpful. It depends on the physician. it depends on the physician As I mentioned, there were some early adopters who participated in the trials, and they recognized the advantages of the drug immediately just by observation of their own patients. as i mentioned there were some early adopters who participated in the trials and they recognized the advantages of the drug immediately just by observation of their own patients There are some physicians who read the literature and are aware of it, and there are some who are strictly I'm just going to do what the answer sheet says. there are some physicians who read the literature and are aware of it and there are some who are strictly i'm just going to do what the answer sheet says And so the short answer to your question is it would be helpful. and so the short answer to your question is it would be helpful

Speaker 1: Okay. Good. I wanted to talk about Fycompa and just what that cadence of generic erosion could look like this year. Since you've already achieved 70,000,000 in the '5, how do we get to 2025 guidance of 90,000,000 to $95,000,000 Sure. Okay. okay Good. good I wanted to talk about Fycompa and just what that cadence of generic erosion could look like this year. i wanted to talk about fycompa and just what that cadence of generic erosion could look like this year Since you've already achieved 70,000,000 in the '5, how do we get to 2025 guidance of 90,000,000 to $95,000,000 Sure. since you've already achieved 70,000,000 in the '5 how do we get to 2025 guidance of 90,000,000 to $95,000,000 sure

Speaker 2: So in our most recent call, we announced our performance in the first half is $70,000,000 on guidance of 90,000,000 to 95,000,000 as you mentioned. So we got a significant amount of push, as you can imagine. What are you expecting? And really, we were expecting our assumptions going into this were there were two first filers, and they would be good actors in the market. And so Teva was one of the first filers, and they actually did get approval, and they launched. So in our most recent call, we announced our performance in the first half is $70,000,000 on guidance of 90,000,000 to 95,000,000 as you mentioned. so in our most recent call we announced our performance in the first half is $70,000,000 on guidance of 90,000,000 to 95,000,000 as you mentioned So we got a significant amount of push, as you can imagine. so we got a significant amount of push as you can imagine What are you expecting? what are you expecting And really, we were expecting our assumptions going into this were there were two first filers, and they would be good actors in the market. and really we were expecting our assumptions going into this were there were two first filers and they would be good actors in the market And so Teva was one of the first filers, and they actually did get approval, and they launched. and so teva was one of the first filers and they actually did get approval and they launched But they launched a month late, month later than we thought. They came to commercial. They had commercial presence. So the second first filer has yet to launch. And so we wait, and we want to be prudent in our guidance. But they launched a month late, month later than we thought. but they launched a month late month later than we thought They came to commercial. they came to commercial They had commercial presence. they had commercial presence So the second first filer has yet to launch. so the second first filer has yet to launch And so we wait, and we want to be prudent in our guidance. and so we wait and we want to be prudent in our guidance But we know that two more will come in November. But we know that two more will come in November. but we know that two more will come in november

Speaker 1: Okay. Okay. okay

Speaker 2: And while our prescriptions are strong, if you look at Aptium, Aptium is another product that went generic in May, another epilepsy product. We outperformed them in June. And we continue to outperform them, but our erosion of branded prescriptions is going down in cadence now with Aptivum. But it's one of the things we say about epilepsy is it's a sticky market. Patients that are on epilepsy products don't like to change from the brand to a generic. And while our prescriptions are strong, if you look at Aptium, Aptium is another product that went generic in May, another epilepsy product. and while our prescriptions are strong if you look at aptium aptium is another product that went generic in may another epilepsy product We outperformed them in June. we outperformed them in june And we continue to outperform them, but our erosion of branded prescriptions is going down in cadence now with Aptivum. and we continue to outperform them but our erosion of branded prescriptions is going down in cadence now with aptivum But it's one of the things we say about epilepsy is it's a sticky market. but it's one of the things we say about epilepsy is it's a sticky market Patients that are on epilepsy products don't like to change from the brand to a generic. patients that are on epilepsy products don't like to change from the brand to a generic They do change eventually, but it's slower. It's a slower erosion. It's not your typical eight weeks and you're 90% down. So it's slower. So we forecasted that in. They do change eventually, but it's slower. they do change eventually but it's slower It's a slower erosion. it's a slower erosion It's not your typical eight weeks and you're 90% down. it's not your typical eight weeks and you're 90% down So it's slower. so it's slower So we forecasted that in. so we forecasted that in We think the prescriptions will be fine to the end of the year, but the two generic two additional generics that come in will actually be selling and loading the channel in October and into November. So while prescriptions may be fine, there's only so many prescriptions. Our share is 100% of us. But as these new come in, wholesalers will start buying less of us and more of the generics. And so while our prescription channel looks fine, we think our position is defensible because we will lose dollar share. And we all know that we can't eat prescriptions, we eat dollars. And that's what we look for. So we want to be sure that we're, again, prudent on that. And we've said consistently we expect to lose share, and we will. It's the nature of genericization of products. We think the prescriptions will be fine to the end of the year, but the two generic two additional generics that come in will actually be selling and loading the channel in October and into November. we think the prescriptions will be fine to the end of the year but the two generic two additional generics that come in will actually be selling and loading the channel in october and into november So while prescriptions may be fine, there's only so many prescriptions. so while prescriptions may be fine there's only so many prescriptions Our share is 100% of us. our share is 100% of us But as these new come in, wholesalers will start buying less of us and more of the generics. but as these new come in wholesalers will start buying less of us and more of the generics And so while our prescription channel looks fine, we think our position is defensible because we will lose dollar share. and so while our prescription channel looks fine we think our position is defensible because we will lose dollar share And we all know that we can't eat prescriptions, we eat dollars. and we all know that we can't eat prescriptions we eat dollars And that's what we look for. and that's what we look for So we want to be sure that we're, again, prudent on that. so we want to be sure that we're again prudent on that And we've said consistently we expect to lose share, and we will. and we've said consistently we expect to lose share and we will It's the nature of genericization of products. it's the nature of genericization of products But we would see an acceleration of dollar share loss toward the end of the towards the middle of the fourth quarter, which we think makes sense for a number in and around our guidance. Again, we want to be prudent because the second player has not entered, and we don't know what their price play is. Teva entered, and Teva was a very, very good competitor coming in at a 17% discount to the brand, which is very healthy. And we were very pleased with that. But we would see an acceleration of dollar share loss toward the end of the towards the middle of the fourth quarter, which we think makes sense for a number in and around our guidance. but we would see an acceleration of dollar share loss toward the end of the towards the middle of the fourth quarter which we think makes sense for a number in and around our guidance Again, we want to be prudent because the second player has not entered, and we don't know what their price play is. again we want to be prudent because the second player has not entered and we don't know what their price play is Teva entered, and Teva was a very, very good competitor coming in at a 17% discount to the brand, which is very healthy. teva entered and teva was a very very good competitor coming in at a 17% discount to the brand which is very healthy And we were very pleased with that. and we were very pleased with that

Speaker 1: Got it. So very sensible guidance. You are not sandbagging. Got it. got it So very sensible guidance. so very sensible guidance You are not sandbagging. you are not sandbagging

Speaker 2: We get accused of a lot of things, but we are prudent. We think we are prudent forecasters. We get accused of a lot of things, but we are prudent. we get accused of a lot of things but we are prudent We think we are prudent forecasters. we think we are prudent forecasters

Speaker 1: All right. Wanted to ask about SG and A investment. So you have started to see some payoff from having dedicated sales force, preferred apps and a GammaRay. What goes into the decision to like how do you expect to evaluate the need or opportunity for future SG and A investments? All right. all right Wanted to ask about SG and A investment. wanted to ask about sg and a investment So you have started to see some payoff from having dedicated sales force, preferred apps and a GammaRay. so you have started to see some payoff from having dedicated sales force preferred apps and a gammaray What goes into the decision to like how do you expect to evaluate the need or opportunity for future SG and A investments? what goes into the decision to like how do you expect to evaluate the need or opportunity for future sg and a investments

Speaker 2: One of things we said was if this ten year realization actually happens, there's a limit to what you can do as far as the number of sales people that you want to put in the field. You can overwhelm an office. Right now in the DMD space, as an example, it's just very crowded. And there are a lot of issues surrounding that. And you when I was in big pharma I worked for four big pharmas. One of things we said was if this ten year realization actually happens, there's a limit to what you can do as far as the number of sales people that you want to put in the field. one of things we said was if this ten year realization actually happens there's a limit to what you can do as far as the number of sales people that you want to put in the field You can overwhelm an office. you can overwhelm an office Right now in the DMD space, as an example, it's just very crowded. right now in the dmd space as an example it's just very crowded And there are a lot of issues surrounding that. and there are a lot of issues surrounding that And you when I was in big pharma I worked for four big pharmas. and you when i was in big pharma i worked for four big pharmas The strategy was you hold them, I'll hit them. That's how we promoted to doctors. Right? We just kept pounding away on the docs, pounding away. And you don't do that in in orphan rare. The strategy was you hold them, I'll hit them. the strategy was you hold them i'll hit them That's how we promoted to doctors. that's how we promoted to doctors Right? right We just kept pounding away on the docs, pounding away. we just kept pounding away on the docs pounding away And you don't do that in in orphan rare. and you don't do that in in orphan rare You lose a lot of credibility. You have to be thoughtful. And so the 16 the 12 sales representatives we have on the DMD side is right. Okay. How we go about reaching the patient through nontraditional means, direct to patient, you know, social, etcetera, that's an area we see a lot of opportunity. You lose a lot of credibility. you lose a lot of credibility You have to be thoughtful. you have to be thoughtful And so the 16 the 12 sales representatives we have on the DMD side is right. and so the 16 the 12 sales representatives we have on the dmd side is right Okay. okay How we go about reaching the patient through nontraditional means, direct to patient, you know, social, etcetera, that's an area we see a lot of opportunity. how we go about reaching the patient through nontraditional means direct to patient you know social etcetera that's an area we see a lot of opportunity Working with the patient advocacy groups is another area where we see a lot of opportunity to gain credibility. Our business model is one where we because we buy and build, we come late to the game. The product is well developed. Other people have done the work and maybe not done as much work as they should. And so we have to make up ground. Working with the patient advocacy groups is another area where we see a lot of opportunity to gain credibility. working with the patient advocacy groups is another area where we see a lot of opportunity to gain credibility Our business model is one where we because we buy and build, we come late to the game. our business model is one where we because we buy and build we come late to the game The product is well developed. the product is well developed Other people have done the work and maybe not done as much work as they should. other people have done the work and maybe not done as much work as they should And so we have to make up ground. and so we have to make up ground So we find out we're getting a product. It's immediately accretive or nearly immediately accretive, which means we have to get in touch with the patient groups really fast. And we generally show up and they're like, who are you? We have to build credibility really quickly. It's very challenging. So we find out we're getting a product. so we find out we're getting a product It's immediately accretive or nearly immediately accretive, which means we have to get in touch with the patient groups really fast. it's immediately accretive or nearly immediately accretive which means we have to get in touch with the patient groups really fast And we generally show up and they're like, who are you? and we generally show up and they're like who are you We have to build credibility really quickly. we have to build credibility really quickly It's very challenging. it's very challenging But it's the model we adopted and we like. So when we think about the opportunity on cancer LEMS and idiopathic LEMS on the Firdapse side, we see this as two markets. We don't think there's a need for more sales representatives. There may be a need for effort at the top of these GPO decision maker, but that's not significant. Okay. But it's the model we adopted and we like. but it's the model we adopted and we like So when we think about the opportunity on cancer LEMS and idiopathic LEMS on the Firdapse side, we see this as two markets. so when we think about the opportunity on cancer lems and idiopathic lems on the firdapse side we see this as two markets We don't think there's a need for more sales representatives. we don't think there's a need for more sales representatives There may be a need for effort at the top of these GPO decision maker, but that's not significant. there may be a need for effort at the top of these gpo decision maker but that's not significant Okay. okay So the SG and A profile is beautiful for this this opportunity, for all of the opportunities we have. Yeah. And we're ratcheting down all of our investment in Fycompa because it is actually generic. We stopped we ratcheted down significantly in January, And it's mostly social media and samples through the end of the year. So the SG and A profile is beautiful for this this opportunity, for all of the opportunities we have. so the sg and a profile is beautiful for this this opportunity for all of the opportunities we have Yeah. yeah And we're ratcheting down all of our investment in Fycompa because it is actually generic. and we're ratcheting down all of our investment in fycompa because it is actually generic We stopped we ratcheted down significantly in January, And it's mostly social media and samples through the end of the year. we stopped we ratcheted down significantly in january and it's mostly social media and samples through the end of the year

Speaker 1: So on your business strategy, you have a strong balance sheet. How are you thinking about potential BD opportunities in terms of therapeutic areas of interest, alignment with your current portfolio versus adding to it, and then stage of development of assets? So on your business strategy, you have a strong balance sheet. so on your business strategy you have a strong balance sheet How are you thinking about potential BD opportunities in terms of therapeutic areas of interest, alignment with your current portfolio versus adding to it, and then stage of development of assets? how are you thinking about potential bd opportunities in terms of therapeutic areas of interest alignment with your current portfolio versus adding to it and then stage of development of assets

Speaker 2: Sure. So our we're a CNS focused company, but we believe that the infrastructure that we have to help patients get on drug, stay on drug, and optimize their dose is applicable to any therapeutic area. We get to ask the question, if you got a new drug, would you put it on top of your current sales forces? My answer is typically no. Because the SG and A is so strong here and so light, we can afford to put 12 people, 14 people, 16 people on the field, and it would be positive payback for us. Sure. sure So our we're a CNS focused company, but we believe that the infrastructure that we have to help patients get on drug, stay on drug, and optimize their dose is applicable to any therapeutic area. so our we're a cns focused company but we believe that the infrastructure that we have to help patients get on drug stay on drug and optimize their dose is applicable to any therapeutic area We get to ask the question, if you got a new drug, would you put it on top of your current sales forces? we get to ask the question if you got a new drug would you put it on top of your current sales forces My answer is typically no. my answer is typically no Because the SG and A is so strong here and so light, we can afford to put 12 people, 14 people, 16 people on the field, and it would be positive payback for us. because the sg and a is so strong here and so light we can afford to put 12 people 14 people 16 people on the field and it would be positive payback for us So when we think about the opportunity to continue to invest BD, we're looking at products that are we're therapeutically agnostic, immediately accretive, nearly immediately accretive. We want to stay below a $500,000,000 peak year sales for us right now. Because when we show up at the auction, if it's above 500, you have other companies that have better balance sheets bigger than us, and then Steve and I are buying them coffee. So we don't really participate. So if we stay below $500 it's a really good opportunity for us. And so we're that's what we're looking at. So when we think about the opportunity to continue to invest BD, we're looking at products that are we're therapeutically agnostic, immediately accretive, nearly immediately accretive. so when we think about the opportunity to continue to invest bd we're looking at products that are we're therapeutically agnostic immediately accretive nearly immediately accretive We want to stay below a $500,000,000 peak year sales for us right now. we want to stay below a $500,000,000 peak year sales for us right now Because when we show up at the auction, if it's above 500, you have other companies that have better balance sheets bigger than us, and then Steve and I are buying them coffee. because when we show up at the auction if it's above 500 you have other companies that have better balance sheets bigger than us and then steve and i are buying them coffee So we don't really participate. so we don't really participate So if we stay below $500 it's a really good opportunity for us. so if we stay below $500 it's a really good opportunity for us And so we're that's what we're looking at. and so we're that's what we're looking at

Speaker 1: All right. And wanted to leave the audience with kind of a parting statement. One year from now, what could be your top one to two achievements for the year? All right. all right And wanted to leave the audience with kind of a parting statement. and wanted to leave the audience with kind of a parting statement One year from now, what could be your top one to two achievements for the year? one year from now what could be your top one to two achievements for the year

Speaker 2: Getting closer to closer. I want to be real clear on this. Closer to full enrollment on the SUMMIT study. Getting closer to closer. getting closer to closer I want to be real clear on this. i want to be real clear on this Closer to full enrollment on the SUMMIT study. closer to full enrollment on the summit study

Speaker 1: Okay. Okay. okay

Speaker 2: All right? And beginning to look at what data we could harvest from that. And then our cancer LEMS initiative has really hit the ground and is really moving forward. Because we have we're on the care pathways. We're contracting with folks. All right? all right And beginning to look at what data we could harvest from that. and beginning to look at what data we could harvest from that And then our cancer LEMS initiative has really hit the ground and is really moving forward. and then our cancer lems initiative has really hit the ground and is really moving forward Because we have we're on the care pathways. because we have we're on the care pathways We're contracting with folks. we're contracting with folks And the patients are, in that concentrated environment, really gaining the benefit of the therapy. And the patients are, in that concentrated environment, really gaining the benefit of the therapy. and the patients are in that concentrated environment really gaining the benefit of the therapy

Speaker 1: Very positive. So with that, thanks everyone for attending. Thanks so much Rich and Steve for being here. Very positive. very positive So with that, thanks everyone for attending. so with that thanks everyone for attending Thanks so much Rich and Steve for being here. thanks so much rich and steve for being here

Speaker 2: Thank you. Thank you. thank you

Speaker 1: Have a great rest of your afternoon. Have a great rest of your afternoon. have a great rest of your afternoon

Speaker 2: Thanks. You too. Thanks. thanks You too. you too