Good afternoon. Hello everyone, thanks for joining us for the first day of the Muscular Dystrophy Symposium, sponsored by Cantor. I am Charles Duncan, I'm a Senior Biotech Analyst on the team, and it's a pleasure to introduce the next presenting company. This is Catalyst Pharmaceuticals, and I have the pleasure of having Mr. Rich Daly, the company's CEO, and Dr. Steven Miller, the company's Chief Operating Officer, on the line today. So Rich and Steve, great to see you, thanks for joining us. Thanks Charles. Thank you all. So I'm excited about this fireside chat. Why? Because we're going to be talking about Catalyst Pharmaceuticals in a way that, frankly, we've never talked about. I've covered the company for several years, and the company has done well by doing good for patients with not one but two commercial products. But it is now launching a product that is relevant to the muscular dystrophy community, the Duchenne muscular dystrophy community, and that is the product called AGAMREE. So we're excited to talk about that today. But before I do, and I've been deep on that product, Rich, you joined the firm about a year ago or so. I know you've been on the board for a while. So tell us a little bit about why you were interested in leading Catalyst. Sure. And Charles, thanks for the opportunity to have both Steve and I with you today. We really appreciate it. We really appreciate the opportunity to talk about AGAMREE as well. I was with the board, as you said, for about nine years, and I had the opportunity to join the firm on January 1st as the CEO. So I had the opportunity to be with the firm and use my years of experience in the industry and work with Catalyst as it was developing its first drug, FIRDAPSE, for Lambert-Eaton myasthenic syndrome. And as the company went from an R&D organization to an R&D&C, or R&D and commercial organization, which is where I've really spent the bulk of my career. So it's really exciting to me as we handed the reins off from Pat McEnany to myself and really to take the opportunity to take the company to the next level, going from one product to three products in a year, building the organization, building the infrastructure. Steve's been integral to that and really excited about this opportunity to pivot the company to multiple products, multiple therapeutic areas, and really looking at increasing our footprint internationally as well and building our business development capabilities as well, and looking to expand and help more patients as we go forward. Wanted to ask Steve a question as well because Steve's been with Catalyst for many years, and I've interacted with him throughout the time of coverage. And it's been clear that with the FIRDAPSE approval and then successful launch, that that has opened up the opportunity set for Catalyst. And so Steve, do you have anything to add with regard to business development efforts in the past and the way that you've been able to access new opportunities and perhaps going forward? Nothing specific, but tell us your perspective. Well, first, you're right. I've been with the company a long time, 17 years. And with the development and commercial launch of FIRDAPSE, we basically had the appropriate experience to do all of that clinical development work and then to ultimately successfully launch the product, as well as develop a very thorough intellectual property estate to protect FIRDAPSE. So we have a lot of the skills necessary for both the development of products and the commercialization of them. And that skill set is very valuable for reviewing future assets for potential acquisition. As Rich will probably explain, the company has shifted a little bit to buy and build for our approach, at least for the short term. Yeah, so why don't we talk about buy and build a little bit? Before we get into the nuts and bolts of AGAMREE and the opportunity set for that drug to serve the needs of muscular dystrophy patients, you had a neuromuscular asset, or you have one, FIRDAPSE. Then you also went into epilepsy. And now coming back to neuromuscular with AGAMREE, I guess, what makes you interested? What are some of the commercial synergies that you see in your current portfolio of products? And what made you interested to go into muscular or DMD? So we see ourselves fundamentally as an orphan drug company that has specialty in central nervous system disorders. And the opportunity to build the franchise, build the company around Lambert-Eaton myasthenic syndrome, or LEMS, was central to the success of the company. So the execution, the ability to continuously execute and execute successfully around LEMS, I think, is a hallmark of who we are. I think, as Steve pointed out, we get a lot of credit on the execution for commercialization, and we're really proud of that. I think where we deserve a lot more credit is on our evaluation from a regulatory, a clinical standpoint, and an IP standpoint, and building our IP portfolio as well. We've done a really good job of looking at assets and bringing those assets in that will build, I think, a solid base of income that will enable us to build our balance sheet and buy more products and help more patients. So going back to the FIRDAPSE success, we were looking for things that would stabilize and diversify our income set. So we bought FYCOMPA as a way to really get a solid foundation, if you will. FIRDAPSE is great. It's continuing to grow at 15%-20% a year. That's a five-year-old product, so solid performance for five years. And we see that continuing for the midterm, next three to five years. We have a solid pipeline of patients we can see that are undertreated in the marketplace. But FYCOMPA then adds a stability element to that. So we build a broader base. We really like that. But coming back foundationally to who we are, we're an orphan drug company that's in CNS. And we saw the AGAMREE opportunity, and we just couldn't pass it up. This is a community that needs a lot. We believe that our skill set in serving those patients is very, very strong. And we believe we're the best organization out there to do that. And so when we had the opportunity, we really seized the moment. And we believe that our service is out there for patients is incredible. And our commitment to this community is really, really strong. And we look to continue to build our commitment in the DMD community into the future. It seems to me that some of the success with the LEMS community, some of the fundamentals, the learnings, if you will, identifying patients and then providing patient access and ensuring access and helping to create clinical value for those patients over time, those are learnings that can be applied, certainly in DMD, perhaps even in epilepsy as well. Yeah, I think you're right, Charles. So when we look at the opportunity we have with the DMD community, the drug was approved in the fourth quarter, as you know. AGAMREE was approved there. And we started enrolling patients prior to the launch of the product, which occurred on March 13th. And we had basically 3.5 months to get those patients on board. And because the drug was approved, we began to help those patients enroll for insurance approval. While we only had about two, 2.5 weeks in the first quarter for sales, if you will, because we did launch on March 13th, so we would expect a pretty thin quarter in the first quarter, we really expect a very solid jump start in the second quarter because we had a very solid line of patients coming to the table saying, "We really want this drug." So we're expecting very solid performance second quarter on. And we stand by our guidance of $25-$30 million. We're very happy with the performance of the drug so far. And one of the things we learned with FYCOMPA, which really serves us well, is initially when you launch an orphan drug, it takes about 4-5 weeks to help those patients move through the insurance process initially. But as you gain maturity and you gain a relationship with that product in the insurance world, you can shrink that time to 4-5 days. And we're seeing that. We're seeing it takes time right now for AGAMREE. But because we have this expertise, we're able to move that time frame very quickly to a much shorter time frame. So again, we expect to be able to do that for the benefit of the patients. Now, before we talk more about AGAMREE and its profile and what it can do for DMD patients, you mentioned synergies with regard to interactions with reimbursement authorities. Or you alluded to that. I guess I'm wondering, is that product-specific, or does Catalyst start to build a brand value that is recognized by some of the reimbursement authorities that help to facilitate progress with those interactions? That's a really interesting question. I think there are some specificities that would go with a certain therapeutic area. Then I think there are generalities. I think understanding the challenges that orphan communities face is a generality that I think we can have expertise in. But each community has its own challenges. I think when you get in with the community and one of the benefits of really being engaged with the community for three months before we launch was truly understanding what are the issues, how can we be helpful, what do you need, and really embedding ourselves with that community and talking with the community is incredibly helpful to understanding how it's different from LEMS and how we can be a better partner for that community. So we really took our time and really tried to understand what were the subtleties and what's really needed for that community. Why don't we talk about AGAMREE or vamorolone's clinical profile a little bit and bring Steve into the discussion because he was part of the diligence team that identified the opportunity and led it. And I guess a question that I have for you is, what is it about vamorolone or AGAMREE that makes it stand out in terms of really providing clinical value for DMD patients? Well, Charles, during the clinical trials, both the caregivers, the parents of the patients, as well as the physicians who were involved in the trial, observed that there were less behavioral problems with the patients who were involved in the study. There also was what appeared to be a better outcome in terms of weight and growth. Some of these doctors had the opportunity through both expanded access programs as well as the trials themselves to use the drug long enough to see a longer-term benefit in the patients. Much of this data is actually published and is available to the community. One final thing is that that community is a very tight community. They talk to each other about new therapies and what works and what doesn't work. As the caregivers and the doctors learned more about vamorolone, word spread throughout the community that the drug appears to have substantive benefits over other therapies that are available to those patients. Yeah, we've read those papers. It's quite clear that relative to prednisone, certainly, this drug stands out. Can you help us understand how it stands out relative to other competitors in the field and what makes a dissociative or dissociated corticosteroid? Well, the dissociative term really refers to what's considered by the pharmacologist to be a disconnection between potential adverse events to the drug and its potential therapeutic benefits. And so, for example, I mentioned the behavioral changes. There are published articles that said that there was virtually no adverse behavioral changes in the patients who were on therapy. The drug itself actually has modifications to it that change its binding characteristics to both the mineralocorticoid and glucocorticoid receptors. And those changes alter both DNA suppression and DNA transcription that results in all of the benefits that you expect to have from the drug, but yet seems to significantly potentially could reduce the adverse events that result from this drug. And in particular, this drug is the only corticosteroid that is a mineralocorticoid antagonist. All of the adverse events, including cardiovascular health, should be significantly reduced with this drug. Only long-term usage will ultimately bear that out. Now, do you anticipate that corticosteroids will continue to be the backbone of therapy with increasing visibility in gene therapies? We talked to a company earlier today that is developing a gene therapy. So as you've conducted diligence in this space, it would seem to me that corticosteroids are important for muscle inflammation. But what's your perspective on? Well, you are actually right about the corticosteroids being a backbone for therapy. Our expectations is that they will continue to be. As you know, the gene therapies and exon-skipping therapies provide some very interesting therapeutic options for patients and some level of benefit for those patients as well. But it's clear from all of the data that's been available that the corticosteroids will continue to remain a backbone of therapy in addition to those promising new therapies as well. I think, Charles, it's really important to realize it's not only our opinion. It's the opinion of the physicians we encounter. They're seeing these therapies come along, which are great therapies. We're seeing good advancements here, which to benefit the patients. And consistently, the physicians we encounter, not just the key opinion leaders, but the physicians across the board, they're continuing to say the steroids will be the backbone of therapy. It's the first drug that patients go on. And it's the drug that they stay on the longest. Yeah. And all the clinical studies of the gene therapies are done on a background stable prednisone or deflazacort or, hopefully, in the future, AGAMREE. That is correct, Charles. Yes. The corticosteroids remain a therapy all throughout those trials. And so those patients who were treated continue to receive the corticosteroids in addition to the other new therapies. Now, when you consider the market dynamics, I understand that there are going to be some changes with regard to the protection for Emflaza. You've clearly worked that into your diligence. But what is your perspective on that? So we did our market research. We went out and talked to 17 active decision makers in the payer space prior to launch. And we said, here's our product profile, target product profile, which matched our label, obviously. Here's the market landscape as it sits right now, plus the introduction of a generic Emflaza. What's your anticipated utilization of AGAMREE? And the utilization was no step through beyond prednisone, which you just expect is going to be the starting point. And so they said, as long as you price it appropriately, which was at or slightly below the price of branded Emflaza. And our assumption on the generic Emflaza was 80% of the branded Emflaza. And when it launched, when the Emflaza went generic on February, I believe it was February 8th or 9th. I believe so. Yeah. The pricing that came out was 80%. So it was in line with our assumption, 80% of the branded and Emflaza. So all of our assumptions were correct and in line with the market actions. So we anticipate the drug will be there. But our forecasts are in line with the assumptions. So we're very comfortable where we sit right now. Remind us of the pricing of AGAMREE and it sounds like your logic behind it. But just put a finer point on that. So the pricing is for 40. I'm sorry, for a 28-kg patient. So this is a drug that goes by a weighted patient. Weight-based dosing. Yeah. Right. It's about $130,000 a year, which, again, is less than the price of competitors. Okay. And that weight-based dosing, obviously, there's a range, a big range. Oh, of course. Yeah. Depending on the weight of the patient. Right. Where do you anticipate AGAMREE to be used first? So when you look at the patient enrollment forms that you have been tracking initially, are they for younger patients, maybe earlier-stage patients, or are they for older patients? So we haven't given patient numbers yet. I'm not sure that we will. But what we're seeing is, actually, it's a conversion. So we're not seeing very many naive patients at this point in time. The majority of patients are coming from prednisone, and we're seeing a pretty healthy split between Emflaza and prednisone. Okay. In terms of switches, it would make sense that they'd be coming from prednisone because, as you mentioned, there's a step through to get there. Right. Right. Right. But in terms of. We'd assume they'd be a little bit older because if they're not naive, then they're on prednisone. The step through makes sense. Some of the clinical benefits that Steve mentioned and I've seen in the literature, behavioral challenges, but other things like bone mineral changes and height, velocity, all that stuff, it seemed to me that that would certainly accrue to the benefit of younger patients, maybe more than older. So where do you see the real interest or demand of starting on AGAMREE coming from? So I think where you're going is a short-term and a long-term benefit. I think behavior is what will be easiest to measure and easiest to see. And if there's a behavioral benefit, you'll see it pretty quickly. And I think that's where the benefit accrues to not just the patient, but the patient's family, the caregivers. If the young boys are being mainstreamed in schools, it accrues to the student population they sit with, the teachers, the teacher's aide, I think everybody around them. So I think that's the fastest benefit that will accrue to the patient and the community around them. And obviously, I think there are psychosocial issues that go along with height. If a patient can grow, I think there's a benefit there. They can grow with their peers. I think there's opportunities there. Bone density, obviously, as Steve pointed out, and Charles as you pointed out, these are things that will take time to see and to see the benefits. But I think the immediate one is improved behavior. Okay. Steve? Comments? Okay. Anything to add? Well, yeah. I was going to add that Richard's exactly correct, that some of those benefits, we'll take time to see. And so we actually have invested in a registry where we're going to track patients for at least five years. It will include about 25 sites, about a quarter of the physicians who treat LEMS. They have to be physicians and sites who are used to doing research and able to do so in appropriate quality to the FDA's requirements. We'll be looking at that data about once a year, perhaps more often, looking for long-term benefits of the drug and proposing changes to the product labeling from time to time as a result of that data. We'll also be comparing it to data that we'll purchase from the FOR-DMD program that enables us to have baseline historical control data for these patients and so that we can use the FDA's guidances on real-world data to make changes and expansions to the label with appropriate real-world data. Okay. So if we go back to your let's see. You had guidance of $25-$30 million for this year of revenue from AGAMREE, correct? That's correct. Are there key performance indicators or metrics that you're tracking that maybe you're not going to share in great detail? In addition to patient numbers, would they be patient age or certain geographic spread? What are things that you're looking at that is going to encourage you to continue to invest in the franchise? There are about 100 DMD centers in the U.S. with 295 writers who write 80% of the prescriptions out there. Tracking the centers and where we're getting the prescriptions from and the physicians who are writing become the core of the metrics we'll be tracking. There's also the mix between commercial and Medicaid. This is typically a 50/50 split. I think that's a key one too. I think age, as you pointed out, is one. The source of patients, where are the patients coming from? This is not a growing market. The opportunity is to take patients from other therapies, obviously. What's the source? Where are they coming from? Are they coming from prednisone? Are they coming from Emflaza? And then what's the age? How early can you get the patient on? Are we truly getting that benefit from the patient for the patient, rather, for their behavior so that the families are seeing that benefit and so they want their son on earlier? I think those are just some of the core metrics we'd be looking at to see success. And Rich, when you think about the LEMS situation with FIRDAPSE, where there was a little bit of noise around competition initially, but that was a little bit of a different situation. It wasn't as competitive. But it does seem to be an ultra-orphan disorder as well, not as many patients. But how does the number of script writers compare in terms of FIRDAPSE versus for AGAMREE? So this is a very insightful question, Charles. So they are fundamentally two different markets. And the LEMS market, the average physician will have one LEMS patient in their career. And so it's just LEMS is widely dispersed. And it's a prospecting market. We go out, and we help the physician understand what LEMS looks like. Many patients are misdiagnosed having myasthenia gravis. And then their patient journey is very, very long until they get an accurate diagnosis. Here, 90%-95% of the patients in the Duchenne's market are diagnosed. And again, they are highly concentrated in these 100 centers with these 250 physicians who are specialists. Prospecting doesn't occur in this market. So we know exactly where these physicians are and the patients are. So they're very, very different markets. So it's a very interesting place to be. And it gets back to your question earlier. Are there core skills that we have that make us good? Yes and no. I mean, there are things about these markets that we bring to it, like helping the patient get on therapy and making sure that the patient stays on therapy. We have copay programs and the like. We guarantee a patient, once they start therapy, regardless of their ability to pay, they will stay on therapy. That's a guarantee. We're sure that we're doing all we can for the community. There are parts of it that are just very, very different. It's incredibly interesting. Working with these communities as they are and approaching them is a hallmark of who we are. It sounds like it's not so much a disease awareness situation, obviously, as it is an awareness of AGAMREE and its clinical profile and how it really is different, very different than prednisone or Emflaza. That's right. I would say that's a very accurate assessment. As we think about, Steve mentioned the registry, but as we think about ways to gauge success, obviously, there's some revenue line. But other ways to look at how you're executing, your team's executing. What are some of the programs that you're considering over the course of the year that we should keep track of? I think physician education, as you said, looking at participation in that. I think patient retention because this is, as we said, it's not a growing market. We are gaining patients from other products right now. The value of the product will be the tail of the tape, if you will, will be is the product that different that we can hang on to those patients and hold those patients because of the value we create for those patients? So looking at the opportunity to be at the scientific meetings, creating data based on the clinical profile and the clinical performance through the registry, as Steve talked about. And then looking at, is this drug, is AGAMREE different enough that it could be the steroid of choice for all orphan conditions that require a steroid? Is there a lifecycle management opportunity here that goes beyond Duchenne's? Because it has these benefits that fundamentally move it away from these adverse events that you experience with other steroids in orphan conditions and exploring those opportunities. And while we're focused on the launch of AGAMREE, I mean, hyper-focused on the launch of AGAMREE, simultaneously, we have another team looking at that as well and being sure that we can focus as we look for opportunities to improve the therapeutic footprint and increase the therapeutic footprint for AGAMREE. So I want to get into that a little bit more deeper in one second because I'm intrigued. It does certainly seem like, yeah, the benefits of corticosteroids is no more broadly within neuroinflammatory disease. But let's first talk about the choice of the use of AGAMREE and to switch from prednisone or even to switch from deflazacort. How much time does a patient or a patient caregiver have to wait to see that benefit? Do you think that a refill rate after six months or a year in terms of persistency is going to give you some insights on getting traction in the market? So just so I understand your question, are you asking how long will it take to see the benefit of AGAMREE? Yeah. It's really how long will it take your team to decide that benefit is being seen with adopting AGAMREE and switching over to AGAMREE? Why? Because patients or patient caregivers are refilling prescriptions. Our understanding of the market is that we can get these switches pretty quickly from other drugs because physicians are filling them on a monthly basis. Yeah. But do they last? How many months? Right. So I think I understand where you're going. So what we'll be able to see is, to get to your point, we'll be able to see pretty quickly if the patient is satisfied with the product because if they're not, they can switch back. So we should be able to see very quickly. Patients are not getting three months or six months worth of a prescription. And then they have to sort of let it wind down. They get a prescription, then they get another and another. So when we are successful, we will know the refill rate, the persistency, to your point, should be there. And so we will know. Really, after, call it, a quarter or so on the market or maybe two, we'll be asking you about persistency. Yeah. I think 2-3, the persistency rate, we should be able to see persistency. Yeah. Okay. Very good. And how about ex-U.S.? I know that you have a license to Canada and Mexico. What is your plan there? Have you launched there, or do you plan to? Well, so we would partner. So we look for partnership opportunities. Our focus is on building our business in the U.S. and then partnering our opportunities when we license a product or we acquire a product. So we would look to license. So we're currently seeking partners in Canada. And we would welcome the opportunity to have those discussions in Mexico as well. Let's see. If you do a partnership, will you need to pay some of that amount? What is the royalty that you pay to Santhera? Can you give us a little bit more color on that collaboration or those licensing terms? Steve, I don't think we've gone into depth on that, have we? I don't believe so. Some of the terms may be in an 8-K. Yeah. We can come back and talk to you about that in the court. Yeah. Okay. So you are looking for extending the franchise. Absolutely. ex-U.S. in Canada and Mexico. Okay. All right. Let's come back to that idea of lifecycle extension or lifecycle management and potentially other indications. I know that you probably don't want to reveal all your cards. But if you had a favorite that you would look to help us understand your creativity, what would that be? Well, I think it's a little too early to say. Again, we're really focused on the launch. But I think our theme here is, can we be the steroid of choice in all orphan conditions or in orphan conditions that require a steroid? And again, just really focused on the launch, making sure that that gets off the ground and then coming back to it and then investing appropriately in opportunities that make sense. That's how we're approaching it. Okay. Now, with regard to the broader Catalyst story, let's get back to epilepsy because this is an area we do a lot of diligence on. We are impressed with the innovation in epilepsy and the potential for rare epilepsies to continue to gain traction and with drugs that have really very unique profiles. So are you considering epilepsy in licensing or other areas of neurology as well? Yeah. I think we're an orphan company in CNS. And if there's a rare epilepsy opportunity, and I would say, Charles, we are considering at any point in time a broad array of opportunities. We have a team in epilepsy that is deeply experienced. And you know this is a highly competitive space. The science here is very, very complex. And so to leverage that team would be phenomenal. So we are looking there. It is, as you know, it's just to reiterate, highly competitive, which means generally, you pay higher rates of discounts. So I think we just have to be very selective if we decide to continue to go in that direction. So it's just a more challenging space from a commercial standpoint than your typical. But if it is orphan, we are all on board. FYCOMPA has a pretty interesting profile. It's the only AMPA-based drug that's approved. So a question that I have for you is, is this a drug that easily goes generic, or is there something about epilepsy that really prevents generic entry? I mean, it seems to me that an epileptic patient, if they're stable, it's hard to switch them. I think it's harder. I think we assume generic entry. Steve can talk about the generic timeline. But we assume generic entry, and we assume that there will be erosion. We assume that it just won't look like your typical rapid decline. It's going to be a softer decline because, unlike a cardiovascular or diabetes drug, the ramifications of not having the same dose on board every day are just unacceptable. So Steve, I don't know if you want to talk about the timeline. Sure. The current composition of matter patent expires in May of 2025. We do have a polymorph patent that we are considering enforcing as well. Eisai didn't for a number of legal reasons actually related to the composition of matter patent. But that is something that is still under evaluation. Okay. That's helpful, Steven. And remind us of the FIRDAPSE IP as well or orphan drug protection. FIRDAPSE has a total of nine patents, six of which are listed in the FDA's Orange Book. There are ongoing patent infringement suits at this time. It has patent protection until February of 2037. Last question we're going to have time for, and that's just bringing it back to AGAMREE and Duchenne muscular dystrophy and your vision. If you were to look out three to five years, where do you see this market? Do you see additional gene therapies? Do you see a role for AGAMREE and corticosteroids? I think the answer is yes there. But what would you like Catalyst to be known for in three to five years? I think we would want to be known as a key player in the Duchenne's market, committed to Duchenne's. We would also want to be known as a player in a broader array of the orphan space as well. We think that we have a lot to offer in the Duchenne's market. I do think that there will be a broader offering in the gene therapy space. We do believe that steroids will be the backbone and continue to be the backbone. We believe we're going to be a central player here for a long time to come. I wonder if you could even go out of steroids into some of these other innovative platforms. I think it could be a launching pad for us. We're very excited about that opportunity to be in this space, be in this community, and be looking at opportunities to contribute in a greater way to the Duchenne's community. Not to be good, but it sounds like the future lies ahead. We're pretty much out of time, Rich and Steve. I appreciate you taking time with us today. Thank you very much for sharing the AGAMREE story, the Catalyst story. We're looking forward to seeing you make an impact, much like you have with FIRDAPSE, in terms of doing well by doing good. Thank you, Charles. Thank you, Charles. We really appreciate your time.