Q2 2025 ACADIA Pharmaceuticals Inc Earnings Call
Good day, ladies and gentlemen. Welcome to the ACADIA Pharmaceuticals conference call. My name is Gerald, and I’ll be your host for today. I would now like to turn the conference over to Al Kilany.
Senior Vice President of Investor Relations and Corporate Communications at Acadia, please proceed.
Thank you. Good afternoon, and thank you for joining us on today's call to discuss. Acadia's, second quarter, 2025 Financial results.
Joining me on the call today from aadia, our Katherine Owen Adams, our chief executive officer who will provide some opening remarks, followed by Tom Garner. Our chief commercial officer who will discuss our commercial Brands debut and New Plaza.
Also joining us today is Elizabeth Thompson, PhD Executive, Vice President head of research and development who will provide an update on our pipeline programs.
And Mark, Schneider, our Chief Financial Officer who will review the financial highlights.
Catherine will then provide some closing thoughts before we open up the call for your questions.
We are using supplemental slides which are available on our website's events and presentations section.
Before proceeding, I would like to remind you that during our call today.
We will be making several forward-looking statements within the meaning of the private Securities. Litigation Reform, Act of 1995.
These forward-looking statements including goals expectations plans prospects growth potential timing of events, future results. And 2025 Financial guidance are based on current information assumptions and expectations that are inherently subject to change and involves several risks and uncertainties that may cause results to differ materially.
These factors and other risks associated with our business, can be found in our filings made with the SEC.
You are cautious not to place undue Reliance on these forward-looking statements, which are made only, as of today's date. And we assume no obligation to update or revise. These forward-looking statements as circumstances change, except as required by law.
I'll now turn the call over to Catherine for opening remarks.
Thank you, Al, and good afternoon, everyone.
Our second quarter performance reinforces the momentum. We're building a cross. All facets of our business from commercial strength to our clinical Pipeline and Global expansion.
We delivered total revenue of 264.6 million this quarter.
Driven by strong growth across our portfolio.
This includes 96.1 million from debut with patient uptake, continuing to grow for the second successive quarter.
And 168.5 million from New Plaza. It costed buy new patient starts and Commercial execution.
Debut has now passed; stabilization moved into growth.
And acceleration.
Our investments in patient support Community engagement and field expansion are starting to make a positive impact as Tom will highlight in more detail.
On New Plaza. We're encouraged by continued growth across all leading indicators.
Bolstered by recent litigation wins enforcing our intellectual property.
These victories validate, our long-term strategy, and reinforce our ability to deliver new closet to patients through 2038.
Our full year Revenue guidance, reflects this confidence.
We are raising the low-end of our new Plaza guidance.
Based upon a foundation of disciplined execution and momentum delivered by our DCC campaigns.
We also hosted our first R&D Day.
Our Milestone event that showcased our exciting and expanding pipeline.
With 9 Des close programs, and development and 5 Phase 2 or phase 3 data. Readouts expected through 2027
Take 5 more within Acadia is palpable?
This will walk you through that shortly. First, let's start with commercial over to Tom.
Thanks Katherine.
Let's begin with debut, Q2 sales were 96.1 million up 14% from a year ago.
We observed steady progress across key performance metrics in the quarter.
In Q2 987 unique patients in the US received paid shipments increasing from 954 in q1 and 920 in Q4 2024.
The upward trend reflects encouraging signs of growth in both new patient starts and persistency.
Long-term persistency remains a key strength for debut.
Our 12-month persistency rate continues to exceed 50%, and we're now pleased to report that our 18-month persistency rate is above 45%.
This trend reflects the growing and stable base of patients who remain on therapy over time.
When we look at length of treatment among patients currently on therapy, 70% have been on treatment for at least 12 months.
Highlighting the sustained benefits that debut continues to deliver and the durability of our growing patient base.
notably, we're seeing more new prescriptions from the community setting signaling progress in broadening reach Beyond academic centers, which was a key element of our Field Force expansion which is now complete
We finished hiring and training earlier this quarter. And we're already seeing encouraging signs that the new customer model is gaining traction.
1 of the most promising indicators in the second quarter is a meaningful. Uptake in our proportion of new referrals coming out outside of Center of Excellence, where a majority of rep patients, receive their primary treatment.
Encouragingly, this increase was underpinned by a further increase in the number of new community-based writers for debut.
with 900, total hcps now, having written
Additionally, we launched a direct consumer campaign for just for debut in July and we are already seeing positive signs of early engagement from the rep community.
During Q2 we welcome Alison McMillan Young Blood a senior vice president of our rare disease franchise.
Allison brings a breadth of commercial experience across the US biofarma business, including many launches,
Her leadership will help to drive this critical business forward.
As we look ahead with the expanded team. Now in place, we're continuing to execute against our strategy to drive long-term, sustainable growth for debut.
We expect this will translate into an increase in new patient Stars towards the back. End of the year.
At the international rep syndrome Foundation meeting in June, our presence, as a leader in the ref therapy. Field was felt as Liz will detail, we shared multiple compelling posters that add to the body of evidence for debut?
Let's now turn our plans to trap outside the US.
In the EU where the prevalent population is estimated to be between 9 to 12,000 rep. Patients, name patient Supply as requested by local hcps is available through clinigen.
we've received requests from multiple countries and are continuing to support patients, where regulatory Frameworks allow
Name patient supply based upon HCP request is also in place in Israel through Rafa and in selective rust world countries through Farmer Mando.
use momentum of debut in the US and with the global interest in where we have already started serving patients through these programs
Now, turning to New Plaza, our commercial clean delivered. Another strong quarter with revenue of 168.5 million up 7% from a year ago, driven primarily by volume.
We saw strength across all key metrics in Q2.
Referrals were up 17% year-over-year driven by continued. Momentum in our direct consumer campaigns.
As a result for the first time ever, both referrals and new prescriptions, increased sequentially from q1 to Q2.
Another highlight of the quarter was shipping the highest number of new positive bottles since launch.
The strong indicator of growing demand. As we continue educating both Healthcare Providers and patients with PDP about the product's unique and differentiated profile.
The DTC campaigns are doing exactly as we intended.
Sparking meaningful conversations with healthcare providers and reinforcing our commitment to educating caregivers about the symptoms of hallucinations and delusions related to Parkinson's disease.
Visits to new plaza.com have increased 17-fold in consumer traffic year-over-year, helping to drive more patients to speak with their physicians.
To carry this momentum forward, we're pleased to extend our agreement with Ryan Reynolds and the multi Parkinson's campaign through February of 2026.
The campaign continues to drive. Meaningful awareness of hallucinations and Illusions in Parkinson's disease.
And we believe a strong portion through year end is essential to build on that success.
This quarter's new planet results reflect the strong executional focus of our field. Teams coupled with the ongoing positive impact of our DTC efforts.
Taken together. We expect to see sustained growth through the second half of the year and Beyond
And with that, I'll hand it to Liz to review our pipeline progress.
Thank you, Tom.
Let me Begin by. Revisiting the highlights from our inaugural R&D Day held in New York on June 25th where we showcased the strengths and breadth of our Pipelines.
We were pleased with how the event came together and I want to thank many of you on this call who are able to join us.
well, we had already disclosed, all the programs on this chart R&D day gave us, great opportunity to provide more details, particularly for 2, new molecules
The first is ACP 211 an orally administered selectively deuterated form of Arne or ketamine which were developing for the potential treatment of major depressive disorder.
Dated State support the potential for efficacy, without significant sedation or dissociation a profile. We will continue to explore in Phase 2 and Beyond
The second is ACP-271, a GPR88 agonist with potential applications in tardive dyskinesia and Huntington's disease.
This is some of the most novel biology in our Pipeline and we believe it to be the first GPR, 88 Agonist that will enter clinical trials.
If you weren't able to participate in our R&D day, I highly encourage you to visit our website where you can access the webcast and the full set of presentations slides.
The event was a clear demonstration of the momentum building across our pipeline. We're advancing with purpose and Clarity and the progress is tangible.
Across our 9 Des programs. We anticipate initiating 7 Phase 2 or phase 3 studies over the course of 2025 and 2026.
Moreover between 2025 and 2027, we expect 5 Phase 2 or phase 3 readouts.
These Milestones underscore both the breadth of our Pipeline and the strength of our R&D strategy.
Specifically, we have several important trial, initiations and data readouts on the horizon.
In Q3 2025, we plan to initiate a phase 2 study of ACP 204 in Lewy Body dementia psychosis.
Also, in the third quarter, we expect to initiate our phase 3 study of traits in patients with rat syndrome in Japan.
In early Q4 2025, we expect to report topline results from the Compass PWS Phase 3 study of ACP-101 in Prader-Willi syndrome following the completion of enrollment in Q2.
In major depressive disorder.
wrapping up the year, we plan to begin a first inhuman, study of act 271 in healthy volunteers before year end
And finally, we also continue to progress through the review process with EMA. For tinit, with agency decision, expected in the first quarter of 2026,
In addition to these upcoming milestones, we continue to make meaningful contributions to the scientific literature for our marketed and pipeline products.
The second quarter was a busy one for ACADIA. Relevant medical meetings included major conferences for Rett syndrome, Prader-Willi syndrome, and Alzheimer's.
First, there were multiple debut related presentations at the international rat syndrome Foundation meeting continuing to expand the body of evidence. Describing debuts long-term impact in the rat community.
Several posters focus on caregiver, reported outcomes, and real world data, which importantly continue to align with what we have. Observed in clinical trials,
Additionally, there were analyses, evaluating treatment utilization, in less commonly studied populations such as males and older patients.
And finally, aadia shares, an analysis of our clinical trial data suggesting that most patients who are going to benefit from debut, show a response within 6 months reinforcing our guidance to Providers and caregivers.
Together these data points, reflect a growing and maturing evidence base, that continues to shape how debut is understood and used in the real world.
In the pipeline, both ACP 101 and ACP 204 teams, had important meetings in the quarter.
In proder Willi syndrome, the end of June marked the United and hope meeting the joining together of 3 separate PWS organizations in a combined meeting.
Those of you who tuned in to R&D day will recall that our PWS panel in part was live from United and post.
as we await data out of our phase 3 trials, we nevertheless are contributing to the knowledge and understanding of PWS with posters about the experience of patients and families, exploring comorbidities, and the associated burden
In the Alzheimer's space. I'm also pleased to report multiple ACP 204 presentations at the Alzheimer's Association. International Conference held last week in Toronto.
This meeting represented, a significant step in our public disclosure of detailed data from non-clinical and early stage clinical studies supporting act, 204's attainment, to date of its desired profile.
presented data included the specificity for 5hd and the supported PK profile, indicating a faster, time to study State, support for daily dosing, the lack of QT, prolongation, and the ability to dose with and without food
We also shared clinical data primarily focused on the 16g dose across. Several Phase 1 trials with supportive safety and tolerability profiles to date including in healthy elderly subjects.
Collectively the data provides support for the potential utility of ACV, 204 and key aspects of its Target profile.
Lastly, I'll touch briefly on ACP 101 where we continue to expect Topline results in early Q4.
Just as a reminder, this is a 12-week, Placebo-Controlled Parallel Group study.
That design is important because if successful, it would allow us to clearly describe to physicians and patients what to expect upon initiating therapy in a Prader-Willi syndrome patient population.
Should the data be positive? We anticipate being in a position to file and the first quarter of 2026.
And we continue to anticipate that this will qualify as a resubmission with the FDA with the associated shorter. Potential review clock with the potential to do for date in the third quarter of 2026.
And now, I'll pass over to mark for a financial overview of the quarter.
Thanks Liz. Let's now review our second quarter, 2025 Financial results
The second quarter was strong across the board, with 264.6 million in total revenues up 9% year-over-year.
In the quarter debut, net product sales of $96.1 million represented a 14% year-over-year growth, including 12% volume growth, primarily reflecting the increase in unique patients receiving shipments in the quarter.
The debuts roasted at adjustment for the quarter was 23.3%.
8.5 million up 7% year-over-year with 5% of that growth attributable to volume.
The new Plaza grows to that adjustment for the quarter was 24.6%.
R&D expenses were 78 million in the second quarter up slightly from 76.2 million in the second quarter of 2024.
Sgna expenses for the second quarter were 133.5 million up from 117.1 million in the second quarter of 2024.
The increase was primarily driven by increased expenditures for both debut and New Plaza in the US including the planned expansion of the debut commercial team.
We ended the quarter with a cash. Balance of 762 million up from 681.6 million at the end of q1 and 756 million at the end of 2024.
Let's turn to our 2025 guidance.
We are raising the low end of our new Plaza guidance range, reflecting the strength of the business and its performance to date.
We now expect New Plaza, net product sales for the year to be between 665 and 690 million.
This Compares with our prior guidance, range of 650 to 690 million.
Accordingly. We are also adjusting our us-only, total revenue guidance, to reflect this change.
As you can see on this slide, we are reiterating all other prior guidance ranges from our q1 call.
We're confident in our ability to execute against these targets, and to continue creating value for patients and shareholders.
I'd now like to hand it back to Katherine for closing remarks.
Thanks Mark.
As you've heard quarter, 2 was a quarter of progress and momentum.
Our teams are executing with urgency and precision.
And we remain focused on accelerating, debuts commercial trajectory.
Sustaining long-term growth and differentiation for New Plaza.
Advancing a deep pipeline through rigorous, clinical development, and continuing to build the pipeline, through Business Development.
And expanding globally to reach more patients in need.
The next major Milestone is ACP 101's data readout in early Q4.
And we're hopeful for what that could mean for families Living With Pride or Willie syndrome.
Thanks for joining today's call and thank you for your continued support of Acadia.
And I'll now open it up for questions, operator.
Thank you. At this time. We will conduct the question and answer session. As a reminder, to ask the question, you will need to press star 1 1 on your telephone and wait for your name to be announced.
To withdraw. Your question, press star 1 1, again please, stand by while we compile the Q&A roster.
Our first question comes from test Romero from JP Morgan. The floor is yours.
Good afternoon team. Thanks so much for taking our questions. So I actually wanted to ask about ACP 1011 today. Liz, maybe. Could you Orient us to the approach? You plan to take with your Top Line? How much detail will you provide? And will this be more qualitative or quantitative in nature?
And, um, are there any secondary or other endpoints that you will think will matter Beyond HQ CT? And then as a follow-up, where are you really most focused from a clinical trial, conduct perspective to manage any key risks. Thanks.
Are, um, expected area, are expected ranges. So, those are the areas that we've been focusing on, in terms of conduct and making sure that we have as consistent, a behavior as possible across the sites.
Thanks, Liz. Thanks for that question.
Thank you for your question.
Our next question comes from reto, Bal from TD Cowen. The floor is yours.
Good afternoon. Thanks for taking the question. Um, I wanted to talk a little bit or ask a little bit.
Um, the new momentum in, in debut. Um, could you, um, could you tell us what percentage of, um, patients or or new patients specifically,
Um came from the community setting and um you mentioned uh the the new number of 900 hcp writers what percentage or at least movement of those writers were in the community, setting on um, a quantitative basis. And then I have a greater Willie follow-up as well.
Thanks, Richie. We'll take the the daily questions first, I'll ask Tom to give you some more details on that. Perfect. Yeah, good afternoon, Rita. Hope you're doing well. So, um, in terms of your first question regarding the, uh, penetration that we've seen as, as a result of the increase in the sales force, which, as a reminder that went live kind of through May. So we're we're kind of fairly early on in terms of seeing the impact,
But we're pleased with what we're seeing already in the quarter. We actually saw that um the number of referrals that were coming directly from our non Coe accounts actually grew to about 3 quarters of our overall number. So that was up from around 2/3. The uh, the quarter before which I think is a a nice increase in terms of um, being able to penetrate that large group of patients that we know exists that fall outside of our Coe. So just as a reminder, roughly 65% of our patients fall outside, the direct care of 1 of the Coe uh centers uh spawn Coe designated centers.
Um with regards to your second question as to the additional writers that we saw through Q2 the vast majority of those new writers fell outside of the coe's which given the fact that we saw this nice uptick in terms of penetration through the quarter. I think again it's a nice leading indicator that the the um new model that we have in place is beginning to pay dividends for us.
Tom, do you want to ask your 101-question routine?
yes, and just
um, can you talk to how the conduct specifically dropouts, um, of the phase 3 prayer, Willie have gone, or they within expectations and all are, the are all the dsmb looks, um, for the trial completed, and if the sap has been finalized with FDA,
So I am not going to comment on data from a currently ongoing trial aside from to say that generally speaking. We are continuing to see this trial, um, unfolding in an acceptable way. But obviously it's blinded. And I'm, I'm not going to comment any further on that at this point. Um, we do have an sap in place. Uh, I I do consider that we have the right to continue to modify until before we unblind the trial. But we do have our planned analyses established at this point.
Great. Thank you.
Thank you for your question.
Our next question comes from Sean lemon from Morgan Stanley. The floor is yours.
Hi. This is Mike. Riyadh on for Sean. Thank you for taking our questions and congratulations on the quarter. Um, so thinking about debut, the 987 new patient ads, um, suggest a good steady growth rate quarter over a quarter.
How should we be thinking about this trajectory like given the relative lower prevalence? Is it reasonable to expect that this would ever accelerate and doesn't need to, or how should we be just thinking about that trajectory, thanks so much.
Quarter of a quarter. And I think now we have this very stable and growing group of persistent patients, many of whom have now been on treatment for 12 months or longer, I think gives us a real sense that, um, we can really continue to grow this brand and take it to new heights.
Thanks so much.
Thank you for your question.
Our next question comes from Jason Butler from citizens JMP, the floor is yours.
Hi, thanks for taking the questions. Uh, just, uh, 1 on the Plaza. Uh, given that you're seeing a return on investment on on, for example, BTC activities. And now that you have the greater visibility with intellectual property, are there more Investments or longer term investments that you're considering for the franchise. Thanks.
Hi Jason, thanks for the question. You know, I think as as we think about our strategy with new Cloud that I track,
I encourage us to sort of bikeit. Um, the commercial strategy that we're putting in place now with the longer term ability to now maximize the brand. So
Direct to consumer decisions that we're making are sort of relative to the 2038 shorter term. We, we, we're seeing, um, impact of the DTC. We would have continued to invest, regardless of the outcome of the of the, um, IP trial because it has a strong momentum for the next sort of 2 to 3 years potentially, depending on how long we continue to invest beyond that. Now, what we have been able
Able to do is think about the longer term strategy for New Plaza. Um, I'm looking forward to sharing a little bit more about that. As we talk as we head towards the back, end of the year, in terms of how we now think about the investment longer term. Um, but for right now, the DTC campaign is certainly paying dividends as you point out and we're excited about the continued momentum and indeed in terms of the, um, mortar Parkinson's campaign and raising awareness of hallucinations and delusions. We're delighted that Ryan Reynolds has agreed to continue supporting that campaign. Because we believe that's been 1 of the, the main drivers of increased awareness. And once a caregiver is aware, they're encouraged to go in and talk to their doctor. So it's been a very strong, uh, impact to caregivers. And we're excited to continue that.
Thanks for the question.
Thank you.
Thank you for the question.
Our next question comes from Brian Abrams. From RBC Capital markets. The floor is yours.
Oh, hey guys. Congrats on the quarter. Thanks for taking my questions. Um, just 2 for me, I guess. Just, um, both on on debut, um, as you've expanded the sales force. Can you talk a little bit more, I guess, qualitatively around your learnings, uh, from, uh, the, the, the patients outside of the centers of excellence, just in terms of position receptivity. Um, number of prescriptions per physician any early persistent signals and, and how well educated the docs are around the titration. And then can you also maybe talk about any
Hints of changes you may be seeing uh in overall debut. Persistence both Court over quarter and bigger picture Trends and just how much the education around AE management and the efficacy message and importance of staying on therapy. Uh is is resonating thanks.
For all that Tom kind of with his thoughts on that. Yeah, thanks. Thanks for the question. So yeah, we we've already had a number of, learnings as we've expanded Beyond, kind of the, the coe's, um, 1 of the key things is there, there is clearly receptivity to debut outside of the coe's. You know, we know that there are Physicians who are treating rep patients, um, who won't necessarily have been called on yet, and we're appreciating that,
They require kind of ongoing education, to make sure that they fully understand the profile of the product. They fully understand how to utilize the product, and we're utilizing the
The full mechanics of the system that we built to make sure that we can educate all of those, those, uh, prescribers as quickly as possible. So, you know, I think, 1 thing that you probably need to be thinking about, as you look at modeling is that the, you know, the the buying process, the number of calls may be slightly longer for this group of prescribers versus those that we see for Coes. Um, and that's purely because they just don't see rep patients as frequently. Um, as you talk about persistency, I mean, we're really pleased with the persistency that we're seeing. So as I mentioned on the call 12 months persistency remain as well above 50%. And as we also mentioned for the first time,
as I mentioned, as we get new patients started,
Tom. Thanks for the question.
Brian.
Thanks a lot.
Thank you for your question.
Our next question comes from Ami fadia from Needham & Company. The floor is yours.
Good evening. Thanks for taking my question. Um, I have 2 quick ones. Uh, firstly, just with regards to new closet,
Uh it continues to remain really strong uh with the uh number of ships bottles that you uh mentioned, uh, can you give us some sense of what's driving the strength and maybe give us some color around where the growth is coming from across channels?
And then with regards to debut, as you, uh, see increased adoption outside the Coe setting.
Can you give us a sense of what you saw in your open level study in terms of persistency out?
You know at you know, 12 or 18 months in um you know with regards to patients that are being treated outside. The co setting
If you have that, thank you.
Oh me, thanks. I'm going to let Tom answer, both of those and uh Les maybe on in terms of the longer term in the trial, but
Tom. So, in terms of new positive, yeah, I mean, we're seeing a nice uptick in terms of uh, referrals mbrx is an ongoing TRX, um, volume. Um, I think as you look at the second quarter and kind of the impact that we saw,
um, we were pleased with the fact that
We were pleased with the fact that um, our mbx volume was kind of reflected across all channels. So we saw it both within the community setting, but we also saw nice increases across the LTC setting as well as a reminder you know, the vast majority of our patients do sit within the community setting about the fact that we have a group of patients that sit within long-term care where we also promote showed kind of just continued strength across the breadth of patients that we serve for new plazas. So very pleased with that kind of ongoing, uh, uptick and the strengths that we have now going into the second half of the year.
In terms of the question around debut and the real world data, um, I think, I think the real world data that we have, and we had a recent publication, I think at irsf that showed that 18 months we had about a 40% persistency rate.
So I think the real world data that we're now seeing, um, through the, the latest data. With a 45% persistence rate through 18 months. I think really kind of begins to match up with that. Very, very nicely. And I think again, gives us a real sense that this drug is, is performing as we've seen in clinical studies and moving forwards. We don't expect to see any kind of significant deviation versus this, very kind of stable kind of plateau that we're now seeing
And I'll just add on, thanks Stella. I'll just add on that, you know, in our actual clinical trial experience. Um, we we really only have about 9 months of data that you can kind of count on from a persistency perspective. Because in the later, part of the open label extension, we had um, patients going off to marketed drug and so coming off of the program if you look to that 9-month experience at that point you've got something like 45% persistency. So I think what we continue to see and what we've seen sort of all along is that actually the real world persistence is a little bit better than we're seeing in the clinical trials.
Thank you for your question.
Our next question comes from Mark.
Goodman from lorinc. The floor is yours.
Can you talk about discontinued for for debut and what you saw in the quarter and was there any um, inventory changes that were of any significance for either product? Thank you.
Just as a reminder, that concept really doesn't exist for debut as our single Specialty, Pharmacy. Really only takes, uh, control of the inventory for like a nanosecond before it goes directly to patients. So, the Devi model has always been really a cellphone
Mark, can you also just Mark, Mark Mark, can you also comment on just the tax rate and how to think about it this year and going forward?
Yes, so, our tax. Um, so right, I guess if you look at our p&l, our book tax rate year-over-year is a little higher, um, because for gaap accounting, we're not able to account for all the credits and nols that were actually using for modeling purposes. You know our cash tax rate remains currently in kind of the mid-teen uh rate you know long term we guide more towards mid 20s. Um and that's before considering anything uh for ob3 which will start to be uh implemented it from from our standpoint in the next quarter. And with that um we have about 400 million dollars of kind of us-based activity. R&D expense that have been capitalized and that in the near term uh we'll be able to have some accelerating expensing for us tax purposes. So in the next year or 2, our tax rate will actually go down
Ob3 is our internal vernacular for 1. Big useful bill just in case.
Thanks Mark. Um, next question.
Thank you for your question.
Our next question comes from Ash Verma from UBS. The floor is yours.
Yeah. Hi thanks. Uh, for taking our questions so, uh, for New Plaza. I know, like you've outlined that this is a largely Medicare patient population, uh, roughly by. When do you think that it will be eligible for Ira price, negotiation and implementation? Uh, and then on, secondly, on the, uh, ECP 101. I, I wanted to ask like, is this the same formulation from fairing that required refrigeration? And do you think that could potentially become a hindrance at all? Uh, in terms of price daily Administration for this patient population? And then, is there a plan for a room? Temperature, stable variant here.
Thanks Ash. I'm going to let um Mark answer the IRA question and none of those on 101 and some of that was a little bit unclear. You broke up for a little bit so you might have to just say your 101 question again for Lisa she can she can make sure she answered correctly but let's start with the IRA. Yeah in terms of potential timing for negotiation under the IRA 2029 would be the first year that new plazas. It is eligible for negotiation unless there's changes
Is in the legislation, like, uh, um, getting rid of the pill penalty. That's probably the year we anticipate, we'd potentially be subject to negotiation, just to remind you as a small company. We'd have a limit on the discount. We'd have to offer, it's in the range of 25 to 34% is outlined in the legislation. And then after that would be subject to negotiation like any other drug. Um, you know, from our standpoint just due to the launch timing of New Plaza. Um, if the pill penalty is removed, that could add another year before negotiation, I would likely be expected.
Small company is the 2-year, right?
2 years. Yes, yes, yes, great. And with respect to ACP 101 and the question there in that obviously um, tell me if there's anything that I didn't catch about your question that I, uh, neglect here. But we are using the same formulation as levo used. And an important thing to remember is that part of what we're doing here is a resubmission to the complete response letter. And so the intent is to provide the new information that FDA requires which is an additional study to demonstrate efficacy while changing a few things as possible about the overall, um, initial presentation. Um, you know, we have seen that to be acceptable and usable in our clinical trials, have not found it to be a concern in terms of your question about next Generation, you know, we're always considering whether there are things that we can do to our product to make them more patient friendly.
Um, and so I anticipate that we'll be thinking about that for Q1 as well as we do for other things.
Thank you for your question.
Our next question comes from David, Huang from December. The floor is yours.
I guess the open-label extension for the Phase 3 study, um, like what you're seeing in terms of rollover rate, and would you ever consider adding, uh, a randomized withdrawal portion to, um, you know, to the plan that I think was used by a competitor with improved products in the market to, uh, you know, to get that product approved. Thank you.
Great questions. Um, so first off on the Ole, I I'm not going to comment to specifically at this point, but I will say that we have seen generally good interest in our open label extension and our continuing to collect, um, information in on patients. Who are enrolling into that.
To the question about the randomized withdrawal. It's a, it's an interesting question, you know, at this point, we really are focused on the results of our current, um, parallel arm study that we're looking at those data coming in in early Q4, and we think a real advantage of that is that that's going to be able to give a clear, um, if it's positive, and it turns out the way we hope, it's going to give a clear demonstration for Regulators for Physicians and for patients and families. And what you could expect, upon initiation of therapy, um, randomized, withdrawal study, could be something that we might consider in the future. But at this point, we think the most important data set really is this uh, Parallel Group study that we're currently running and eagerly anticipating the data.
Thanks David.
Thank you for your question.
Our next question comes from oi ear.
From mizo.
The floor is yours.
Hi, thanks for taking our question. This is Leo on for oi and congrats on the quarter, uh, for each of your Brands, new plasma debut. What is the right way for us to be thinking about 2026 from a growth perspective? What are the key factors in drivers? We should be thinking about maybe, you know, on the heels of the recent R&D day, you know, excitements clearly growing in the pipeline, um, which pipeline programs is a team most excited about
Thanks.
And then Liz to tell us, which is her favorite child and have Pipelines.
Yeah, thanks for the question. So um let me start with debut. So as as we've mentioned during the call, you know we've seen very nice continued growth for the last 3 quarters in terms of activations, we do anticipate through the second half of the year that the the rate that we are growing. That number will accelerate as we see the impact of our new Field Force model, really begin to pull through as a reminder, you know, our our penetration rate. Um, in general across the entire Rec Community remains, kind of in the, the low 30%. So we've still got a
Significant opportunity here for this brand, to continue to grow and that's our goal through 2026 and Beyond, is to really make sure that we engage with the patient community in the right way, we really meet patients where they are, which is what we are doing with our new customer model. And we can really make sure that we take debut to the uh, the height that we know. It can be. So that's our goal for 26. So it's it's really a story of continued continued and we plan for accelerating growth through the year as it relates to new plazas. Um, it's a similar story in a way, you know, I think we've seen this year and this quarter in particular some very nice numbers in terms of leading metrics, um, the team in the field continue to execute very well, our campaigns are working for us very well and they're giving us a nice Tailwind as we think about the second half of this year. And we believe that that sets this very nicely up for 2026. So I think the outlook for both brands
and the commercial point of view is 1 of strengths and we really look forward to really capitalizing on that as we head into 2026,
Katherine. T this
Favorite child.
Going to say is I would never say who my favorite child is. Um, you know, I think that across our pipeline, we have a nice mix of assets that are um relatively de-risked from a mechanistic perspective, things like ACP 204 where we are following in learning that we have from New Plaza as well as some areas of really novel biology like ACP 71. Um, I think that we are
Excited about. And the fact that we've got a number of different ways, we could potentially serve patients living with rare and neurological diseases. So there's a lot in our pipeline that we're very enthused about and I'm not going to take a favorite child today. Maybe I'll just come on top of that with, you know, I think what was exciting for us R&D day was to be able to share
All five of the new products could hit, um, blockbuster potential.
And we believe 3 of them have the ability to, um, achieve over 2 billion dollars, should they be successfully approved? So, we're moving into bigger markets with still high on that medical need.
And we're excited to continue to focus, our development on really differentiated assets. And ensuring that we're
Um, developing a pipeline of of valuable uh Innovation that patients from those underserved communities will really feel adds to their opportunity to see um more memorable moments with their families. So we're excited for that.
Awesome. Thanks.
Thank you for your question.
Our next question comes from tazeen. Hamed from Bank of America. The floor is yours.
Hi, good afternoon. Thanks for taking my question. Um, I wanted to ask about whether the 204 study in Lewy Body. Dementia has started, I think your guidance had been that it's supposed to start soon or this quarter and then I also wanted to get your thoughts about the ADP data that's expected in the middle of next year. If that's positive um wouldn't what's your view about uh the likelihood of the Louis body study working? You know, knowing that you had the company had looked at Lewy Body as part of a previous study with with Puma a few years ago. Thanks.
Okay. Um, the first easy 1, which is we do continue to anticipate that the Louis body study will get started this quarter. So, um, hasn't gotten first patient randomized yet, but we are confident, we'll get it in the quarter, uh, in terms of ADP and potential for read through, you know, I guess, what? I'll
Its numbers that we have from New Plaza, is pretty supportive of Lewy Body again. There's a relatively small number of patients in that study, but you know, roughly 20 patients in the active arm and 20 patients in the placebo arm. And there was a marked difference in relapse rate of patients on Placebo versus patients who continue to on drugs. So I think we have some good reason to believe just based on the existing New Plaza data set, you know, a positive adpd is as said, is certainly going to make me feel better, um, in particular because that that gives you clear evidence that this particular molecule is active, though. We, of course, expect it to be based on all the non-clinical and, um, Phase 1 work that we've done to date. So certainly, we are anticipating that ADP readout, um, with a great deal of anticipation and feel good about Lewy Body and its potential for success.
Thanks. Thanks Justine.
Thank you for your question.
Our next question comes from sumat Kami, from canaccord, the floor is yours.
Good afternoon. Nice to see the progress. This quarter and thanks for taking our questions. Could you give us some specifics on how ACP 2591 fits into your plans for red syndrome relative to your current efforts with debut?
Ah, thanks for the question. We haven't had a 2591 question for a while, so I'll let this answer that.
Yeah, as excuse me, as we think about ACP 25591. I mean I think what originally attracted us to the program was the fact that there are some similarities mechanistically speaking to debut. And so that the risks it with a potential for a differential penetration from a, a brain perspective. So there's a possibility that you got a, A different on the benefit risk profile. There obviously that's going to have to play its way out in red patients in order to know how that would um how we would use that in the context of Debut. What we're doing right now on this 1 is some additional work to verify how the information we need to specifically take forward in red and I look forward to providing some additional updates on that and appropriate time.
Thanks.
Thank you for your question.
Our next question comes from saline Richter, from Goldman Sachs. The floor is yours.
Good afternoon. Thanks for taking my question. Um, for um,
For PWS here, Beyond HQ CT, and, and CJIS and CGIC. Do you plan to evaluate functional endpoints, such as hyperglycemia control and weight loss, in the phase 3 trial? And, um, what would be a clinically meaningful bar for success in this study? Thank you.
Is similar to the magnitude that was demonstrated with the 3.2 milligram dose in the prior study. That would be um I think we feel confident that that's going to be a meaningful change. And we feel confident, not just from ourselves but from talking to Physicians as well as you know, patient advocacy organizations, we think that that would be a meaningful note. I mean, I will say that the I think your other question was about things like weights or
Uh, weights circumference. We aren't specifically looking at that. We do think that this is a complex interplay of the disease itself, as well as the mechanisms that families have put in place to manage their, uh, their children and their access to food. So that hasn't been a focus here. Um, we are looking at, you know, Adverse Events, um, and and you know typically at blood profiles and at least based on the data that we saw on the prior study, we have no reason to anticipate that routine, monitoring is going to be necessary, that's going to be subject to the data we see in this study.
Thank.
You for the question.
Our next question comes from yatin, SIA from Guggenheim, the floor is yours.
Hey guys, thank you for taking. My question, question is on uh, 204 ACP 204? Would you just comment on the pharmacology that you think is better addressed with this molecule? Which was not addressed with new Plaza, specifically, as it relates to this ADP population, just trying to get a sense. Um, in terms of how the setup is, or how different the setup is into this ADP readout versus the DRP study that you ran with new project. Thank you.
So I think, um, so thank you for the question as I think about the differences with 2 or 4, they kind of go into a couple of different categories. There's a molecule differences and then there's program design differences. And I do think both of those play into what we could be looking at at the ADP study. Um, so on a molecule perspective just hitting on this really briefly, you know a new Plaza does have that QT prolongation, it's not significant, it's not clinically impactful but in an elderly and frail patient population, you do need to think about it. But also it limited our ability to dose range and we do see some differences in the exposure response, that suggests that higher levels of exposure could get us to higher efficacy. We think that this is a reason to think that we've got increasing reason to believe was 204 in our ADP program and then on, on the design of the program itself, you know, I think 1 of the biggest things that we learned from our prior experience
Experience, was the importance of having our program specifically focused on the disease that we are studying? Um, I I don't want to go through the whole history of DRP, but obviously, that was 1 of the challenges that we had there. So the program that we've designed with ADP is very specifically. Identifying that patient population. It's specifically looking at a patient population. That's a little more severe in their psychosis which we also found to be more responsive and we're biomarker confirming that patient population. So if we take all of these things together, we think that we have set up, um, we think we have a good setup for ACP 204 to show what it can do. And so good reasons to think that it might be more likely to be successful.
Thank you for the question.
Our next question comes from Evans, sermon for BMO, Capital floor is yours.
Hi, I'm Mark. Mothman on for Evan, thanks for taking our question. Uh back to debut. I noticed some improvement in the percent of active patients, who have been on therapy for 12 months or longer from 3 to 5 percent to 70% this quarter? Can you just expand on this a little bit further, is this more a factor again or just providers, handling treatment becoming more comfortable with the GI profile and how to manage it over time?
I would appreciate any caller there. Thanks.
Yeah, I'll say that question. So thank you. Um, I I think it's a few different factors. I mean, as I, as I mentioned, a few minutes ago, you know, we are learning more about the product. As we go. We are clearly educating the both the patient community and the caregivers and the hcps regarding, you know, how they should go about utilizing the product.
Knowledge. I think we continue to educate in the right way. We we make sure that all of the learnings that we have from our Coes are now being Amplified into the community as well. And I think that this is all being reinforced with our kind of uh updated customer model and strategy as we think about daily view, moving forwards.
And I think just finally the, you know, the persistency that we continue to see sort of amplifies that. So the 65 to 70 is really a recognition of everything that Tom has just talked to, um, just a much more stable base than we were a quarter ago and then we were a year ago. So again, um,
Lots of steadiness, and now we're driving the momentum into debut.
Thank you for the question.
Appreciate it. Thanks guys.
Thank you for the question.
Our final question, comes from Paul Matthias from stifle, the floor is yours.
Hey there, this is Julian on for Paul, thanks so much for, for taking our question and congrats on the progress. Um, just wanted to Circle back to something that was, you know, mentioned earlier in the Q&A about the sap for sap, a for ACP 101. And the, how you guys, quote, unquote retain the right to modify the sap while you remain blinded. Just curious. You know what, what types of modifications could, you know, potentially qualify or it could be sort of, you know, in the realm of of possibilities, just curious if you could expand on that.
Um, and I have 1 quick follow-up as well on debut.
Yeah, no, no planned modified.
I purely meant that as a just a from a practical point of view until you have unblinded your database. You can consider your sap subject to the possibility to change. But no there are no plans on vacations.
Got it, that's helpful. Thank you for clarifying. And then uh, on debut it just sounds like, you know, things are going well. You're starting to see, you know increased, um,
Uh, scripts in the community, in addition to, um, you know, higher persistence or I guess, you know, greater line of sight to the persistence of your patient population. Um, I guess you're just thinking about the second half of the year. You've sort of messaged how, you know, you expect to see greater growth. Um, you know, why the decision to not narrow guidance, you know, this quarter? It just seems like, um, you'd kind of easily hit if you continue to, you know, add the patients that you have this year. Um, just curious if that's being conservative, you know, out of, you know, sake for being conservative or, um, if there's anything else to that. Thank you.
Yeah, I wouldn't read anything in the guidance. I think the, what I would say maybe on the the reason why we adjusted New Plaza is, we started the year, you know, with a large, a wider range than we normally do kind of coming into the year. Uh, not knowing all the puts and takes for the Medicare Part B redesign. So, when we looked at the new Plaza range, we just thought it was just too wide. Kind of halfway through the year as we look at all the other ranges we left them, including debut, and we'll revisit those um in the third quarter as we would typically it would be a typical point to uh to start narrowing.
Thanks Scott.
Thank you, operator. So we really appreciate everybody joining us today.
And we look forward to updating you on our progress next quarter.
Thank you for your participation. In today's conference, this does conclude the program. You may now disconnect