ACADIA Pharmaceuticals Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 2, 2023

There are 18 speakers on the call.

Operator

Good day, ladies and gentlemen, and welcome to Acadia Pharmaceuticals Third Quarter 2023 Financial Results Conference Call. My name is Kathy, and I will be your coordinator for today. At this time, all participants are in a listen only mode. We'll be facilitating a question and answer session toward the end of today's call. I would now like to turn the presentation over to Al Caddani, Senior Vice President of Investor Relations and Corporate Communications at Acadia.

Operator

Please proceed.

Speaker 1

Thank you, Kathy. Good afternoon, and thank you for joining us on today's call to discuss Acadia's Q3 2023 earnings. Joining me on the call today from Acadia are Steve Davis, Our President and Chief Executive Officer, who will provide some opening remarks followed by Brendan Kian, our Chief Operating and Head of Commercial, who will discuss the DAVEY launch and NUPLAZID execution Doug Williamson, our Head of Research and Development, will provide an update on our pipeline programs and Mark Schneier, our Chief Financial Officer, will review the financial results. Steve will then provide some closing thoughts before we open up the call for your questions. In addition, both Kathy Bishop, our Head of Rare Disease and External Innovation and Parag Maswani, Senior Vice President, trofinetide, Rare disease franchise will be available for the Q and A session.

Speaker 1

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 form act of 1995. These forward looking statements, including goals, expectations, plans, prospects, Growth potential, timing of events or future results 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 cautioned not to place undue reliance on these forward looking statements, which are made only as of today's date.

Speaker 1

I'll now turn the call over to Steve. Thank you, Al. Good afternoon, everyone, and thank you for joining us. Please turn to Slide 5. Acadia is entering a transformational period of growth delivering record revenue in the 3rd quarter.

Speaker 1

Our rapidly growing franchise in Debut complements are highly profitable NUPLAZID business and our robust R and D pipeline provides rich opportunities to fuel growth even higher levels in the years ahead. We plan to capitalize on this opportunity set by executing on our strategic priorities. Let me touch briefly on First, we are extraordinarily pleased with the success of DABUT. We generated $66,900,000 of net sales in the 3rd quarter, our first full quarter since launch, demonstrating a high level of excitement in the RED community as soon as DABU became available. As we'll discuss today, we're seeing strong indicators across all launch metrics underlying these results, including demand, persistency and access.

Speaker 1

2nd, our NUPLAZID franchise continues to be highly profitable and strongly cash flow positive. NUPLAZID revenues for the quarter were and $44,800,000 These results reflect our ability to continue to gain market share and grow the revenue base. 3rd, in addition to these successful marketed products, we have a deep and growing pipeline including our Phase 3 ADVANCE 2 study of pimavanserin in negative symptoms of schizophrenia, where we expect to have top line results in the Q1 of 2024. Our Phase 3 study evaluating ACP-one hundred and one in Prader Willi syndrome, where we expect to commence enrollment later this month. In leveraging our learnings from NUPLAZID, we have developed ACP-two zero four, our next generation 5 HT2A blocker.

Speaker 1

Here too, later this month, we will commence enrollment of patients in our seamless Phase II, Phase III program, studying ACP-two zero four in Alzheimer's disease psychosis. 4th, the success of Dabu's U. S. Launch underscores the opportunity we have to expand in the global markets following our expanded license agreement for DEBUT announced in July, which granted us worldwide rights to the asset. Our early U.

Speaker 1

S. Experience serves as an important reminder of the significant unmet medical need worldwide with no approved treatments for Rett syndrome outside the United States. We're meeting with Health Canada this month to discuss a planned new drug submission for trofinetide in Canada. In addition, we're engaging with European regulators in advancing plans in other geographies. 5th, we have a deep early stage portfolio that includes disclosed and undisclosed programs Focused on neuropsychiatric and rare disorders, they represent significant opportunities to continue to build on our current growth.

Speaker 1

In addition, we continue to remain very active in business development to further expand our portfolio and build on our success with DEBUT and UPLAZID in CNS and rare disease. Let's discuss our debut launch and some of the launch dynamics on Slide 6. 5.5 months into the launch, We've provided hope to a community that has never had an approved treatment for Rett syndrome. We've seen Rett patients We've established critical relationships in the medical and caregiver communities and we provided a strong base for future continued success. During this time from mid April through September, We booked approximately $90,000,000 in revenue.

Speaker 1

In preparing for the approval and launch, we made significant investments in both the medical and caregiver communities, In medical education, in launch preparations and the result, everything about the launch thus far has gone either according to plan or exceeded our plans and in some measures greatly exceeded our pre launch expectations. Prior to launch, we expected demand to be high. It has been. In fact, one area where we significantly Our pre launch plans is just how fast the demand came immediately upon launch, particularly from centers of excellence. Another area where we have exceeded pre launch expectations is the speed at which we've established access with payers.

Speaker 1

As Brendan will speak to you shortly, this access has come faster both in terms of the pace at which payers have adopted formal coverage plans and the pace at which they have approved treatment under letters of medical necessity prior to the adoption of written plans. These two dynamics pent up demand from centers of excellence and more rapid access than anticipated, coupled with high levels The patient persistence on therapy have resulted in just over 800 patients on DABU as of September 30. Importantly, these dynamics have produced a substantial foundation that will continue to benefit our launch as we move forward. We've established significant breadth and depth of physician experience with DABUT in a short amount of time. A high number of patients and families are sharing their positive experiences at this early stage, enabling us to gain momentum sooner.

Speaker 1

And establishing access well ahead of plan enables us to further build on that momentum. Turning to persistence, we previously reported that we're very encouraged by what we're seeing And that continues to be the case. As Brendan will discuss shortly, our real world evidence to date indicates 81% of patients starting DEBUT remain on therapy 4 months after treatment initiation and only an additional 6% are beyond 60 days since their last scheduled refill. In comparison, in our LIHALAC-one open label extension study, 65% of patients who started debut treatment after rolling over from placebo remained on therapy 4 months after initiation. To sum up, we continue to operate at or ahead of plan.

Speaker 1

Prior to launch, we projected a linear growth curve producing attractive revenue growth year after year. This is what we typically see in rare disease launches. Of course, what we didn't anticipate with the debut launch is the surge in demand that accelerated our penetration and the associated contributions to revenues in these early quarters. As we move forward, we'll continue to leverage this strong foundation as we increase breadth and depth in all sectors of the Rett community. Let's turn to a snapshot of our current products and pipeline on Slide 7.

Speaker 1

We see as an overview of our 2 successful commercial products and our pipeline of late and early stage programs that represent substantial long term growth opportunities for Acadia. As we've noted, New Plazid continues to be a highly successful franchise generating significant cash flow. In Parkinson's disease Our commercial team is continuing to successfully leverage the real world evidence studies rolled out earlier this year and the impact is becoming increasingly evident each month and quarter. Building on this, Daybut provides dramatic growth potential to our business And we have a robust pipeline behind that, we'll be commencing late stage studies in 2 programs later this year. In our negative symptom schizophrenia program, There's something rarely seen in this indication that is a positive pivotal study.

Speaker 1

There are no FDA approved treatments to treat the negative symptoms of schizophrenia. The unmet need is high and we look forward to having results from our 2nd pivotal study in the Q1 of next year. Later this month, we will commence our Phase 3 study with ACP-one hundred and one in Prader Willi syndrome. Prader Willi is a rare and debilitating genetic disease for patients who have an unrelenting drive to eat. Here too, there are no FDA approved treatments.

Speaker 1

November will be a busy month for us as we also commence our CMOS Phase II, Phase III program with ACP-two zero four in Alzheimer's disease psychosis patients. And behind that, as I've mentioned, we have a rich pipeline of early stage disclosed and undisclosed programs that position us for long term growth. I'll now turn it over to Brendan to provide additional insights on our debut launch execution and NUPLAZID's commercial performance on Slide 8.

Speaker 2

Thank you, Steve. Let me provide additional commentary on our 2 commercial franchises, Debut and New Plaza and the terrific performance Both delivered in the quarter. Let's begin with Debut on Slide 9. We have 3 commercial execution priorities I'd like to discuss today: Demand, persistency and conversion to paid treatment. DABU's launch has outperformed across each parameter since approval.

Speaker 2

As Steve noted, we have seen a surge in demand for DABU since the launch in mid April, resulting in just over 800 patients on DABU as of September 30. This initial wave of demand for us was a bit unique, likely due to several factors including a very strong patient advocacy community. 800 patients on therapy is a great start, but we still have a lot of RET patients who can benefit from DABU, many of whom have fewer interactions with centers of excellence and with patient advocacy organizations. This segment represents the majority of patients who have not yet been prescribed DayBu. As we previously described, Approximately 25% of RET patients are treated in centers of excellence, another 60% are treated in high volume institutions and the remainder in community practices.

Speaker 2

As we look to engage this next significantly larger segment of the Rett market, we expect a more linear adoption curve as we've seen with Other orphan rare disease genetic rare disease launches. New patient requests are coming in at the rate we expected at this point in the launch and will support the continued attractive growth of DABUT. Our experience so far with new starts, coverage, Efficacy and persistency reinforce our conviction that Daybut will achieve a high degree of penetration in the rent market. To penetrate this larger segment of the market, we are now employing a number of initiatives, including delivering HCP and caregiver webinars, which bring together experienced HCP treaters and caregivers with loved ones already on therapy to speak about their successful experience starting and staying on DABU. We are participating in local RET events, especially during RET awareness month in October that provide an Opportunity to bring together patients and families already on debut treatment with those that are still looking to learn more.

Speaker 2

In addition, we're delivering HCP peer to peer programs to have experienced treaters help newer to brand HCPs with When we measure persistency based on confirmed discontinuations, 81% of patients who started DEBUT remain on therapy at 4 months since treatment initiation. When we measure persistency based on confirmed discontinuations plus Any other patients who are more than 60 days past their last scheduled refill, the real world persistence is 75%. In comparison, in our LIHALAC-one open label extension study, 65% of patients who rolled over to drug from placebo remained on therapy at the end of 4 months. This is important because we believe high levels of persistence early on translate into a higher number of patients staying on therapy long term. Further supporting this outlook is the Extremely high level of persistency we've seen from patients that transitioned from the open label extension to commercial therapy.

Speaker 2

This stronger than this stronger early persistency does not surprise us. We expect the real world to be different from what we observed in our clinical trials where caregivers and patients did not have all the information we now have about proven clinical benefits as well as proper GI management, including specific language in our label that instructs HCPs and caregivers to discontinue anti constipation medications prior to initiating DABU, further supporting a better initial clinical experience. I'd now like to turn to a discussion of conversion and dose compliance on Slide 11. In addition to outperforming on demand, we A significant proportion of this surge converted to paid in the 2nd quarter, but most converted in the 3rd quarter. This leaves a smaller number of yet to convert patients carrying over into the Q4 as we continue shortening the time between a script being written and converting to paid treatment.

Speaker 2

To quantify our progress with payers, our early foundational work has resulted in payers adopting formal plans covering almost 80% of lives to date. Now let me turn to titration and compliance to dose. Two factors that have been helpful in starting and keeping patients on therapy. Compliance to dose through 3 months is in the range of 75% to 80% of the labeled dose. As we expected, the majority of patients begin debut 50% of their prescribed dose and titrate up over a period of 4 to 6 weeks.

Speaker 2

We believe this compliance range provides insight into using titration to find the most appropriate dose so caregivers and patients can benefit long term. Finally, I'd like to turn to what matters most. The real world benefits caregivers are sharing with us about the improvements they're seeing in their loved ones being treated with DABU. Please turn to Slide 12. We are excited and gratified to see the real world daily stories of the impact DABU is having on the lives of Rett syndrome patients through the caregiver testimonials you see on this slide.

Speaker 2

A few representative examples of the day to day important benefits families are describing include improvement in speech or even speaking for the first time in years, broadening vocabulary and improved engagement in conversations, improved gait and feet placement, Purposeful eye contact and improved communication through the patient's eyes, decreased hand ringing and stereotypies coupled with more purposeful use of hands. We also regularly hear feedback about the loved ones increased alertness with patients now being able to better follow conversations or complete activities they previously unable to complete. These testimonials all speak to the promise of treatment with DayVu and we'll continue to monitor I'd now like to move to Slide 13 for a discussion of the great progress We're seeing it in our New Plaza franchise. As Steve noted, the New Plaza franchise is increasingly cash flow positive and continues to provide a strong foundation for our overall business. Leveraging the emerging real world evidence data, We continue to increase our PDP market share versus off label atypical antipsychotics in a contracted overall Parkinson's disease market.

Speaker 2

Product sales of NUPLAZID in the Q3 were $144,800,000 The broad educational campaign we started at the beginning of the year leveraging the real world evidence studies continues to deliver increasing value. The more often we are able to speak with customers about these important regarding NUPLAZID and off label antipsychotics, the greater their confidence in choosing NUPLAZID over other alternatives. These results reinforce the impact these data sets have on our efforts to drive new patient starts. Looking at the broader market dynamics, We see that carbidopalevodopa prescriptions remain essentially flat over the 1st 3 quarters of 2023 versus the 1st 3 quarters of 2022. While NUPLAZID continues to grow, outpacing the market by a wide margin.

Speaker 2

As you can see on this slide, in the office space channel during this timeframe, We've grown new patient starts 9%, while all other PDP products have increased only 1%. Turning to the LTC channel where we delivered a record bottle quarter for New Plaza, we also continue seeing improvement in new resident admissions, indicating some degree of market recovery. In this channel, New Plaza has substantially outpaced the class, growing 16% during this time period, while all other products used to treat PDP in the long term care setting have grown just 7%. Understanding that the large majority of revenues we record during any given quarter are the result of refills by continuing patients, These significant increases in Newplazid new patient starts in both market segments are encouraging. I'll now turn it over to Doug Williamson, our Head of Research and development to provide an update on our pipeline programs starting on Slide 14.

Speaker 3

Thank you, Brendan. In addition to our 2 commercial products, we have a strong pipeline of clinical programs providing us with several opportunities to further expand our growth. Let's start with pimavanserin as a potential treatment for the negative symptoms of schizophrenia on Slide 15. Predominant negative symptoms remain one of the largest unmet needs in schizophrenia. And as of today, There are still no approved treatments for these symptoms.

Speaker 3

Our adjunctive pimavanserin program is designed to treat patients whose positive psychotic As we stated last quarter, we have completed enrollment in ADVANCE-two, our second study of pimvanserin in negative symptoms of schizophrenia. We look forward to sharing top line results in the Q1 of 2024. Please turn to Slide 16. We also have 2 pipeline programs with entirely new compounds in development, ACP-one hundred and one and ACP-two zero four. I'll begin with ACP-one hundred and one.

Speaker 3

We have completed all the necessary steps to initiate the Phase 3 study evaluating ACP-one hundred and one for the treatment of hyperphagia in Prader Willi Syndrome. This is a program we're very excited about. Prader Willi syndrome is a rare genetic neurobehavioral syndrome that affects approximately 8000 to 10000 patients in the United States and represents a significant unmet need. There are currently no therapies approved to treat the characteristic hyperphagia in patients with PWS. On this slide, we've laid out the design of the Phase 3 global Multi center, randomized, double blind, 12 week, placebo controlled study evaluating the efficacy and safety of ACP-one hundred and one approximately 170 prior year only patients.

Speaker 3

The primary efficacy endpoint is improvement of hyperphagia as measured by the Hyperphagia Questionnaire for Clinical Trials or HQCT Scale. Those patients who complete the study will be eligible to enroll in an open label long term extension study. If data from this Phase 3 study is positive, we plan to submit a new drug application for the treatment of hyperphagia in PWS to the FDA. We look forward to working with the Prader Willi community and clinical experts as we continue to advance development of this program. Please turn to Slide 17.

Speaker 3

We continue to advance ACP-two zero four, our next generation 5 HT2A compound, which we're developing as a potential treatment for Alzheimer's disease psychosis. As we previously described, ACP-two zero four works primarily as an inverse agonist of the 5 HT2A receptor. Our experience with pimavanserin suggests that this mechanism is very well suited for elderly populations with multiple comorbidities and concomitant medications, providing antipsychotic efficacy with a very attractive tolerability profile and low drug drug interaction liability. With ACP-two zero four, we're seeking to build on those learnings and believe it has an exciting future. Our work completed to date includes a comprehensive Phase 1 program and supports our target product profile for ACP-two zero four.

Speaker 3

As you can see on the slide, ACP-two zero four's profile could represent a significant improvement over an already strong product profile for pemvanserin. Please turn to Slide 18. Armed with this strong data from Phase 1, we're preparing to start our seamless Phase 2, Phase 3 program for ACP-two zero four in the coming weeks. Our plan includes an initial Phase 2 study with over 300 patients, which is designed to roll seamlessly into 2 Phase 3 studies. This Phase 2 study has been designed and sized in such a way But if successful, it could be considered a pivotal registration study.

Speaker 3

As a reminder, with this Accelerated development plan, we can eliminate dose finding and move from Phase II directly into 2 Phase III studies with the same sites continuously enrolling patients. As each site completes their Phase 2 site allocation, They will move directly into recruiting patients for 1 of the Phase 3 studies. Once the full study allocation of patients for Phase 2 is complete, We will analyze and report Phase II results, by which time the 2 Phase III studies will already be underway. This plan, which we've aligned on with the FDA, will ultimately provide 3 potential pivotal studies for a submission. Overall, we are very excited with the progress of this program.

Speaker 3

It represents an important component of our strategy to continue to provide attractive opportunities to grow our business. And now, I'll turn it over to Mark for a financial update on Slide 19.

Speaker 4

Thank you, Doug. Let's review our quarterly performance on Slide 20. In the Q3, we recorded $211,700,000 Total revenues. NUPLAZID net product sales were $144,800,000 in the quarter compared to 100 $7,000,000 in channel inventory reduction in the prior year that did not recur this year, dollars 4,000,000 attributable to lower 340B volumes $3,000,000 as a result of 2% demand growth. Our gross to net adjustment for New Plaza was 19.9% for the quarter as compared to 18.6% in the Q3 of last year.

Speaker 4

The increase to gross to net was primarily attributable to increased rebates associated with the Inflation Reduction Act, partially offset by the reduction in 340B volumes I just mentioned. Turning to DABU. Net product sales were $66,900,000 in the 1st full quarter of commercialization, reflecting the significant early demand and strong commercial performance described earlier by Steve and Brendan. R and D expenses increased to $157,000,000 in Q3 2023 from $81,300,000 in Q3 2022. The increase was mainly due to the $100,000,000 upfront payment to Neuren Pharmaceuticals for worldwide rights to trofinetide, partially offset by a reduction in other R and D expenses.

Speaker 4

SG and A expenses increased to $97,900,000 in Q3 2023 from $78,100,000 in Q3 2022. The increase was driven by commercial costs associated with our stronger than expected debut launch, partially offset by reductions in our spend on New Plaza. We ended the quarter with a cash balance of $345,900,000 On a year to date basis, excluding the upfront payment for worldwide rights to trofinetide, we were cash flow neutral as a company. With our successful launch of DEBUT, we were cash flow positive in the Q3 also when excluding the recent trofinetide upfront payment. Going forward, We expect to be cash flow positive over the long term subject to the size and scope of future business development.

Speaker 4

Turning to Slide 21, we are raising the bottom end of our full year New Plaza net sales guidance and our new range is $537,500,000 to $545,000,000 We are also raising the bottom end of our full year New Plaza gross to net guidance and our new range is 24.5 to 25%. Additionally, we are providing 4th quarter debut net sales guidance of $80,000,000 to $87,500,000 On the expense side, we are narrowing our guidance ranges as we near the close of the year. Our full year R and D guidance $340,000,000 to $350,000,000 narrows to the middle of our prior range. Our full year SG and A guidance of $390,000,000 to $400,000,000 narrows to the upper half of our prior range. And now, I'd like to turn the call over to Steve for closing remarks on Slide 22.

Speaker 1

Thanks, Mark. Acadia is entering a transformational period of growth as we continue to execute across all our strategic priorities and we I would like to thank our employees for their accomplishments and their ongoing commitment and passion as we continue our mission to elevate life. Operator, I'll turn it over to you to start the Q and A.

Operator

Yes, thank I repeat, please limit yourself to one question. Please stand by while we compile our Q and A roster. Our first question comes from the line of Ritu Baral of TD Cohen. You may proceed.

Speaker 1

Rita, you're coming through very muffled.

Operator

Could you turn up your volume, please?

Speaker 1

Yes, I'm sorry, Ritu. We can't hear you talking. We can't understand what You

Operator

could get in the queue again and try again, but we'll bring our next questioner up. Just a moment please. Your next question comes from the line of Tess Romero with JPMorgan. You may proceed.

Speaker 5

Good afternoon, everyone. Congratulations on all the progress. Our question is around Cadence of patients starting debut specifically in 3Q and how that looked exiting the quarter? And then second question, if I could. Any trends with respect to the type of patient that might drop off earlier than the average?

Speaker 5

Thanks so much.

Speaker 1

Yes. Thanks so much for the question. Brendan, do you want to take this?

Speaker 2

Yes. Sure. Tess, thank you so much for the question. I think the right way to think about our demand early on is a pull forward of the demand we would have expected to see in the first place. We had a highly engaged group of families that got started early.

Speaker 2

It's important to note that prescriptions that came in the 2nd quarter, many would be filled in the 3rd quarter simply due to conversion. And then in the Q3, continued to see in the early part of that quarter that surge, many of which were then filled in the 3rd quarter. Some I I think we'll also carry over into the Q4. So what we're seeing is, I think we've brought the launch to sort of a Stepped up basis in terms of number of families and patients already on therapy and what we would expect to see leaving the Q3 is more linear growth, But on the same slope, very familiar with what you've seen in other rare disease launches, though some of those didn't have the early step up that we've seen.

Speaker 1

And Brendan, I think the second part of the question was regarding any Difference we see in terms of patients that discontinue.

Speaker 2

Sorry, great question. And no, I would say that it's been What we would have expected in terms of discontinuations, as with any rare disease product launch with subjective endpoints, Some patients aren't going to see benefit early enough to stay on therapy. Some will discontinue due to tolerability, but we've seen that across

Operator

Our next question comes from the line of Yatin Suneja with Guggenheim. You may proceed.

Speaker 6

Hey guys, thank you for taking my question. Very impressive results today, particularly on debut. So I have a question on the guidance. Could you comment on some of the push and pulls into that guidance of $80,000,000 to $87,500,000 Like what constitutes the lower versus the high end, what are some of the drivers that can help you maybe be closer to the higher end versus the other end? Thank you.

Speaker 1

Yes. Thanks so much for the question, Jan. Mark, you want to take that?

Speaker 4

Yes. So as we've been discussing, I think we've looked at Kind of all the key parameters that kind of build up to a financial forecast, persistency, demand, access and conversion and All of those together kind of come together to the range. Obviously, higher it gets you towards the higher end, lower towards the lower end. One other thing kind of potentially on the lower end, we've yet to go through the Christmas holiday period with debut. So We don't know if the last couple of weeks of Q4 could impact bottles and sales on the lower end of the range.

Operator

Thank you. Our next question comes from the line of Ritu Baral with TD Cowen. You may proceed.

Speaker 7

Hi, guys. Can you hear me now?

Speaker 1

Yes. Yes. Thank you.

Speaker 7

Okay. I'm so sorry about that Train station noise. No worries. Apologies. So obviously, congratulations on a series of consensus leading quarters.

Speaker 7

I wanted to ask about insurance coverage. As I'm sure you know, we've been conducting tracking surveys of the debut launch. And one of the things we hear over and over again from doctors is complaints about coverage denials. Despite the good numbers, it seems like there's still patients left on the table because of Denials around efficacy, denials around this, denials around that. Wanted to see what you guys were hearing around Denials, maybe how long it takes for these exception letters to come through?

Speaker 7

And now that we're 6 months out, what are you seeing on reauthorization? Thanks.

Speaker 1

Thanks, Ritu. Brendan?

Speaker 2

Sure. Thanks, Ritu. And thanks for the question. So what we've seen is that published coverage policies have been very helpful. Obviously, in the early days, there is going to be a bit of a backlog in patients because we'll get the prescription in, we have to work through the prior authorization process.

Speaker 2

And this is probably a bit compounded by the size of the rare disease treating audience. As you know, this tends To be even in centers of excellence maybe a single physician and an assistant doing some of this work. That's why we've provided them with so much support in the prior authorization process and the work that our hub can do on their behalf. What I can say is that Approvals have continued to accelerate as we moved further into the launch, coupled with these coverage policies that we've seen now covering Approximately 80% of lives. For your specific question around reauthorizations, those have very much been consistent with our expectations and are reflective of what we've seen for other rare disease specialty products.

Speaker 2

They generally require a physician attestation that the patient is receiving clinical benefit from debut. And most of those reauthorizations are at a 6 or 12 month period. Some of them are at 3 months and then annually thereafter. But thus far, we have not had a final denial for a reauthorization for any DEVU patients.

Speaker 1

Just to maybe annotate on that last point. There are denials and there are denials. So it's not uncommon, particularly the plan doesn't have a formal coverage policy in place when a prescription comes into issue an initial denial. And so that is kind of noise in the system much of the time, but still doctors and they're all set to deal with that as we do in terms of supporting them. But as Brendan mentioned, in terms the other kind of denial is a denial where a plan just does not move off of that.

Speaker 1

That is very rare. And So it does create a little bit of noise and administrative effort in the system, which does then subside as more and more plans conform policies in place.

Operator

Thank you. Our next question comes from Gregory Renza with RBC Capital Markets. Your line is open now.

Speaker 8

Hi, this is Anish on for Greg. Congrats on the quarter and thanks for taking my questions. Firstly, from your interactions with physicians treating RET, what should one expect on seasonality of diagnosis, And also if I could, how should we be thinking about potential value unlock for debut availability ex U. S. Specifically by region?

Speaker 1

Yes. Thanks so much. I'm going to ask Parag Viswanee to take the first question and the second question. Rob?

Speaker 9

Sure. Thanks for the question. So what I'll say at the outset is that overall physician feedback regarding JVU is encouraging and points to a high level of Regardless of age, gender, weight range, so on and so forth. So we feel very confident about their view of the product profile. In terms of diagnosis rates, we don't really see any variance or differences as to when patients are diagnosed.

Speaker 9

What we do see are differences When patients are coming in for their follow-up assessment. One of the things that Brendan mentioned in his prepared remarks is that there is a different cadence of when patients are going in to see with their clinicians and over time we expect that to accelerate as more and more patients become familiar with DayView and DayView's product profile.

Speaker 4

Yes. Thanks, Brock.

Speaker 1

Brendan? Yes. Thanks, U. S.

Speaker 2

So for outside the United We are obviously excited about the opportunity to be able to bring trofinetide to a broader audience. Our first progress will be in Canada. As we've described, we're already in conversations to bring DaveView to Canada in the next 18 months. We've also began our conversations with regulators in taking scientific advice in Europe and are also prepping for Japan. We will look opportunistically at the rest of the world as well for opportunities for us to continue our global expansion from there.

Operator

Thank you. Your next question comes from Jeff Hung with Morgan Stanley. Please proceed.

Speaker 10

Thanks for taking my question. You said that centers are treating existing prevalent patients during planned clinic days. So I guess with the expectations of more linear trajectory going forward, Have the number of clinic days remained stable or are they starting to decrease the centers work through existing prevalent patients? Thanks.

Speaker 1

Thanks for the question, Jeff. Parag, you want to take that?

Speaker 9

Yes. Happy to take that question. So I would say that it's variable. We know that a number of centers of excellence have actually increased their clinic days as a consequence of the approval of JVU. Some have gone from a clinic day a month to a clinic day a week.

Speaker 9

Others don't have the infrastructure yet. As Brendan mentioned before on the payer question, there may be just 1 or 2 clinicians who are for everything from clinic visits to payer access issues and managing clinical trials as well. So over time, we are seeing more and more clinic days being added. We're encouraging that and So we're taking those work with connection through centers of excellence and through the community as well. But this is a new muscle that they're learning to flex in many ways.

Operator

Thank you. Your next question comes from the line of Charles Duncan of Cantor. Please proceed.

Speaker 11

Hey, good afternoon, Steve and team. Congrats on a great quarter on the launch I think I'll ask a couple of questions on the pipeline since other good questions been asked On the commercial, on the pipeline, first of all, question regarding ACP-one hundred and one, helpful context in terms of the number of Can you give us a sense of how quickly you would anticipate that study to enroll? And for ACP-two zero four, I guess I'm assuming that they're going to be primarily Alzheimer's disease Patients that are later on in their journey and how will you confirm that there are Alzheimer's patients versus other etiologies or is that not an issue Yes.

Speaker 1

Thanks, Charles. As tempting as to answer the first question, We just typically don't comment on projected enrollment until we get until we actually get some enrollment experience. So we'll have to get back to you on that. And then I'll ask Doug to answer the second question regarding Alzheimer's patients.

Speaker 3

Yes. And it's a great question, Charles, because that's actually we had with the FDA and their outcomes assessment group are increasingly interested in validating the diagnosis of Alzheimer's. So we've agreed with them that we'll conduct a biomarker, a blood based biomarker into the diagnostic procedure to just add extra an extra level of

Operator

Thank you. Your next question comes from the line of Ash Verma with UBS. Please proceed.

Speaker 12

Hi, thanks for taking our questions. So just in terms of persistency on debut, I wanted to get your thoughts on how you This could trend over time. I mean, it's very encouraging to see this 4 month data and get this type of granularity. But how do you think this might look at like 6, 9 or 12 months. And any updates on the LILAC-two study?

Speaker 12

And would that be a more representative data point for what we are seeing in the real world? Thanks.

Speaker 1

Yes. Thanks much. I'll take the first question, I'll ask Kathy to take the second. So with respect to the first question, It's early still. We do have, as we've mentioned, 800 patients with therapy.

Speaker 1

So now we have a meaningful amount of Data on multiple parameters including persistency. So we have a strong number of patients that have completed 4 months of therapy and as we go forward we'll continue To monitor this and update, we have obviously not as many patients at 5 months as we have before and 6 months and 5, etcetera. But I would say that as we look at

Speaker 4

the data that we have,

Speaker 1

with the separation between what we're seeing in the real world And what we're seeing from the LIHALAC-one open label rollovers, which again as we said, we think is the best clinical trial comparison because those are patients Starting therapy, knowing they're starting on debut, we continue to see the separation. And so we'll look forward to updating you as we go forward, but we're highly encouraged by what we're seeing. We expected to be able to improve on our clinical experience. We put significant investments in it and so far we are. Kathy, you want to take the second question?

Speaker 13

Yes. With regards to the LILAC We just completed that study as we had those patients continue on therapy until the drug was approved and they had the opportunity to go on commercial therapy. Over 90% of those patients did roll over directly onto commercial therapy. We now have those results in hand and we actually plan on presenting them in early December, so in about a month, the American Epilepsy Society meeting, but I will say in lilac 2, we had very, very few dropouts. So as long as they enrolled in lilac 2, they stayed in lilac 2.

Speaker 13

And I think that's consistent with what Brendan mentioned is We think if we can sort of get them through that first period, then they continue to stay on terfenucide or debut.

Operator

Thank you. Your next question comes from the line of Tazeen Ahmad with BofA Securities. Please proceed.

Speaker 5

Hi, guys. Good afternoon. Thanks for taking my question. Maybe I'll switch topics for a bit. On the upcoming ADVANCE-two study, Can you just remind us what type of data to expect at that top line readout in 2024?

Speaker 5

And what would you consider to be good data? Thanks.

Speaker 1

Thanks for the question. Doug, you want to take that?

Speaker 3

Sure. So the top line data we We're using the NSA 16, and the primary outcome is the overall change in the NSA 16, which is Very well validated and accepted scale for assessing negative symptoms. In terms of what does good data look like, The effect size that we saw at the 34 milligram dose in ADVANCE-one was around about 0.3 I think it was 0.34 Coincidentally, Nalik, 0.34 was the effect size. So I think that's Consistent with what generally constitutes a clinically significant change in psychiatric studies. So if we saw that replicated, I think we'd regard that as good data.

Speaker 1

Operator, can you hear me?

Operator

Yes. Thank you. Our next question comes from the line of Yu Ihrer with Mizuho. You may proceed.

Speaker 6

Hey, guys. Congrats on the good quarter. I guess I'll switch On NUPLAZID, you mentioned that you benefited from the 340B program this quarter. Just wondering if this is a one time thing or do you expect this benefit to persist going forward? Thanks.

Speaker 1

Thanks much for the question. Mark, you want to take that?

Speaker 4

Yes. So generally speaking, kind of the 340B volumes as part of our mix has been Slowly growing. I think what you saw in the results this quarter was the artifact of kind of a one time increase last That didn't happen this year. So that provided kind of a net change benefit in revenue when you're comparing the quarters. I think as we go forward, we'll continue to expect slow to modest growth in the 340B volumes as we've seen over the last couple of years.

Operator

Thank you. Our next question comes from the line of Mark Goodman with Leerink Partners, you may proceed.

Speaker 14

Hi, thanks. This is Madhu on

Speaker 15

the line for Mark. Couple of other follow-up questions on DABE from us. First, are you seeing any increases in overall diagnosis rates or numbers of rep patients who have been identified in the community or when might you start to expect to see that? And then For the side effects, are you hearing anything on whether the side effects are better tolerated in specific patient subgroups like older versus younger patients? Thanks.

Speaker 1

Brittany, why don't you take the first question? Parag, you want to take the second?

Speaker 2

Yes. So thanks for the question. Can you forgive me, can you repeat the first question? I apologize. The point was did we see Have we seen any increase in Forgive me.

Speaker 2

So diagnosis rates, yes. We are so we're about 2 quarters into the launch of the drug. And as we look at data at this point, we're not seeing meaningful changes in diagnosis rates. I think it's safe to say that it's Early on, our first goal is obviously to try and close that gap between the prevalent population, which we know is 6000 to 9000 And the 4,500 or so diagnosed and treated patients in the U. S, we will continue to track that very closely.

Speaker 2

We know as a first to market drug is approved, you tend to see increases you can see increases in the diagnosed population. And I think we'll continue to share those insights over time.

Speaker 1

Thanks, Vin and Parag?

Speaker 9

Sure. And the second question, what I'll say at the outset is that we're very encouraged with the real world experience both from an efficacy perspective and from a tolerability perspective. Remember, we learned a lot from our Phase 3 program, perspective, remember we learned a lot from our Phase 3 program and that informed the basis of a very comprehensive approach to educating both providers and caregivers at the outset post approval. And I would say that that's been received really favorably. We've designed a footprint inclusive of a patient services team that is paired with every single new caregiver, every single new family that goes on debut and we supplement that with extensive education to providers as well.

Speaker 9

So that is started at launch. It remains a core part of our communication point Again and down with providers and with caregivers and because of that the experience that we're seeing and hearing both from a prescriber perspective and from a caregiver perspective is that a much better tolerability experience with tools to mitigate any tolerability issues as they arise, whether that's diarrhea or other issues as well.

Operator

Thank you. Your next question comes from the line of Salveen Richter with Goldman Sachs. You may proceed with your question.

Speaker 16

Hey, thanks. This is Matt on for Salveen. You all highlighted New Plazards increasingly cash flow positive. Could you Share what kind of margins you have right now and where you see that headed longer term? And then on the litigation, the patent litigation for New Plaza, I think we were expecting an update by the end of the month.

Speaker 16

Do you have any update there for us? Thank you.

Speaker 1

Yes, I'll take the second question first and I'll ask Mark to answer the first one. So the district court has not issued a decision yet. We think highly likely that the court will by the end of the year for sure. And just in terms of Update or commentary, it's litigation. There's nothing really that we have to say at this point other than I'll just say what we said before and that is We believe the law is on our side and we're highly confident in our position.

Speaker 1

So we're eager to get to a decision as well. Mark, you want to take the Yes.

Speaker 4

On the cash profitability of NUPLAZID, we generate approximately $300,000,000 of Cash flow on a fully allocated cost basis of the franchise, we expect that to continue to grow over time. Obviously, on a year to year basis, as we go through our budget plans and investment cycles on a for year over year, we'll decide to The level of support and investment for the franchise with the focus being both on top line growth and near term profitability.

Operator

Thank you. Your next question comes from the line of Sumant Kumkari with Canaccord Genuity, you may proceed.

Speaker 6

Good afternoon. Thanks for taking my question. Assuming that pimavanserin is successful in negative symptoms of Suprenya, how are you thinking about potential pricing given the product is already in the market? And do you think you're appropriately resourced from a sales force perspective?

Speaker 1

Two part question. Britton, why don't you take the second part? Actually, why don't you take both of them if you don't mind?

Speaker 2

Yes, sure. So for us, The in terms of pricing, we're not going to discuss where we are at this point other than to say we certainly will be looking at pimavanserin's value across our portfolio of indications. When we stop to think about resourcing, we are a CNS focused company and there's a fair amount of Crossover between the treaters of negative symptoms of schizophrenia, which will allow us to kind of capitalize on core resources we have within the organization. But we will obviously logically expand our footprint to cover both

Operator

Thank you. Your next question comes from the line of Amy Fadia with Needham and Company. You may proceed.

Speaker 14

Good evening. Thanks for taking my question and congratulations on the strong performance of debut. Maybe just one follow-up on debut from me. Can you talk about sort of the time to approval for patients, whether they are under medical exception or whether they are under an insurance plan where they do have been put on the formulary. And I'm trying to sort of get a better sense of where you are in the launch as you've indicated that the growth from here will be more linear.

Speaker 14

Is this more to do with sort of the initial bolus of patients already getting on DaeVu and now it's more about Really blocking, impacting and waiting for new patients to be diagnosed. So if you can give any color on that. Thank you.

Speaker 1

Thanks so much for the question, 2 part question. Brendan, you want to take the first and Prague the second?

Speaker 2

Sure. That would be great. So, thanks so much. What we've seen and unsurprisingly and very similar to other rare disease launches, The initial approvals have come through letters of medical necessity, letters of medical exception. That was in the second quarter and Somewhat in the Q3, as I pointed to, we now have almost 80% of covered lives that have Written plan coverage for DayView.

Speaker 2

And as we've seen almost on a month by month basis, We've seen more in the same month approvals, 2nd month approvals, 3rd month approvals over time as these written plans have gone into place. So as I think we said on our last call, by the end of the year, we'd expect most of those But we are pleased with the coverage we already have and the increasing pace at which those enrollment forms are becoming prescriptions. Do you want to take the second piece, Bharat?

Speaker 9

Yes. Amy, repeat the second question, please, if you don't mind. Steve, do you want to repeat that?

Speaker 2

I think it was the linearity of The adoption of DABU as a function of sort of what's happening for treatment dynamics in the Q4 Sure. As opposed to Serge.

Speaker 9

Yes, sure. So at this stage, as Brandon mentioned in his prepared remarks and in the Q and A as well, Through a really effective pre launch education and connecting closely with the rec community with providers, we had led to a it led to a step up and update during the 1st couple At this stage, our plan for growth is to really identify physicians and patients that have a solid number of patients And patients that aren't having the same degree of frequency as visits in the clinics as they had in the 1st couple of quarters So our goal moving forward is to establish a strong foundation that we've had so far in terms of demand, which is a significant breadth and depth of physician experience so far, A high number of patients at Danly's sharing positive experience in the marketplace and access that has happened much faster than our initial pre launch expectation. So looking ahead, given this early demand, we're expanding our efforts beyond COE to reach physicians who have fewer rep patients as well as family that aren't as connected

Operator

Thank you. We have time for one more question, which comes from the line of Jay Olson with Oppenheimer. You may proceed.

Speaker 17

Hey, congrats on the quarter and thank you for taking our question. Based on the impressive debut launch dynamic So far, could you please share your latest thoughts on the longer term commercial potential in terms of what peak sales are achievable? And How does the commercial value of the ex U. S. Opportunity compare to the U.

Speaker 17

S. Opportunity? Thank you.

Speaker 1

Thanks so much for the question, Jay. Mark, you want to take that?

Speaker 4

Yes. Clearly, Jay, thanks for that. We're very excited about What we've experienced and launched so far at Debut as we've said many times, we expect this to be a very, very meaningful product In terms of size, in the U. S, it could be similar to pimavanserin, just as a matter of and higher, But as a matter of kind of just our company policy in this, we don't put out peak sales estimates. The size of the opportunity outside the U.

Speaker 4

S, there are more patients just by population In Europe than there are in the U. S, little less in Japan than there are in U. S, the pricing dynamics in those markets could be a little different, But we're still working through that as we advance towards our regulatory efforts. So more to come to discuss in the coming

Speaker 17

Yes. Thank you very much.

Operator

Okay. Thank you. This completes our question and answer session. Mr. Davis, please proceed to closing remarks.

Speaker 1

Great. Thanks much, operator. Thanks again everyone for joining us today. We look forward to updating you on our progress next quarter.

Operator

Thank you again for your participation in today's conference call. This concludes the presentation and you may now disconnect. Good day.

Powered by Quartr
Earnings Conference Call
ACADIA Pharmaceuticals Q3 2023
00:00 / 00:00