Q3 2024 ACADIA Pharmaceuticals Inc Earnings Call
Ladies and gentlemen, thank you for standing by. Welcome to the third quarter 2021 Acadia Pharmaceuticals earnings conference call. At this time, all participants are in a listen only mode.
After the speaker's presentation, there will be a question and answer session.
to ask a question during the session. You would need to press star at one one when you're telephone. You would then hear an automated message of advising your hand to raise.
2 withdrawal your question, please press star 1 1 again.
Speaker Change: Please be advised that today's conference is being recorded. I would like now to turn the conference over to Al Kildani, Senior Vice President, Investor Relations, and Corporate Communications. Please go ahead.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Al Kildani: Thank you. Good afternoon and thank you for joining us on today's call to discuss Acadia's third quarter 2024 earnings results.
Al Kildani: Joining me on the call today from Acadia are Catherine Owen-Adams, our Chief Executive Officer, who will provide some opening remarks, followed by Brendan Teehan, our Chief Operating Officer and Head of Commercial, who will discuss our Strong Commercial Franchise's debut in New Plaza.
Also joining us today is Liz Thompson, PhD, Executive Vice President, Head of Research and Development, who will provide an update on our pipeline programs. And Mark Schneyer, our Chief Financial Officer, will review the financial results. Catherine will then provide some closing thoughts before we open up the call for your questions.
Speaker Change: We are using supplemental slides, which are available on our website's events and presentation section.
Before proceeding, I would like to remind you that during our call today, we will make several forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.
Speaker Change: These forward-looking statements, including goals, expectations, plans, prospects, growth potential, timing of events, future results, and financial guidance,
Speaker Change: are based on current information assumptions and expectations that are inherently subject to change and involve several risks and uncertainties that may cause results to differ materially.
Speaker Change: These factors and other risks associated with our business can be found in our filings made with the SEC.
Speaker Change: You are cautioned 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, beginning on slide four.
Speaker Change: Thank you, Al. Good afternoon, everyone, and thank you for joining us. By way of introduction, I'm Catherine Owen-Adams, and it's an honor to be speaking with you as the new CEO of Acadia.
Speaker Change: I want to take a few moments to share a little about my background and how it aligns with Acadia's goals and explain why I felt so drawn to join Acadia.
Speaker Change: To begin, I have over 30 years of experience in the pharmaceutical industry, most recently serving as Senior Vice President and General Manager of the U.S. Business at Bristol-Myers Squibb. I'm proud of that, leading their international major markets business.
Speaker Change: Before that, I spent 25 years at Johnson & Johnson working in both their pharma and med tech businesses in the EU and US.
Speaker Change: Throughout my career, I've led teams that have successfully launched and managed drugs across many therapeutic areas, including rare disease and neurology.
Speaker Change: In the last five years, I've had the privilege of being involved in nine drug launches in the U.S. and internationally, including a number of successful Rye Disease launches.
Speaker Change: I joined Acadia because I was excited by the foundational business.
Speaker Change: and the Future Potential of the Pipeline.
Speaker Change: I'm both inspired and humbled to have the opportunity to lead Acadia in advancing therapies that can make a real difference in people's lives.
Speaker Change: As I look toward that future, I see incredible opportunities ahead to grow our current portfolio.
Speaker Change: and advanced innovations in our pipeline including two late-stage assets serving new communities of patients with high unmet medical needs.
Speaker Change: I see significant potential to enhance shareholder values as we continue to execute and evolve our commercial priorities and advance our pipeline assets.
Speaker Change: Please turn to slide five, where I will elaborate a little on each of these areas and underline why I chose to join Acadia against our three core pillars.
Speaker Change: First, on the commercial front, we have two growing franchises, which together, based on our Q3 results, are now tracking to over $1 billion in annualised sales, an extraordinary milestone for a biotech company of our size.
Speaker Change: Second, our robust pipeline includes two late-stage assets in Prader-Willi Syndrome and Alzheimer's disease psychosis, complemented by many promising early-stage programs.
Speaker Change: And third, our financial strength is fuelled by our positive cash flows and a growing cash balance, which now stands at $565 million.
Speaker Change: I'll now briefly highlight our commercial and R&D pillars before handing off to our leaders who will dive deeper into the details.
Speaker Change: Turning now to slide six.
Speaker Change: Beginning with debut, where we've had another solid quarter, generating sales of $91.2 million, up 36% year-over-year and 8% sequentially.
Speaker Change: I'd like to take this opportunity to share some high-level observations on Debut based on my experience with multiple rare disease launches and to give some context for our launch results to date.
Speaker Change: Six quarters in, with $429 million generated in sales, I think we can say this has been a successful launch.
Speaker Change: Every rare disease launch is unique, but I have observed some key commonalities with day view.
Speaker Change: Especially important is the focus on the patient's journey, both to start and stay on therapy, and concentrating on how this journey is fully supported.
Speaker Change: We've reached a steady state of new patient starts with debut and have strengthened our focus on growing this in the next several quarters.
Speaker Change: This will take time.
Speaker Change: and growth will be driven by continuing to lean into the breadth of our clinical data.
Speaker Change: illustrating and describing to HCPs the impact Debu has on patients.
Speaker Change: bringing to life the scoring systems used in clinical trials and generating real-world experience and insights from our ongoing Lotus study.
Speaker Change: We will also continue to focus on the early part of the patient journey as families and doctors work together to find the dose for their child.
Speaker Change: I'm extremely confident about the potential for debut growth and further penetration beyond our COEs into the diagnosed patients who can benefit from its impact both in the U.S. and beyond.
Speaker Change: I'll now turn to New Plazid, which has had an outstanding quarter with net sales of 159.2 million, up 10% year-over-year.
Speaker Change: These results were achieved on the basis of underlying trends we described last quarter, including the impactful real-world evidence studies and last year's label clarification, combined with what we now see as a stable Parkinson's disease psychosis market.
Speaker Change: In August, in collaboration with Ryan Reynolds, we announced our unbranded More to Parkinson's campaign to raise awareness of Parkinson's-related hallucinations and delusions.
Speaker Change: We are excited about the early impact of this campaign that we're seeing with patients and caregivers and HGPs.
Speaker Change: Separately, we launched a new Plazid direct-to-consumer campaign, which we believe will help us accelerate growth for New Plazid in 2025. Brendan will discuss these campaigns in more detail.
Speaker Change: Turning now to slide 7 for discussion of our pipeline.
Speaker Change: In addition to our successful commercial brands, Acadia's pipeline is an important reason why I was so excited to join the company.
Speaker Change: as I believe the pipeline is truly underappreciated.
Speaker Change: We have a Phase III asset in ACP101 that's being evaluated as a treatment for Prader-Willi syndrome, a rare and debilitating genetic disease with no FDA-approved treatment.
Speaker Change: Behind that in our pipeline is ACP204, our second generation 5HT2A blocker, where we are currently conducting the Phase 2 portion of the program.
Speaker Change: This exciting asset has the potential to extend and expand on your psychiatry franchise.
Speaker Change: Both of these drugs will allow us to offer new options for patients in underserved communities with patient numbers larger than our current portfolio.
Speaker Change: We also have a number of exciting earlier stage assets we look forward to discussing more in the future as they advance in their development.
Speaker Change: We're also actively pursuing opportunities to expand this pipeline further through business development.
Speaker Change: which remains a strategic imperative of the company and an important focus for me as we look to set our growth trajectory for the future.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Speaker Change: I'll now turn it over to Brendan to discuss our commercial performance with these brands beginning on slide 8.
Brendan Teehan: Thank you, Catherine. Let's take a look at both our debut and New Plaza franchises, starting first with debut on slide nine.
Brendan Teehan: In Q3, we continue to drive growth in our debut business with $91.2 million in net sales.
Brendan Teehan: That growth was primarily driven by continued penetration of the prevalent population and increased demand as measured by dispense rates among our continuing patients.
Speaker Change: During the quarter, 923 patients received paid shipments, which compares to 917 receiving shipments in Q2 and 871 in Q1.
Speaker Change: Importantly, the average number of bottles each patient received increased as our patient cohorts mature and begin reaching a dose that they will likely remain on longer term.
Speaker Change: Weekly patient discontinuations in the quarter were similar to Q2, and continue to be down significantly from Q1.
Speaker Change: Importantly, we now have over 60% of all active debut patients on treatment for 10 months or more since treatment initiation.
Speaker Change: This is significant as the persistency curve for patients out past 10 months on treatment is flattening, suggesting a strong, enduring patient base benefiting from treatment over time.
Speaker Change: In fact, looking out 12 months and beyond, we see persistency rates at 50% or higher. This compares favorably to other chronic therapies at similar stages.
Speaker Change: Turning to Prescriber Dynamics.
Speaker Change: We have continued to expand the breadth of prescribers with now approximately 800 unique prescribers having written a prescription for debut.
Speaker Change: We have further increased our penetration of the 21 RET Centers of Excellence or COEs, and we see an increasingly large proportion of new starts coming from non-COE, high-volume institutions, as well as community-based practices.
Speaker Change: This is important as approximately 70% of all red patients are treated outside of COEs.
Speaker Change: We are focusing on driving depth of prescribing among those HCPs who have prescribed Day-Bu to one or two patients where we know they have additional patients under their care who can also benefit from Day-Bu.
Speaker Change: Let's discuss our ongoing efforts to drive future adoption. Please turn to slide 10.
Speaker Change: As Catherine mentioned, to expand treatment to more of the prevalent Rett patient population, we are focusing on further educating prescribers about Debut's efficacy and the real-world day-to-day benefits patients and their families are experiencing.
Speaker Change: This is especially important outside of COEs where HCPs do not have as deep an understanding of Rett Syndrome or DEBU's efficacy profile.
Speaker Change: These caregiver and HCP-reported real-world benefits include improved quality of life, improved mood, more purposeful use of hands,
Speaker Change: Increased alertness and better engagement in conversations and improvement in both verbal and nonverbal communication skills, which among other benefits are the driving force behind continued penetration of the RET population.
Speaker Change: As an example of these tangible daily benefits, I was excited to speak to one of our caregivers at a recent program about what her daughter is now capable of doing on her own now that she's been treated with DayView.
Speaker Change: She shared that since her eight-year-old daughter started treatment, she can now grab and hold on her own everyday items like utensils and other objects, including, much to her delight, ice cream cones.
Speaker Change: Other caregivers tell us about their loved one's improved ability to communicate non-verbally with their Tobii eye gaze device.
Speaker Change: Each of these benefits are helping create better connections between Rett patients and their caregivers.
Speaker Change: These are just a few very tangible and exciting examples of the real-world benefits we want all HCPs and caregivers to hear, understand, and pursue.
Speaker Change: We are sharing these stories within the REDD community where these types of improvements will surely resonate.
Speaker Change: One of our best sources of real-world efficacy is our ongoing LOTUS observational study that continues to yield valuable insights for both HCPs and families about the number and breadth of clinical benefits caregivers are reporting in their loved ones, as well as their GI management experiences.
Speaker Change: We are sharing this data with HCPs to allow them to make more informed prescribing decisions for their RET patients.
Speaker Change: In summary, our primary focus for growth is on demonstrating DEBU's efficacy and real-world benefits in our sales materials, peer-to-peer engagements, and our caregiver programming, sharing these benefits with all HCPs and families.
Speaker Change: and we plan to continue to drive growth while helping HCPs and families better manage tolerability challenges and improve the early treatment journey.
Speaker Change: With now over 30% of the 5,000 and growing diagnosed RET patients in the U.S. having started debut, we have a substantial opportunity ahead of us to continue growing the brand.
Speaker Change: Now let's turn to New Plaza on slide 11.
Speaker Change: Q3 was another outstanding quarter for the New Plazid franchise with $159.2 million in net sales, representing the highest ever quarterly sales for the brand with 10% growth year over year.
Speaker Change: We achieve this growth by increasing active patients on Neuplazid across all market segments.
Speaker Change: This strong performance in the quarter follows what we've seen throughout the year as we've grown demand quarter over quarter throughout 2024.
Speaker Change: Q3 volume growth was driven by two key initiatives that have been the focus of our discussions with HCP.
Speaker Change: and Stephen Davis. This is an uncorrected transcript. To see the full interview with Stephen Davis, Douglas Williamson, click on the link in the
Speaker Change: First, leveraging the published real-world evidence demonstrating the important differential outcomes Parkinson's disease psychosis patients have experienced with Nuplazid, including a lower overall risk of all-cause mortality versus all other off-label antipsychotics, most notably low-dose quetiapine.
Speaker Change: and educating the market about last year's label clarification, which helps HCPs understand that they can treat Parkinson's disease patients experiencing hallucinations and delusions with or without comorbid dementia.
Speaker Change: These two levers helped us increase HCP conviction in Neuplazid as their first line therapy of choice for PDP, leading to a higher demand and market share.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Speaker Change: Looking at the broader market, we are seeing a return of Parkinson's patients as measured by in-office visits as well as long-term care resident census numbers.
Speaker Change: This market dynamic supports continued future growth for New Plaza.
Speaker Change: Please turn to slide 12.
Speaker Change: As we announced in August, we launched an unbranded disease state education campaign featuring Ryan Reynolds and his mother, Tammy, focused on the non-motor symptoms of hallucinations and delusions that can often accompany Parkinson's disease.
Speaker Change: The early results of this disease state awareness campaign suggest it is among the most successful campaigns ever in this category. Here are just a couple of the impressive early measures demonstrating this.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Speaker Change: Over 3.9 billion media impressions in just the first 8 weeks or so of airing, and nearly 200 media placements including approximately 75% with headline mentions of the campaign.
Speaker Change: These are very encouraging early indicators.
Speaker Change: Concurrently, we also launched a branded campaign featuring Neuplazid as the first and only treatment for PDP.
Speaker Change: In just the first two weeks post-campaign, we saw a nearly three-fold increase in Parkinson's disease patients who visited NewPlaza.com and subsequently visited a PD specialist.
Speaker Change: As a reminder, we expect the vast majority of the benefit of these campaigns to be seen in 2025 as new patients schedule appointments with their specialists and seek treatment for hallucinations and delusions with Neuplazid in the months ahead.
Speaker Change: In summary, we're excited about the growth we're seeing in New Plazid and will look to capitalize on that momentum with our consumer campaigns in the weeks and months ahead. I'll now turn it over to Liz on slide 13.
Liz Thompson: Thanks, Fran. Please turn to slide 14.
Liz Thompson: I'd like to start today highlighting some good news on the global expansion front. We recently received approval from Health Canada for debut as the first and only approved therapy for patients in Canada living with Rett Syndrome.
Liz Thompson: Our next focus is in the EU, where we are targeting a submission of the marketing authorization application in the first quarter of next year. We're now building up our team and expertise in Europe in order to plan for that launch.
Speaker Change: In addition to European expansion, we've had productive conversations with PMDA, the regulatory agency in Japan, regarding the potential to bring Debut to patients there. We very much look forward to continuing to collaborate with PMDA and Japanese experts to progress our clinical program.
Speaker Change: Continuing on regarding support of our marketed medications, I'd also like to touch upon some of the analyses that we continue to generate and share about new plazas on slide 15.
Speaker Change: At the recent Movement Disorder Society meeting, we presented data on sedation and sleep. Both sleep quality and the avoidance of unwanted sedation are key areas of interest for physicians treating Parkinson's disease psychosis.
Speaker Change: In some of the studies of Pimivanserin, across healthy volunteers, patients with PDP, and patients with neuropsychiatric symptoms related to a neurodegenerative disease, we've included various exploratory measures of sleep.
Speaker Change: Taken together, the data across these trials suggested that pemivanserin may be associated with low levels of sedation and other sleep-related adverse events.
Speaker Change: Just last week, we also presented data at the PSYCH Congress meeting examining patients in our PDP studies who had complete resolution of their hallucinations and delusions.
Speaker Change: The literature suggests that symptoms of Parkinson's disease psychosis will tend to worsen over time if left untreated, but there's little information about the impact of treating earlier versus later.
Speaker Change: In an integrated analysis, including 135 patients with PDP, of whom 21 reported no symptoms after receiving Pimivantrin,
Speaker Change: Treatment initiated sooner after onset of hallucinations and delusions was associated with a higher probability of achieving a complete response than was later treatment.
Speaker Change: Turning to slide 16, I'll discuss our late-stage clinical programs, starting with the ACP 101 program in Prader-Willi syndrome.
Speaker Change: As a reminder, Prader-Willi is a rare genetic neurobehavioral disorder. Roughly 8 to 10,000 patients in the U.S. are living with Prader-Willi.
Speaker Change: As we've described before, the defining characteristic is hyperphagia, which is an unrelenting hunger. This manifests very early in life and can lead to obesity and myriad complications like type 2 diabetes or heart disease, as well as behavioral changes like anxiety and aggression.
Speaker Change: And unfortunately, life expectancy is currently only around 30 years old, largely due to cardiovascular disease.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Speaker Change: Our Phase III study, called COMPASS-PWS, is currently enrolling. This study is global, multicenter, randomized, double-blind, and placebo-controlled. We built on prior Phase III experience in terms of both dose and endpoint selection.
Speaker Change: We've been truly pleased with the enthusiasm we are seeing in the Prader-Willi community and we look forward to continuing to work with them and clinical experts as we advance through the study. I anticipate providing more specific guidance on timing early next year.
Speaker Change: Now, turning to our second late stage clinical program, ACP 204 on slide 17.
Speaker Change: Here we have utilized our extensive neuropsychiatry expertise.
Speaker Change: and Pathway Understanding to develop a next generation 5-HT2A compound designed to build upon the strong product profile of Niplazid.
Speaker Change: In particular, to date we've seen no sign of QT prolongation at the doses we are studying, a wide dose range, supporting the potential for a dose equivalent to approximately twice the approved Nuplazid 34 mg dose.
Speaker Change: and Steady State PK achieved in less than half the time of New Plaza, suggesting the potential for an earlier onset of activity.
Speaker Change: Currently, ACP 204 is in development as a potential treatment for Alzheimer's disease psychosis in a master protocol that includes a phase 2 and 2 phase 3.
Speaker Change: The phase two is over 300 patients. We continue to plan that the two phase three studies will be of roughly equivalent size
Speaker Change: Once the Phase 2 study data are collected, we'll analyze and report results, by which time the two Phase 3 studies will already be underway. I look forward to also providing more specific timing guidance on this program early next year.
Speaker Change: And now, I'll turn it over to Mark for a financial update beginning on slide 18.
Mark Schneyer: Thank you, Liz. Let's review our quarterly financial performance on slide 19.
Mark Schneyer: In the third quarter, we recorded $250.4 million in total net sales, up 18% from the third quarter of last year.
Speaker Change: Debut net product sales were $91.2 million in the third quarter.
Speaker Change: up from $66.9 million in the third quarter of last year.
Speaker Change: Sequentially, debut sales were up 8% from the second quarter, comprised of 4% volume growth and 4% net price growth.
Speaker Change: New Plaza Net Product Sales were $159.2 million in the third quarter, up 10% versus the prior year's third quarter, comprised of 7% volume growth and 3% net price growth.
Speaker Change: Rose Tenet for New Closet was 24.9% in Q3.
Speaker Change: R&D expenses decreased to $66.6 million in the third quarter of 2024 from $157 million in the third quarter of 2023.
Speaker Change: The decrease in research and development expense was related to a reduction in business development expenses as we made the $100 million upfront payment to NIRIN for ex-North American rights to Trinitide in Q3 of last year.
Speaker Change: SG&A expenses increased to $133.3 million in the third quarter of 2024 from $97.9 million in Q3 2023.
Speaker Change: The increase was mainly due to the ongoing New Plazid consumer activation campaign, as well as one-time costs related to our CEO transition.
Speaker Change: We ended the quarter with a cash balance of $565.3 million, which increased $64.4 million versus the prior quarter.
Speaker Change: I also have an update to share on our Rare Pediatric Disease Priority Review Voucher. Yesterday we entered into an agreement to sell the PRV for $150 million.
Speaker Change: We expect this transaction to close in the fourth quarter, and as a reminder, as part of our licensing agreement with Nuren for Trophinatide, we will owe Nuren one-third of the net proceeds received from this transaction.
Speaker Change: Please turn to slide 20 for discussion of our latest 2024 financial guidance.
Speaker Change: For Debut, based upon our third quarter results and the dynamics Brendan described, we are narrowing our guidance range for Debut and now expect net sales of $340 to $350 million.
Speaker Change: For New Closet, we are narrowing our guidance range to the high end of our previous range and now expect net sales of $600 to $610 million.
Speaker Change: As Brendan said earlier, this guidance does not rely on a meaningful impact from our recently launched DTC campaigns, the benefit of which will largely be achieved in 2025.
Speaker Change: We are also narrowing our full year new plaza gross net guidance and our new guidance range is 26 to 27 percent.
Speaker Change: On the expense side, based upon year-to-date results, we are reducing our R&D guidance to $280-$290 million and increasing SD&A guidance to $480-$495 million.
Speaker Change: Lastly, we are raising our cash guidance range to $600 to $640 million, reflecting our expectations for our operational performance.
Speaker Change: This range does not reflect the anticipated net proceeds from our sale of our PRV.
Speaker Change: And now I'll turn the call over to Catherine for closing remarks.
Speaker Change: Let's now please turn to slide 21.
Catherine Owen-Adams: As we head towards the end of the year, our business today is built on a strong foundation and I am excited for us to drive further growth in 2025 and beyond.
Catherine Owen-Adams: We will continue to execute on the significant opportunity that remains in front of us for both Debut and New Plaza to drive that growth.
Catherine Owen-Adams: We will also work diligently to enroll our two late-stage trials, as well as our pipeline programs and potential for business development deals.
Speaker Change: We're pleased to be generating sustainable and expanding cash flow from operations to fund future growth.
Speaker Change: As I stated up front, I truly believe that the deeper I dive into the company, the more
Speaker Change: The more enthusiastic I am about Acadia's incredible potential for future growth. With that, I'll turn it over to the operator for our Q&A. Operator?
Speaker Change: Thank you. Thank you. Thank you.
Speaker Change: Thank you. As a reminder to ask a question, please press star 11 on your telephone and wait for your name to be announced and to withdraw your question please press star 11 again.
Speaker Change: And our first question comes from Gregory Renza with RBC Capital Markets. Your line is now open.
Gregory Renza: Great. Good afternoon, Catherine and Katie.
Gregory Renza: Congrats on the progress. Catherine, welcome. Welcome aboard and thanks for taking my question.
Speaker Change: of joining Acadia. You mentioned business development and certainly the early stage pipeline I'm sure is of interest to us. I just wonder if you could just add a little more color about how you break down the strategic framework, what the capabilities are for Acadia to sort of welcome in some of those business development assets as well as really nominating and fleshing out the early stage pipeline that was of interest to you.
Speaker Change: Great. Thanks for the question, Greg.
Speaker Change: So yes, I'll ask Brent to talk about Ryan Reynolds at a more appropriate point, but I will focus on business development.
Speaker Change: So yes, in terms of the focus of growth for the company, I believe business development is going to play an important role in our future.
Speaker Change: As you know, it's played an important role up until now in our growth with the acquisition of Trofenatide. And so, as we think about the framework that you asked around, obviously we have a very strong footprint in neuropsychiatry and continue to develop drugs in that area, and I believe that will be part of our future and possible additional business development areas.
Speaker Change: Rare Disease, we now have a year and a half under our belt, strong successful launch with debut and Rare continues to be a strong focus for the company.
Speaker Change: Beyond that, I'm working with the team to discuss further areas of interest and we'll be sharing that probably at a slightly later date. But just to confirm that we are
Speaker Change: in a strong financial cash position as Mark has outlined and we feel very strongly that we are in a great position to look at business development deals as they come forward in the next few months.
Speaker Change: That's helpful. Thank you. And maybe a question for for Brendan, just on the the latter part of 2024 with respect to to debut. And, you know, as I think about coming into the holiday season, and we reflect on some of the December to January to February patient visits and.
Speaker Change: and debut utilization from patients and families. Now I should be thinking about some of the patterns when it comes to us entering Thanksgiving and the December holiday season and how it relates to transitioning to 2025. Thanks again, guys, and congrats.
Brendan Teehan: Sure, Greg, thanks. Thanks so much for the question. And just as a reminder, this will be just our second first quarter transition as we as we head into 2025 and a lot has changed.
Speaker Change: in the debut franchise since then.
Speaker Change: First of all, we have a much better understanding of the re-verification process and requirements for our patients, and we've built out those capabilities both within our hub, but also our Family Access Manager team that's working very closely with each of our families during that transition.
Speaker Change: I think a very important difference is our patient mix heading into 2025, which is quite different than the mix we had in the first quarter of 2024.
Speaker Change: as I mentioned in our prepared remarks.
Speaker Change: We have over 60% of our current debut patients currently on treatment for 10 months or longer.
Speaker Change: which obviously wasn't even possible in the first quarter of 2024. There were far less than 10% of patients at that time that had been on therapy that long. So I think that provides a much more dependable base of business for first quarter demand.
Speaker Change: In terms of patient dynamics, I would think that, just as we've seen in Parkinson's disease, I don't think that patients tend to schedule as many appointments in January. So that wouldn't be a surprise to me. But otherwise, we fully expect to grow patients and sales in 2025 over the longer period.
Speaker Change: Fantastic. Thanks so much.
Speaker Change: And the next question comes from Jason Butler with Citizens. Your line is now open.
Jason Butler: Hi, thanks for taking the questions and congrats on the quarter. I guess just have a couple on.
Jason Butler: New Plaza.
Jason Butler: Can you give us a sense of the profile of patients that are initiating therapy now in the context of the branded and unbranded campaigns? I guess, and also in terms of label change, are you seeing more older patients initiate on therapy versus to what extent you've seen patients come on board earlier in the diagnosis or early in the psychosis symptoms?
Speaker Change: Thanks for the question Jason, I'm going to let Brian answer that for us.
Brian: Thanks, Jason, for the question.
Brian: I would say first we're in very early days post launch of both of those campaigns, but I can tell you from what we're seeing for patient dynamics thus far, the mix of prescribers still seems to reflect what we see for what from what we saw in the second and third quarter.
Speaker Change: So we're seeing the same kind of prescribers that are our targets.
Speaker Change: and a similar number of new positions that are new to the brand.
Speaker Change: In terms of the age of our patients, it's been largely what we would expect. I think the caregiver campaign has helped to help connect families.
Speaker Change: to identify perhaps more readily subtle changes that they're seeing in their loved ones. But I don't think we've seen a substantial change in the age of our patients so far.
Speaker Change: http://TheBusinessProfessor.com
Speaker Change: Okay, great. Thanks for taking the question.
Speaker Change: And our next question comes from Amy Faria with Needham and Company. Your line is open.
Speaker Change: Hi, this is Purna on for Ami. Thank you for taking our question. You've suggested that you'll be having 1 billion in sales in 2025. I'm just wondering, are you anticipating any decline in debut sales relative to 2024? What is like the mix of the two products? Thank you.
Speaker Change: Thanks. I'm going to let Mark answer that for us. Yes. So let me just clarify that. Thanks for the question. I think what we're saying is right now we have $250 million in sales in the quarter and that equates to a run rate of a billion dollars.
Speaker Change: So we're not at this point suggesting, guiding yet for the full year 2025, but we're pleased to kind of have a run rate milestone that's over a billion dollars, and then we'll guide into next year, but certainly at this time can share that we expect growth in both franchises and growth over that in 2025.
Speaker Change: Thank you.
Speaker Change: and Stephen Davis. Thank you. Thank you.
Speaker Change: And our next question comes from Mark Goodman with Lyrinc Partners. Your line is open.
Speaker Change: Hi, this is Basma on Primark. Thank you for taking our question.
Speaker Change: We have a question regarding the 50 or 60% of the patients remaining on therapy. Do we know the proportion of patients who respond to the treatment and actually demonstrate clinical benefits? Should we assume that all of them at this point are responders and basically benefit from the therapy?
Speaker Change: And on the flip side, I wonder if you could provide us with more color on the discontinuation data and share with us what is the key factor that really drives the discontinuation of therapy? Is it the A-profile or is it the lack of treatment effect? Thank you.
Speaker Change: Thank you, Bhatna. I think I'm going to ask Brent to take us through those two questions. Sure, no problem. And thank you for the question.
Speaker Change: First, just to confirm, yes, we have over 60% of patients at 10 months or longer, and what we see there is the flattening of the persistency curve out to 15 months for the latest cohort that we're looking at, which is very encouraging.
Speaker Change: What that would suggest to us is that patients have worked through the treatment journey and have found the dose that they're settling in on and are benefiting.
Speaker Change: Our Family Access Manager team and our clinical nurses at the HUB would confirm that these patients are benefiting and continuing. So I think that answers that question. The second question around discontinuations.
Speaker Change: We still see that the majority of discontinuations happen in the first one or two fills of treatment, and that is consistent in terms of the reasons for discontinuation.
Speaker Change: tends to be diarrhea or vomiting and not so much a lack of treatment effect. It's really the tolerability in the early fields.
Speaker Change: Thanks, friends.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Tess Romero with JP Morgan. Your line is open.
Speaker Change: Hi team, this is Caroline Pocher on for Tessa Romero with J.P. Morgan. Thanks for taking our questions.
Speaker Change: So first in your prepared remarks you had mentioned that the debut launch is now at steady state of new patient flow. We're just curious if you could just clarify this statement. Does this mean steady net ads?
Speaker Change: And then you mentioned it will be a focus to grow this over the next few quarters. Is there anything that has fundamentally changed in your strategy to accelerate new patient starts? And are you considering changing or pivoting any facets of the launch strategy going forward? Thank you.
Speaker Change: Thanks Carolyn. I'll take some of that in terms of the perspectives that I've developed with the team over the last six weeks. So just to go back to the original part of the question,
Speaker Change: In terms of the steady state, yes, we're seeing a steady state of patient ads after a very strong start to the debut launch.
Speaker Change: And that's not unusual in rare disease launches. As you know, you see a big burst of patients out the door with with the high unmet medical need and then tend to sort of go to a more plateau phase.
Speaker Change: But we are looking to see how we can recharge that growth and start to see more patients coming in the top of the funnel. And we believe that there is a strong possibility of driving that growth. And let me just tell you what the areas that we're focusing on in order to achieve that.
Speaker Change: The first is efficacy. What we've learned over the last six quarters is that the
Speaker Change: Scales that we use in our clinical trials, the RSPQ and the CGI, they need to be brought to life for physicians and they need to understand for their patients the impact that Debut can promise them.
Speaker Change: for their changes in cognition, their changes in communication, their changes in hand wringing. So bringing the efficacy to life is our first real focus, as well as starting to enhance the data that we're generating around the Lotus study and ensure we're amplifying this message.
Speaker Change: to physicians and helping them understand the longer-term impact of Debut on their patients.
Speaker Change: The second Brendan's just sort of mentioned, which is managing the patient journey with all rare diseases, we need to really start thinking about the patient journey that surrounds the patient when they start on therapy and as they continue to persist.
Speaker Change: treatment right out beyond 15 months now and ensuring that our families are supported throughout that whole journey.
Speaker Change: A couple of things. First of all, we've seen the diagnosis rate increase since the launch of Debut. We've seen that increase by around 10%.
Speaker Change: which obviously allows us to go after more patients and make them aware of the opportunities with Debut. And secondly, 70% of our patients are treated outside our COEs, so we see a strong opportunity for growth outside of our centres of excellence.
Speaker Change: So, if I compound those three factors, enhancing the efficacy.
Speaker Change: really focusing on the patient journey at the beginning.
Speaker Change: and then driving the growth beyond our COEs, I feel very strongly that we will see strong debut growth as we move into 2025.
Speaker Change: Great, thank you so much.
Speaker Change: And the next question comes from Joel Beatty with Baird. Your line is open.
Joel Beatty: Hi, thanks for taking the question. What do you make of the ratio of the patients on
Joel Beatty: therapy to the number of unique prescribers. And what I mean is that it looks like there's been about 1,500 patients who have started therapy at some point with about 800 unique prescribers. So that's a little bit less than two patients per prescriber. And perhaps I would have expected it to be a little bit more concentrated.
Speaker Change: Thanks, Joel. That's a great observation and one that I know we've been looking at. So Brendan, do you want to share a little bit more about that? For sure, Joel. Thanks. Thanks for the question. And I think it's, I think it's right. There's a concentration and a long tail.
Speaker Change: And I think for a first mover in a rare disease area, the centers of excellence still have the vast majority of prescribers that have multiple referrals that are written for debut. High volume institutions also have a concentration of physicians that have.
Joel Beatty: three or more referrals that have been written and we're continuing to penetrate those. In the community, as we've gone further and further into the community, it is not uncommon to see a physician that may have one patient or one or two patients that they see less frequently. And there you see kind of a long tail of not necessarily pediatric neurologists anymore. You're seeing primary care physicians and some pediatricians who likely will have one patient to offer.
Joel Beatty: We are, however, focused on those physicians that have written one or two prescriptions for debut, and we know they have additional patients for follow-up.
Speaker Change: Thank you.
Speaker Change: And the next question comes from Keith Tapper with BMO Capital Markets. Your line is open.
Keith Tapper: Thanks and good afternoon team and thanks for taking my question. Welcome and congratulations to Catherine.
Keith Tapper: So per debut, can you remind us what to expect from the Canadian approval, maybe in 2025 and 2026 in terms of launch expenses, material impact revenues, if all goes well, and then have you got it for timelapse revenues in Europe and Japan?
Keith Tapper: And then separately, I know it's early, but could you talk about 2591, which you have rights to in RET and Fragile X? Is it a similar strategy versus Bayview, the drug itself? I know your partner, Naren, showed interesting data recently in different indications. Anything you can share will be helpful there. Thank you.
Speaker Change: Thanks Keith. I'm going to ask Brent to elaborate a little bit more about our strategy in Canada, Europe and Japan and then Liz perhaps take the 2591 questions for Brent. For sure, thanks. We're obviously excited to have an XUS approval for Canada. Very much looking forward to serving that patient population.
Speaker Change: and the public payer process tends to take a bit longer.
Speaker Change: In the interim, we have a great opportunity to work.
Speaker Change: with the centers that have RET patients in Canada.
Speaker Change: and begin to generate their interest in signing patients up for when we do have reimbursed approval. So we'll be doing that in the interim. And otherwise, we are very much looking forward to making DayView accessible to patients as soon as we can while we're actively engaging those necessary stakeholders to get to coverage.
Speaker Change: and to comment a little bit on 2591. I think I'll, I'll start out by commenting generally, you know, we've, we think we've got a first great treatment for patients living with RETS with Dayview, but are obviously interested in continuing to find new ways to serve that community. So 2591 and other things that you may see come from us in the future, really show our commitment to this patient population.
Speaker Change: At this point, it's probably premature to talk too specifically about what we're thinking about from a clinical perspective. I'll just generally say that we are learning from NIRIN and their interesting data that they have compiled so far, as well as putting together our own set of information that we think is necessary to have a RET specifically.
Speaker Change: specific strategy with this asset. So more, more that I'll be able to talk about in future.
Speaker Change: And just to keep in terms of Europe and Japan, we are we are preparing our MAA as Liz has already referred to in the prepared remarks. We've started to build out a team in Europe to support that launch and we're looking forward to
Speaker Change: Not only making our debut product available for patients in Canada, but in Europe and beyond. And in terms of Japan, Liz alluded to, and it was on the slide, our discussions with the PDMA. So all of those discussions are currently active and ongoing.
Speaker Change: and Stephen Davis. Thank you.
Al Kildani: And our next question comes from Tazeen Ahmed with Bank of America Securities. Your line is open.
Tazeen Ahmed: Hi, good afternoon. Thanks for taking my questions. And Catherine, welcome for me as well. I wanted to get your thoughts about how to think about the European launch. In your previous roles, you did handle ex-U.S. launches. Are there certain rules of thumb to be aware of as it relates to rare disease launches in Europe that you can share with us today? And can you talk to us about your general plan forward in that region? Thanks.
Speaker Change: Sure. Thanks, Jazine.
Speaker Change: So, as I've just said, we are preparing for our MAA application early next year in Europe. As you know, there's a set time clock in Europe. It normally takes up to a year for that document to be assessed.
Speaker Change: Then we start the clock with each of the national HTAs on reimbursement and we would normally expect to launch in
Speaker Change: in Germany first, as well as Switzerland, and then other countries come online after that, depending on how long our local negotiations take with both the national authorities and then some of the regional authorities in places like France, Italy and Spain.
Speaker Change: In terms of managing a rare disease launch in Europe, from my experience, it's relatively similar to managing a rare disease launch in the U.S. except that
Speaker Change: involvement in the patient journey. The access to medicines once it's prescribed is a lot easier. We're not dealing with payers on a sort of a case-by-case basis and so while we need to wrap around support of the physician and patient it just looks slightly different in terms of that individual patient journey.
Speaker Change: But with my experience and the team already being built up in Europe, I feel very confident that we will have Debut available post-registrational approval and we're looking forward to making that launch successful as well.
Speaker Change: Our next question comes from Jeff Hung with Morgan Stanley. Your line is open.
Jeff Hung: Thanks for taking my question. For ACP 204, I was wondering if there's any updates on whether EU regulators have agreed to your master protocol and can you just remind us of the strategy there? Thanks.
Speaker Change: Yeah, I'll ask Liz to take that one for us.
Liz Thompson: Sure. So we haven't reengaged with them about the master protocol, the phase three portions in particular. We anticipate doing that when we're a little further into the phase two portion of the study. That said, you know, again, all this really means is that Europe may be a little bit slower up and running on the phase three portion of the on the phase three portions of the master protocol. But the advantage is that potentially we'd be in a position of starting up phase three there with data in hand. From from the phase two portion, which is always helpful in terms of getting investigator interest. So, you know, it's a little bit of a bump in the road, but we don't see this having an overall implication on strategy or timeline.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Ritu Bhara with TV Cohen. Your line is open.
Athena: Hey guys, this is Athena on for the two. Thanks for taking my question. I had another follow-up on
Liz Thompson: high value academic centers and community practices. How should we think about forward adoption rates here? And how comfortable are these positions when it comes to titrating Debut? And what learnings are you applying from the earlier stages of Debut's launch to your conversations with these folks? Thank you.
Speaker Change: Thanks, Athena. I'll let Bren elaborate on that. Yes, sure. Thank you for the question.
Liz Thompson: As I think I alluded to in my prepared remarks,
Jeff Hung: COEs were instrumental in the early part of the launch, and they still continue to contribute.
Jeff Hung: and we're driving further penetration there. I'm pleased with what we've seen for high volume institution penetration growth over the past year. I'm similarly pleased with our ability to get into the community to find those physicians that have RET patients and see them continue to start to prescribe Debut.
Jeff Hung: The further you get from a COE, the more you're doing education on the particulars of Rett syndrome because they have infrequent visits with these patients.
Jeff Hung: a clinician and several caregivers that have had longer-term successful experiences on Debut to tell them what to expect in real-world performance of the product and what they're seeing in their loved ones, and that's been a very successful combination for us.
Speaker Change: Thank you.
Speaker Change: And our next question comes from Paul Matisse with Stifel. Your line is open.
Speaker Change: Hi, this is Julian for Paul. Thanks so much for taking our question. Just really quick one. What are your expectations for ROI on the DTC campaign in 2025 for New Plaza? Obviously, you've, you know, demonstrated strong execution in 2024. Just curious if you have any more commentary you can provide for next year. Thank you.
Jeff Hung: Thanks. So, Brendan, do you want to talk about the direct consumer campaign? For sure, and thanks for the question, Julian. So, we initiated this campaign in the middle of August, and it is really just...
Jeff Hung: patient visits, starting to see their neurologist or movement disorder specialist. So I think the vast majority of the benefit we'll see will begin in 2025. But as a reminder, the lifetime value of our Neuplazid patients
Jeff Hung: both in the community and long-term care will extend beyond 2025. So we'll start to see
Jeff Hung: both an increase in new patient starts, but also their continuing value throughout 2025, 26 and 27, perhaps beyond.
Jeff Hung: and so I don't think we'll fully realize all of its its value in 25 but we're enthusiastic about what we're seeing about early visits and some of the some of the early returns.
Speaker Change: Thanks Bren.
Speaker Change: The next question comes from Charles Duncan with Cantor. Your line is open.
Charles Duncan: Good afternoon, Catherine and team. Thanks for taking our question and Catherine, congrats on the new opportunity with Acadia. I had a quick question regarding ACP 204. In terms of the data released
Jeff Hung: I guess I appreciate the master protocol in terms of facilitating enrollment, but I'm a little bit confused as to
Jeff Hung: whether or not you'll be releasing any information from Phase 2 in terms of efficacy, etc. as the enrollment of the Phase 3s are ongoing. So please provide a little color there. Thanks.
Jeff Hung: http://TheBusinessProfessor.com
Speaker Change: Thanks for the question, Charles. I'm going to let Liz enhance that. Yeah, our current expectation is that we would be releasing some data at an appropriate time. And the expectation was always that we could use the Phase 2 data to make modifications to Phase 3 as needed.
Jeff Hung: Thank you for watching.
Speaker Change: And our next question comes from Jay Olsen with Oppenheimer. Your line is open.
Jay Olsen: Well, hey, thank you for providing the update and thanks for taking our question.
Speaker Change: For Catherine, could you please talk about your due diligence and any key considerations that you were contemplating?
Speaker Change: as you considered taking on the role of CEO at Acadia.
Jeff Hung: Also, maybe elaborate on your longer-term vision for Acadia. Where would you like to see this company go in the future? Thank you.
Speaker Change: Thank you, Jay. So in terms of the approach I had to looking at an opportunity.
Speaker Change: I was really looking for the combination of three things. First was a strong commercial base where I could come in and add value based on my experiences of 30 years so far in the commercial side of the pharma business. And that's what I found with New Plazid and Debut, very exciting.
Jeff Hung: core brands that we can look to grow and develop over the coming years. The second important area I was looking at was a pipeline, and that I was really excited by, especially with our two later stage clinical trials in Prada-Willi.
Jeff Hung: and Alzheimer's disease psychosis.
Jeff Hung: and as I got into the due diligence was able to see a little bit more in the pipeline and I'm still very excited by that.
Jeff Hung: a really strong board and strong investors and so those three things together.
Jeff Hung: made Acadia for me a very attractive and exciting option. And I can tell you six weeks in, I'm even more motivated and excited than I was when I first walked in the door here in San Diego. I'm very excited about the future and really believe that this company has only to go from strength to strength.
Speaker Change: And our next question comes from Summit Kukarni with Canaccord. Your line is open.
Summit Kukarni: Good afternoon, thanks for taking our question, another one on debut. Other than patients being at centers of excellence versus not, what's your understanding of the key bottleneck that may be preventing new patients from starting debut and what's the main variable you need to focus on to see an inflection on debut sales from current levels?
Speaker Change: Thanks so much. Very sad question so I'm going to let Brent answer that. Thanks so much for the question.
Speaker Change: I think we're focused entirely, once you get beyond centers of excellence and some of the academic centers that are that are very much like COEs.
Speaker Change: on Education on the Efficacy in the Real World Setting.
Speaker Change: And so it really is a combination of focusing on physicians and explaining to them what the translation of CTII, RSVQ into daily improvements that families are seeing, which we're doing through a combination of peer-to-peer programming, as well as
Speaker Change: Having caregivers that have had their loved ones on therapy for two or three years, talk about what they've been able to see in their loved ones over time.
Speaker Change: And then similarly, leveraging those experiences to speak to caregivers that may be on the sidelines, need to know more about what they should expect in their loved one. And we have a number of programs that look at patients between the ages of two and five.
Speaker Change: 5 to 10, preteens, teenagers, and patients over the age of 20.
Speaker Change: so that we can really speak the language of the caregivers from similar experiences that some of the caregivers that have already started debut are well ahead on. And those are really our two primary areas of focus to elucidate efficacy and what I can see for clinical benefit for both of those audiences.
Speaker Change: Thank you, Brendan.
Speaker Change: Thank you.
Speaker Change: And the next question comes from Danielle Brill with Raymond James. Your line is open.
Speaker Change: Yeah, great. Thank you, Alex. I'll let Brendan do that one as well. For sure. Great, great question. It's important to realize that we stay in contact with every family.
Speaker Change: and as you know, and probably are pointing out here, there are some patients that will discontinue after just one or two fills, mostly due to tolerability challenges.
Speaker Change: with those families were obviously checking to see what their GI management experience was like and looking to further educate them on strategies both around GI tolerability and engaging their HCPs to make sure they have a strategy in place but also understanding more about their their dosing and treatment journey because there could be alternate approaches where they could work themselves to a dose that would be more effective.
Speaker Change: and so that will that will certainly be something we continue to focus on over time. Up to now I would say that the for restarts in terms of patients that have restarted is still significantly less than 10% of our of our overall patient base.
Speaker Change: Thanks, Brent.
Speaker Change: And at this time, I would like to turn the call back over to Katherine Owen-Adams for closing remarks.
Katherine Owen-Adams: Great, thank you, Operator. So thanks again, everyone, for joining us today. Really appreciate the welcome that you've given me into the Acadia community. We look forward to updating you further on our progress in the next quarter.
Speaker Change: Thank you. Thank you.