Good morning, everyone. I'm Andrew Fine. I'm one of the research analysts at HC Wainwright. Thank you very much for joining us today. I'm happy to be here with Catalyst Pharmaceuticals, one of the great success stories from our coverage list. Thank you guys for both being here.
Maybe the best place to start is just broad. Maybe kind of get people up to speed, you know, maybe a little bit less familiar with the latest that's going on at the company, and then we can get into more specific questions.
Andrew, thank you again for the opportunity. Catalyst Pharmaceuticals is a company in the CNS space, and presently in this that are rarely immediately creative, and we bring them into the great execution engine for commercial, for the opportunity to leverage the patients.
Great. Maybe we can start with FIRDAPSE. Obviously, a hugely successful product. Maybe you can talk about where you've been and where you see yourselves going. Growth is seemingly more challenging now, but you've got a couple of things going on that could potentially help drive things longer term. Maybe we can start there.
FIRDAPSE is used in Lambert-Eaton myasthenic syndrome, or LEMS, and it's a very effective therapy and a pretty easy therapy for patients to take. We've been in the business on the market for a little more than six years, and the product's doing very, very well. We had 15 quarters in a row of 15% or more growth until the Change Healthcare cyberattack happened. If you look at first half of 2025 versus first half of 2024, we still see a 19% growth in the product, or, I'm sorry, 17% growth in the product. The product continues to perform very, very well in a bigger phase. The opportunity in Lambert-Eaton myasthenic syndrome, really, when you position it appropriately, is about a $1.2 billion addressable market. In 2024, we had about $300 million in sales, so we're about at a 25% incursion rate with the product.
We're really excited about the continued growth. Recently, what we've done is we've actually split the brand team so that it's, think of it as one molecule, two brands. You have the idiopathic business, which is non-cancer related, and then the cancer related. Now we have people getting up every day focused on idiopathic and another team focused on oncology. We really believe there's a tremendous opportunity to continue the growth at the same pace for the foreseeable future, even on a bigger base. Obviously, nobody ever gets 100% of the addressable market. That never happens. Understanding the value of the product, and you know, we have some IP challenges that are out there, and Steve can talk to that. We're pretty excited about the long runway we have for the product and really looking forward to continuing to grow.
Maybe you can talk about the NCCN guidelines and the impact they might have.
On the cancer side of our business, we have basically a four-step. First was we wanted patients to be able to go to an office to get a BPCC test and then confirm through physical diagnosis that they actually had. The second part was to make sure that we were included in the NCCN guidelines. NCCN guidelines are pretty much the bottom line. Not only did we get that, we got months early. We got frictionless testing through a contract with Quest Labs, a national lab. The patient can get the test in the office, and the results are returned to the physician and the patient. NCCN guidelines came at the end of July. Now the opportunity is to take those NCCN guidelines and impose them on the care pathways in each of the largest oncology practices. When you look at oncology today, it's evolved significantly.
80% of all oncologists are in three GPOs, McKesson being one of the largest, US Oncology being a sub of McKesson. Getting into the care pathways, physicians are then judged on how they follow the care pathways. That's really, really important. We're looking for the opportunity to contract with some of these larger GPOs at a minimal discount because we're the only game in town. We're the only evidence-based therapy that's out there. We believe we can really leverage that position. Those are the four steps that we're looking for in the success of oncology. We've had that concentration for about a year now, and we're really excited about it.
On the idiopathic side, one of the goals, one of the stated goals of the company has always been to kind of shorten the timeline it takes to get a patient onto the drug. You spoke about the frictionless testing as one example. Are there other ways you can continue to build awareness of LEMS within the physician community and try and make it easier for doctors to kind of identify the patients that are potentially suitable for therapy?
A great question. One of the key differences between the idiopathic side and the cancer side is idiopathic is a prospecting market. You've got to go out and find those patients. We did this pool of about 500 or more patients that we look for, and half of our patients come from this pool. We look for patients through AI and machine learning, and then also using market data to determine where the patients might be. We look at that opportunity. We're identifying these patients, and then we prioritize within the 500. We prioritize 60 to 100 patients, and we get half of our patients in any quarter from this prioritized list. That's one. The second opportunity is myasthenia gravis. I don't think people realize that for LEMS, the number one misdiagnosis is myasthenia gravis.
All of the opportunity that's happening out there now on MG with these new drugs that are coming out and the promotion, more patients will get diagnosed with myasthenia gravis. By default, more patients will be misdiagnosed with myasthenia gravis, and eventually they'll come to us. Using data and then using looking at the opportunity for MG, we refer to it as the best money we never spent. The opportunity is really there for us. It's a really strong opportunity. We're looking to accelerate that as well.
How broadly are we MG docs of LEMS and FIRDAPSE?
This is the biggest challenge. This is why it's a prospect in market. The average physician will see one LEMS patient for an entire number of years. It's an effort. Prospecting is really the question where you need to do that. These markets, the idiopathic and the cancer, are about the same size. They're about to split 50-50. The thing we like about the idiopathic side is we can do this pool of patients and hyper-prioritize certain patients. What we like about the cancer side is it's a more concentrated market. If you see small cell lung cancer, which is the most frequently associated cancer with LEMS, we're going to be testing those patients. We believe we can find more patients more and quickly. Those patients don't generally live as long, but there's more of them.
There are about 900 LEMS patients who could be diagnosed each year with small cell lung cancer. They actually live longer than a small cell lung cancer patient without LEMS. They live about twice as long. At any point in time, there's about 1,700 of those patients available to us.
Do you have a sense of how long on average it's taken for other companies to kind of feel or experience the impact from the inclusion in the NCCN guidelines on average?
No, we don't really have a fear for that so much as we're looking at what's the cadence of NCCN guidelines translating into care pathways. Because this is an orphan condition, we'll have to work at that. We believe that this is more of a 2026 and beyond impact, even though we got it in July, late July. We're working right now. We have dedicated assets in the oncology community, working at the very top of the GPOs to look for education opportunities, again, to look to get into the care pathways. We believe it's going to be a 6 to 12 month opportunity for us.
Okay, that's helpful.
Yeah.
Steve, on the patent side of things, there was news recently about settling another case. Where do things stand and how should we think about things going forward?
All right. Just in summary, we've settled three of four lawsuits now. Two of those three were first filers. The remaining one, which is Hetero, is the remaining last first filer. At this, it's ongoing litigation, so there's not a lot I can say about it. I think the best thing that I can say is that we do talk to the litigants from time to time. When they have something that we feel is in the best interests of the shareholders, we act on it. Certainly, that's what happened with regard to Teva and the recent settlement with Lupin, and then the original settlement with MSN early on. If Hetero has something useful to say, we will. I can assure you we also will defend our intellectual property if we have to.
I just want to point out to build on, the products have been on the market for six plus years, seven years. The settlements we have with the first three take us to February of 2023. In my experience, having watched more than 10 products, you know, if you get seven or eight years in your entire life, that's amazing. We're looking at a potential for an extension of an additional 10 years. We really believe that there's opportunity to continue to grow. Yeah, I'm really excited about it.
Great. You know, maybe we can switch gears a little bit and speak about AGAMREE.
Sure.
Maybe just kind of brief people in terms of where things are at there, how you're thinking about the opportunity there, and what folks can expect coming going forward.
Sure. AGAMREE is a, we believe, a differentiated steroid that's used in the treatment of Duchenne muscular dystrophy. To set the stage, steroids are the foundation of therapy. Patients' first diagnosis is the drug they should get on first. To think about it, it's a drug that drugs should get added to, not the drug that gets added. It's the foundation. 11,000 to 13,000 boys in the U.S. have Duchenne muscular dystrophy. 95% of them have been on a steroid at one point in time, but only 7% are currently on steroids. We believe this speaks to the unmet medical need that's out there. Steroids are incredibly effective in helping the patients to continue to walk years and longer. The drop-off is a problem. Patients should stay on this therapy for life. The launch is going really, really well.
There are about 100 centers of excellence in the U.S., and we have penetrated or had prescriptions in 93% of those centers of excellence. 250 physicians represent more than 90% of all prescriptions written. We're at about an 85% penetration rate there with at least one prescription. We've gone there, and the goal now is to go deep. There are challenges in this market because of the non-steroid noise that's going on, specifically with gene therapy. It's truly unfortunate to know it's been happening. It creates a lot of noise for us. A really fortuitous thing we did was in April of this year, we actually split our sales forces and made them dedicated. We have a dedicated FIRDAPSE sales force and a dedicated AGAMREE force.
The share of voice is not an issue on the steroid side because you've got a couple of generics and you've got a brand that recently went generic. We have almost 100% of the share of voice here in this market, which is really important. However, the background noise that's going on is really difficult to crack through this. We're working diligently. Our dedicated sales force really is a prime move for us. We think it really will help continue to grow the product. The product's incredible, incredibly strong belief and uptake. We've been on the market a little more than a year and a half. We're continuing to see good growth.
What's the process by which physicians choose which steroid to use?
Right now.
First, I've had.
Yeah, of course. Right now, almost all the patients have experienced either prednisone or deflazacort. Generic deflazacort came to the market before we even. We are seeing about 45% of our patients come from deflazacort and 45% of our patients come from generic prednisone. That's been consistent since the launch of the product. We initially thought we'd be working fighting with deflazacort for market share. That we think is about 35% of the market. There's not a lot of data available in this market, prescription data. It really looks like now we're playing in 100% of the market, which is really good for us and really good for growth. A patient's got to get generic prednisone first. The average age of our patients is about 12 years of age. That's the point at which they lose the ability to walk or ambulate. Those patients have already been on prednisone.
They've already been on deflazacort. There's really no block for us at this point in time. About 500 patients, 700 patients come onto the market each year. As those patients mature, we expect to be taking on those patients. They'll experience that generic prednisone. We want to be that next drug. It's always going to get a generic first. We want to be that next drug. We believe the side effect profile of the product is superior to what else is out there. Steven can speak to the side effect profile, but it's a really good opportunity for us as well.
Yeah, please.
With regard to the side effects, there's a lot of published literature on AGAMREE over the years that it was developed showing that patients have better bone health, bone density, growth, stature. It's based on the mechanism of action. It should have better cardiovascular safety. Based on other published information, it looks like it doesn't cause the anxiety and aggression that's very common with the other steroids that are used. All of those are benefits to the patient community.
You guys have spoken in the past, I think, about trying to find an immunosuppressive dose. Where does that stand? Maybe just educate people about the importance of that.
Yeah, so in gene therapy, as an example, one of the immunosuppressive doses is steroid. Currently, the label, the AGAMREE label, does not have that. We are doing work of that, bifurcating work. Two things. Number one, we're looking for an immunosuppressive dose, but we're also looking for a translational dose. If you come off of prednisone or influenza, you know, what's the right dose to go to? Currently, our label says start at the highest dose and titrate down. We want to be sure that we can give good guidance, working on that. That work, the trial is complete and the analysis is ongoing. We expect to have the data sometime late this year or early next year. There's no promise we'll actually find an immunosuppressive dose. We just continue to want to see if we can find it.
That plays into the lifecycle management of the product where we go next.
If you're successful in finding it, how long does it then take to have that reflected on the label?
That depends on how the data works out and are there any other steps, but it would take, I think, a significant amount of time. However, publication indication versus publication to promote something off label, that's not what we do. Once it's published, the physicians can make the determination about whether or not they want to use it. The difference is when is it going to be on the label versus when is it going to impact more. That could be, those could be significantly different.
Okay. Maybe we can speak about, you know, business development because that's always been an area, right, for discussion. Obviously, you guys have been hugely successful, you know, going out and finding assets over the years. What's the state of your current process? Just in broad terms, I guess, is the macro environment conducive to finding something or making it more challenging or somewhere in the middle?
We've said consistently that this is probably one of the best buyers in the market. Equity markets are obviously very tight lately. We look for three things when we start with our diligence. Number one, we want a differentiated product. Number two, we want a product that makes money. Number three, we look for the social element. I've been involved in business development for the past 30 years and alliances or licensing agreements. That ability to actually get along with the other management team and facilitate that is more important than most people realize. I actually had the opportunity early in my career to run two major integrations, companies coming together. Consistently, people said our cultures are exactly the same. It's not. Those issues loom large. We go deeper into the due diligence around the product.
We really can't say a lot except we're really busy. 80% to 90% of our opportunities are inbound because of the success of the company, the commercial success of the company. We want to continue to build and diversify our income so that we can actually get further into the pipeline, back into the pipeline. Right now, it's immediately accretive or nearly immediately accretive, it says. We really want to be able to have that conversation with you and your team about here's what's in our pipeline. We need just a little bit more income diversity before we do that.
Do you see yourself staying within the neuromuscular realm or looking at a kind of a broader swath of potential therapeutic areas?
This is a very insightful question because if you look at the CNS space, all of the opportunities for growth in the next five years are coming from movement disorders. We like to keep our peak year sales under $500 million because that certainly moves the needle for us, but doesn't move the needle for bigger players. If we go into movement disorders, those are big opportunities. Steve and I like to make the joke that if we do that and we show up at the auction and bigger companies are there, we're buying them coffee because we can't compete. As strong as our balance sheet is, we just can't compete. We want to keep it under $500 million and look for those opportunities that make a difference for us and we can make a difference for patients.
Are there categories that fit neatly in the context of the current portfolio? Does that matter? Can it just kind of touch within the, kind of stay within the umbrella?
We are therapeutic agnostic. We believe the infrastructure we have behind the sales organization, getting the patient on, keeping the patient on, and optimizing the dose is really important. All the support services that we give behind the back of the house, what we call the back of the house, really we believe can be applicable to any therapeutic area. CNS obviously would be the easiest one, but we think we can field the team that has expertise in whatever therapeutic area and then use the tools that we have and apply that there.
Great. I think we're out of time. I guess with the final few seconds, any final thoughts you want to leave people with?
We're just really excited about the opportunity to grow the company, and we really appreciate your support and support of our show. Thank you.
Thank you very much.
Thanks, Sarah.