Yeah, so I think we'll get started. Welcome to the afternoon of the second day of the Cantor Global Healthcare Conference. My name's Charles Duncan. I'm a senior analyst here at the firm, and I cover a lot of neuroinnovators. And these are companies that develop and commercialize drugs for the treatment of central nervous system disorders, both psychiatric as well as neurological. And Catalyst Pharmaceuticals is a company that has a long history, and I've known this team for quite some time, and it's been variously called a neuroinnovator. But in this last two years, it has transformed itself. Now, that was on the back of an approved drug, which they did very well with, but subsequent to that, they have broadened their pipeline. They've also broadened their commercial portfolio. They have revenue stream that is profitable, and now they have a new CEO. So it's a pleasure to introduce Rich Daly, the company's CEO, and Dr. Steven Miller, the company's chief operating officer and head of R&D, and they're gonna tell us a little bit about Catalyst and the future that lies ahead for you. Thanks, Charles. Good to be here. Thank you, Charles. Thanks for having us. Well, thank you. Thanks for being here. Thank you ... with us. Great, long day. Well, yeah. Had enough espresso. Give me a break. Yep. But this is a great story that, I think is underappreciated by the Street, in part because of how Firdapse came to be, and some of the, you know, call it mishigas around that, but you really have done well by treating patients well. You've done good by treating patients well, if you will, or somehow that works. And the bottom line is that you have a drug now that has kind of a new lease on life, Firdapse, with a new dosing regimen, and then a couple of other drugs, one which is pretty exciting, could be meaningful in the treatment of DMD. That's Agamree. So when you think about this last year, Rich, and you've only been here for a year, what are you most proud of having achieved there at Catalyst? So, Charles, thanks for the opportunity to be here. We're really excited, and we really appreciate the support of Cantor and your support as well. I think what's most exciting. I've been- I was on the board, as you know, for nine years of the company, and maintaining the sales and the growth of Firdapse, which I think is a really exciting product, while we're launching another product, I think was probably the biggest challenge that we had. You know, can a company that is an emerging company continue to grow its core business while it takes on another product? Because as a buy-and-build company, what we wanna do is we want to attract other companies to us. When they say they have a product that they want us to partner with them on, can we prove that we can actually maintain our core business while we take on these other products? And that's a big challenge as you transform your company, and this is a positive transformation. So we look at this as a real challenge and a real opportunity, and the launch of Agamree has been an incredible success while we're continuing to grow Firdapse at 15%-20%. So we're really excited about that, and we keep our underlying business solid and growing, and we can successfully launch products. That's been, I think, probably the most exciting thing. Steven, you've been here for quite some time. Do you have perspective on the last year and your most proud moment thus far? I think our most proud moment was being able to do the due diligence on the new acquisitions that were brought in just in the last year or so, and the fact that we were able to, upon acquiring them, successfully commercialize them. Yeah. So we're gonna talk about that successful commercialization because for Agamree, it's fairly new. So I'd love to hear why you believe that it's been successful, but just touching on Firdapse and being able to continue to grow it, what gives you confidence in your ability to grow Firdapse over the coming year? When we look at the way that we have our sales force deployed, what we do is it's really a prospecting opportunity. Our sales organization is out, and our medical organization is out talking to physicians, and the number one misdiagnosis for Lambert-Eaton myasthenic syndrome is myasthenia gravis. And when we see certain prescriptions that are written in physician offices, we will send a sales representative in and talk to that physician because the average physician will only see one Lambert-Eaton myasthenic syndrome patient in their entire career, and so the treatment journey- Very rare. Very rare. Very. The treatment journey for that, I should say, the diagnostic journey for that patient is eighteen months from first symptom to actually getting therapy. So we have to work with the physician to say, "Hey, this is what it looks like. It's not myasthenia gravis. It's very closely associated, but you need to think about this differently." So we look for surrogate markers of prescriptions, and then we apply a sales organization and a medical organization to that, and we have a pipeline of 500 patients. HIPAA compliant, we don't know the names of the patients or anything, but we see the prescription written, and we can talk to the physician about those patients and say: What are you seeing? Help us understand. And half of our patients this month, this quarter, and this year come from that pipeline of patients. So we can continually say to somebody, "We believe we have 15%-20% growth next quarter, next year, and the year after," 'cause we continually refresh that pipeline with those patients. So we're always looking to the future, and that's what gives us great confidence. ... So really is doing well by doing good- Yeah -for patients for a really rare neuromuscular disorder. Yeah. What, three thousand? Anywhere from three to five thousand patients out there, yeah, and so- In the United States? Yeah, and it's a lot of work- Yeah -to keep that pipeline fresh. But you've been able to grow the drug and do so for a couple of reasons. One is that it works, and you've been able to identify patients, but you have a new growth opportunity that's in front of you with Firdapse, and that recently been granted a broader label in terms of dosing, the maximal dosing. Sure. So tell us a little bit about that. Steven? Sure. We recently had a supplement approved- Mm to increase the maximum daily dosage from 80 milligrams a day to 100 milligrams per day. There were about 40% of our patients who were already on the 80 milligram per day dosage, and our expectation is that a number of them have expressed and a number of others probably will want to increase their maximum daily dosage. Even patients on lower doses may also increase their dose simply because their doctor feels that since there's a larger maximum dose, that it leaves a little bit of headroom to treat that patient in case they get worse. As a result of that, we believe over the next few quarters that we will see about a 5%-10% increase in the sale of Firdapse. And that wasn't a walk in the park. That wasn't just- Uh Filing some paperwork. You had to prove that A-absolutely ... you should do that, you could do that. That's absolutely correct. The supplement included information that was efficacy data all the way up to 100 milligrams, as well as safety data that we had to accumulate over a number of years for patients at 100 milligram dose, and we also acquired Ruzurgi, and so we wanted to take advantage of their product, the data from that old product as well, and so we did a bio study to prove that the two products were bioequivalent. What other drivers to growth for Firdapse do you see that you can identify beyond just the pipeline that we talked about and the potential for a little bit higher dosing? Other drivers. First of all, any pricing increases that you anticipate or can speak to? Probably wouldn't, but really, it's a demand-driven event, or growth. Yeah, I think that we split the LEMS world into two different segments. Really, we think about it, and it... This is very simplistic, Charles, so we think about non-tumor LEMS or idiosyncratic LEMS. Yep -and then tumor LEMS. And this non-tumor LEMS is this prospecting world I just talked about. You gotta go out, and you gotta find the patients. It's, it's hard work. I mean, it's really, really hard work. And then there's the tumor LEMS, which is closely associated with small cell lung cancer. That's what I wanted you to talk about. Yeah. Yeah. This is a really interesting world. Yeah. There are 30,000-40,000 small cell lung cancer patients diagnosed every year. 3% of them have LEMS. The world thinks that a small cell lung cancer, it's a horrible diagnosis, absolutely, and the world thinks that these patients are gonna live seven months, and on average, they do. But if you get small cell lung cancer, you actually want LEMS. If you have LEMS, and you have small cell lung cancer, you're gonna live 17 months on average. The challenge is that- So it has prognostic value? Yeah, but the problem, one of the challenges is that nobody's testing for LEMS in the oncology community. Sure. And so helping- No one knows about it. Right. It's a neuromuscular disorder. Right, and the challenge that you have is that these patients are significantly compromised in their mobility, and 60%-90% of them have a Modified Rankin Scale of three, four, or five, which means they can't walk or they're bedridden, up to three, four, or five. So you've got a significant opportunity where simply taking an oral drug three times a day, they could be mobile, and in the 17 months that they have on average, you could have a patient who's living a higher quality of life. But because the percentage is so small, they're just lumped into the greater majority that's only gonna live 7 months. So we have a big initiative to say, "Hey, what can we do for these patients? How can we help the oncologist better understand what's going on with this segment of patients?" So this is, I think, a really good thing for us to sort of ferret through. So how do you break into the oncology world with your existing sales force? What can you do to enable that? Do you have some testing ability? We do. We actually provide the test for free. Can we get on the panel? Can we get into the care pathways? Can we figure out how to, you know, become a regular part of the physician's world? And quite frankly, I think that's gonna be really a challenge for us. But even without that opportunity, we expect to be able to grow 15%-20%, so this is an opportunity for us to grow even more, and so we're exploring this, and we're trying to figure out how we can be a little bit more, I would say, appropriately aggressive in this environment. We're gonna talk about IP, but beyond IP. Maybe we should talk about IP first, and then we can talk about ways to extend the franchise beyond IP, like with innovation. Why don't we first talk about the intellectual property behind Firdapse and, you know, its durability? Steven, you have some experience in this. I do. I manage that estate. I suspect you'll be cautious about strategy, but tell us your current perspective on the life cycle of Firdapse. Firdapse is currently protected by six patents. Okay ... listed in the FDA Orange Book, with expiration dates out to February of 2023. Catalyst is, You said, Steve, you said February of 2023? Oh, I'm sorry. He's gonna say, "Whoo! February 2037. Holy smokes. Thank you for correcting me. Well, we- Thirty-seven, he said. Thirty-seven. We're confident in the strength of that intellectual property, and we feel that the product has a long lifetime ahead of it. Why do you have that confidence? I see here, according to my associates, that some stuff has happened lately, like lawsuits being settled, and- Mm-hmm. Ah, it happens all the time. That's a very good question, Charles, and obviously you're watching not only our SEC filings but the public records related to the ongoing litigation, and you are exactly correct. There are some late-breaking news with regard to those lawsuits. First, we recently settled a lawsuit against one of the Paragraph IV filers, and their entry date in that settlement is February of 2037, which is the last expiration date of the last patent. Very good. And the second- That tells you something. Yep. The second thing that happened is- Yep ... one of the first filers has, we've dismissed the case against them, for one of the families of patents that end in 2034. And the reason we dismissed that case is because, they have basically given up, challenging those patents. And that's very interesting because they clearly have given up challenging those patents, in all likelihood because they felt that, it was unwinnable, the case. But more importantly, that filer, is one of the first filers, which means that not only do they feel it's unwinnable, but they feel that they're not giving up their chance to be among the first on the market as a generic because, they probably feel that the others can't win it as well. And so- Interesting ... that goes back to what I've been saying for quite some time, that we think we have a very strong intellectual property estate to protect Firdapse. Prior to Catalyst, you were at what company, and what did you do in terms of IP? Prior to Catalyst, I was at Watson Laboratories- Ah! more than seventeen years ago. Okay. With regard to intellectual property, in that regard, I actually was running the R&D department and was developing new products, and so I would work on with the patent attorneys to develop new intellectual property for those new products. Okay. So this is helpful and gives me a perspective. So when would you guess that Firdapse franchise could come to an end, if it did come to an end? Then we can talk about extending it. You're actually asking me to comment on the ongoing litigation, so I can't give you a- Okay ... specific date, but the information I just gave you about the late breaking news seems to indicate that it will certainly be sometime in the 2030s. Yeah. I also want to point out that we are pragmatic about this. We listen to what the defendants have to say, and if they have something useful for us that we believe is in the benefit of the shareholders, we'll act on that information. Okay. That's helpful. It's beyond our current model, so we're interested to hear this. Mm-hmm. Beyond the existing Firdapse, candidate or drug, what else can you do to, you know, extend that franchise? What can you do for patients to further innovate in Lambert-Eaton myasthenic syndrome? I think the real focus is trying to get to more patients and get to them faster. Like I said, on the idiopathic side of the non-tumor LEMS side, it's eighteen months. So our business is. Our business in general, everybody in this room, is very complex, but really, it boils down to get them on sooner, you know, increase dose if it's appropriate, keep them on longer, or lifecycle manage the product. I think for LEMS, I don't think there's much for Firdapse, I don't think there's much in the way of lifecycle management that we can do. So how can we shorten that time, that eighteen months? That's really, really critical. These patients will live a normal life on the idiopathic side. They have seventeen months on the tumor LEMS side. Getting more time with that patient, improving their quality of life, really critical. Upping them in dose, we've handled the hundred milligram. Yeah. Keeping them on longer, the number one reason why a patient drops off is because they have passed away from other causes. Product is relatively safe, and it provides an increased, enhanced, and quality of life. So we're really looking at shortening that time to diagnosis. That's our biggest driver, our quality driver right there. With 100 milligram, we've done, you know, I think we've done really good work, so getting them on sooner is our real driver now. Doing well by doing good. So you have this system called Catalyst Pathways. Yes. Help us understand that and what it does for patients to not only enable initiating dosing but also staying on drug. So this is a really good lead into what I think is probably a future question that you have, and I think everybody has, is business development. We believe that- Yep ... the inherent value that a company like ours delivers is for the patient and for the patient community. Mm-hmm. And people look at companies like ours, and they say, "Well, you know, you've got a sales force, you have MSLs, and you have products. Okay, what else do you offer?" The infrastructure we have that very few people see is what helps the patient get on drug and helps the patient stay on drug. And a lot of companies that are developing orphan drugs don't understand and don't see the cost and the rigor that's behind that, and this is what makes us a really good partner, is it's already there, it's highly developed, and it's excellent. And so by having this Catalyst Pathways, which we apply to Firdapse, and we apply to Agamree, it's a proprietary pharmacy. It's also reimbursement services. It's patient advocacy services, helping them understand educational services, what's available for them, and co-pay services, and directing them to insurance or any type of services or co-pay systems that might help them reduce their burden. It's the entire suite. When we talk about IP as an example, people always ask, "Well, what do you think will happen to your share when you face generic competition?" 'Cause eventually, we all face generic competition, and in the orphan world, it's quite compelling. But you look at what happened to us, we've, in fact, already faced generic competition with Ruzurgi. Yes. Right? So Firdapse faced Ruzurgi, and Ruzurgi got no traction. They had a product that was essentially half the cost of our product, and they got no traction because they had no back office. Yeah. They had no services that the patient really wanted, and so I think this is consistent with the orphan model. It's very powerful for the patient and the support of the patient, and this is where value is really, really developed and really is, is found for the patient, so. You're right in terms of anticipating the segue- Okay. And this is why I ask about Catalyst Pathways. First of all, I was around. I know that it was very enabling in terms of helping with the successful launch of Firdapse. Right. But let's fast-forward and talk about what has changed in the last year or so, right? So AGAMREE. Right. Let's first of all talk about what Agamree is, and secondly, with that drug, you are already facing generic challenge- Yes ... if you will, or you're walking into a mature market. Right. How can Catalyst Pathways help you there? First of all, the setup on Agamree. So, DMD is a highly organized community, very advocate-driven. So we came into the market, and we came in very humble. So first of all, you decided to in-license it prior to it being approved. Yes. So Agamree is a dissociative corticosteroid. It's a non-traditional steroid, and it's been since approved for the treatment of DMD patients, Duchenne's, but you didn't know that it was gonna be approved beforehand. That's correct. So you in-licensed it, so tell me about that diligence and then- Sure ... and what convinced them, that company, to decide to work with you? So two things in your question. Number one, we are looking to diversify our income stream and make sure that we have, you know, solid stability and income stream, and this requires now that we begin to work our way back in the portfolio, right? Yeah. Into the pipeline, which- A little bit earlier. Right, which we're taking appropriate risk. Now that we have three products, three revenue streams, we can work our way backward, which we're really excited about. And what convinced our partner, Santhera, was the success we had with Firdapse. Yeah. The opportunity with the infrastructure that we had that can support a product, and we... Quite frankly, you know, we're an execution machine. We're out there every day working it, and, you know, the success we've had, if you were to say, "I want a company that's in CNS orphan and is profitable," you might get one company. Mm-hmm. Maybe two. I think, you know, we've done a scan. We don't find very many. So when you say I- Neurocrine may be the other one. Okay. Yeah. And so you look at it and say, "I'm gonna give my baby to you," 'cause one of my favorite things to say is, "Relaunch is the ugliest word in the English language." That's not a pretty word. You'd never want to relaunch a product. You have one shot, so you give it to somebody you can trust, and Catalyst Pathways plays a big role in that, making sure that you can work successfully with the advocate community, the patients, the caregivers, and the healthcare community, healthcare providers, and we play in all those spaces with Catalyst Pathways. You were able to take the risk Santhera got that you could launch successfully. You're right in terms of the drug being approvable. Right. And so you launched one. Right. And so, but Steven talked in reference to the due diligence process, and we're really proud. That's what Steven said he was. You know, that's. You can talk about that. But that was a risk for us because, you know, Firdapse, we took on before it was approved as well, but this is the first time now, and we're taking that risk, and again, we want to work our way back, Steven. I don't know if you want to talk about it. I'll elaborate a little bit on that. Yeah. Rich is right. We actually developed Firdapse and had a lot of regulatory experience. Sure ... clinical development experience, CMC experience. We had to basically develop the entire product and get it launched, and of course, we had the commercial execution experience. But you brought that in- But, uh- - at a time when the company was very different. That's correct. Yes. A lot of those people are still with us, and so when it came time to evaluate Agamree. Mm-hmm ... for regulatory approvability- Yeah ... and for CMC readiness and for, all the other aspects that would be necessary to make sure that that product was gonna be a success, including the pharmacology, to make sure the drug's actually gonna work. I mean, patients aren't gonna buy drugs that don't work. Yeah. That is the experience and expertise that we had within the company that allowed us to do a successful due diligence for Agamree. Mm-hmm ... and pick a product that we could recognize from looking at the regulatory filings as they existed at that time, was likely to get approved, and upon approval, was likely to be manufacturable and launchable and successible, successful. But then you are stepping into a competitive market, unlike Firdapse- Correct ... which was a non-market beforehand. You had to create the market. Right. I think there are a couple of drugs- There was a few. ... that are used in DMD? Before we launched, actually- Sure ... a generic came in from Emflaza, right? Right. So, So you have a branded. Right ... you have a real competitor, and then you have a bunch of generic. Right. So what gave you confidence that you could take share? So we did a fair amount of market research in our due diligence on the product profile, and one of the things we did was we went and talked to fifteen active decision-makers in the payer space, and we said, "Look, here's the product profile of, of Agamree. What do you think?" And they said, "If this is the product profile, you know, if it has the, the challenge or the challenges that exist in the marketplace, if it addresses those challenges, which are behavior, bone density, and stature or growth, then we're willing to put this out in the marketplace and relatively unchallenged. Background therapy, steroids, whack all three of those out, and these patients have to be on drug because they have profound inflammation of musculature for a long time. Right, and the issue in the marketplace. There are three issues in the marketplace because of the way that steroids work. Steroids are foundational therapy. The issue is patients start too late, their dose is too low, and they stop too soon. Because of these issues? Yes. Yeah. Yep, they behave poorly. It affects the parent, it affects the classroom that the young man and the boy is in. It affects their entire community they're around. It affects the young man because their bones are weak, they don't grow. And sometimes physicians will even say, "You know, once they're in a wheelchair, I don't give them steroids anymore because nobody cares about their height." The young man cares about his height, even if he's in a wheelchair. You ask the young man, they're like, "Yeah, I care about my height, even if I'm in a wheelchair." So we want a drug that they can start earlier, they can be on the appropriate dose, and they can stay on longer. Mm. Gets back to the things I talked about. You know, how do we grow our business, and this drug addresses all of those things. And do you think that physicians are starting... So you launched recently, right? March. First? Yeah, first quarter, okay, and yet you said it's going well. How do you know? So we thought we would really cannibalize Imflaza's business. We thought that's what we would do, but when we talked to these payers, they said, "Hey, we think this is something we wouldn't block." Then. So in December, we started enrolling patients prior to the launch, and we thought we'd get almost exclusively Imflaza patients. We saw a fifty-fifty split between Imflaza patients and generic prednisone. Mm. That continues to today. It's roughly 50-50, about 45 Imflaza, 45 prednisone, and about 10% not on any current therapy. We continue to see that, and that's a good sign. But we also see the response from the physicians about behavior. We can't tell bone yet, bone density or stature, we can't see that. Too early ... but behavior, right away. Really? The physicians are reporting back, "I see it," and the retention rate- Uh-huh ... patients staying on drug, are they coming back month after month, is very high. So we're really excited about that. Interesting. So So it'll be six months soon. You'll be in the market. We'll be talking to you about how it's going. Do you think that you'll be able to be in a position to provide guidance in the new year? Yes. Really? Yeah. Very good. So you talked a little bit about it so far, but that was early launch metrics. What gives you confidence to provide guidance soon? So- -on Agamree? With our Catalyst Pathways and our proprietary pharmacy, we can see right into the marketplace. Okay. We have this great insight. The enrollments, as they continue, we'll be able to provide that guidance. We feel really confident about that, and the feedback- So, patient enrollment forms being submitted- The feedback we're getting, so we feel really confident. Very good. Nice to hear. That's an example of innovation that was kind of not expected. Mm-hmm. Surprising. Yeah. Last one, and we only have a couple of minutes, Fycompa. Sure. So you also entered the rare or the not so rare epilepsy space, but many of your folks have good epilepsy experience. Right. Fycompa's different in that it's the only AMPA modulator that's approved. Tell us a little bit about it, where you licensed it from, and what you expect to do with it, and does epilepsy or rare epilepsy, it would seem, make sense to you to do any more? Yeah. I'll let Steve answer the where it fits, but as a positioning, we're not that interested in the general retail epilepsy market. This was a financial play for us. We were really- Okay. We had one product at the time, Firdapse. We were looking to increase the diversity of our income stream. This was a perfect play for us. Gave us access to steady income stream, gave us access to great talent, and we really like the talent that we have. Rare epilepsy, we would definitely be interested in. As we talked about before, we like our orphan space, we like the SG&A, we like the gross to nets, we like all of that about the model, and so we would be interested in that. But as far as where it fits, In terms of fit, of course, as both Rich and you pointed out, we are a rare disease company, first and foremost. Yeah. The epilepsy space is undergoing somewhat of a transformation in that. Yeah ... the causes of various kinds of epilepsy are being elucidated, and upon determining that, each of the respective mechanisms is in and of itself a smaller rare disease subset of epilepsy, and so we do remain interested in potential rare epilepsy treatments. Now, you have a couple of players there, Jazz with Epidiolex. You have, UCB, arguably with Fintepla. Mm-hmm. But, you know, frankly, it seems like that's a place that you can play because, you know, what, there are three or four genetic epilepsies that have been identified and approved. Not identified, but have approved agents? Yeah. Twenty-some others that don't. I mean, it seems like there are assets out there that you could grab. That's true, and yet, you know, there actually was a publication last year in which a number of rare epilepsies were studied with various on-the-market epilepsy medications, and seven of them were found to respond very well to Fycompa as well. Oh, interesting. Now, unfortunately- Yeah ... it's far too long into the life cycle of that product- Sure ... to make any label changes at this point. But, it is an interesting finding and points to the fact that there are opportunities in rare epilepsy. Okay, so you're getting traction in rare epilepsy. You're getting traction in neuromuscular. Really, probably the driver in the near term is Agamree, but more to, more to come in terms of, buy and build or in-licensing? Right. Okay. Yeah. Excellent. It's been a good year for you this last year. If we're sitting here in a year, answer me this: What will you be most proud of having achieved in the last year? Accelerating the growth on Firdapse- Okay ... continued success with Agamree, and new products in the portfolio. All right. Well, that's a good set of goals. I think we're pretty much done. The red light is buzzing. So Steven, Rich- Charles ... great to spend time with you. Thank you to the audience for your interest in Catalyst Pathways, and thank you for spending time with us. Thank you, Charles.