Praxis Precision Medicine (NASDAQ:PRAX) released second-quarter financial results and hosted an earnings call on Thursday. Read the complete transcript below.
Benzinga APIs provide real-time access to earnings call transcripts and financial data. Visit https://www.benzinga.com/apis/ to learn more.
Access the full call at https://edge.media-server.com/mmc/p/f6feqzkr
Summary
Praxis Precision Medicine is transitioning into a commercial company with two NDAs in late-stage review, expecting approvals within six months.
The FDA completed mid-cycle communications for Ulixacaltamide, identifying no efficacy-related significant issues, and does not plan to request an advisory committee meeting.
For relitarging, the FDA deemed the submission of additional sensitivity analysis a major amendment, extending the PDUFA target to December 27, 2026.
The company has built comprehensive patient support programs and commercial teams for upcoming launches.
Operating expenses for Q2 2026 were $96.9 million, with $1.4 billion in cash, cash equivalents, and marketable securities, supporting the runway into 2028.
Praxis is finalizing plans to amend and restart POWER 2 and POWER 3 studies for Vermetra gene, informed by lessons from POWER 1.
The company received a Breakthrough Therapy Designation for Healthy Nursing for seizures associated with SCN2A DE.
Praxis expects no further regulatory updates until the expected action dates for their NDAs.
Full Transcript
OPERATOR
Good day, and thank you for standing by. Welcome to the Praxis Precision Medicine Second Quarter 2026 Financial Results 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, you will need to press star 11 on your phone. You will then hear an automated message advising that your hand is raised. To withdraw your question, please press star 11 again.
Please be advised that today's conference is being recorded. I would now like to hand the conference over to our first speaker today, Daniel Ferry, Managing Director of LifeSci Advisors. Daniel, please go ahead.
Daniel Ferry, Managing Director, LifeSci Advisors
Good morning, and welcome to the Praxis Precision Medicine's second quarter 2026 financial results and business update conference call. This call is being webcast live and can be accessed on the Investors section of the Praxis website at www.praxismedicines.com. Please note that remarks made during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These may include statements about the company's future expectations and plans, clinical development timelines, and financial projections.
While these forward-looking statements represent Praxis' views as of today, they should not be relied upon as representing the company's views in the future. Praxis may update these statements in the future, but is not taking on an obligation to do so. Please refer to Praxis' most recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business. Joining us on today's call are Marcio Souza, President and Chief Executive Officer of Praxis, and Tim Kelly, our Chief Financial Officer.
After providing updates on our key programs, we'll move to a brief Q&A session where Marcio and Tim will be joined by Steve Petrou, President of Research and Development, and Megan Sniecinski, Chief Operating Officer. With that, my pleasure to turn the call over to Marcio.
Marcio Souza, President and CEO
Thank you, Dan. Good morning, everyone. Thank you for joining Praxis' second quarter 2026 conference call. Three months ago I told you this would be the year Praxis becomes a commercial company. This quarter is the one where that stopped being a plan and started materializing into the organization. We have two NDAs in late-stage review with the FDA, with both approvals expected in about six months. It's extremely exciting to bring both Ulixacaltamide to ET patients and relithrogine to SCN2A and 8A patients.
We have commercial leadership in place, a field force for the first launch hired and trained, and a distribution network established and inventory being built. I want to spend most of my time today on some key regulatory developments and what we have been building. Let me start with Ulixacaltamide. Essential tremor affects over 7 million Americans and there is still no FDA-approved therapy developed specifically to treat the condition. With the potential approval coming up by January next year, Ulixacaltamide is poised to change that.
Speaking about the NDA review, the FDA completed its mid-cycle communications with us, and we are very pleased with the progress and discussions with the agency. In that meeting, the agency identified no efficacy-related significant issues and stated that it does not plan to request an advisory committee meeting. I would characterize the discussion as expected and very forward-looking. On the commercial build itself, leadership is in place and all core capabilities are where we expect them to be at this stage.
We will be ready ahead of PDUFA to launch Ulixacaltamide for ET patients. We're set up for a very successful launch and continue to think many years in the future as we intend to continue to serve patients with ET and other neurological conditions. As part of that, you should expect updates from us in the near future about life-cycle opportunities for T-type calcium channel inhibitors. One of those steps is the collaboration we just announced with Reimagine, which would extend the reach of elixir further.
Their work is about expanding the value for patients and practices way beyond the initial launch year. Turning to relitarging, SCN2A and SCN8A are amongst the most severe epilepsies we know of, seizure onset in infancy, profound developmental delays, and no approved treatment. The addressable population is roughly 10,000 patients in the United States, as we disclosed last quarter. We submitted additional sensitivity analysis of existing clinical data, and the FDA deemed that submission a major amendment, and the review period was extended, with a new PDUFA target now of December 27th this year.
In the mid-cycle meeting for Lulutrigine, very similarly to Ulixacaltamides, as I just discussed, the agency also confirmed they do not intend to hold an advisory committee meeting. If approved, Raulgine would be the first therapy for SCN2A and 8A D and would be eligible for a pediatric review voucher. Just like for elixir, launch preparation here is further along than the calendar might suggest. Commercial and medical teams are fully hired, the supply chain's established, and we have built a comprehensive patient support program, all pointing to a very structured and successful launch.
The broader opportunity keeps getting clearer. Enrollment in Emeralds, our study in broad GES, exceeds its target with approximately 200 patients enrolled, spanning more than 50 distinct genetically defined etiology, amongst many others not genetically defined. There is a trial population that did not exist as a cohort even five years ago. Assuming the study will be positive and the initial review for Lithuania 2a and 8a also positive, Emeralds would serve as the base for supplemental NDA approval in 2027.
It's also worth mentioning a quick regulatory update that spans both programs. During the quarter, the FDA conducted a BIMO inspection of Praxis as a sponsor for both Ulixacaltamide and relutragen applications. The scope was very comprehensive, including corporate and clinical operations, safety reporting, data integrity, statistical analysis, and the interim analysis for both programs, amongst other areas of the BIMO program. We're incredibly pleased that the inspections concluded without any findings, and therefore no Form 483 was issued.
Considering how complex both programs are, with multiple studies and the first-in-its-kind decentralized study 483, as well as the interim analysis, we're extremely pleased with the outcome of the inspections. One note on how we communicate from now on. Given the stage of discussions on both applications, we do not intend to provide further regulatory updates until the expected action dates. I would ask you to read our silence between now and January as discipline rather than a signal of any kind.
Let me turn to Vermetra gene. In June, we reported top-line results from POWER 1 in a highly refractory focal on-stat seizure population. As you know, the study did not meet its primary endpoints of reduction in monthly focal seizure frequency from baseline to week 12. It did meet a key secondary endpoint, with a significantly greater proportion of patients on barmetregine achieving at least 50% reduction in seizure frequency. That result tells you something specific, and we have spent the last several weeks making sure we took the right lessons from it rather than the comfortable one.
The responder find says the drug is doing something real in a population where very little works. The primary endpoint miss says our dose and a few elements of our design were not matched to the question we're asking. Those are design problems and therefore fixable. We're finalizing the plans to amend and revamp both POWER 2 and POWER 3, informed directly by what POWER 1 taught us about the dose and entry criteria, and we intend to have both studies up and running by the fourth quarter of this year.
We will further describe the amendment and impact on the design once they are final in the very near future. Switching gears to Healthy Nursing. In June, the FDA granted us BTD designation for Healthy Nursing for seizures associated with SCN2A DE caused by gain-of-function variant, based on the results of the EMBRAVE Part A study. That's our third breakthrough designation since July last year. Three designations across three different assets on two different platforms is basically unheard of for a company like Praxis.
We're taking advantage of the access to the FDA that the designations give us and discussing a comprehensive plan with the agency in the near future. Parallel to that, EMBRAVE 3 continues to roll out, with top-line results expected next year. We're incredibly pleased with all the progress made on all fronts this quarter, and we look forward to a successful rest of the year. Let me now turn the call to our CFO, Tim Kelly. Tim.
Tim Kelly, Chief Financial Officer
Thank you, Marcio, and good morning, everybody. Thank you for joining today's call where you've heard about the good updates that we have going on. I'll provide a quick summary of our second quarter financials. In Q2, our operating expenses were $96.9 million, with $69.4 million of that for R&D and the remaining $27.5 million for G&A, which compares to $76 million in operating expenses for the Q2 period in 2025. During the second quarter, Praxis spent $78 million in operating cash compared to $55 million in the second quarter of 2025, with the increase reflecting greater spend in both R&D and G&A. We expect G&A will pick up more in the second half of this year to support our planned upcoming launches. This will be driven by adding two teams of commercial field-facing headcount, rolling out disease state awareness campaigns, building sufficient inventory, and ensuring solid business systems and infrastructure. We ended the second quarter with $1.4 billion in cash, cash equivalents, and marketable securities, compared to $926 million as of December 31, 2025, and we maintain that this is adequate to support our runway into 2028.
With that, I will hand the call back over to Marcio.
Marcio Souza, President and CEO
Thank you, Tim. Really appreciate it. The updates. Now we're going to move into Q&A. Operator, thank you.
OPERATOR
At this time, we will conduct the question-and-answer session. As a reminder, to ask a question, you will need to press star 11 on your phone and wait for your name to be announced. To withdraw your question, please press star 11 again. Please limit one question per analyst and hop back into the queue for further questions. Please stand by while we compile the Q&A roster. Our first question comes from Yasmin Rahimi from Piper Sandler. Your line is open.
Yasmin Rahimi, Analyst at Piper Sandler
Good morning, team. Congrats to an incredible update that I think was very, very timely and important to us, especially as some bears have been creating some noise around, you know, adcom. So thank you for letting us know that you had a successful mid-cycle review along with inspection. Given now that that is behind us, maybe help us understand sort of with the sales team that is being hired for Lugitrigine, what is the phenotype of the sales force that you have in place, the size of it, and how do you see the cadence of hiring for Elixircaltamide?
So Tim, that was really helpful, but if you could dig a little bit deeper around some of the matrix and if you also envision sort of patients have been warehoused as we're getting very close to launch early next year. Appreciate your color and congrats again.
Marcio Souza, President and CEO
Thanks, Yaza. Absolutely share the sentiment that you just expressed there. Right. Like, incredibly complex programs, as we discussed on the remarks, to actually check all the boxes, collaboration with the FDA has been exceptional. The real questions we got throughout have really been, I would say, very straightforward and really very similar to what we've been discussed before publicly. So check that box quite nicely as well. And of course the cherry on top.
It's always good to get the FDA in the house checking everything, making sure that they agree. We always knew we were doing everything correctly, but that they agree with our assessments that, from data integrity, documentation, communications procedures, safety of subjects in these studies, everything was checked there. So we turn a page to talk about what we're really talking about, the millions and millions of Americans that are not served currently in the United States.
We've been very diligent hiring a world-class sales, marketing, market access, medical, of course, team at Praxis Precision Medicine. And I can say this is probably an opportunity of a lifetime if you are in one of those positions to launch these drugs to transform patients' lives. But I'll hand over to Megan to discuss a little bit the phenotype and what we are seeing at the stage we are.
Megan Sniecinski, Chief Operating Officer
Thanks, Marcio. So yeah, as Marcio is sharing, right, not unexpectedly, these are hot launches upcoming. They each represent the first targeted therapies for indications with huge unmet needs. So it's allowed us, from a hiring perspective, to be very selective and we're incredibly pleased with the caliber of the talent. So certainly from the phenotype, individuals with multiple launch experience, the rare neuro space, the hunters that are going to go out there and really do a phenomenal job for us in the case of relutrigene.
Now we've got the team hired and trained, so we have the next few months to really be active in doing the account profiling, which will set us up quite well from a launch readiness perspective. And then the ulixacaltamide field force build-out is well underway and also on track for where we'll be from a launch perspective.
Marcio Souza, President and CEO
Thanks, Megan. Thanks, Yas, for the question.
OPERATOR
Thank you. Our next question comes from Ritu Baral of TD Cowen. Your line is open.
Ritu Baral, Analyst at TD Cowen
Good morning, guys. Thanks for taking the question. Marcio, I wanted to dig down into your comment about the ulixacaltamide mid-cycle review meeting, if you'll let me. You mentioned the word forward-looking. I guess first, could you comment on if there were any surprises during the meeting, any new topics that were unexpected? And two, I guess how do you define what you're calling forward-looking? Have you been able to discuss what we spoke of previously, which is the potential inclusion of alternate titration regimens to improve the ulixacaltamide experience?
And if I could ask a quick follow-up to that last point, the ulixacaltamide experience, what aspects of commercial launch prep are you preparing to optimize that commercial experience to optimize compliance? Thanks.
Marcio Souza, President and CEO
Yeah, absolutely. And I appreciate the vagueness of what forward-looking might be there, so I'll take that one. But it was not meant to be vague, was really meant to be when you look into forward-looking here in the context of this application. Right. So we are late stage now: approval, labeling, promotion, making sure these patients have access. I think that's what I meant by that. In the conversation, I would say, Ritu, the conversation itself in the room there, it's a rare type of feeling when you are in a discussion with the FDA—at least in my view—where you actually feel very peaceful.
And that's the way I would describe how I felt in that discussion, where they know for a fact that they will be incredibly transparent, collaboration is incredibly high, and really all the elements that are necessary to make a decision have been on the table. So on your sub-question about surprise, I would say none, really. Maybe my surprise on the meeting is just how much of the discussion has turned into proper use. Argument by proper use is when you discuss labeling and things like that, normally later in the process, all you're really trying to do is proper use.
So when you are actually marching towards proper use in conversations like this, I consider exceptionally positive the discussions, the level of collaboration and integration, and the understanding of the application, the depth and the breadth of the discussion, the number of people in the meeting, the presence of leadership and the support from leadership, all of that. What I meant is it is an application that matters for them as much as it matters for us.
And it was good to see that overall the topic of titration did come up, to your point, as completely expected. Right. It's something we propose to have. And once again, I was positively surprised by how much further along our alignment is in that regard. While we cannot and should not predict what's going to end up on a label, I can tell you right now, unequivocally, that there is a very good understanding that when patients start ulixacaltamide, they will sometimes, in about 30% of the cases, have some tolerability issues that do not transfer to safety issues.
But if they stay on, that goes away, and they have this, quite phenomenal in my view, efficacy that is just not there for any other compounds. And any reasonable person—and I think that is incredibly reasonable—and certainly we believe we will look into that as an opportunity to maximize the suffering on this incredibly difficult indication by figuring out a way for patients to get there. And I think we're really, really close to figuring that out, how this translates to commercial.
And I'm going to hand back to Megan on this as well. Right. You can imagine that 70% of the patients on 7 million or even 2 or 3 million at launch, anyone would say plenty for. But we want every patient to have the best possible experience, and you want to make sure every patient stays on drug if they desire to and if their physicians believe they should. So maybe Megan can talk a little bit about what we are doing there.
Megan Sniecinski, Chief Operating Officer
Sure. Thanks, Marcio. So maybe just to recap, again, the focus out of the gates for the launch will absolutely be on ensuring the high-quality first experience so that we build the physician confidence and ensure that we have that durable patient persistence. I think in the context from the provider's perspective, and as we've been doing advisory boards and also engaging with the physicians, I think it starts with them setting up that first conversation with clear expectations.
As they see the ulixacaltamide data, the ability to tell a patient there's going to be a rapid onset of effect with a meaningful change, and that there might be some tolerability issues, which, as we hear from the neurologists, they're very comfortable with managing the patients through that. In terms of some patient programs and services that we're building, Tim mentioned in his remarks, we're well underway in establishing the infrastructure we need to support this.
We're basically building a hub of the future, which is fully integrated from the front end to receive the prescription all the way pulling through the channel to ensure that we have line of sight to where we are with fulfilling that first Rx, but then also having certain programs and services on the specialty pharmacy side, with our integrated network, to ensure that we're able to deliver the support to the patient to get them started on treatment as quick as possible and then titrate through those early weeks.
So it's absolutely a priority for us, Ritu, and we're feeling really good about where we are with that build, and the excitement and enthusiasm from the physicians is there to get as many patients started on this therapy.
Ritu Baral, Analyst at TD Cowen
Thank you.
OPERATOR
Thank you. Our next question comes from Francois Brisbaugh from LifeSci Capital. Your line is open.
Francois Brisbaugh, Analyst at LifeSci Capital
All right, thanks for the question. So, just on relutrigine, I was just wondering, I think you mentioned that there's about 50 separate genetic etiologies involved here. Would you say that the study population is relatively enhanced for indications which either have a history of sensitivity to sodium channel blockers or whatnot?
Marcio Souza, President and CEO
Yeah, thanks, Franco, for that. I hand over to Steve to discuss a little bit.
Steven P. Petrou, Chief Scientific Officer
Yes, I mean, you know, looking at the size of the trial, that spread of etiology is precisely what you would think to get when you look at the distribution of prevalence in that group of patients, and the precise mix of people that we fully anticipate would be pharmacosensitive to a sodium channel mechanism is represented in that cohort.
Francois Brisbaugh, Analyst at LifeSci Capital
Okay, great. And then you mentioned at all. Can you comment on the powering of Emerald here? I think based on the... the study number, is there like a placebo kind of level or median percent change that you're looking for for stat sig?
Marcio Souza, President and CEO
Yeah, with the caveat, right, that two—like, multi—both genetically diverse and non-genetically diverse—the study has not been run so far, but there are many that we can borrow from. And when you go for that analysis, I think what we know is, like, there are kind of three levels here. So the first, when you look into the overall response—and let's define whatever, 50%—that benchmark is very clear. It's, like, very small for placebo. Of course, these patients are so severe.
I'll give you a number. The median baseline countable seizures in Emerald is over 50 for 28 days. So imagine that kind of burden and just how little it is the possibility that these patients are going to naturally regress. But the second is, as we move upwards the ladder—like 75% response, 90% response—those numbers become, like, very, very ridiculously small for placebo. So as we are looking into the distribution, it was very simple, I would say, to model from a power perspective, and I would say very straightforward.
The expectations, as you can imagine, as you heard from me before, you hear from Steve now: we're very pleased not only with the a priori powering, but, quite importantly, the posteriori mix of patients that are pharmacosensitive to the mechanism. So stay tuned—soon to come are the results—but I think we should be as bullish as we are on what we're going to see on the other ends.
Francois Brisbaugh, Analyst at LifeSci Capital
Great, thank you very much.
OPERATOR
Thank you. You're back. Our next question comes from Kevin Strang of Goldman Sachs. Your line is open.
Kevin Strang, Analyst at Goldman Sachs
Good morning. Wanted to ask, on formatrigine, you alluded to the design being more important versus the drug itself. Do you mind walking us through sort of some of the specific learnings from Power One on dose or design that gave you confidence to restart the program? Thanks.
Marcio Souza, President and CEO
Yeah, absolutely. So we're going to reserve it, and I hope you don't see this as hedging, because it's not, since we're going to be discussing this a little bit more in the future. But a couple of things that, as we look into, like, in very detail, and at the same time keeping ourselves from seeing things that are not there, a really disciplined approach to what we're going to do next. Right. Let's think about the value right now, at least external value for the company.
One could argue four-fifths of the value is on ulixacaltamide and relutrigine. So, of course, there's a huge potential for upside and huge residual value for vermetregine, but we really want to measure. That's one of the reasons why we're not focused today, call, on vormet. Dose clearly played a role. Duration at the dose clearly played a role. We sometimes say dose, it looks like it was only the 20 or the 30, but actually six weeks and six weeks played a role, and I would say a pretty significant role, on that.
I think a few other things that you're going to be hearing further, including the number of failures that was extremely high to prior medications, that could be tightened up, and a few things here and there. But every single parameter—maybe that's the message I'm going to leave you with—that we look into are very easy to adjust and to fix. And then once we do, without overstretching, without drinking the Kool-Aid, without seeing things that are not supposed to be seen there, the effects on the other side for Power Two and, of course, eventually Power Three, are at or higher than what I would expect for this drug on those populations.
So great way to look into this. We're finalizing a few things internally and With our key advisors. You're going to see an update, a fulsome update about that in the near future and we're starting up this study.
OPERATOR
Thank you. Our next question comes from Tiago Foth of Raymond James. Your line is open.
Tiago Foth, Analyst at Raymond James
Great. Thanks for taking the question just on Emerald. Right. So you know, for Dravet, conventional sodium channel blockers are contraindicated. Sometimes they can make seizures worse. Yet you had really strong preclinical data in Dravet models. Right. So what does that example tell you about the mechanism relative to conventional sodium channel blockers and what does that imply about the potential to work across other indications? We've been getting a lot of questions on the enrichment criteria that you have on seizure burden being enough to offset some of the unknowns or risk from non-ion channel DES that can be in the mix of Emerald.
So how should we think about that overall?
Marcio Souza, President and CEO
Yeah, absolutely. I'll start with and then hand over to Steve here, Thiago. The first is I find a little ironic, I'm going to say, to be the classical me in calls like this. Then no one asks about how many serotonergic mutations are when this is being discussed, the serotonergic one. But it's very easy to say sodium channels for us. So maybe one must revisit their own understanding of neurobiology. But having said that, I'll hand over to the person who really knows neurobiology here.
That's not me, that's Steve. So Steve.
Steven P. Petrou, Chief Scientific Officer
Thanks, Marcio. I think when you look at the role of sodium channels in determining the behavior of neurons normally and in epilepsy, clearly they are the gatekeepers of excitability in a neuron. And because of that role, if the sodium channels themselves are altered in their behavior as a result of mutations, as we saw in the involved study, they are a clear target. But beyond that they are also the most downstream element in the etiology of other disorders that result in disease, whether it's other genetic mutations or acquired conditions.
Because of that very unique role, they are also targets where a lot of the physiology converges. So we got a lot of confidence that it doesn't really matter what the etiology is, even in the cases of loss of function. And there's always a lot of chatter about that. Clearly, even though we've lost sodium channel function as the primary mutation, we still have excitability issues. And the way to control excitability is through modulation of sodium channels.
When these loss of function mutations occur, that can result in the upregulation of other elements in the neuron. So we're confident that our preclinical data shows that the initial... And this is all because sodium channels are concentrated in a very specific part of the neuron where the axon emerges, called the axon initial segment. It's a pretty much crystalline structure of sodium channels and other elements. And that is the little part of the neuron that decides from my experience, from everything that's upstream, what am I going to do, how am I going to respond to that input?
And we know that program is modulated a lot by sodium channel modulation. One other thing I want to say about sodium channels is the manner in which the sodium channel modulator interacts with the actual sodium channels themselves is very important. And we know, we've talked about this a lot, that the mechanism of action and the profile of ralutrigine distinguishes itself from any other agent in the market now. And that was the initial therapeutic hypothesis.
We started with ralutrigine and we are following that through the trials right now.
Tiago Foth, Analyst at Raymond James
Yeah, honestly, very helpful. Appreciate it.
OPERATOR
Thank you. Our next question comes from Douglas Zhao of H.C. Wainwright. Your line is open.
Douglas Zhao, Analyst at H.C. Wainwright
Hi, good morning. Thanks for taking the questions and congrats on the progress, I guess. Marcio, I just want to maybe start with formatrigine for a minute because it was interesting that you sort of are going to be restarting both Power Two as well as Power Three. I'm just curious, do you think that those two studies would be enough to support a potential filing? Just given the fact that they are going to be very different studies in terms of their design and what they're trying to demonstrate.
Thank you.
Marcio Souza, President and CEO
No, thanks, Doug. Yeah, we do. That's... that's the... maybe the short answer to that. There are steps between now and then to have conversations with the agency and to discuss exactly which label potentially that would result. But the bottom line is both from a historical perspective and most importantly from a policy perspective as it firms up right now and even considering like the progressive nature of the division, that’s all epilepsy falls within right now as just restructured a couple weeks ago.
I think we feel incredibly bullish about it, but to be seen.
Douglas Zhao, Analyst at H.C. Wainwright
Okay. And if I can ask a follow up in terms of Lucia gene, I'm just curious because obviously that program, or in particular with Emerald, is enrolling and there's, you know, obviously the Bexacazirin program ongoing as well. And I'm just curious if you have heard any feedback in terms from clinicians, if there's any kind of sort of pattern in terms of what types of patients they're referring to each particular study. Meaning is there any kind of sort of subconscious enrichment, perhaps ongoing in terms of picking a study in which they think a patient might be best to respond to, just given the different MOAs of the drugs.
Thank you.
Marcio Souza, President and CEO
I get it. And I would say we always have to take with a grain of salt anecdotal conversations we have with one or two physicians here and there. But it is not unexpected, right, that you would say, oh no, like let's say we start with serotonergics here. There are drugs approved. There are a lot of stuff that's being done on that space. Hand-to-hand combat with multiple drugs for Dravet and LGS. Yeah, if we're going to try another drug, let's try on the ones that are there.
I would humbly say that, yeah, that's maybe okay for a trial execution. It's a terrible strategy once you got to the markets, but I'll leave it there. I think likewise for us, the Emerald White, as we said, like about 200 patients finished randomization like a while back. And it is kind of obvious by what Steve just mentioned that when you look into the final mix that either by chance or not, that this seems to be some of the most potentially active on this mechanism historically.
So whether or not there was a conscious or unconscious kind of segmentation when there were sites that were enrolled in both studies, it happens naturally. You fast forward a few years from now, both mechanisms work. Right. I think we know that. And on a market with like anywhere between two and 400,000 patients, the discussion is bringing on 10 other mechanisms. Right. Like this is number one, should be a dream for anyone on this space. It's an ability to help incredibly sick kids and young adults to control seizures.
And any one of us that thinks that one mechanism is going to do this should be institutionalized. So I think it is more than reasonable to expect that multiple mechanisms are going to be. I keep going back to the same thematic. You heard me saying this a thousand times, I'm going to do 1001: the zero-sum game idea in disease and epilepsy is purely serving to people who don't want patients to get drugs, has nothing to do with either drug development or market potential.
So if anything else, I'm going to say I'm going to be cheering every day for Luanbec to be incredibly successful, just like you're going to be so we all can help patients with these conditions.
Douglas Zhao, Analyst at H.C. Wainwright
Okay, great. Thank you so much, Marcio.
OPERATOR
Our next question comes from Andrew Tsai of Jefferies. Your line is open.
Andrew Tsai, Analyst at Jefferies
Hey team, good morning. Thanks for all the great set of updates. Back to Essential Tremor. There really to me at least seems to be a chance maybe ET could be approved earlier than expected. Especially if this mid-cycle review is done, inspections are done. Is it the right thinking that you will be entering final labeling discussions soon? Or if not, can you just remind us what the key steps generally are from here? And then how prepared would you guys be to launch in Q4 if there was an early approval?
Thank you. I appreciate you might not be able to share too much, but just thought I'd ask. Thank you.
Marcio Souza, President and CEO
Appreciate it. So the next formal steps here are the quick late-cycle discussion. I'll tell you, that's in the books. Label negotiations. That's in the books. So the reason why we mentioned in my prepared remarks is that we're not going to be giving updates because you can imagine that this discussion as we move forward is very dynamic, right? So there's a lot of back and forth. There's a lot of really cool discussions there. We set the goal to be ready for launch way ahead of the PDUFA for multiple reasons.
One, it's the right thing to do. Two, thanks to a lot of you listening to this call, we have the capital to do it. Three, I would challenge absolutely everyone in this call to name one market with millions of Americans that don't have a treatment right now that are getting every single day requests from physicians and patients to when is this drug going to be available? So it's just a responsible thing to do. So we'll be ready. We are basically ready and we're going to continue to be ready to maximize in the case of the great fortune that the agents finish the review earlier and we are blessed with that approval earlier than the PDUFA.
Andrew Tsai, Analyst at Jefferies
Thank you. Fingers crossed. Thank you.
OPERATOR
Thank you. Our next question comes from Yatin Suneja of Guggenheim. Your line is now open.
Yatin Suneja, Analyst at Guggenheim
Hey guys, thank you for taking my questions. And again, excellent updates today. So just staying with the Essential Tremor, could you maybe talk a little bit about the payer work you have done. Marcio, in the past you have talked about pricing. Love to get the feedback that you are hearing from the payer perspective. And in terms of the step edit, how should we think about it? I mean most people are on generic stuff so there should not be much. Love to sort of understand all of those dynamics.
And in terms of the commercial build out, could you maybe outline for us when is that plan in terms of, you know, how big of a salesforce you would need, all of that stuff. Thank you so much.
Marcio Souza, President and CEO
Yeah, absolutely. So from a payer perspective, very, very active. So we did a lot of pre-work to shape like our general understanding. Of course that is a lot of analytical work that can be done with... done that benchmarking work. And we moved on the last several weeks to a different phase, right, where both proactively we want to talk to some of those plan administrators, but I would say the latest wave is that they want to talk to us. And I would say there was a lot of those interactions.
I would even argue, I was positively surprised with one, their understanding that absolutely there's a need here and that they're not going to put a lot of stuff, not a lot of blocks in the way. The second is just like they want to be ready day one. Just like we want to be ready day one. So that's good news. Our planning assumptions includes step edits through propranolol. Not at all, by the way, what we're hearing across the board is going to happen.
It's just a prudent thing to do or look into this. Now we know we did extensive work here from a medical perspective and claims and so on that a lot of these patients, they're just severe bradycardia or something else that prevents them from ever going into beta blockers. So about half of the market cannot magically take propranolol. One can call that low-hanging fruit, but I guess you call a million patients low-hanging fruit a little bit oxymoronic.
So I'm not going to do that. So that is a very clear part of the markets. I think the other parts they just had exposure to that. I'll remind everyone on the stratified predefined use of propranolol on the Essential3 study showing that on top of propranolol ulixacaltamide is incredibly efficacious. And so there is really no restrictions here one way or another. And welcome. Do we believe that in the long run that's going to be needed to stay involved? No, but that's a belief. We welcome all the patients at day one here, and physicians are incredibly excited about hearing that, which is normally what payers actually want to hear. So a lot of work's been done on the payer space. I know you ask about pricing. I think the more we talk to payers, the more we realize that our initial pricing assumptions are very, well, I would say, grounded.
We talked about a little bit over maybe 50 to 100,000 per year. There was a lot, as you know, from clients of yours and from people without you, a little bit of pushback and you actually go that high. I think right now, while we're not going to disclose the price specifically, I think we're actually very confident that that's the right range to operate in general.
OPERATOR
Thank you for your question. Our next question comes from Cambiz Yazzie of US Bancorp. BTIG, your line is open.
Cambiz Yazzie, Analyst at U.S. Bancorp
Morning, Dean. Thank you for the question. How are you thinking about the ralutragine efficacy in Emerald relative to what was observed? And in BOLD, from a biological level, how should we think about ralutragine performance in broader DEEs compared to the SCN2A and 8A population? Thank you.
Marcio Souza, President and CEO
Yeah. So thanks, Ken. Good to hear. In front of you, I would start with the what is necessary and then what is possible. And I think those are two completely different things here. Right. I mentioned earlier in the call on the background seizure burden for these patients, it is absolutely insane. I cannot even imagine as a parent to have to deal with something like that. This patient strides and this parent strides everything they could possibly imagine all those.
So logically, no matter what he wants to believe, reducing consistently a part of those seizures and therefore statistical significance. When you think about a study, that should be a bar. Right. So the bar here is significant on this study. But of course, we want to go about much, much higher than the bar. Right. So bar for success. No doubts whatsoever. Physicians patients are saying, help me control a little bit better. Let me give a little bit more hours without being on top of these kids nonstop, afraid of complications, you name it.
And that would be a big win. Biologically, though, by what Stephen just discussed, there are reasons to believe that it could be similar, if not better than what you're saying on in BOLD. I think it's hard to imagine. Right. Being better than BOLD. But we need to stay true to the science and to what you're saying so far. We're going to discuss a lot more about this in the near future as well. But again, going to have to stay true to what is possible.
Not necessary, but would love nothing more than help these patients to an extreme. Thank you so much. You bet.
OPERATOR
Our next question comes from Jay Olson of Oppenheimer. Your line is now open.
Jay Olson, Analyst at Oppenheimer
Oh, hey, congrats on all the progress and thanks for taking our questions. We have another ralutragine question and just wanted to follow up on something that you've commented on in the past, Marcio, that you've seen in the pooled masked data from Emerald that you've observed dynamics that are profoundly different from what a meta analysis of historic DEE placebo groups could accommodate. Can you talk about the most important factor behind this observation and how would you compare the information in proportion of patients with a 50% reduction versus 75% reduction versus 100% freedom from seizures.
Thank you.
Marcio Souza, President and CEO
No, thank you very much. I think that all those parameters you mentioned, right, this continuous of response, 50, 70%, 75%, 90, 95, whatever you want, 100 are incredibly important. And we've been tracking and we've been, I would say quite pleased about the entire distribution. Maybe one point here that we haven't discussed as much. It is quite interesting as well to see what happens when they transition to the open label. And study's been going on for a bit and we enrolled relatively fast.
So there is a very large proportion of patients that have multiple months now in the open label. So when you put all of that together, the initial response of the double blind, the information we are able to get from the open label, I would say they depart a lot from what historical expectations would be. And hey, who here hasn't been burned by blinded data right from the first rock? So I'm not saying this is completely proof of any possibilities of not being a misread, but it is just very hard to believe that we would read this incorrectly considering how severe this disease is and how high the seizure burden is.
So very happy across the boards. But we're going to stay vigilant until the end of this study.
Jay Olson, Analyst at Oppenheimer
Super helpful, thank you.
OPERATOR
Our next question comes from Ami Fatia of Needham and Company.
Ami Fatia, Analyst at Needham & Company
Hi, good afternoon. Thank you for taking my question and congrats on all the positive updates this morning. I had one question on Alexa and one follow up on Emerald. As you think about the uptake of Alexa, can you talk about the mix of patients that you expect across maybe the commercial Medicare Medicaid setting? And where do you see the initial patients coming from? Is it sort of older patients that have been sort of, you know, suffering with ET for a very long time or do you also expect younger patients to start to take elixir earlier in the launch? And then with regards to the Emerald study, across the 50 etiologies that you talked about, from a mechanistic perspective, is there a reason to believe that the response rates would be similar or could it be varied across, you know, across the different etiologies?
Thank you.
Marcio Souza, President and CEO
Yeah, no, absolutely. I mean the. So for this launch is going to likely have like several states. If you look into the. I believe we've been quite responsible, defining the addressable population at time of launch, around 2 million patients and that is mostly, I'll say 3/4 or so of those patients would be the Medicare, Medicare Advantage. Like arguably slightly older patients there. Maybe the phenomena that we are seeing more and more is the family members.
Right. And the interests of those patients. And so I would say for the phase two, very likely we're going to see is like a migration continue to increase these patients on these 65 plus a lot of the 40s to 65 patients there, of course there's a different payer mix, there's different dynamic on those patients. Maybe the part we don't talk as much about, we kept this number static, but it's not static. Right. The population demographics in the United States and globally, but particularly in the United States is shifting quite a lot.
And when you look into the prevalence of essential tremor in the overall population, it's a little bit about like 2, 2.5%. When you get to 60s, that is about two and a half times the overall prevalence. And then about every 10 years after that it doubles. So we haven't discussed, but you're going to hear us discussing a lot more is actually the completely organic growth of these markets that is about double digits. And we just don't have drug launch on multimillion patient markets growing organically as a market, double digits are moving forward.
So that changed a little bit the mix. I know you had a Emerald question there as well. Efficacy across the etiology. Oh, the efficacy across etiologies. Thanks, Tim. Of course it's not going to be the same like I think it would be. Actually. I think my math teacher would tell me would remove my diplomas if I say it's going to be the same on an heterogeneous population. But we do expect that would be consistently positive and I think that that's what we should be expecting at this point in time.
Ami Fatia, Analyst at Needham & Company
Thank you.
Marcio Souza, President and CEO
Thank you.
OPERATOR
Our next question comes from Brian Scorney of Baird, your line is open.
Brian Scorney, Analyst at Baird
Hey, good morning guys. Thanks for taking the question. Maybe if I could just kind of ask you to characterize some of the areas of the focus for the agency in the mid cycle review meeting for you look so like, like who took the most time on the side of the FDA? Was it like the clinical review team, the stats group, the safety group? Is it mostly handed by the lead reviewer? Like are Emily Friedlich and Teresa Baraccio in the meeting? And I don't know if this is something that comes across in the context of a mid cycle review meeting, but any insight into FDA's thinking about whether or not they're going to look for DEA scheduling here?
Marcio Souza, President and CEO
Yeah, so I would say those meetings are comprehensive. This is not exactly like, oh, they stayed quiet for several months and they come and dump a meeting on us. Quite the opposite. Right. There's been dialogue and overall it's an opportunity. As you might recall, when Senate with heavy lobby from the industry requested mid cycle meetings to be implemented as part of a PDUFA authorization was to actually give up as the applicants an opportunity to have that discussion how things are going.
And I would say very, very little on areas that are of, I would say interest for people that don't have an interest on this drug getting to the markets like some of your clients. A lot of the interest here was actually how to act against this drug to help patients. All the areas were represented that you named there. As is normally the case, of course senior leadership was represented since this is not only an important application, but as one with breakthrough designation.
No drug approved mechanistically for essential tremor ever, only one approved. So you would imagine that that fits exactly the agenda for the FDA from a public health perspective in the United States. And what I would say is, and as we said in the prepared remarks, which by the way, we're legally obliged to be complete as you know. So I find some of the questions, to be honest, a little bit annoying, is that there was no major like comments here or there.
So we see this as overall incredibly positive. There we are. It's not over yet. It's never over. But one must take the stage. We are right. The questions before this call were what happens in the mid cycle? Is the FDA going to have an advisory committee is this, this and that? So maybe it's time to flip the page towards how large of an opportunity essential tremor is and burn the ships as one say in Kartaga, and start moving forward towards conquering new worlds.
Brian Scorney, Analyst at Baird
Thanks, Marcia.
OPERATOR
Our next question is Danielle Brill of Truist. Your line is open.
Alex (for Danielle Brill), Analyst at Truist
Hey guys, this is Alex on for Danielle. Thanks for taking the question. Another question on the mid cycle reviews, just given that you have these two mid cycle reviews in close proximity, any noticeable differences in the tenor, pushback, body language, et cetera between the FDA reviews for Elixir vs. Rauluchigine? Thanks so much.
Marcio Souza, President and CEO
I would say no on the body language. I think we all that is very collegial discussion throughout the group at the agents and ourselves and exemplified by the fact that we are really the only company that probably know every person that room by name and actually have a trust and rapport with each one of them because there are multiple INDs and multiple NDAs under review. Much much larger. Right on the as you can imagine, the application for Elixir Altamide hydrochlorate is so much larger.
So there's a lot more people involved on that. But. But if anything I'm a paranoid by nature person so I never expect people to be very happy on meetings like this. But I'll venture to say that I think it's very calm. As I said, it's very peaceful and body language is incredibly positive across the board and it reflects the collaboration throughout the review as one would expect.
Alex (for Danielle Brill), Analyst at Truist
Thanks so much.
OPERATOR
Our next question comes from David Hoang of Deutsche Bank. Your line is open.
David Hoang, Analyst at Deutsche Bank
Hi there. Thanks for the updates and taking my questions. So I want to go back to Olixa's potential commercial launch. Could you talk a little bit about The prescriber base for the drug — and remind us, will this be primarily neurologists writing for it? Or would a primary care doc, let's say, feel comfortable to write for this drug? And what size of sales force would you need to support a successful launch? And then if you could just remind us of your latest assumptions on peak sales for Alexa. Thank you.
Marcio Souza, President and CEO
Sounds good. We start with the last part and I’ll hand over to Megan, the big sales here. I think we’ve been very conservative when you look at the size of the opportunity in general — from number of patients, from not really having anything else, the growth we just mentioned that we have not been adding, general feedback from physicians, you name it — we set that floor at around 10 billion. And I would say the more we move forward, the more we feel comfortable that that's really a fairly conservative number.
Let me hand over to Megan to discuss the other topics.
Megan Sniecinski, Chief Operating Officer
Yep, absolutely. Thanks, David, for the question. So from a target perspective, you're right — neurologists are our focus coming out for the launch, with us targeting them primarily because of their strong ET influence and just the patient volume. So with a call target sizing in the 13,000 to 15,000 range, that puts us in place of having a field force around 300. And as I shared earlier at the start of the Q&A, we're well underway with our hiring.
And again, the context we're heading into — with the first targeted therapy, huge unmet need, and just the opportunity to have the most successful launch here in neurology — we're definitely attracting top-caliber talent that want to be a part of this. And again, the focus for the buildout will allow us to be out in the field doing the account profiling, so we're very well prepared upon PDUFA.
OPERATOR
Thank you. Our next question comes from Leonid Timishev of RBC Capital Markets. Your line is open.
Josh (for Leonid Timishev), Analyst at RBC Capital Markets
Hey guys, Josh on for Leo. Thanks for taking my question. So for the initial patient population that you'll be targeting for ralutrigine, are you planning on going after the most severe patients or do you think you'll go more broadly earlier, and how might that play with how clinicians typically may use a novel seizure agent? Thanks.
Marcio Souza, President and CEO
Yeah, I would say to call any patient with this condition non-severe is probably something I’m never going to be able to do. So the population is the population here, right? We are. Steve represented us at the SCN2A Family Foundation meeting last week, and we had several updates after that in discussions with them, and many clinicians gave us feedback based on how they are waiting for this. Some of these hospitals in America — centers of excellence — have very large, either the largest or second-largest cohorts of GEs.
Why we should never be suffering is suffering, and we shouldn’t compare, but when you look into other GEs that there are three or four companies going after, they are way, way, way less severe, and the majority of the patients are being treated there. So we don’t see a segmentation per se here, but really careful use. I don’t think we would want everyone to just start right away without doing the proper assessments of these patients, making sure their background medications are optimized before getting into ralutrigine.
So that is what’s going to dictate the launch. I think our medical education, exchange discussions are going to focus on proper use, because proper use is what leads to maximum penetration and maximum retention and, of course, maximum benefit for patients. And the other part that we’re all interested in is maximum revenues that can return to all of us and get more drugs to the market. So that is the strategy here, and I couldn’t be more pleased with the feedback we’re getting from physicians and patient groups.
OPERATOR
Thank you. Our next question comes from Rudy Lee of Wolfe Research. Your line is open.
Rudy Lee, Analyst at Wolfe Research
Thanks for taking my question. For Ulixa, what gives you confidence that titration can help improve discontinuation in practice? Like what data or evidence do you have to support your titration proposal? And how should we think about discontinuation rates in the real world? Thanks.
Marcio Souza, President and CEO
Now that is a fantastic question and one that we spent a lot of time ourselves — and of course discussing with the agency. That is why I can’t possibly go through every line of evidence here. One thing that is quite key that we haven’t — and I’ll take full responsibility for not actually discussing this properly publicly before — is: if the patient is steady, odds of staying on drug and responding, if you just stay a day or two more after arriving at the R, are disproportionate.
So that evidence, and the mathematical evidence, is very, very clear. Right. So imagine a study: when we conduct these studies, we wanted to make sure we’re not biasing these patients. When a patient goes to an office to discuss with their physician, it is very different — conversations like, "This is the possible benefits and this is the possible risks." Right. But the benefit question is there. In a clinical study, the benefit question is not there.
So that is a key driver. So we have a fair bit of data showing that if patients stay on the drug, and if they stay a little bit longer — not a lot longer — they’re going to be able to tolerate and get fantastic, in my words, benefits. On the other side of that, our proposal in the label — notwithstanding the fact that label has to be approved by the agency and so on and so forth — is that physicians are instructed to, if they have concerns because they know their patients.
There are patients that chronically don’t respond so well in terms of tolerability. They can keep the patients for a little bit longer. Right. It is important because they’re going to see 70% of the patients doing really well, and then their desire is going to turn into, "I want to get all my patients to do really well." And that’s the bridge we want to— what Megan mentioned before about the hub of the future. Right. It is really — and we’re going to be talking about it in our commercial day coming up soon, going to be announcing — it is really a state-of-the-art way to help the practice manage the patients and get all the tools to maximize tolerability. We could be here saying, "Why do we care about those patients?" Right. It’s completely irrelevant from a big revenue perspective, but it’s not, because we know this drug works and we want to make sure it’s there with each one of those patients. So I really appreciate it. It is something very close to our hearts. Our team worked incredibly hard to make sure every percentage point is not only a percentage point in revenue, it’s a lot more patients being able to get benefits that they cannot get any other way.
Rudy Lee, Analyst at Wolfe Research
Very helpful. Thanks for the color.
Marcio Souza, President and CEO
Of course.
OPERATOR
Our next question comes from Ben Burnett of Wells Fargo. Your line is open.
Orfi Azonifu (for Ben Burnett), Analyst at Wells Fargo
Hi. Good morning, team. This is Orfi Azonifu. Ben, congrats on over-enrolling EMERGE. I had one question on RELU and one on your cash runway. First, on RELU, are you able to share what proportion of EMERGE patients are on Xcopri or another sodium blockade at baseline? And what are your expectations for incremental efficacy in patients who are already on cenobamate? Then secondly, on your cash runway, given that both Velo and LSW are eligible for pediatric vouchers, are your current plans to monetize those on approval, and is that contemplated in your cash runway?
Thank you very much.
Marcio Souza, President and CEO
Yeah. So I think we got a very representative distribution of all the background meds here. Very happy. I’ll tell you, discontinuation, for example — it’s a good surrogate — they have been extremely low in this study. Tolerability being very good. We know, unfortunately, a lot of these patients failed pretty much everything. So you name a drug, I’m going to tell you they failed, or they are on it. But we’re confident not only on the effects, which is important, but on the safety as well, to get to a positive benefit-risk.
And then I’ll leave the last question to Tim, who is anxiously waiting for a financial crash and protocol.
Tim Kelly, Chief Financial Officer
Thanks for the question about the runway. And I think part of what we talked about with the runway is it gives us this great flexibility and ability to launch into these launches the way we’re investing with field force and all the activities that Marcio and Megan have taken us through. With respect to the PRV, because we do anticipate approval for ralutrigine for 2a and 8a, where we do have orphan designation and are eligible for a PRV, we would expect to receive that as well.
It is not a meaningful impact to our runway, but it does ensure that we can continue to invest in these launches. And you’re right also about elzanursen down the road, because that also has orphan designation. We believe that would be our second product that could be eligible for a PRV. But thank you for the question.
Orfi Azonifu (for Ben Burnett), Analyst at Wells Fargo
Got it. Thank you. Congrats again.
OPERATOR
Thank you. This concludes the question and answer session. I would now like to turn it back to Marcio for closing remarks.
Marcio Souza, President and CEO
Yeah. Thank you so much. I appreciate — I hope you're seeing — we tried to be very comprehensive today, giving updates on— it’s just absolutely amazing, palpable energy that we get every single day here in the office with all the now sales team as well and being there and talking to physicians, giving us a lot more information. I would say as we move this page towards commercial, a lot of you helped us along the way to make a successful clinical development for these drugs.
As we're going to discuss less and less the clinical and regulatory, I just want to take a moment to thank all of you who are certainly my biggest critics and my biggest supporters when we got into certain conversations. And I appreciate every feedback being given to the company — made us to where we are right now. Couldn't be prouder on behalf of patients when we get these stories every single day. And trust me, we get them every single day — from the patients who transitioned on EMERGE to the open label, or the ones who are on BOATS, or the ones that are on emergency access of one of our medicines, or particularly these days for the ones wanting to be on. That's what keeps us going. Thanks enormously for your support, and really looking forward to the conversations later today and in the near future.
OPERATOR
Thank you for your participation in today's conference. This does conclude the program, and you may now disconnect.
Disclaimer: This transcript is provided for informational purposes only. While we strive for accuracy, there may be errors or omissions in this automated transcription. For official company statements and financial information, please refer to the company's SEC filings and official press releases. Corporate participants' and analysts' statements reflect their views as of the date of this call and are subject to change without notice.
Login to comment