On Wednesday, 19 March 2025, Acadia Pharmaceuticals (NASDAQ: ACAD) took center stage at the Stifel 2025 Virtual CNS Forum. The company’s leadership team provided an optimistic overview of its strategic initiatives, highlighting robust financial health and ambitious growth plans. While Acadia is poised for expansion, challenges remain in raising awareness and driving sales for its key products. Key Takeaways
Acadia forecasts over $1 billion in revenue for 2025, supported by strong financials with over $750 million in cash and no debt.
The company is focusing on increasing awareness and market penetration for its products NUPLAZID and DAYBUE.
Expansion plans include broadening the company’s focus beyond neuropsychiatry to rare cardiovascular, endocrine, and metabolic diseases.
Acadia’s pipeline is advancing with promising developments in treatments for Alzheimer’s disease psychosis, Lewy body dementia psychosis, and Prader-Willi syndrome.
The company is confident in its intellectual property position and is actively pursuing business development opportunities.
Financial Results
Acadia reported a strong balance sheet with over $750 million in cash and no debt, positioning the company for potential acquisitions and expansions.
The company is guiding to achieve over $1 billion in revenue this year, with expectations for significant growth in the latter half of 2025.
Operational Updates
NUPLAZID, used to treat Parkinson’s disease psychosis, is benefiting from increased promotional efforts and real-world evidence supporting its efficacy.
DAYBUE, for Rett syndrome, is seeing stable patient retention, with over 62% of users remaining on the drug for more than a year.
The company is expanding its field force to reach more physicians and increase DAYBUE’s market penetration, especially outside centers of excellence.
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Future Outlook
Acadia is exploring opportunities beyond its current neuropsychiatry focus, considering rare cardiovascular, endocrine, and metabolic diseases as potential areas for expansion.
The company is preparing for the launch of a new product for essential tremor, with further details expected at the upcoming R&D day.
Pipeline Developments
ACP-204 is in clinical trials for Alzheimer’s disease psychosis and Lewy body dementia psychosis, aiming to improve upon NUPLAZID with better efficacy and safety profiles.
ACP-101 is in Phase 3 trials for Prader-Willi syndrome, showing promising data in earlier studies.
Q&A Highlights
CEO Catherine Owen Adams emphasized the importance of increasing physician engagement to boost initial prescriptions.
CFO Mark Schneier expressed confidence in the company’s intellectual property strategy, particularly regarding upcoming patent cases.
Acadia’s management remains optimistic about the company’s growth prospects and strategic initiatives. For further details, readers are encouraged to refer to the full transcript.
Full transcript - Stifel 2025 Virtual CNS Forum:
Paul, Host: Great. Thanks.
Thanks very much for listening in. It’s my pleasure to be introducing the Acadia Pharmaceuticals management team. With me is Catherine Owen Adams, CEO Elizabeth Thompson, Head of R and D and Mark Schneier, CFO. I’m sure most folks listening in here know the company well, but maybe I’ll ask Catherine to just give a quick couple minutes snapshot of where Acadia is at with the commercial business and the pipeline, and then we’ll do q and a. So thank you very much, and take it away.
Catherine Owen Adams, CEO, Acadia Pharmaceuticals: Well, thanks so much for having us. Yes. So Acadia, we have a focus on neuroscience and neuro rare. We have two, commercialized products right now guiding to over a billion dollars this year. We have NUPLAZID for the treatment of hallucinations and delusions in Parkinson’s disease and debut for the treatment of Rett syndrome.
We have a strong balance sheet and are looking to be active in our BD space as well this year and beyond that we have a really great pipeline with two products in late phase two, phase three in Prader Willi syndrome and, ACP two zero four, which is in clinical trial for, Alzheimer’s disease psychosis and also the psychosis of Lewy body dementia and, recently acquired a new product for essential tremor. But all of that will be revealed in more detail on r and d day on the June 25. That’s my elevator speech, Paul. Alright. Paul, Host: I like it. Thank you. So let’s talk about debut for a bit. Right? So the launch has been was up, up, up and then kind of flattish to down for a period.
And it feels like we’re at kind of an inflection point, right, where we’re either at a steady state or we’re going to see a new sort of inflection based on an evolving commercial strategy. What gave you the confidence to give guidance for Day2U that implies a real rebound in organic growth by RMAT? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: Yes. So let’s sort of break that down into your component part. So yes, we came out the gate very strong. Lots of demand for debut amongst the centers of excellence treating rep patients. And then once we’d hit a lot of the centers of excellence, we moved out into the community.
And that’s when our kind of patient demand, I would say, plateaued. And the last two or three quarters has been very steady, not unlike many other rare disease launches. But the key to a rare disease launch is getting to those patients that are treated outside the centers of excellence. And we know for Rett syndrome that’s about sixty five percent of patients who will be treated by physicians outside of a center of excellence. And when I when I came in and looked at our field force and our go to market model, we were underpowered to reach those physicians and have the right conversations with them.
So immediately what we’ve done is to increase the size of our And so that’s going into place now and, will allow us to reach that extra sixty five percent of patients at the right reach and frequency, I believe, to drive additional growth. So we’ve guided to, we’ve guided to that with seeing that growth coming in towards the back end of this year. And I feel very confident that we will be able to achieve that, having led quite a few commercial launches now and understanding how, how it works. We were just underpowered. And so I feel very good that we can we can reach the the rest of our of our market and really see that increase towards the back end of this year. Paul, Host: Okay. Great. Sorry. The, automatic light just turned off my office, so I’ll have to throw something across.
3rd party Ad. Not an offer or recommendation by Investing.com. See disclosure here or remove ads. Catherine Owen Adams, CEO, Acadia Pharmaceuticals: All the time. Happens all the time. Paul, Host: Yeah. That’s great. Well, okay. That’s a that’s a great overview. I guess, you know, there’s there’s a couple of variables here.
Right? One is the NRX variable. One is the persistence variable. On the latter, persistence, like, do you feel like we’ve seen or we’ve hit, like, the curve or to some degree on on an average basis with the existing patient base where we’re, like, seeing a plateau in the discontinuation rate? Like, how are you thinking about that? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: So let’s think about the journey of the patient on debut. And what we’re seeing is, over time that at around twelve months, we are we still have over fifty percent of patients still taking debut and seeing a clinical effect. And so for me, that’s a pretty high, persistency rate for any product, particularly, in in the rare disease space. So over fifty percent at twelve months. Over time, we’ve been launched nearly two years now.
Our patient base has become more stable and the amount of patients on product at a steady state is increasing, right? So if you look at a point in time now, over sixty two percent of our patients have been on debut for more than a year. So again, we’ve got a persistency rate that’s stable. We’ve got a steady state of patients that are increasing their time on debut. And so for us, that sort of base is a lot more consistent than it was even two quarters ago.
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And so now we’re building on that. And so now the strategy is to get more referrals in the top of the funnel. And that’s why we’re increasing the field force to drive those increased referrals. Paul, Host: Yeah. Yeah. Okay. And why your commentary that you’re really thinking about this being back half loaded, why is that? Like, why wouldn’t you?
I mean, you have a lot of experience in the commercial side. Like, why wouldn’t it be you know, a more immediate impact? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: It takes time for reps to reach their doctors and have the right number of conversations to drive that first script. You know, this is a this is a conversation that needs to happen three, four times before a doctor feels confident, in the main to start debut. There’s obviously some that need less and some that need more. So just having led a lot of launches and field forces and looked at the numbers, it takes about two or three months for the field force to start having an impact on prescribing. And so, you know, we’ve factored that in.
And we’re being pragmatic, and we’re being, I think, achievable in the way we’ve guided. And I want us to achieve all of our guidance this year. Paul, Host: Fair enough. Anything about these non COEs where the marketing message has to be, like, fundamentally different? You know, I mean, the academics, right, they’re much more aware of the clinical data. What what do you like, what are the questions you get from these community doctors? Are they different?
Catherine Owen Adams, CEO, Acadia Pharmaceuticals: Actually, they’re not different. It’s just that they see fewer rep patients, so they need to ask more questions, and they need to have more understanding of the clinical data. And I think, you know, the medical affairs and the MSL side of things becomes more important. They need to have somebody to call on and to understand and to ask questions of. And for us, it’s bringing that data to life.
If you only see two or three rep patients a year, you really need to understand, what our data means. What does the CGI mean in real life? What do the changes in schools mean in real life? And again, we’re evolving our messaging, to your point, to make that more tangible and more, just more obvious to physicians that treat less rep patients. So, again, that they’re armed with all the questions that the families will ask them. Paul, Host: Fair enough. Do you want to talk, Catherine, about your efforts ex US and Catherine Owen Adams, CEO, Acadia Pharmaceuticals: Sure. So we have approval in Canada, as you know, and we have filed for approval in the European Union. In Canada, we’re in talks with the with the health authorities right now for reimbursement. We expect to get some outcomes of that in the next few months. In Europe, we’re preparing to launch and we are getting our teams ready.
We have MSLs out there in the countries. Our big countries are, as you would expect, based on population are Germany and France and Spain and Italy, and nothing strange there. So we’re building a team out in Europe. We’re going to be launching this ourselves. I’m very comfortable launching products outside The US.
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And so we’re building that team up, but we’re being judicious and we’re being, we’re looking at it on a country by country basis. But RET is treated pretty similarly in most European countries. There’s a lot of localization and centers of excellence in most countries. So it’s not that different to The US. It’s just making sure we’re in the right countries at the right time. Paul, Host: Makes sense. You know, when I when I look at the rare disease space and, you know, look at very different drugs for very different diseases, sometimes it feels like it’s almost arbitrary. Like, what drugs are able to kind of hold US pricing in Europe and what aren’t? Right? I mean, I can think of examples like amazing medications, like things for cystic fibrosis where they’re nowhere close or medications where the effect size is really, really small like an ERT, but the pricing is very, very tight.
How much visibility do you have into this at this point as to whether or not you can get US pricing ex US? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: I don’t think there’s any markets really where there’s US pricing ex US at the end of the day because as you know, you start in some countries and you come down. So you have to look at you have to look at the European Union over time, knowing that prices will always sort of erode. But I think, you know, we feel very strongly about the value story that we we have for for debut. We feel very strongly that we have a lot of global demand for where we’re getting requests every day from physicians in these countries, which is why we’re also opening up our named patient program, as soon as we can. So there’s a lot of demand for debut, and I feel back to the price that we’re gonna get, we’re gonna get a good price, but every market will take on its on its individualities and we’ll make decisions based on that.
Paul, Host: Great. Let’s talk about New Plaza for a few minutes and then we can talk about the pipeline. But for New Plaza, I mean, I followed this drug for a long time. I sat through the adcom nervously. So I have a lot of the history there.
And I think it’s really impressive what you guys have done to reinvigorate growth, like this late in the life cycle of the drug after it went through a number of years where growth kind of language a bit. What has like been so effective about your new strategy and how sustainable is it? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: I think the team already knew that this was a promotionally sensitive market. And what I mean by that is patients and caregivers need to be continually educated that hallucinations and delusions are associated with Parkinson’s disease. It’s relatively low awareness when a loved one gets Parkinson’s that they might suffer from these symptoms. And so keeping that awareness up is really important. The team stopped doing a lot of DTC during COVID and started in the middle of last year when we realized that the levels of awareness were actually pretty low.
And so that has really been impactful and, is really starting to pay dividends now in terms of more patients coming into offices and asking for New Placid. And we’ve also had a lot more real world evidence generated for the efficacy of NUPLAZID versus off label antipsychotics, which continues to look really strong. And we had some label clarity about whether you could use NUPLAZID with patients with dementia, which was always a little bit of a gray zone for a few physicians. So with the label clarity, the real world evidence and the DTC campaign, I feel very confident that we can really continue to drive growth into NUPLAZID. We have a 25% market share, and I think there’s a ways to go yet.
Paul, Host: Makes sense. Do you want to comment briefly on the two upcoming IP cases that we’re gonna get results from? I know there’s not I know there’s not a lot you can say, but I think, you know, the question I get most commonly is sort of framed as a version of this. It’s framed as a version of, you know, the the your, you know, your your predecessor, Catherine, would was guiding to generics coming to market in 02/1930 for a while, right? And then now there’s this potential optionality where it could be later than that.
So is there a reason to think that like not that something changed, but that this was IP optionality that was discontinued that was discounted by Steve, but actually might be something where there’s a real upside driver? Like how are you guys thinking about that? Catherine Owen Adams, CEO, Acadia Pharmaceuticals: I’ll let Mark answer that for you. Mark Schneier, CFO, Acadia Pharmaceuticals: Yeah. I’ll jump in here. I think for us the way we look at it is it’s really bookends, right? So I think we’re on the October 2030, the kind of the short end of the bookend, we remain highly confident that will prevail in the composition of matter appeal which is yet to still yet to be scheduled. And to use your words on kind of optionality, time and litigation is advanced, right?
So the formulation patents which lots of people just discount by nature, We think as we went through the trial and the steps leading up to it, we have the better argument on both invalidity and non infringement. And so obviously we’ll have to see how that plays out. But we think we have the better position and we’ll get a ruling on that in the first half of this year. It’s our expectation.
Paul, Host: Okay. Ruling on both that and Mark Schneier, CFO, Acadia Pharmaceuticals: Both should come out, right? I think we’re a little surprised on that the appeal hasn’t happened yet. So, kind of stop guessing on the timing there. But we should again, there’s no like PDUFA clock for trial rulings, but our expectation and advice from counsel is we’ll hear sometime in the first half of this year on formulation as that trial took place last December. Paul, Host: Yes. Okay. And Mark, while I have you answering questions, I did get a question from an investor who’s listening in who was curious to hear about, you know, and it’s actually not clear to me which franchise he’s talking about. So I think it’s Debuting, but we can just maybe comment on both for Debuting and Neapolitan. What were the gross to net dynamics, for 1Q ’twenty four?
And should we be considering those as coming into play again for 1Q25? Mark Schneier, CFO, Acadia Pharmaceuticals: Yes. So I think what we’ve specifically said, it will change for NUPASA, right? So NUPASA is a high Medicare patient population, about 75% is Medicare. So where the gross to net dynamic changes with the Medicare Part D redesign. And so what we saw in the past was a very high gross to net in the first quarter as we had to pay our manufacturing obligation for both new patients in the quarter and essentially every patient that was renewing therapy from the previous year.
In the new design, it’ll be more even over the course of the year. So we don’t expect kind of quarter to quarter fluctuations and we do for NUPLASID expect a benefit in gross to net as we qualify as a small company manufacturer on the inflation reduction axle. Our gross to net will go down this year and that’s factored into our guidance. So I think that’s it. If there’s a debut question, you know, I think debut again we guided up a little bit due to a number of changes from the Medicare impact on that as well as just pricing dynamics that have taken place year over year.
But again for that, we expect that to be mostly consistent quarter to quarter. You always have fluctuations as patient mix for payer in any quarter can change so that has fluctuations for our medicines just like any other medicine. Paul, Host: Yeah. Okay. Great. Alright. Maybe I can ask a few questions about the pipeline, if that works.
Okay. Yeah. So I’d love to ask about 02/2004, and the ADP program. And, this is my this is my weighted question. So I I’d love to hear the counter case, and I’m open minded to counter case.
But my my perception was always that the ADP parallel group data for pimavanserin was kind of messy, right? Like it hits that thing at week six, but not four, nine and twelve. And there was a whole discussion we have to get into about the FDA ad comment about how that data was somewhat noisy. So I guess in the context of that, maybe a couple of things. One, why should we be confident that this five HT2A selective mechanism truly works in ADP?
And two, are there things on the design side that you can do with two zero four to better maximize probability and success? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: Okay. Thanks for that. I’m going to start back just a tiny bit even though I know you all you know all of this. But, you know, quick grounding, ACP two zero four is our new five HTa five HT2A inverse agonist. We designed it to improve on neplasid in three ways.
You know, we’re looking to reduce or eliminate QT prolongation, achieve higher exposures, and shorten time to study state. And so far, preclinical and clinical data that we have are supportive that we are accomplishing all of these. We’re getting some of those data out into the public medical literature, and we’ll be sharing more of it at R and D Day. So that’s a little bit of basic grounding more for the audience than for you, really. There was a primary there was a a prior parallel group study in ADP.
And as you note, you know, it did meet its primary endpoint. Week six was the primary endpoint for that trial, and there was efficacy demonstrated there. But there was less consistency across other time points. Some of them numerically look like they separate but didn’t meet statistical significance. And, you know, some of them didn’t even quite do that.
So I think that, you know, as we look at a number of learnings from the PUM program broadly, which we considered when we were looking at 02/2004. So that’s information from the parallel group trial as well as other trials. Really, I think at heart, we think that what we need here is more robust efficacy to get greater consistency across the dataset. We do think that the data set that we have are supportive of signals of efficacy in Alzheimer’s, but we need to have stronger efficacy in order to more robustly demonstrate that and demonstrate that across numerous endpoints. So as I think about that, there are a couple of things in the molecule that contribute to that.
You know, prior work on PIM does show some suggestion of an exposure response relationship from an efficacy perspective. And like I said, we demonstrated two zero four for the possibility to get to greater exposure. So that could contribute to that. The other thing that we did was designed to get to a shorter time to steady state that, you know, it’s not definite that that’s going to translate into earlier onset of efficacy, but it could. And as you note, you know, some of those earlier time points, there was some indication of efficacy but not statistical significant separation.
So we think that those are a couple of important things about the molecule itself that gives us the potential for more robust efficacy. There are some aspects to trial design as well, you know, certainly looking at a more severe patient population and focusing in specifically on the Alzheimer’s disease subset in an adequately powered way. Paul, Host: Is there something about this mechanism that makes it more potent for Parkinson’s? I think even in the randomized withdrawal study, the hazard ratio in PDP was 0.09, which is unbelievable, right? And in Alzheimer’s, it was good. I’m not dismissing it, but it was closer to maybe 0.6 or zero point six five. Like, why do you think there’s been that discrepancy? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: Yeah. I will say that the data Parkinson’s and, in Parkinson’s has been striking, obviously, most striking there. I don’t think that we have a clear explanation as to why that would be the case. It doesn’t seem, for example, that it’s a difference in exposure levels in the different patient, in the different disease populations. We explored that.
But again, I think even within Alzheimer’s, it’s a question of how much more can you do with this mechanism. And the exposure response we have is suggestive. You know, we’re testing this in our current trial. We’re looking at higher exposures to allow us to actually identify whether higher exposures do get you better efficacy. But the exposure response analyses we have to date suggest that that’s a possibility.
So we’re looking to maximize that with two zero four. Paul, Host: Okay. Okay. Do you want to talk briefly about Lewy Body dementia and the thought process there? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: Absolutely. Jumping a little bit off of what you just said, part of what we like about this is the fact that this is an area with small subsets, but some strong data from a PIM perspective. But again, I’ll take a step back and say Lewy body dementia is, you know, it’s associated with abnormal deposits of alpha synuclein. These Lewy bodies can impact thinking, movement, behavior, mood. Psychosis is a huge, huge issue in this patient population.
The hallucinations and delusions here are really significant. And I’ll say that there are antipsychotics that are commonly used in other psychoses that can actually be harmful for patients with Lewy Body. So the unmet need is significant. As we look at data from PIM, and I’ll be very clear that this is a small subset of patients. It’s about twenty patients per arm from the HARMONY study, and that was our withdrawal study.
What you see there is in patients who had Lewy body dementia psychosis in that patient population, those who remained on PIM, only about five percent of them relapsed, whereas those who were on placebo, about fifty five percent of them relapsed. So we do think there’s some good reason to believe from our historical PIM data, and we think that 02/2004, again, has the possibility of being able to bring potentially more here as well as an Alzheimer’s. Paul, Host: Yeah. What what are can you leverage the same endpoints in DLB, like the SAPS, or do you need something different? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: I anticipate well, you know, we’re going to be in phase two with DLB. And so what we’re going to be doing here is looking at a variety of different endpoints to see which one is most useful. But we will be looking about at Zaps as well as others. Paul, Host: Yeah. Okay. Okay. Great. Do you wanna briefly talk about, one zero one, the Prader Willi program and just give us a quick intro before I have a couple follow-up? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: I will do it as quickly as I possibly can. So ATP one zero one intranasal carbetocin, as you know, this was in a prior phase three trial that did not meet its primary endpoint, which was with the high dose. But a lower dose did show, did suggest that there was efficacy. There was a complete response letter for this, but we are running a new randomized placebo controlled Phase three trial looking at the dose that did seem to be effective in the prior trial.
Paul, Host: That is a quick intro. I like it. You know, why why did Acadia look at that data and think that the inverse dose response was not noise, but indicative of a real drug effect at the lower dose? Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: So, you know, I look to I like to look at a few things in this instance. Do we believe the mechanism makes some sense? Do we see some internal consistency to the dataset? And do we have any kind of alternative hypothesis for what could have happened here? So, you know, we do think that mechanistically, there’s some there’s reasonable reason to believe here about the relevance of oxytocin and Prader Willi.
We looked at the prior phase three and did see some consistency across time points, but also across end points that was reassuring. And, you know, while it’s not proven, there is at least a plausible hypothesis for the underperformance of the higher dose, which is about off target, off target impacts through the vasopressin receptor. So it’s a reasonable reason to believe at the end of the day we’re going to need a we expect that we’ll need a positive trial in order to support registration. We designed our currently running trial with those learnings from the prior Phase three in mind and we’ll see what comes out of it. Paul, Host: Okay. Fair enough. Catherine, maybe one last question for you. You mentioned business development in your opening remarks. Anything you can say there about Acadia’s strategy and what we should expect in 2025?
Catherine Owen Adams, CEO, Acadia Pharmaceuticals: I’ll start with the strategy and let Mark maybe add something. But in terms of our strategy, what we’ve decided to do is open the aperture a little bit beyond just neuro, psych and neuro rare to adjacencies of rare disease. We believe we have the capabilities to be successful beyond neuro rare. So we’re looking at, rare cardio, rare endocrine, rare metabolic, etcetera. And we have a pristine balance sheet, which Mark is eager to put in the right place.
And I’ll let him maybe share a little bit more about our strategy there. Elizabeth Thompson, Head of R and D, Acadia Pharmaceuticals: Yeah. I Mark Schneier, CFO, Acadia Pharmaceuticals: think financially we’ve got over $750,000,000 in cash, no debt. And, you know, debt positions are strong to execute to expand the portfolio. And, you know, as long as we find the right assets and they beat our financial metrics and everything, things are actionable, we’ll execute. Paul, Host: Anything you can say about like capacity for size? Mark Schneier, CFO, Acadia Pharmaceuticals: Yeah, I think we don’t Paul, Host: go ahead. Catherine Owen Adams, CEO, Acadia Pharmaceuticals: No, no, please go Paul, Host: ahead. Mark Schneier, CFO, Acadia Pharmaceuticals: Yeah, I mean, for us, I mean, our bias is to invest, right? I think for us, we’d lean in and I think what we’ve said since I started as CFO and we’ve turned cash flow positive, our bite size gets bigger every day as we kind of add cash to the balance sheet and you kind of don’t put an upper limit on it. Obviously there’s a certain level of just actionability in transactions that we’ve said kind of you kind of wouldn’t do things, we’re not likely to do things more than half your market cap. But if we find things that we are excited about, we’ll deploy the cash, we’re not afraid to raise capital if there’s things above our cash bite, but but that would have to be exceptional or not likely to happen, but things that we still look at and if something’s excited, we’ll lean in as I said.
Paul, Host: Fair enough. All right. Well, thank you very much. Thanks for engaging with me and thanks for joining the event. We appreciate it.
And thanks to everybody for listening in. Thank you so much.
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