Acadia Pharmaceuticals Inc (ACAD) May 12, 2026 Conference Transcript

Okay, guys.

Good afternoon. Welcome back to the Bank of America Healthcare Conference. I'm Tizia Namad. I am one of the biotech analysts here at the bank. It's my pleasure to have our next presenting company, Acadia Pharmaceuticals. Sitting up here on stage with me are a couple members from the management team. Of course, Catherine Owen-Adams, who is CEO of the company. Welcome, Catherine. And also Liz Thompson, who is head of research and development. Nice to see you again, Liz. So we'll go into the specifics of a couple of data catalysts that people have their eye on. But maybe, Catherine, you can start off by giving us an overview of the company. You know, you brought in a new era to Acadia. And maybe you can talk to us about changes that have happened to the company since you became CEO.

Sure. So I've been enrolled now just over 18 months. And, you know, the initial focus for us was to stabilize the commercial business and also look for new growth opportunities and bringing in Tom Garner as the new CCO. I think we've looked at the business through new eyes and reassessed the opportunity and have made some significant changes for both New Placid and Debut in terms of the field for sizing and the commercial strategy. and as a result of that at JP Morgan this year we were able to guide to a 1.7 billion opportunity for both of those commercial brands by 2028 and then Liz who came in about two months or three months ahead of me we've been working together Liz particularly building her team and looking at how we can invigorate the pipeline both looking at how we can accelerate the phase two and phase three opportunities but also look at the whole pipeline and start to make decisions on where we're going to invest. And then in terms of BD, we've definitely got a nice balance sheet, 851 million, and we're no debt. And so we've been looking actively at BD opportunities and really in two areas. One is later stage that we could add to our commercial portfolio within the next couple of years. And we've been looking, as many other people are, in the areas of rare disease to try and sort of ensure that we can have something beyond debut and look to build on the on the great commercial infrastructure we have there. And we're also looking slightly earlier in the pipeline to add to that. So we're looking at sort of preclinical. And then we have a really nice bolus of phase two and phase three products, which I'm sure we'll spend a lot of time on today, which are going to come to fruition in the next couple of years. Okay, great.

So maybe let's talk about currently marketed products just for a couple of minutes. So for New Placid, that's a maturing launch. It's been on the market now for some time. But you felt it important to continue to focus attention and time on this launch. You had a sales force increase in order to focus on areas that could increase demand. Can you talk to us about how that's been going and what early indications of upside you're seeing?

So, you know, when I came into role, the peak sales, depending on the analysts, was, you know, $650 to $700 million for New Placid. As I said earlier, Tom and I looked at it again and we felt we could really drive some incremental growth into that figure. And we've guided now to a billion dollars in 2028. And the confidence behind that is based on the fact that we have a great brand that has a strong clinical profile. And we didn't feel that it had really been looked at in terms of its commercial opportunity through the lens that we wanted to see. And so we have increased the field force by 30%. And the focus of that is to really get to about 4,000 to 5,000 new physicians. So we have been looking at the impact of our direct-to-consumer campaigns, and they've really raised the awareness of hallucinations and delusions amongst the caregiver population. And we've been seeing new types of prescribers for Newplazid beyond neurologists. And that's in sort of PCPs and MPPAs that are now treating more patients with Parkinson's disease. So we've really almost doubled our sort of physician population that we're going after with the field force, as well as revamping our direct to consumer campaign, working with a new agency and signing Ryan Reynolds for the unbranded campaign, which has been incredibly impactful. we've had a fourth fourfold increase in the awareness of hallucinations and delusions since we started working with ryan we have a new campaign coming out next week which we're really excited about and so both of those two things together gives us confidence that we really have a lot of room to grow new plazard in this market we're roughly depending on how you look at the market basket about 20 to 25 percent share and we think we have the um the ability to go way beyond that. Okay.

And as you think about, you know, the IP runway, so you did win some important pieces on patent challenges. Yeah. So how should the street be modeling sales in terms of just strict IP expiry now?

So we have Composition Matter out to October 2030 and MOU out to 2038. So we're looking at our runway to 2038. And the I guess the the biggest sort of inflection point for New Plaza over the next sort of five years or so will be the 29 to 31 timeframe where we will highly likely get IRA price reductions. And so we do qualify for the small company benefits. So we step into those price reductions over the first two years. And then we do expect those price reductions to come to New Plaza. But even so, we still think there's a strong runway beyond that in terms of having New Plaza on the market. And so that's how we're looking at it in terms of out to 2038. Okay.

And then for debut and Rett syndrome, the launch had started off with a lot of enthusiasm and demand from the community and from physicians.

Talk to us about, you know, sort of like the intermediate stage of the launch what you saw what tweaks you think were needed and and what you're seeing as a result of making some changes yeah so um one of the initial tweaks was to increase the size of the field force we didn't feel we were getting to enough of the community physicians 65 percent of rep patients are treated outside of centers of excellence and we really didn't have enough reach and frequency on those physicians to get to them um so we increased our field force initially and then we've also been evolving our patient support model to ensure that we're really thinking about the patient journey right from actually prior to the prescription starting to all the way through and beyond and we've really increased our focus from our family support educators and our msls in terms of educating the physicians and we've seen a nice stabilization in terms of those initial sort of first i think few quarters post the launch hump where we saw this sort of increase in in in dropouts on on debut due to the GI side effects. We've learned a lot through the process. We've got a white paper now out now on how to titrate patients looking at how how you can increase patients over time. And we've got a very steady persistency rate now, which is actually increased a little bit since we first came on, which is 55 percent at 12 months. And we've just launched debut sticks. And so we've had a lot of community interest and physician interest in that. And we're really excited to see what incremental opportunities that now gives us to penetrate the market.

Yeah. So maybe explain what DB6 is for those who may not know.

Yeah. So it's a powder for oral solution. It comes in a sachet, a little bit like a liquid IV that you get to add to your water. And it's much more flexible for our caregivers. So it doesn't have to be refrigerated. It's obviously a lot smaller in terms of the size. it can be importantly mixed with any liquid apart from dairy that the child prefers so apple juice lemonade iced tea whatever it might be and importantly also the volume that the caregiver can choose to to mix it to is is about half of the volume of of the liquid and we've also removed red dye 40 and maltitol so from an overall patient experience perspective it's just got a lot more to offer the patient and the caregiver. And because of that, we're seeing three types of patients now coming back to debut. First of all is switch patients, patients that are currently on liquid who are looking to switch over. The second is new patients who would not start debut because of mainly the maltitol and red dye 40. A lot of these girls are on a keto diet and their parents didn't want to start them. And then the final group is patients that have discontinued debut in the past who are now coming back to try debut sticks and so those the three groups we're now seeing who have a lot of strong interest in in the oral solution.

Is there one of those groups that's more represented?

So in terms of the the first quarter we launched mainly in the centers of excellence because we wanted to get experience with physicians who understood how to use debut we had 250 prescriptions for debut sticks of which 30 percent were either new to debut or restarts on debut. And so the rest of them were switches, so 70-30. And then for the foreseeable three years, we've guided to about 450 incremental patients that we believe will come to debut as a result of the powder for oral solution. And so we're tracking slightly ahead of that right now, and we'll see how Q2 plays out. Okay.

So maybe let's just move to the pipeline. um so acp204 remlefanserin liz i'm sure this is now super near and dear to your heart with all the questions that you're getting so just remind us this is a study that's a phase two that we're expecting to see top line data for between august and october of this year um just remind us of that study design and then we'll go into a few more questions so briefly just sort of set the stage of remlefanserin, ACP204, is a cousin of pimavanserin.

It was informed by a pretty vast amount of data on pimavanserin, both from a molecule perspective and from a program perspective. And so, you know, nuplazid is a good drug, does good things for people. It does have a QT prolongation signal. And so that can be important in and of itself in an elderly patient population, but it also limited our ability to dose range with pimavanserin. That's important because we've seen some indication that there's an exposure response relationship for pimavanserin with efficacy in Alzheimer's disease from some of our many prior studies there. So here with remlifanserin, we have the opportunity to look at higher exposures and see if we're able to get more efficacy. And also we have a faster time to steady state, which could translate into quicker onset of efficacy. We've also focused our Alzheimer's program. And so now I'll talk a little bit more about the study design aspects. One of the main learnings from our prior history in Alzheimer's was the importance from a regulatory perspective about having a study that is devoted entirely to the patient population you're looking to get indicated in. So in our phase two and the follow on phase threes, we're requiring a clinical diagnosis of Alzheimer's, but we're then confirming that with biomarkers, which we think is going to be hopefully helpful from a technical perspective, but I think very important from an eventual regulatory perspective to be able to prove that we have the population that we want. Our primary endpoint is at week six. It's something called the SAPS H&D, which focuses in on hallucinations and delusions. It is an instrument we have used in the past with Pimavanserin. What other things are important to note? The three-arm study placebo-controlled, the lower dose that we're using of remlefanserin is roughly equivalent to the exposures you get with the currently marketed dose of nuplazid. And the higher dose that we're using is roughly twice that exposure. So it's going to give us the opportunity to see if we can get more efficacy out of higher exposures.

Okay. So a couple of things. Is six weeks enough time to detect if you're seeing efficacy?

So we'll say if we look back at the pivivanserin data set in Alzheimer's in a phase two study, we did have a positive primary endpoint at week six. That was part of what led us to choose this. And we've seen efficacy by week six in other aspects of the new plazid program. Hopefully with a faster time to steady state and potentially faster onset of efficacy, we will see more consistency efficacy across that time frame. But we think week six gives this mechanism time to work.

Okay. And then as far as the doses that you've chosen for the higher dose where you have double the exposure, is there any concern about safety profile that comes into play there?

Yeah, I mean, you always have to think about the there's exposure response for efficacy, and oftentimes there's exposure response for safety. What I can say is a couple of things. You know, overall, aside from the QT prolongations and signal, the nuplazid safety database has been fairly reassuring, but of course, that's the exposure for the lower dose. We've used remlifanserin in a variety of phase one trials, and some of them have gone as high as 180 milligrams of dosing, but of course that is in short-term and limited numbers of patients, but seemed to be generally supportable there. And always it's a blinded thing, so it's going to depend how things sort out, but at least at a blinded aggregate level, so far the safety profile appears to be we're not seeing anything concerning in blinded data. We'll see how that sorts out eventually. But thus far, we think there are promising signs. Anything on QT prolongation? So we feel pretty good about the QT prolongation based on the phase one work that we've done. So I feel pretty confident from that perspective. Okay.

Now, what should we be expecting to see when you release that data later this year? Level of data to expect in the press release, assuming it's on a press release. And what are you going to hold for, let's say, a medical meeting?

So I don't know exactly what we're going to hold for a medical meeting. What I can say is that we're currently anticipating that we would be sharing a comment on the primary endpoint that week six SAPS H&D, as well as general commentary on the safety profile. That's sort of the minimum set that you should expect. We may very well share additional information as needed to sort of contextualize what we're seeing. There's a number of aspects of the clinical data that we're looking at in terms of thinking about what a successful product could be and what we want to see.

Have you talked about what effect size you would think would be clinically meaningful?

Well, so what we've talked about is the effect size that we're currently powered for. And I will say that I think that something a little bit below that probably would also be clinically meaningful. But what we're powered for at this point is a 0.4 effect size or a moderate effect size, 80% power. So again, this is a phase two. Our primary goal here is phase three enabling. But we feel confident in the KOLs we worked with as we put the program together, felt that if we saw the level of data we're powering for, that would be a win. Okay.

So you get your data, you top line it. What would be the next steps? Making arrangements to go talk to FDA? Or have you already had some preliminary discussions about what a phase three would look like?

Yeah. So we have a bit of an unusual situation here in that we have a master protocol that covers a phase two and two phase threes. It's operationally seamless And so what that means is once we stop enrolling in the phase two, we start enrolling in the phase three. And so I'm saying, but it is statistically separate. So we're able to evaluate the phase two and apply learnings to phase three. The reason I'm saying this is we talked to FDA before we went into the phase two, phase three program. We have the opportunity to learn from the phase two and potentially make modifications to the phase three. Depending on those modifications, we may want to go talk to FDA again to make sure those are still in line with thinking. but that's going to depend a little bit on the scope of what they are. I'll say in general terms, the kinds of things I think about that we might consider modifying. Right now, we do have, I said, the placebo 30 and 60 milligrams. If there were evidence that made us feel really good that one of those doses was the right one to take forward, we might well consider dropping one of those doses. That has a number of advantages, but one would be presumably speed. There is the possibility that we may need to modify our estimates around effect size, and that could have sample size implications. And there could be things that we would think about from an endpoint perspective. We are looking at SAPs and H and D, but we are considering other endpoints as well.

Okay. So I guess why choose to kind of have an outline already as opposed to wait to see this phase two and then figure out what phase three looks like?

Yeah. So I think part of it was we felt good about many aspects of this. And so while I'm saying these are things we could do, that doesn't mean these are things that we definitely think we're going to have to do. Right. So the intent was to have the benefit of the time savings of starting up that enrollment directly, but then the opportunity to make modifications if we need to.

Okay. And how are you thinking about the competitive landscape for ADP?

So I'll make a couple of comments and then hand it over. I mean, I think one thing is that this is a pretty substantial unmet knee. There's a large amount of patients out here. I think that there are some other later stage programs also looking at psychosis. There are some related but separate things that are looking at agitation. We think we have something that's mechanistically distinct in terms of those agents that are currently looking at the psychosis component. actually, overall. And so I think that if we have the kind of profile out of remlefancerin that we're hoping for, we think that could be a really powerful agent for patients. Okay.

You bring up a good point on agitation, and I wanted to talk about agitation versus psychosis. And do people use these terms interchangeably? And are AA drugs viewed by physicians as eligible to treat psychosis?

So they may use the terms interchangeably, but they are not interchangeable. Agitation can come from a number of different sources. It's a pretty complex phenomenon. It can be driven by pain. It can be driven by confusion. It can be driven by psychosis. It can be driven by just separate mechanistic anxiety-related behaviors. And so by and large, the agents that are targeting agitation don't generally target the underlying driver of that agitation. So I think that, you know, as I think about it, something that is effective in agitation may or may not be, and frankly, probably isn't effective in treating the underlying manifestations. On the flip side of the coin, as we look at psychosis, I don't expect that we would be able to address agitation broadly. What I do think, and we've got some data from New Plaza that suggests that in patients who have significant psychosis and associated with that, they also have significant agitation, In those patients, when their psychosis got better, their agitation got better. And so I think that we have a possibility within the psychosis realm, I think we could treat the underlying and the resultant agitation potentially, but again, not agitation more And the agitation agents are by and large not going to positively impact psychosis. And some of them based on mechanism may actually have not helpful impacts on psychosis.

And I think just as a market as a whole, I think probably people are very well aware, but, you know, there are 7 million patients with Alzheimer's in the US and about 30 percent of those will have a psychosis episode during their diagnosis. So for us, there's a large market of patients who currently don't have an approved medication because of that. I think there's a lot of competitive focus on the space. And that's great because patients need choice. And we all know that within just psychiatry generally, different MOAs, different patient types, there's co-prescribing, there's all sorts of ways to treat these patients. So just in terms of the competitive environment, of course, we expect competition, but there's a lot of patients and we believe that we hold a specific space in terms of the type of patient that we can treat. We'll see with the data what that looks like. But I feel confident that there's room for quite a few players in the space, and it will be better for patients to have those different choices.

Now, several years ago, Pima Vanserin did do an umbrella study where ADP was looked at, and we've talked about this a few times.

But just to put a bow on this conversation, if you will, the difference in what you're doing with with 204 mechanistically what gives you more confidence that it will have the desired effect that pima did not so i would say that for me at least it's a little bit less a mechanistic question and more a a little bit of the molecule parameters we talked about with the potential for greater efficacy greater speed to efficacy etc but a lot is about the you know the umbrella drp trial wasn't powered for any of the individual subpopulations. And I think that, you know, I'm not trying to condense all of the challenges in that indication down to that one thing, but I think that was a pretty substantial component. So the biggest thing that we're doing differently here is having a very Alzheimer's specific program that then again goes that sort of extra mile with the biomarker confirmation. Okay.

But the flip side of the argument is that Lewy body did work, even though it wasn't that umbrella study was empowered. So, you know, we feel particularly bullish on that study being positive in 2027, I believe is the readout. So wanted to get your thoughts about, you know, is that observation from Pima the reason why you chose to do a Lewy body study for this asset as well?

Yeah, so I was very, the Lewy body study is the first program I started when I got to Acadia. I was very intrigued by the data. Small patient population, exactly as you know, but pretty striking data. So I think that that is a really exciting opportunity for remlefanserin. And I will note that it is also, while it is a smaller patient population than Alzheimer's, it's about a million patients. It's still quite large. It's disproportionately impacted by psychosis. 50 to 75 percent of patients with Lewy body are going to have psychosis as part of their journey. And from a clinical development perspective, there's less going on there. So I think this is a really impactful constellation of something where I have some good data to believe in from a mechanism perspective and some really substantial unmet medical need what do patients with louis body take now if anything it is well it is um a lot of off-label yeah yeah which you know given that they do have motor impact can be very problematic for them the cognition impact can be really difficult um so yeah it's there are good options so in terms of sites that you use, what was the overlap in ADP sites versus Lewy body sites?

And can you compare the pace with which these enrollments occurred?

So we haven't yet shared our expectations on when Lewy body is going to be done. So what I can say is, you know, we are looking at a somewhat smaller study for Lewy body. And in part that is because we're expecting a somewhat larger effect size. I anticipate that it will probably not take as long as the Alzheimer's program, but part of that may be sample size. There is some overlap in sites and some distinct sites that we're working with. Okay.

And is it the case that for studies where you're looking at these types of indications, the same concerns might pop up as for study sites for, let's say, schizophrenia, where there's concern about, you know, certain patients being enrolled in too many studies, for example?

I think that we see maybe not to the same extent those dynamics, but I think we are very cognizant of doing everything we can to ensure data quality that the patients are, you know, appropriate patients. I think in the Alzheimer's trial in particular, the fact that we're biomarker confirming these, we're pretty confident these are the right people to have in here. So there can be some of that dynamic, but I don't think it's as significant as you see in some of these other spaces.

Okay. And in a few minutes we have, maybe let's talk about 2-1-1 in a major depressive disorder. Sure.

Yep.

Okay.

Okay. So ACP-211 is our selectively deuterated are nor ketamine. We are looking at this in major depressive disorder. It is in a phase two trial right now. The sort of promise of this is ketamine-like efficacy with a different patient experience. We base our expectations of that on preclinical and early clinical data. Preclinical data suggesting efficacy and absence of sedation. It's kind of hard to measure dissociation in preclinical studies. But in early phase one, we have seen no sedation and only low levels of dissociation at the highest doses. And that's in healthy volunteers, which can be a little bit more sensitive to these things. We designed our phase two in part, of course, to look at efficacy, but also to try to help us rule out unacceptable levels of sedation and dissociation. And we are looking for the proof of concept readout in that in roughly mid-year next year.

Okay. And on that, just because there's so many mechanisms that are being developed for MDD, how are you thinking about where this could fit in?

I mean, I think that you're right. There's a lot going at this. I think that this is widely understood all the way up to the president's executive order around psychedelics, widely understood as a massive unmet medical needs space with huge numbers of patients suffering. I think that a profile that is consistent with what we're hoping that 2.11 can be could be pretty impactful for patients. And I think that's true, again, to Catherine's point earlier about these are spaces where having a variety of mechanisms handy is a good thing for patients. I think that's going to be true here. And I think this profile is pretty compelling.

Yeah, I think the oral nature of the product is an additional benefit to patients. And to the point that Liz just made, there's plenty of competition already, but, you know, the ketamine-like efficacy without the dissociation and the monitoring required of the total patient experience is something that we're really holding out hope that we can launch into this market.

I did want to ask you, I mean, there are companies that are leaning into psychedelics. And so how do you think that this is, you know, a type of disruptive treatment, you know, to go against the grain on traditional, you know, antidepressants? There's obviously a certain subset of patients that don't respond to any treatment so far. And do you think that, you know, allowing things like psychedelics could also in the in the longer term also help, you know, products like what you're trying to develop become a little bit more seamless in how FDA chooses to look at data?

You know, I think psychedelics are obviously the flavor of the moment a little bit right now, both with the administration and also the companies that are in that space. I think for us, there's room for many opportunities. And I don't want to talk specifically about whether I think psychedelics are good or bad, other than there's obviously a high unmet medical need. What is kind of important about getting these products to patients is the schedule and the control nature of getting drugs to patients. And so psychedelics will be highly controlled no matter what happens. And so that's going to be a very specific channel in terms of commercialization. you know having had quite a lot of experience with uh with fentanyl um from my gerogesic days um it's it's a specific commercial um execution yeah opportunity that you have to really be very careful about and it's a very um it's you have to have high ethics and and high monitoring of of the whole supply chain to get these products to patients and so it's quite a complex a complex area. And so I feel like the ketamine-like efficacy with the patient experience that we're aiming for is something that we can achieve both clinically through our clinical study, but also commercially. And I think those two things together are actually quite important. You need both to be successful.

Okay. And I guess last question, based on today's update, there looks like there's going to be staffing changes at the FDA again. And so does that impact any of your expected interactions with the agency near a term?

Yeah, I guess what I'd say is, you know, putting on my just biotech hat, I think that getting to a place where we've got a little bit more stability in the agency is going to be a good thing for all of us. Wearing just my Acadia hat, I will say that a good part of our portfolio has been we've had pretty consistent review teams sort of throughout. We've been most of our products, certainly our later stage ones are in the division of psychiatry. And Tiffany's been a pretty consistent force throughout and the review team's been pretty consistent. So thus far, not expecting an Acadia specific impact, but it would be great to get to a little more stability.

Okay. With that, I'll say thank you for joining us here today. And thanks everybody for listening.