Good afternoon, everyone. Thank you for joining us at the Barclays Healthcare Conference and continuing our Specialty Pharma track for the day. I'm delighted to have the management team from Catalyst. We have Richard Daly, the CEO, and Steve Miller, the Chief Scientific Officer. Steve, and Richard, thank you so much for joining us today.
Thank you.
Thanks for having us.
For the sake of full disclosure, we do not cover the stock and so do not have a rating. That said, Steve, Richard, we'd love to invite you to some opening comments and maybe help provide some details around the business model, the type of drugs that you're working in the neuromuscular space, and orient the audience to the company.
Sure. Love the opportunity. Balaji, thank you for having us. We really appreciate the opportunity to speak with the audience and join you here at the conference. Catalyst Pharmaceuticals is a buy-and-build, orphan-focused company. We have three products on the market. We have Firdapse, which is our lead product for Lambert-Eaton myasthenic syndrome, launched in 2019. Incredibly successful. We have 14 quarters in a row of 15% or more growth. We're really excited about the product. It continues to grow and continues to perform. Second product is Fycompa, which we purchased from Eisai. It's an epilepsy product, and the product continues to deliver, and we are very excited about that product as well. Third product is the product we launched last year in March, AGAMREE for Duchenne muscular dystrophy. It's a steroid. It appears to be a differentiated steroid and is doing very, very well in the marketplace.
We continue to look to add to our portfolio and grow the company both organically and through business development. We feel we're an excellent partner for companies looking to gain a foothold in the US, and we're really excited about our opportunities here.
Great. With all of these launches, what is the main tool for your outlook for 2025, and what are the key priorities for you as you look to execute this year too, and what do we need to look out for?
Great. The key priorities for this year really are as we look at Lambert-Eaton myasthenic syndrome, this is a market that's split 50/50 between cancer-associated Lambert-Eaton myasthenic syndrome, or LEMS, and non-cancer-associated LEMS. We are pivoting, keeping our focus on the non-cancer-associated LEMS, where we've really grown and done a very good job, but also looking to pivot to the cancer-associated LEMS side of the business. This is a really interesting side of the business. It's a highly concentrated part of the market. Obviously, you see these patients in the cancer and the oncology practices and community. These patients, a very interesting part of this market is that it's highly associated with small cell lung cancer. Patients with small cell lung cancer typically live about 7-10 months because they're diagnosed so late. A patient with cancer-associated LEMS lives about 17 months.
Nobody knows why, but there's a really interesting opportunity to improve the quality of life for these patients, these patients who live a lot longer than their peers with just small cell lung cancer. Pivoting here, we think we have a great opportunity. Less than 10% of these patients with cancer-associated LEMS have been diagnosed with LEMS. We're very excited about this opportunity. We feel like we can grow this business. The LEMS part of our business has a market potential of over $1 billion, and we're really excited. Last year, we sold about $300 million, so our market penetration is just 30%. As a player with about 10 years left of our market life, we see a great opportunity for upside in this market.
Got it. Great. In your opening remarks, you spoke about the launch and the growth that you've seen over consecutive quarters. What's going to allow you to continue to maintain that very impressive trajectory on into 2025? Maybe taking a step further on, you spoke about this broad market opportunity, the billion-dollar market opportunity. How would you be able to realize that?
It is two factors. The first factor is we track in a compliant way. We track about 500 patients on the non-cancer-associated LEMS part of our business, and we watch them as they're diagnosed. The number one misdiagnosis for LEMS is myasthenia gravis. We track these patients through certain market surrogates, again, in a compliant way, and we watch them as they go through their diagnostic journey and therapeutic journey. We work with the physicians on helping the physicians understand what it means to have LEMS and what that looks like because it is so frequently misdiagnosed. 50% of our patients come from this pool of patients that we observe in the marketplace. We can predict with great certainty how we're going to grow over the course of time. That is number one.
The second part of this is really looking at the opportunity for cancer-associated LEMS. We are very close to signing an agreement with a national lab on testing for LEMS, for cancer-associated LEMS, and then working with these oncology practices to test these patients who are so deeply in need of improved care. We have worked very closely with the oncology community to look at this opportunity to help improve care. The oncology community is, again, very excited about this.
Got it. Maybe a quick comment around the recent settlement with Teva on the product and what's your view around the longer-term potential too?
Sure. Steve is our resident expert on patents, and I am glad he is here to join us. Steve, it is for you.
Thank you, Rich. The settlement you're referring to is the settlement with Teva Pharmaceuticals. That was a good settlement for both of us in terms of having certainty on the future for the product. There are two remaining companies in which the litigation is continuing, Lupin and Hetero. With Teva out of the game, so to speak, we believe that we should reach settlement with the others potentially in the future. What I want to emphasize is that we think we have very good patents. If we are forced to go to the finish line, so to speak, I think that we'd be in a very good position as well. We're working diligently to bring the litigation to a conclusion in as favorable a manner as possible.
Great. Having covered the generics industry too for a long time, what we have seen typically is that one settlement with a major generic company kind of precedes other settlements too. Is that a reasonable assumption in this case?
It is.
It is. Okay. Great. Can you just remind us of the quick terms of the Teva settlement?
Basically, Teva will be entering the market no earlier than February of 2035.
Got it. When would we expect to hear about the other two companies? Any progress?
Unfortunately, it's ongoing litigation, so there's not a lot of details I can give you. Again, as I said, we're looking to bring these cases to a conclusion as quickly as possible.
Fair enough. Maybe shifting to AGAMREE and Duchenne, could you talk about how AGAMREE fits into the treatment paradigm for DMD patients currently?
Sure. Yes. When you think about AGAMREE and steroids, this is kind of the polar opposite of the Lambert-Eaton myasthenic market. In the Lambert-Eaton myasthenic market, almost none of the patients are diagnosed, and their diagnostic journey is very long. On the DMD side of the Duchenne's market, the diagnostic journey is quite rapid. 95% of all patients with Duchenne are diagnosed, and 90% of all these patients have at some point in their journey been on a steroid. You want to think of steroids as foundational therapy. The easiest way to think about it is steroids are the drugs that drugs get added to, not the drug that gets added. Regardless of what's been happening in the marketplace, this is what all drugs get added to. All of these great advancements that have been happening in the Duchenne's market, which are phenomenal for patients, it's great.
This is a great time for patients. Steroids are base therapy. It's outstanding. This is where this fits in. The challenge for this marketplace has been that while 90% of patients have been on a steroid at some point in their life, only 70% of patients are currently on a steroid. This gap speaks to the need in the marketplace. These patients need something that's better. The hope and the promise of a drug like AGAMREE is that it can potentially address this gap. There are problems with steroids, long-term steroid treatment and adverse events. There's poor behavior, aggression. Patients on long-term steroids don't have the same stature. They don't grow like their peers who are not on long-term steroids. They have poorer bone health, poorer cardiovascular health. The number one reason for death of a Duchenne muscular dystrophy patient is, in fact, cardiovascular disease.
Cataracts are another issue too. This is one of the reasons why we are doing the SUMMIT study. We're looking at the use of AGAMREE versus natural history control. We're looking at the opportunity to see if AGAMREE can improve the standard of care that's out there versus natural history. We're really interested in seeing this. We believe that there's an opportunity to improve the care for patients out in the marketplace.
Understood. As you target trying to get this 30% of patients onto a corticosteroid, what are the key steps and milestones that we can look forward to over the course of this aim or goal? What are the key steps we can look forward to?
I think Steve can speak to the SUMMIT study. I think we're going to look for data to come out of the SUMMIT study that will look at the opportunity to improve the care. Steve, I don't know if you want to speak to some of the steps we're going to take with SUMMIT.
Sure. We've got a SUMMIT study, which is an open-label study where we are examining a number of safety outcomes of patients to determine some additional safety characteristics of AGAMREE so that we can show some of the safety advantages of AGAMREE relative to the known safety characteristics of the other products on the market. Because it's open label, we are aware of what the progression is of the patients. In addition to that, it has the ability to analyze the data against control, which in this case is natural history, and the FDA has guidances for that. This is a submission quality study. We will be analyzing the data, just doing descriptive statistics on a number of the endpoints to make it publicly available at various times because the endpoints progress at different speeds.
On a periodic but relatively less frequent basis, we will analyze the data against the natural history data as controlled study outcomes and take a look at that data and determine whether or not to submit it to the FDA or continue to gather data. We will limit the number of those analyses because you sacrifice alpha every time you do a controlled comparison.
Sure. Got it. Maybe just to add another layer of questioning around this segment with newer therapies, be it siRNA or gene therapies being developed for DMD, how do you see the longer-term view for how the treatment paradigm for DMD, the whole landscape is going to evolve? How do you suggest it progress?
I think this is a really exciting time for patients with Duchenne's. We see a recent filing for cardiovascular, potentially improvement in cardiovascular health. We see opportunities where some recent data has come out for a new gene therapy that's not yet on the market that may even be better. It's just an incredible time. For us, the real value is looking at the long-term potential of a better steroid that's going to underlie all of these therapies and continue to be there. Regardless of what comes out, we're really excited. We think this is a really golden age for Duchenne's, and we look forward to all of these improved therapies coming out. For the better care for patients, it's like no other time for Duchenne's. It's really exciting.
Got it. Yeah. Shifting from AGAMREE to Fycompa, speak about 2025 and how we should think about the product and potential generic entry. I know you also spoke about the erosion trajectory in the setting. What kind of details do we need to look for that can help us gauge the product out?
I'm sorry.
What details can we look for gauging?
We gave guidance in our call a couple of weeks ago. One of the things we like about Fycompa is that what I say all the time is Fycompa is like the mailman. It just delivers. It just continually delivers. Last year, we had $137 million in sales, and we expect generic entry in late May of this year. We expect to be able to deliver consistent with last year's plan, last year's sales up through the end of late May. The remainder of the year, we expect probably, according to our guidance, probably about $35-$36 million or so. As you see with all products that experience generic competition, you're going to see a loss of volume and of price.
Epilepsy products are different because unlike cardiovascular drugs or diabetes drugs, when you have a problem with an epilepsy product, the ramifications are so significant. You have a seizure, you lose the ability to drive for three months or six months in a given state. It is significant. Patients are more likely to stay on a branded molecule for longer, not for forever, but for just longer. We expect to have a little bit more legs on the product for at least the balance of the year. We think that we can hold on. If you were to look at a generic incursion, as I said, in diabetes or cardiovascular, you would lose 90% of your share in eight weeks. We expect to hold on to a significant amount of share.
Our guidance was that we would go from $137 million in 2024 to about $90-$95 million in 2025. That speaks to being able to hold on to a significant share and pretty good price in the second half of 2025.
Okay. Shifting towards other parts of the business, you have around $500 million plus cash in hand on the balance sheet. Speak to us about your business development plans for the next couple of years and what are your key target areas and assets that you are pursuing.
Sure. While we can't talk about specifics in business development, we can talk about our general philosophy about business development. With $500 million on the balance sheet and when we look at forward operating profits, we think about what we can afford. Our philosophy is we want things that are orphaned because our business model supports that. We want opportunities that are differentiated, and we want things that are immediately accretive or nearly immediately accretive because our commercial model is very robust. We like the opportunity to be out there. We like the opportunity to make a difference for patients. This really fits into our model. We are focused in central nervous system disorders. When we look at central nervous system disorders today, the growth in the long term is coming from movement disorders.
When we look at movement disorders, we see a lot of bigger players at the table, and we don't want to compete with bigger players. Our focus is really looking at a broader spectrum of opportunities. Because our infrastructure is so robust, helping the patients find the right therapy, helping the patient get on the right therapy, and helping the patient stay on the therapy, essentially the back of the house that almost nobody can see, patient support and patient advocacy, we believe that applies to almost any therapeutic opportunity we can find. We are agnostic to therapeutic opportunity as long as it's in the orphaned space. Our eyes are on those opportunities that are out there. We believe we have lots of opportunity.
When we look at the size of the deal, again, we do not want to be competing with bigger players, and we think we can look at opportunities that are in the $250 million-$500 million peak year sales. Because that does not move the needle for bigger companies, we think that that is an opportunity for us that those companies will not be at the table for. We like that kind of approach.
Understood. Do you see enough opportunities to pursue?
Yeah.
Maybe some comment around the valuations that you're seeing. What kind of valuations are you seeing? Is it reasonable?
I think that most valuations are reasonable. The key issues are social issues and fit. You need to be sure that the fit is a good fit because having worked in business development and partnerships for the better part of 30 years, when you sign a deal, you're getting into a deal for the long term with a partner that you're going to be with, and it has to work. That means senior management and every part of your management team is going to be working together. Fit becomes one of the primary issues. It's not just about making money and helping patients. How are you going to spend your time as a senior management team and as a management team across your company? Valuations are reasonable. There are some who are unreasonable. There's no doubt about that.
There are reasonable opportunities out there, but then it's about fit and making sure that you can work together for the long term.
Understood. Maybe a final question around your plans to invest in portfolio diversification and what does this mean, what does this entail, and any focus there?
We would like to begin to build beyond just CNS, but we would also like to have the opportunity to work our way back in the portfolio as well in the pipeline. Right now, we are looking at a set of immediately accretive or nearly immediately accretive. Over time, one of the things that was really important to us is to look at opportunities that go into phase three or further back into the pipeline because we think that is where the real value inflection comes from. Not right now, but eventually work our way back because we see peers that are out there that look like us but have higher valuations. The main difference between us and those peers is really portfolio.
You ask a question, which I think is a really good one, Balaji, which is, tell us about BD, and I really can't answer that question. If we had a pipeline, we could have a discussion and disagree over the PTS, the probability of technical success. I could say it's 80%, you could say it's 60%, and then we could have a really robust discussion about that. We can't do that around BD. We really can't talk about the future. We need to be able to do that and have that discussion about the future and be forward-leaning on the future. We would like to begin to work our way back into that in an appropriate way.
We're really focused on that, building that income statement, building that balance sheet, and then working our way back into the pipeline to build a really robust picture of the future.
Got it. Great. Richard and Steve, thank you so much for joining us today.
Thank you.
I do wish you a very productive conference.
Thanks.
Appreciate you joining the conference.
Thank you for the opportunity.
Thank you.