PRAX

Image source: The Motley Fool. Thursday, Aug. 6, 2026 at 8:30 a.m. ET President and Chief Executive Officer - Marcio De'Souza Chief Financial Officer - Tim Kelly President of Research and Development - Steve Petrou Chief Operating Officer - Megan Sniecinski Operator: Good day, and thank you for standing by. Welcome to the Praxis Precision Medicine Second Quarter 2026 Financial Results Conference Call. [Operator Instructions] 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: Good morning, and welcome to the Praxis Precision Medicines 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 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 time lines 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 De'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, it's my pleasure to turn the call over to Marcio. Marcio? Marcio Souza: 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 become 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 relutrigine to SCN2A and SCN8A patients. We have commercial leadership in place, a field force for the first launch hires and train 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 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 it 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 Remagine Labs, which would extend the reach of ulixacaltamide further. Their work is about expanding the value for patients and practice way beyond the initial launch year. Turning to Relutrigine. SCN2A and SCN8A are among 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 27 this year. In the mid-cycle meeting for relutrigine, very similarly to ulixacaltamide, as I just discussed, the agents also confirmed they do not intend to hold an advisory committee meeting. If approved, relutrigine would be the first therapy for SCN2A and SCN8A-DEE and would be eligible for a pediatric review voucher. Just like for ulixacaltamide, launch preparation here is further along than the calendar might suggest. Commercial and medical teams are fully hired, the supply chain is 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 EMERALD, our study in broad DEEs exceeded 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 relutrigine in SCN2A/SCN8A also positive, EMERALD 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 relutrigine 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 for ET as well as the interim analysis, we're extremely pleased with the outcome of the inspections. One note on how it communicates from now on. Given the stage of discussions on both applications, we do not intend to provide further regulatory updates until the expected action date. 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 vormatrigine. In June, we reported top line results from POWER1 in a highly refractory focal onset seizure population. As you know, the study did not meet its primary endpoint of reduction in monthly focal seizures frequence from baseline to week 12. It did meet a key secondary endpoint with a significantly greater proportion of patients with vormatrigine 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 findings say the drug is doing something real in a population where very little works. The primary endpoint miss say our dose and a few elements of our design were not matched to the question we are asking. Those are design problems and therefore, fixable. We're finalizing the plans to amend and revamp both POWER2 and POWER3, informed directly by what POWER1 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 amendments and impact on the design once they are final in the very near future. Switching gears to elsunersen. In June, the FDA granted us BTD designation for elsunersen for seizures associated with SCN2A DEE 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. It's basically unheard of for a company like Praxis. We're taking advantage of the access to the FDA that the designation give us and discussing a comprehensive plan with the agency in the near future. Parallel to that, EMBRAVE3 continues to enroll well with top line results expected next year. We're incredibly pleased with all the progress made in all fronts this quarter, and we look forward for a successful rest of the year. Let me now turn the call to our CFO, Tim Kelly. Tim? Tim Kelly: 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: Thank you, Tim. Really appreciate the update. Now we're going to move into Q&A. Operator? Operator: [Operator Instructions] Our first question comes from Yasmeen Rahimi from Piper Sandler. Yasmeen Rahimi: Congrats on an incredible update that I think was very, very timely and important to us, especially as some there have been creating some noise around 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 relutrigine, what is the phenotype of the sales force that you have in place? What is the size of it? And how do you see the cadence of hiring for ulixacaltamide? Marcio Souza: Thanks, Yas. I 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, we checked 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're doing everything correctly, but that they agree with our assessment that from data integrity, documentation, communications, procedures, safety of subjects in the studies, everything was checked there. So we turn a page to talk about what we really like talking about that 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, Tim at Praxis. 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 in the stage we are. Megan Sniecinski: Thanks, Marcio. So yes, as Marcio was sharing, right, not unexpectedly, these are hot launches upcoming. They each represent the first targeted therapies for indications with huge unmet need. 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. And in the case of relutrigine, 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. Operator: Our next question comes from Ritu Baral of TD Cowen. Ritu Baral: Marcio, I wanted to dig down into your comment about the mid-cycle view, let mid-cycle review meeting, if you 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? Marcio Souza: Yes, absolutely. 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, it 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, like 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, it's a rare type of feeling when you are in a discussion with the FDA, at least in my view, what you actually feel very peaceful. And that's the way I would describe how I felt on that discussion where they know for a fact that they will be incredibly transparent, like collaborations be 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, not really, maybe my surprise on the meeting is just how much of the discussion turns into proper use, I was going to call of the drug. And 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, right? So when you're 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, that's completely expected, right, is something we propose to have. And once again, I was positively surprised by how much like further along our alignment is on that regard. While I cannot and should not predict what's going to end up saying 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 20% of the case, have like some tolerability issues that does not transfer to safety issues. But if they stay on that, that goes away and they have this quite phenomenal, in my view, right, 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 are, we'll 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 million or 3 million at launch, anyone would say plenty for a very, but we want every patient to have the best possible experience and you want to make sure every patient stay 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: 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. And what they're, as they see the ulixacaltamide data, right, the ability to tell a patient, there's going to be a rapid onset of effect, right, 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 SP, the 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. Operator: Our next question comes from Francois Brisebois from LifeSci Capital. François Brisebois: 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 what not? Marcio Souza: Yes. Thanks, Francois, for that. I'll hand over to Steven to discuss a little bit. Steven Petrou: Yes. I mean, 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. François Brisebois: Okay. Great. And then you mentioned at all, can you comment on the powering of EMERALD here? I think based on the study number, is there like a placebo kind of level or median percent change that you're looking for testing? Marcio Souza: Yes. With the caveat, I think that's a true like multi both genetically diversity and non-genetically diverse the DEE study has not been run so far, but there are many that we can borrow from. And when you go through 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 or 28 days. So imagine that kind of burden and just how little it is the possibility that these patients are going to naturally regress, right? The second is, as we move upwards the ladder, like 75% response, 90% response, those numbers become like very ridiculously small for placebo. So as we are looking into the distribution, it was very simple, I would say, to model from our perspective. And I would say very straightforward, the expectation that you can imagine, as you heard from me before, you hear from Steven Petrou now, we're very pleased not only with the priority powering, but quite importantly, the posteriori mix of patients that are pharmacoensitive to the mechanism. So stay tuned soon to come up the results, but I think we should be as bullish as we are on what we're going to see on the other end. Operator: Our next question comes from Kevin Strang of Goldman Sachs. Kevin Strang: I wanted to ask on for vormatrigine. 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 POWER1 on dose for design that gave you confidence to restart the program? Marcio Souza: Yes, absolutely. So we're going to reserve, and I hope you don't see this as hedging because it's not like since we're going to be discussing this a little bit more in the future. But a couple of things as we look into like in a 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? Looking about the value right now, at least external value for the company, one could argue 4/5 of the value is on ulixacaltamide and vormatrigine. So of course, there's a huge potential for upside and a huge residual value for vormatrigine, but we really want to measure. That's one of the reasons why we're not focus today's call on format. Dose clearly played a role. Duration at the dose clearly play a role. We sometimes say dose, it looks like it was only the 20 or the 30, but actually six weeks and six weeks play a role. And I would say a pretty significant role on that. I think a few other things that we'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 matter we're going to give 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 like seeing things that are not supposed to be seeing the effects on the other side for POWER2 and of course, eventually POWER3 are at or higher than what we would expect for this drug on those populations. So great way to look into this. We're finalizing a few things with internally and with our key advisers. You're going to see a fulsome update about that in the near future and we are starting up this study. Operator: Our next question comes from Tiago Fauth of Raymond James. Tiago Fauth: Just on EMERALD, right? So for Dravet, conventional sodium channel blockers are counterindicated sometimes it 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 DEEs? We've been getting a lot of questions on the enrichment criteria that you have on seizure burden be enough to offset some of the unknowns or risk from non-ion channel DEEs that can be in the mix of EMERALD. So how should we think about that overall? Marcio Souza: Yes. Absolutely. I will start with and then hand over to Steve here, Tiago. The first is I find a little ironic, I'm going to say to be the classical me in calls like this, that no one asked about how many serotonergic mutations are when this is being discussed as 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. Steve. Steven Petrou: 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 INVOLVE 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 DEEs, whether it's other genetic mutations or acquired conditions. Because of that very unique role, they are also targets that where a lot of the physiology converges. So we've 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 the loss of function mutations occur, that can result in the upregulation of other elements in the neuron. So we're confident of that. Our preclinical data shows that. The initials, 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, 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 relutrigine distinguishes itself from any other agent in the market now. And that was the initial therapeutic hypothesis we started with relutrigine, and we are following that through the trials right now. Operator: Our next question comes from Douglas Tsao of H.C. Wainwright. Douglas Tsao: Congrats on the progress. I guess, Marcio, I just want to maybe start with vormatrigine for a minute because it was interesting that you sort of are going to be restarting both POWER2 as well as POWER3. 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. Marcio Souza: No. Thanks, Doug. Yes, we do. That's 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 all epilepsy falls within right now as just restructured a couple of weeks ago, I think we feel incredibly bullish about it, but to be seen. Douglas Tsao: Okay. And if I can ask a follow-up in terms of relutrigine. I'm just curious because, obviously, that program in particular with EMERALD is enrolling, and there's obviously the ulixacaltamide 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? Marcio Souza: I get it. And I would say we always had to take with a salt anecdotal conversations we have with one or two physicians here and there. But it is not unexpected, right, that you would say, like, let's say, we will start with serotonergics here. There are drugs approved. There are a lot of stuff that's been done on that space, hand-to-hand combat with multiple drugs for Dravet and LGS. Yes, people are going to try another drug, let's try on the ones that are there. I would humbly say that, yes, that's may be okay for a trial execution. It's a terrible strategy once we get to the market, but I'll leave it there. I think likewise, for us, the EMERALD right, 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 it seems to be some of the most potentially like 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 200,000 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, I think 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 not, I keep going back to the same thematic. You heard me saying this 1,000 times I'm going to do 1,001. The zero-sum game idea in diseases and epilepsy is purely serving to people who don't want patients to get drugs. As long as it 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 Lundbeck to be incredibly successful just like you're going to be, so we all can help patients with these conditions. Operator: Our next question comes from Andrew Tsai of Jefferies. Lin Tsai: Thanks for all the great 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 the 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? I appreciate you might not be able to share too much, but that's just thought I'd add. Marcio Souza: I 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. But you can imagine that this discussion as we move forward is very dynamic, right? 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 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 we're getting every single day request 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. Operator: Our next question comes from Yatin Suneja of Guggenheim. Yatin Suneja: 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 have talked about pricing. I'd love to get the feedback that you are hearing from the payer perspective. And in terms of the step at it, 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 how big of the sales force you would need, all of that stuff? Marcio Souza: Yes, absolutely. So from a payer perspective, very, very active. So we did a lot of prework to shape like our general understanding, of course, there 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 they're not going to put a lot of stuff and not a lot of blocks out 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, we're looking 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 super cardiac or something else that it prevents them from ever going into a beta blocker. So about half of the market cannot medically take propranolol. One can call that low-hanging fruit, but I guess to call million patients low-hanging fruit to live with oxymoronic, so I'm not going to do that. So that is a very clear part of the market. I think the other part, they just have exposed 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, right? 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 on both? 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 is being done on the payer space. I know you asked 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 $50,000 to $100,000 per year. There was a lot, as you know, from clients of yours and from people we talked to a little bit of pushback. In fact, we 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: Our next question comes from Kambiz Yazdi of U.S. Bancorp BTIG. Kambiz Yazdi: Question. How are you thinking about relutrigine's efficacy in EMERALD relative to what was observed in EMBOLD? From a biological level, how should we think about relutrigine's performance in broader DEEs compared to the SCN2A and SCN8A population? Marcio Souza: Yes. So thanks, Kambiz. Good to hear from you. I would start with 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 of seizure burden for these patients, right? Like it is absolutely insane. I cannot even imagine as a parent to have to deal with something like that. These patients tried and these parents tried everything they could possibly imagine on 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 significance on the study. But of course, we want to go about much, much higher than the bar, right? So bar for success, no doubt whatsoever, physicians, patients are saying, help us control a little bit better. Let me give a little bit more hours without like being on top of these kids, nonstop afraid of complications to that, you name it. And that would be a big win. Biologically, though, by what Steven just discussed, there are reasons to believe that it could be similar, if not better than what you're seeing on EMBOLD. I think it's hard to imagine, right, being better than both, but we need to stay true to the science and to what we are seeing 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 we love nothing more than help these patients to an extreme. Operator: Our next question comes from Jay Olson of Oppenheimer. Jay Olson: On all the progress. We have another relutrigine 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 mask 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? Marcio Souza: No, thank you very much. I think that all those parameters you mentioned, right, this continues of response, 50%, 70%, 75%, 90%, 95%, whatever you want, 100 are incredibly important. 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's quite interesting as well to see what happens when they transition to the open label. And study has been going on for a bit, and we enrolled relatively fast. So there's a very large proportion of patients that have multiple months now in the open label. So when you put all of that together, I say initial response was double blind, the information we're 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 the data, right, for the first rock. But, so I'm not saying this is like 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 board, but we're going to stay vigilant until the end of the study. Operator: Our next question comes from Ami Fadia of Needham & Company. Ami Fadia: On all the positive updates this morning. I had one question on ulixacaltamide and one follow-up on EMERALD. As you think about the uptake of ulixacaltamide, 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 suffering with ET for a very long time? Or do you also expect younger patients to start to take ulixacaltamide 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 rate would be similar? Or could it be varied across the different etiologies? Marcio Souza: Yes. No, absolutely, This launch is going to likely have like several states. If you look into this, I believe we've been quite responsible defining the addressable population at the time of launch around two million patients. And that is mostly, I would 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 interest of those patients. And so I would say for the Phase II, very likely what we're going to see is like a migration continue to increase these patients on the 65 plus. was a lot of the 40 to 65 patients there. Of course, there's a different payer mix, the 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 2.5x the overall prevalence. And then about every 10 years after that, it doubles. So, we haven't discussed what you're going to hear us discussing a lot more is actually the completely organic growth of this market that is about double digits. And we just don't have drug launch on multimillion patient markets growing organically as a market double digits moving forward. So that changed a little bit the mix. I know you had a Emerald question there as well. Tim Kelly: The efficacy across etiologists. Marcio Souza: Thanks, Tim. of course, it's not going to be the same. Like I think my message here would tell me would remove my promise if I say it's going to be the same on a heterogeneous population. But we do expect that would be consistently positive. And I think that's what we should be expecting at this point in time. Operator: Our next question comes from Brian Skorney of Baird. Brian Skorney: 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 ulixacaltamide, 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 Frelik and Theresa Buracchio in the meeting? And I don't know if this is something that comes across in the context of the mid-cycle review meeting, but any insight into FDA is thinking about whether or not they're going to look for DEA scheduling here? Marcio Souza: Yes. So I would say those meetings are comprehensive, right? This is not exactly like, oh, they stayed quiet for several months and they come, and the meaning 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 reauthorization was to actually give us as the applicants an opportunity to have that discussion on how things are going. 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 market like some of your clients. A lot of the interest here was actually how to actually get 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 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 also, 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 that we are. It's not over yet. It's never over. But one must take a stage we are, right? The questions before this call or what happens in the mid-cycle. Is the FDA going to have an Advisory Committee? Is this and that? So maybe it's time to flip the page towards how large of an opportunity the essential tremor is and burn the ships as one say in Carthage and start moving forward towards conquering awards. Operator: Our next question from Danielle Brill of Truist. Unknown Analyst: This is Alex on for Danielle. Another question on the mid-cycle reviews. Just given that you have these 2 mid-cycle reviews in close proximity, any noticeable differences in the tenor pushback, body language, et cetera, between the FDA reviews for ulixacaltamide versus relutrigine? Marcio Souza: I would say no on the body language. I think we all, that is a very collegial discussion throughout the group at the agents and ourselves and amplified by the fact that we are really the only company that probably know every person in the room by name and actually have a trust and report with each one of them because there are multiple INDs and multiple NDAs under review, much, much larger, right, as you can imagine, the application ulixacaltamide hydrochloride is so much larger. So there's a lot more people involved on that. 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 venture to say that I think it's very common. As I said very peaceful and by language is incredibly positive across the board. And it reflects the collaboration throughout the review as one would expect. Operator: Our next question comes from David Hoang of Deutsche Bank. David Hoang: So I want to go back to ulixacaltamide's potential commercial launch. Could you talk a little bit about the prescriber base for the drug and remind us if this will be primarily neuros 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 ulixacaltamide. Marcio Souza: Sounds good. I'll start with the last part and I hand over to Megan. The peak sales here, I think we've been very conservative on when you look into the size of the opportunity in general, from a number of patients from not really having anything else, the growth we just mentioned that we have not been adding in general feedback from physicians. You name it, we set that for into around $10 billion for, and I would say the more we move forward, I think the more we feel comfortable that, that's really a fairly conservative number. Let me and I hand over to Megan to discuss the other topics. Megan Sniecinski: Yes, 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 sort of a call target sizing in the 13,000 to 15,000 range, that puts us in a place of having a field force around 300. 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. So, and again, the focus for the build-out will allow us to be out in the field doing the account profiling. So we're very well prepared upon PDUFA. Operator: Our next question comes from Leonid Timichev of RBC Capital Markets. Unknown Analyst: Josh on for Leo. So for the initial patient population that you'll be targeting for relutrigine, 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? Marcio Souza: Yes. I would say to call any patient with this condition on severe, it's probably something I'm never going to be able to do it. So the population is the population here, right? We are still represented into the SCN2A Family Foundation meeting last week, and we had several updates after that and discussions with them and many clinicians gave us the feedback based on how they are waiting for this. Some of these hospitals in America Centers of Excellence have like very largest, either the largest or second largest cohorts of GEs they have. While 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 for like everyone to just start right away without doing the proper assessment of these patients and making sure their background medications are optimized before getting into relutrigine. 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 parts that we're all interested 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 to the feedback we're getting from physicians and patient groups. Operator: Our next question comes from Rudy Li of Wolfe Research. Unknown Analyst: For ulixacaltamide, so what gives you confidence that titration can help improve discontinuation in practice? Like what data evidence you have to support your titration proposal? And how should we think about discontinuation rates in the real world? Marcio Souza: Yes. 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 stay the odds of staying on drug and responding if you just say a day or two more a rising and tolerability are disproportionate. So that evidence and the mathematical evidence is very, very clear, right? So imagine a study when we conducted the 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's very different conversations like this is the possible benefit 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 agents 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 from, in terms of tolerability, they can keep the patients off for a little bit longer, right? It is important because they're going to see 70% of the patients doing really well. But, and then their desire is going to turn into like, 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 in our Commercial Day coming up soon, we're going to be announcing, it is really a state-of-the-art way to help the practice manage the patients and getting 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 peak 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 percent point is not only a percent point in revenue, it's a lot more patients that are being able to get benefits that they cannot get any other way. Operator: Our next question comes from Ben Burnett of Wells Fargo. Unknown Analyst: This is Orfia for Ben. Congrats on over enrolling EMERALD. I had one question on relutrigine and one on your cash runway. First on relutrigine, are you able to share what proportion of EMERALD patients are on XCOPRI or another sodium blocker at baseline? And what are your expectations for incremental efficacy in patients who are already on? And then second, on your cash runway, given that both relutrigine and elsunersen are eligible for pediatric vouchers, are your current plans to monetize those on approval? And is that contemplated in your cash runway? Marcio Souza: Yes. So I think we've got a very representative distribution of what the background meds are here. Very happy. I'll tell you, discontinuation, for example, it's a good surrogate there being extremely low on this study. tolerability being very good. We know and unfortunately, like 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 effect, 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's been anxiously waiting for a financial question for the call. Tim Kelly: Yes. 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, but 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 relutrigine for SCN2A and SCN8A, where we do have orphan designation and are eligible for 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 elsunersen 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. Operator: This concludes the question-and-answer session. I would now like to turn it back to Marcio for closing remarks. Marcio Souza: Yes. Thank you so much. I appreciate, I hope we try to be very comprehensive today, given 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 of 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 this drug that we're going to discuss less and less, the clinical 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 get into certain conversations. I appreciate every feedback being given to the company made us to where we are right now. I couldn't be prouder on behalf of patients. when we got these stories every single day, and trust me, we got them every single day from the patients who transition on ER to the open label or the ones who are on BOLT or the ones that are on an emergency access of one of our medicines or particularly these days for the ones wanting to be on ulixacaltamide. 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. 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Our analysts just identified a stock with the potential to be the next Nvidia. Tell us how you invest and we'll show you why it's our #1 pick. Tap here. Management characterizes the company's transition from clinical to commercial as fully underway, with field forces hired and distribution networks established for two upcoming launches. The FDA's mid-cycle review for ulixacaltamide in essential tremor (ET) concluded with no efficacy-related issues identified and no plans for an advisory committee meeting. A comprehensive BIMO inspection of Praxis as a sponsor for both lead programs resulted in no Form 483 findings, validating data integrity and clinical operations across decentralized and complex study designs. Relutrigine's review period was extended to December 27, 2026, following a major amendment for additional sensitivity analysis, though management reports launch preparations remain ahead of the revised calendar. The EMERALD study for relutrigine exceeded enrollment targets with approximately 200 patients across 50 genetic etiologies, positioning it as the foundation for a supplemental NDA in 2027. Vormatrigine's Phase 2 miss in focal onset seizures is attributed to suboptimal dosing and trial design rather than drug failure, leading to a planned revamp of POWER2 and POWER3 studies by Q4 2026. Strategic focus is shifting toward life cycle opportunities for T-type calcium channel inhibitors, including a new collaboration with Remagine Labs to extend ulixacaltamide's value beyond the initial launch. Management expects PDUFA action dates in late December 2026 for relutrigine and January 2027 for ulixacaltamide, with potential for earlier action given the completion of mid-cycle reviews. Commercial strategy for ulixacaltamide assumes a target price range of $50,000 to $100,000 per year, supported by payer feedback acknowledging the high unmet need in the 7 million-patient ET market. The company anticipates G&A expenses will increase in the second half of 2026 to fund two distinct commercial field teams and disease awareness campaigns. Guidance for ulixacaltamide's launch assumes initial step edits through propranolol for some payers, though approximately half of the market is medically ineligible for beta blockers, creating a clear entry segment. Cash runway is projected to extend into 2028, supported by a $1.4 billion balance and the potential monetization of two eligible Pediatric Review Vouchers upon approval. The FDA designated the submission of additional sensitivity data for relutrigine as a 'major amendment,' triggering a three-month PDUFA extension. Management explicitly stated they will maintain 'regulatory silence' until the final action dates to maintain discipline during late-stage labeling and promotion discussions. Vormatrigine's POWER1 study failed its primary endpoint of monthly focal seizure reduction, though it met a secondary 50% responder rate endpoint, which management is using to justify the program's redesign. Ulixacaltamide's commercial success depends on managing a 20% rate of early tolerability issues through a 'hub of the future' patient support program and potential titration labeling. One stock. Nvidia-level potential. 30M+ investors trust Moby to find it first. Get the pick. Tap here. Management confirmed that titration regimens were discussed with the FDA to address the 20% of patients who experience early tolerability issues. The commercial strategy involves a 'hub of the future' to manage the first prescription and titration phase, ensuring patients stay on drug long enough to see rapid efficacy onset. The study includes a diverse mix of 50 genetic etiologies, with management asserting that sodium channel modulation remains a valid downstream target even for non-sodium channel mutations. The high baseline seizure burden (median of 50 per 28 days) makes the study well-powered to show statistical significance even with a heterogeneous population. Payer interactions suggest a willingness to provide access given the lack of approved alternatives, with management sticking to a $50,000-$100,000 annual price range. The launch will initially target 13,000 to 15,000 high-volume neurologists using a field force of approximately 300 representatives.

Interested in Praxis Precision Medicines, Inc.? Here are five stocks we like better. FDA reviews are progressing for ulixacaltamide and relutrigine, with no advisory committee meetings planned and no findings from a BIMO inspection. Expected FDA decisions are due by Dec. 27, 2026, for relutrigine and January 2027 for ulixacaltamide. Praxis is preparing for potential commercial launches by hiring sales teams, establishing distribution and patient-support infrastructure, and building inventory. The company has discussed potential annual pricing of approximately $50,000 to $100,000 for ulixacaltamide, although no final price has been set. Second-quarter operating expenses rose to $96.9 million, while Praxis ended the quarter with $1.4 billion in cash, cash equivalents and marketable securities—enough, management said, to fund operations into 2028. Pipeline updates included above-target EMERALD enrollment, anticipated elsunersen data next year and redesigned studies for vormatrigine after a primary-endpoint miss. Praxis Precision Medicines (NASDAQ:PRAX) said it is preparing for potential commercial launches of ulixacaltamide for essential tremor and relutrigine for SCN2A- and SCN8A-related developmental and epileptic encephalopathies, or DEEs, as both new drug applications remain under FDA review. Chief Executive Officer Marcio Souza said the company has commercial leadership in place, has hired and trained a field force for its first launch, established distribution infrastructure and begun building inventory. Praxis expects FDA decisions on relutrigine by Dec. 27, 2026, and on ulixacaltamide by January 2027. → Meta’s Earnings Drop Shows Wall Street Wants More Than Ad Growth For ulixacaltamide, Praxis said the FDA completed mid-cycle review communications and identified no significant efficacy-related issues. The agency also said it does not plan to convene an advisory committee meeting for the application, according to Souza. Ulixacaltamide is being developed for essential tremor, a condition that Praxis said affects more than 7 million Americans. Souza said there is currently no FDA-approved therapy developed specifically for essential tremor. → 4 Oil and Gas ETF Plays as Prices Stay Sky-High Praxis also reported that the FDA does not intend to hold an advisory committee meeting for relutrigine. The FDA extended relutrigine’s review period after Praxis submitted additional sensitivity analyses of existing clinical data, which the agency classified as a major amendment. The revised action date is Dec. 27. Relutrigine is under review for SCN2A and SCN8A DEEs, severe epilepsy conditions that can begin in infancy and are associated with developmental delays. Souza said the addressable U.S. population is roughly 10,000 patients. If approved, the drug would be the first therapy for those indications and would qualify for a pediatric review voucher, according to the company. → Ulta's Growth Is Real, But So Are the Risks During the quarter, the FDA conducted a Bioresearch Monitoring, or BIMO, inspection related to both applications. Souza said the inspection covered corporate and clinical operations, safety reporting, data integrity, statistical analysis and interim analyses, among other areas. The inspection concluded without findings and no Form 483 was issued. Praxis said it does not intend to provide additional regulatory updates on either application before the expected FDA action dates. Chief Operating Officer Megan Sniecinski said the commercial and medical teams supporting relutrigine are fully hired, while the ulixacaltamide field-force buildout is underway. For ulixacaltamide, Praxis expects to target neurologists initially, with a call target of approximately 13,000 to 15,000 physicians and a field force of about 300 representatives. Sniecinski said the company is conducting account profiling ahead of potential launches and is building patient-support capabilities, including an integrated prescription and specialty-pharmacy support system. In discussions with analysts, management said it expects physicians to focus on appropriate patient assessment and background-medication optimization when using relutrigine. For ulixacaltamide, management said launch preparations are intended to support patient persistence and help patients manage early tolerability issues. Souza said Praxis’ planning assumptions include potential payer step-edit requirements through propranolol for ulixacaltamide, though he said some patients cannot use beta blockers because of other medical conditions. He added that Praxis has discussed an annual pricing range of roughly $50,000 to $100,000, while noting that the company has not disclosed a final price. Praxis said enrollment in its EMERALD study of relutrigine in broader DEEs exceeded its target, with approximately 200 patients enrolled across more than 50 genetically defined etiologies as well as non-genetically defined conditions. The company said that, if the study is positive and relutrigine receives initial approval in SCN2A and SCN8A DEEs, EMERALD could support a supplemental NDA in 2027. Management said it expects top-line results from EMBRAVE3, a study of elsunersen, next year. In June, the FDA granted breakthrough therapy designation to elsunersen for seizures associated with SCN2A DEE caused by gain-of-function variants, based on EMBRAVE Part A results. Praxis also discussed its vormatrigine program following top-line results from the POWER1 study in focal-onset seizures. POWER1 did not meet its primary endpoint of reducing monthly focal seizure frequency from baseline through week 12. However, the study met a secondary endpoint showing a significantly greater proportion of patients on vormatrigine achieved at least a 50% reduction in seizure frequency. Souza said the company believes dose, duration at dose, enrollment criteria and other design factors contributed to the primary-endpoint miss. Praxis is finalizing amendments to the POWER2 and POWER3 studies and intends to have both trials operating by the fourth quarter of 2026. Chief Financial Officer Tim Kelly said second-quarter operating expenses totaled $96.9 million, including $69.4 million in research and development expenses and $27.5 million in general and administrative expenses. That compared with total operating expenses of $76 million in the second quarter of 2025. Operating cash use was $78 million during the quarter, compared with $55 million a year earlier, reflecting increased spending in research and development and general and administrative activities. Kelly said general and administrative spending is expected to increase in the second half as Praxis adds commercial field personnel, expands disease-awareness efforts, builds inventory and develops business infrastructure. Praxis ended the quarter with $1.4 billion in cash, cash equivalents and marketable securities, up from $926 million at Dec. 31, 2025. Kelly said the company expects its current capital to support operations into 2028. Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on discovering and developing precision therapies for disorders driven by neuronal excitability. The company applies translational neuroscience and genetic insights to design small molecule drugs that target specific ion channels and receptor subtypes implicated in neurological and psychiatric conditions. Its research aims to address unmet needs in rare epilepsies, essential tremor, treatment-resistant depression and other central nervous system (CNS) disorders. The company's pipeline includes several lead candidates at various stages of development. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. The article "Praxis Precision Medicines Q2 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for August 2026.

This article first appeared on GuruFocus. Operating Expenses: $96.9 million in Q2 2026, compared to $76 million in Q2 2025. R&D Expenses: $69.4 million in Q2 2026. G&A Expenses: $27.5 million in Q2 2026. Operating Cash Spend: $78 million in Q2 2026, compared to $55 million in Q2 2025. Cash Position: $1.4 billion in cash equivalents and marketable securities as of Q2 2026, up from $926 million as of December 31, 2025. Warning! GuruFocus has detected 3 Warning Sign with PRAX. Is PRAX fairly valued? Test your thesis with our free DCF calculator. Release Date: August 06, 2026 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Praxis Precision Medicines Inc (NASDAQ:PRAX) received positive feedback from the FDA during mid-cycle reviews for both ulixacaltamide and relutrigine, with no efficacy-related significant issues identified and no Advisory Committee meetings planned for either drug. The company successfully completed comprehensive FDA BIMO inspections for both ulixacaltamide and relutrigine applications without any findings or Form 483 issued, validating data integrity and operational procedures. Praxis Precision Medicines Inc (NASDAQ:PRAX) is well-capitalized with $1.4 billion in cash, providing a runway into 2028 and enabling full commercial launch preparation for both upcoming product approvals. The company has made significant commercial progress, including hiring and training a field force for the relutrigine launch and building a comprehensive patient support program and distribution network for both products. Praxis Precision Medicines Inc (NASDAQ:PRAX) received a third Breakthrough Therapy Designation for elsunersen, highlighting the strength of its pipeline across multiple platforms and assets. The EMERALD study for relutrigine exceeded its enrollment target with approximately 200 patients, including over 50 distinct genetic etiologies, positioning the company for a potential supplemental NDA filing in 2027. Management expressed confidence in the vormatrigine program, attributing the POWER1 miss to fixable design and dosing issues, with plans to amend and restart POWER2 and POWER3 by Q4 2026. Praxis Precision Medicines Inc (NASDAQ:PRAX) reported a significant increase in operating expenses, with Q2 2026 operating expenses reaching $96.9 million compared to $76 million in the same period of 2025. The company's vormatrigine program failed to meet its primary endpoint in the POWER1 study for focal onset seizures, representing a major clinical setback for that asset. The FDA deemed the additional sensitivity analysis submitted for relutrigine a major amendment, extending the review period and pushing the new PDUFA date to December 2027. Praxis Precision Medicines Inc (NASDAQ:PRAX) faces potential tolerability issues with ulixacaltamide, as approximately 30% of patients may experience initial side effects that could impact patient persistence and commercial uptake. The company has chosen to cease providing regulatory updates until the expected action dates, creating a period of uncertainty for investors regarding the approval timelines for both ulixacaltamide and relutrigine. Management acknowledged that the vormatrigine program represents only a small fraction of the company's external value, indicating limited near-term upside from this asset. The company's cash burn increased significantly, with operating cash spend of $78 million in Q2 2026 compared to $55 million in Q2 2025, reflecting higher costs associated with launch preparations. Q: Given the successful mid-cycle review and inspections, what is the phenotype and size of the sales force being hired for the relutrigine and ulixacaltamide launches, and how are you preparing for the early 2027 launch?A: Marcio Souza (CEO) and Megan Sniecinski (COO) stated that the company is being very selective in hiring, attracting top-caliber talent with multiple launch experiences in the rare neuro space. The relutrigine team is fully hired and trained, allowing for active account profiling. The ulixacaltamide field force build-out is well underway and on track. The focus is on ensuring a high-quality first experience to build physician confidence and ensure durable patient persistence. Q: Can you elaborate on the mid-cycle review meeting for ulixacaltamide? Were there any surprises, and did you discuss the potential inclusion of alternate titration regimens to improve the patient experience?A: Marcio Souza (CEO) described the meeting as "peaceful" and "forward-looking," with no efficacy-related significant issues identified and no Advisory Committee planned. The discussion focused on proper use, labeling, and patient access. The topic of titration did come up, and the CEO noted a strong alignment with the FDA on managing the ~30% of patients who experience initial tolerability issues, emphasizing that staying on the drug leads to significant efficacy. The company is building a "hub of the future" to support patients through the early titration weeks. Q: What are the specific learnings from the POWER1 trial for vormatrigine that give you confidence to amend and restart the POWER2 and POWER3 studies?A: Marcio Souza (CEO) explained that the primary endpoint miss was a design problem, not a drug problem. The key secondary endpoint (?50% reduction in seizure frequency) showed the drug is active. The company identified that the dose, study duration (6 weeks was too short), and entry criteria (high number of prior medication failures) were not optimally matched. These are all fixable design elements, and the company plans to have both studies up and running by Q4 2026. Q: Regarding the EMERALD study in broad DEEs, what is the powering and what are your expectations for the placebo response given the diverse patient population?A: Marcio Souza (CEO) and Steve Petrou (President of R&D) noted that the trial enrolled ~200 patients across more than 50 distinct genetic etiologies. The median baseline countable seizures is over 50 per 28 days, making a natural regression to the mean very unlikely. The placebo response rates for 50%, 75%, and 90% reductions are expected to be very small. The company is confident in the a priori powering and the mix of patients who are pharmacosensitive to the sodium channel mechanism. Q: How should we think about the potential efficacy of relutrigine in the broader EMERALD population compared to the SCN2A and SCN8A populations, and what is the bar for success?A: Marcio Souza (CEO) stated that the bar for success is statistical significance, but the company aims much higher. Biologically, sodium channels are the most downstream element in the etiology of DEEs, making them a target regardless of the upstream genetic cause. Steve Petrou (President of R&D) added that even in loss-of-function mutations, excitability issues remain, and sodium channel modulation at the axon initial segment is key to controlling neuronal output. Q: What is the expected patient mix for the ulixacaltamide launch across commercial, Medicare, and Medicaid, and where will the initial patients come from?A: Marcio Souza (CEO) stated that the addressable population at launch is ~2 million patients, with about three-quarters being on Medicare/Medicaid (likely older patients). The company expects a migration of patients aged 40-65 and notes the organic growth of the ET market, which doubles every 10 years after age 60. The launch will likely have several phases, with a focus on patients who cannot take beta-blockers (about half the market) and those who have failed other treatments. Q: Can you provide details on the payer work done for ulixacaltamide, including pricing assumptions and the potential for step-edit requirements?A: Marcio Souza (CEO) stated that payer interactions have been positive, with payers understanding the high unmet need. The company's planning assumptions include step-edits through propranolol, but they note that ~50% of the market cannot take beta-blockers. The pricing range of $50,000-$100,000 per year is well-grounded based on payer feedback. The company believes the drug's efficacy on top of propranolol will support broad access. Q: What is the size of the sales force needed to support the ulixacaltamide launch, and what are the peak sales assumptions?A: Megan Sniecinski (COO) stated that the target call base is 13,000-15,000 neurologists, requiring a field force of around 300 representatives. Marcio Souza (CEO) reiterated the peak sales estimate of ~$10 billion, which he believes is conservative given the size of the opportunity, the lack of approved treatments, and the organic market growth. Q: What gives you confidence that a titration regimen can improve discontinuation rates for ulixacaltamide in real-world practice?A: Marcio Souza (CEO) explained that data shows if patients stay on the drug a little longer after initial tolerability issues, the odds of responding and staying on therapy are disproportionate. The company's label proposal includes instructions for physicians to manage patients through the initial weeks. The "hub of the future" patient support program is designed to provide tools to maximize tolerability and persistence, ensuring more patients can benefit from the drug. Q: Are there any noticeable differences in the tenor or pushback between the FDA reviews for ulixacaltamide and relutrigine?A: Marcio Souza (CEO) stated there were no differences in body language or tenor; both meetings were collegial and positive. The ulixacaltamide application is larger with more people involved, but the company has a strong rapport with the FDA across multiple programs. The CEO described the discussions as "peaceful" and reflecting the collaborative nature of the review process. For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Mid-cycle meeting completed for ulixacaltamide HCl; FDA identified no major safety or efficacy concerns to date and does not plan to request an advisory committee meeting; PDUFA date of January 29, 2027 Mid-cycle meeting completed for relutrigine; FDA identified no major safety or efficacy concerns to date and does not plan to request an advisory committee meeting; PDUFA date of December 27, 2026 FDA Bioresearch Monitoring (BIMO) inspections of Praxis for both ulixacaltamide and relutrigine completed with no Form FDA 483 observations Commercial infrastructure build-out accelerating for ulixacaltamide HCl in Essential Tremor and relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) ahead of expected approvals Praxis received its third Breakthrough Therapy Designation following the positive results from the EMBRAVE Part A trial of elsunersen for the treatment of seizures associated with SCN2A DEE caused by gain of function variants Cash and investments of approximately $1.4 billion as of June 30, 2026 maintains runway into 2028 Conference call today, August 6, 2026, at 8:30am BOSTON, Aug. 06, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today provided a corporate update and reported financial results for the second quarter of 2026. “This quarter reflected meaningful progress toward becoming a multi-product commercial company, with both ulixacaltamide and relutrigine advancing closer to patients through ongoing constructive dialogue with the FDA with positive mid-cycle meetings and BIMO sponsor inspections completed for both programs. We have also taken significant steps in launch preparations: our commercial leadership is in place, we are standing up our state-of-the-art commercial systems, and the momentum is tangible with field force hiring well under way,” said Marcio Souza, president and chief executive officer. “Across the rest of our portfolio, the Breakthrough Therapy Designation for elsunersen, our third in twelve months, further validates our position as a leader in CNS disease, our Solidus™ ASO platform for treating devastating CNS disorders and the depth of our CNS therapeutic development engine. Lastly, we’re excited to be continuing the POWER2 study with renewed conviction, informed by what we learned in POWER1, as we work to transform care for patients with focal epilepsy with vormatrigine.” Recent Highlights and Anticipated MilestonesCerebrum™ for Small Molecules Ulixacaltamide for Essential Tremor (ET): ET is one of the most common movement disorders, affecting approximately seven million patients in the U.S. Ulixacaltamide is the first and only investigational therapy to demonstrate positive results in a Phase 3 program in ET and was granted Breakthrough Therapy Designation by the FDA in December 2025. NDA review is progressing as expected, with a PDUFA date of January 29, 2027. The mid-cycle meeting was successfully completed and the FDA identified no major safety or efficacy concerns to date. The agency confirmed that no advisory committee meeting is planned. Launch preparation activities are accelerating, with commercial organization leaders hired and infrastructure, marketing and disease-education programs, medical information capabilities, physician targeting and an established distribution network with commercial inventory build in progress. A strategic collaboration was initiated in July 2026 with Remagine Labs to develop a transdermal patch of ulixacaltamide for ET. The transdermal formulation is designed to broaden ulixacaltamide’s addressable patient population, strengthen its competitive positioning, and support the long-term growth, durability, and value of the ulixacaltamide franchise. Relutrigine for DEEs: Relutrigine is a sodium channel modulator designed to precisely target the hyperexcitable state of sodium-channels, with therapeutic potential across developmental epilepsies. Relutrigine has been granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA. If approved, relutrigine will be the first therapy for SCN2A and SCN8A DEEs and will be eligible for a Pediatric Review Voucher. Following the submission of additional sensitivity analyses of existing clinical data, which the FDA deemed collectively to be a major amendment, the FDA extended the review period for relutrigine’s NDA for the treatment of SCN2A and SCN8A DEEs and set a new PDUFA target action date of December 27, 2026. The mid-cycle meeting was successfully completed and the FDA has identified no major safety or efficacy concerns to date. The agency confirmed that no advisory committee meeting is planned. Preparations for the commercial launch of relutrigine are gaining momentum, with commercial and medical teams hired, building sufficient inventory, establishing a comprehensive patient support program and engaging with payers to ensure timely market access upon potential approval. Enrollment in the EMERALD study in broad DEEs exceeded the planned target with approximately 200 patients enrolled, spanning over 50 distinct genetically defined pathological etiologies in the trial population. Topline results are anticipated in the fourth quarter of 2026 and assuming successful initial NDA approval of relutrigine, the EMERALD study, if positive, would serve as the basis for a supplemental NDA submission in 2027. NDA-related activities and updates for Ulixacaltamide HCl and Relutrigine Praxis operations were subject to inspections by the FDA in accordance with the BIMO program related to its NDAs for ulixacaltamide HCl for Essential Tremor and relutrigine for SCN2A and SCN8A DEEs. The scope of the inspections was comprehensive, spanning overall quality and clinical operations, data integrity, including statistical and interim analyses, and safety, amongst other standard areas in the BIMO program. The inspection was completed successfully, and no form 483 was issued. Acknowledging the late-stage discussions with the FDA in relation to both applications, Praxis does not intend to communicate regulatory updates going forward until the expected PDUFA dates. Vormatrigine for Focal Onset Seizures (FOS) and Generalized Epilepsy: An estimated 3.5 million people in the U.S. suffer from common epilepsies. Sodium channel therapy is the cornerstone of treatment for patients with epilepsy, yet currently approved drugs have significant safety and efficacy limitations. Vormatrigine is the most potent sodium-channel modulator ever developed for epilepsy and is designed to precisely target the hyperexcitable state of sodium-channels in adult common epilepsies. In June, Praxis announced topline results from the POWER1 Phase 2/3 study in highly refractory patients with FOS (link). Praxis is finalizing the plans to restart the POWER2 study and initiate the POWER3 study in the fourth quarter of 2026 based on the learnings from POWER1. Solidus™ for Antisense Oligonucleotides (ASO) Elsunersen for early-seizure-onset SCN2A DEE: Early-onset SCN2A-DEE is a rare, genetic epilepsy characterized by early-onset seizures and severe impact on development. Praxis remains on track to nominate development candidates for several early-stage ASO therapeutic initiatives in 2026. Second Quarter 2026 Financial Results:As of June 30, 2026, Praxis had $1.4 billion in cash, cash equivalents and marketable securities, compared to $926.1 million in cash, cash equivalents and marketable securities as of December 31, 2025. This increase of $447.8 million was primarily attributable to net proceeds from Praxis’ January 2026 follow-on public offering and interest income on marketable securities, partially offset by cash used in operations. The Company’s cash, cash equivalents and marketable securities as of June 30, 2026 are expected to fund operations into 2028. Research and development expenses were $69.4 million for the second quarter of 2026, compared to $63.0 million for the second quarter of 2025. The increase in research and development expenses of $6.4 million was primarily attributable to $6.7 million in increased expenses related to Solidus™, $4.4 million in increased personnel-related costs and $1.8 million in increased indirect costs, partially offset by $6.5 million in decreased expenses related to Cerebrum™. General and administrative expenses were $27.5 million for the second quarter of 2026, compared to $13.1 million for the second quarter of 2025. The increase in general and administrative expenses of $14.4 million was primarily attributable to $7.2 million in increased professional expenses and $6.3 million in increased personnel-related costs. Praxis incurred a net loss of $83.7 million for the second quarter of 2026, including $11.3 million of stock-based compensation expense, compared to $71.1 million for the second quarter of 2025, including $7.8 million of stock-based compensation expense. As of June 30, 2026, Praxis had 27.9 million shares of common stock outstanding. Conference CallPraxis will discuss second quarter 2026 financial results and business highlights on a conference call taking place today, August 6 at 8:30 am ET, which can be accessed by dialing (800) 715-9871 with passcode 7796789 or by registering for the webcast, here. The live audio webcast will also be available through the Events & Presentations page of the Investors + Media section of the Company’s website. About UlixacaltamideUlixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide has received Breakthrough Therapy Designation from the FDA and is the most advanced program of Praxis’ Cerebrum™ small molecules. About RelutrigineRelutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs). Relutrigine is a functional state-selective sodium channel (NaV) modulator that preferentially modulates NaV channels under the conditions associated with pathological neuronal hyperexcitability, including sustained depolarization, repetitive firing and increased persistent current where present. By preferentially targeting pathological NaV channel activity while sparing normal NaV function, relutrigine is designed to provide broad efficacy across DEEs with differing etiologies and improved tolerability relative to traditional sodium channel blockers. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMERALD study, please visit Emerald | Resilience Studies. About Vormatrigine Vormatrigine is a next-generation small-molecule sodium channel modulator currently being developed as a once-daily oral treatment for adults with focal-onset seizures and generalized epilepsy. Vormatrigine is designed to preferentially inhibit the pathologically increased neuronal firing that underlies seizures while relatively preserving normal physiological activity. Preclinical data demonstrate differentiation from current standards of care and support its potential to be a best-in-class treatment for focal epilepsy. In vitro, vormatrigine has demonstrated preferential modulation of NaV channels under the sustained depolarization and repetitive-firing conditions associated with seizure activity. In vivo studies of vormatrigine have demonstrated unprecedented potency in the maximal electroshock seizure (MES) model, a highly predictive translational model for efficacy in focal epilepsy. Data from patients in the RADIANT study demonstrated a robust seizure reduction and generally well tolerated profile. To learn more about the POWER2 study, please visit POWER studies. About ElsunersenElsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models. Elsunersen has received BTD, ODD and RPDD from the FDA, and ODD and PRIME designations from the European Medicines Agency for the treatment of SCN2A-DEE. The elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc. To learn more about the EMBRAVE3 study, please visit Embrave | Resilience Studies. About PraxisPraxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders for small molecules through Cerebrum™, and for antisense oligonucleotides (ASOs) through Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four late-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, LinkedIn and X/Twitter. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the potential market opportunity and commercial potential of Praxis’ product candidates, the anticipated timing of regulatory submissions and interactions, the anticipated timing of clinical trials, the development of Praxis’ product candidates and plans to initiate new clinical programs, and our projected cash runway, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2025 and other filings made with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise. CONTACT: Investor Contact: Praxis Precision Medicines [email protected] 857-702-9452 Media Contact: Dan Ferry LifeSci Advisors [email protected] 617-430-7576

Track your investments for FREE with Simply Wall St, the portfolio command center trusted by over 7 million individual investors worldwide. Praxis Precision Medicines (PRAX) is back in focus after mixed topline results from its Phase 2/3 POWER1 trial of vormatrigine in focal onset seizures, prompting a pause in the related POWER2 study. The trial did not meet its primary goal on seizure frequency, but hit a key 50% responder rate and showed stronger seizure reduction at the higher 30 mg dose, while maintaining a generally favorable safety profile. See our latest analysis for Praxis Precision Medicines. After a sharp reaction to the POWER1 update, the stock’s 1-day share price return of 7.5% and 7-day gain of 4.95% sit against a 30-day share price decline of 22.18% and a very large 1-year total shareholder return. This indicates strong longer term momentum while near term sentiment has cooled. If you are looking beyond a single CNS biotech story, this is a good moment to broaden your watchlist with 40 healthcare AI stocks With Praxis still loss making, carrying a US$7.43b market cap and trading at a steep intrinsic discount, investors now have to ask whether the recent pullback leaves upside on the table or whether the stock already reflects future growth. Praxis Precision Medicines' most followed narrative anchors on a fair value of $449.13, which sits well above the last close of $266.58 and frames the current discount. Read the complete narrative. Curious how that potential portfolio effect translates into numbers, timelines and margins? The narrative focuses on rapid revenue expansion, a sharp swing into profitability and a rich future earnings multiple. The tension between those assumptions and today’s loss making status is what drives the fair value gap. The narrative also sets out detailed expectations for revenue growth, profit margins and earnings by 2029, along with a specific P/E level that would need to hold in the long run to justify that fair value. These moving parts are all discounted back using a 7% rate, which is the same rate used in the Simply Wall St company report, to arrive at a fair value well above the current share price. Result: Fair Value of $449.13 (UNDERVALUED) Have a read of the narrative in full and understand what's behind the forecasts. However, this bullish story still hinges on clean late stage epilepsy readouts, as well as on clinicians actually switching patients from existing multi drug regimens to vormatrigine. Find out about the key risks to this Praxis Precision Medicines narrative. The first narrative leans on future earnings and price targets, but the SWS DCF model presents a different perspective, with PRAX at $266.58 versus an estimated future cash flow value of $2,918.05, which appears heavily undervalued. The gap is large, so which story do you trust more? Look into how the SWS DCF model arrives at its fair value. With sentiment clearly split between risk and reward, this is a moment to look at the data yourself and move quickly to form an independent view using the 2 key rewards and 2 important warning signs. If you stop at a single stock, you miss the bigger picture. Let the screener surface fresh ideas before the next wave of opportunities moves without you. Spot potential mispricing early by scanning 44 high quality undervalued stocks that pair quality fundamentals with attractive entry points. Prioritize sleep at night by reviewing 71 resilient stocks with low risk scores that score well on resilience and business stability. Get ahead of the crowd by checking the screener containing 20 high quality undiscovered gems before they sit on everyone else's radar. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include PRAX. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]

On May 15, 2026, Exome Asset Management reported selling out of Wave Life Sciences (NASDAQ:WVE), liquidating 421,488 shares in an estimated $5.49 million trade based on quarterly average pricing. According to an SEC filing dated May 15, 2026, Exome Asset Management sold its entire stake of 421,488 shares in Wave Life Sciences. The estimated value of the transaction is $5.49 million, calculated using the average closing price during the first quarter of 2026. The quarter-end value of Exome’s position in the company dropped by $7.17 million, a figure that includes both trading activity and price fluctuations. Exome Asset Management LLC fully exited its Wave Life Sciences position, which was previously 3.4% of the fund’s AUM. Top holdings after the filing: As of May 14, 2026, shares of Wave Life Sciences were priced at $6.90, up 8% over the past year, underperforming the S&P 500 by nearly 20 percentage points. Wave Life Sciences develops stereopure oligonucleotide therapies targeting neurological, hepatic, and genetic disorders, with clinical candidates including WVE-004 (ALS/FTD), WVE-003 (Huntington's disease), and WVE-N531 (Duchenne muscular dystrophy). The firm operates a clinical-stage biotechnology business model focused on proprietary drug discovery and development, leveraging its PRISM platform and strategic collaborations to advance a pipeline of RNA-targeted medicines. It serves pharmaceutical partners, research institutions, and patients with rare and serious genetic diseases, primarily in neurology and hepatology. Wave Life Sciences is a clinical-stage biotechnology company specializing in the design and development of stereopure oligonucleotide therapeutics. It leverages its proprietary PRISM platform and strategic partnerships to advance a diversified pipeline targeting neurological and hepatic indications. The company’s focus on precision genetic medicines positions it to address unmet medical needs in rare and complex diseases, supported by collaborations with leading global pharmaceutical and academic partners. The performance of early-stage biotechs is highly contingent on clinical execution, meaning investment outcomes can hinge on a handful of data releases and regulatory decisions rather than steady operating performance. That’s notable here because Wave shares took a massive tumble in late March, collapsing roughly 50% in one day after new data showed that a higher dose of its obesity candidate, WVE-007, failed to show meaningful improvement in reducing a type of belly fat.Nevertheless, the firm seemed optimistic in its latest earnings release. CEO Paul Bolno said the company is "accelerating" development of WVE-007 following encouraging early body composition data and remains on track across several pipeline programs. Financially, Wave generated $38.2 million in first-quarter revenue, up from $9.2 million a year earlier, while narrowing its net loss to $26.1 million from $46.9 million. It ended March with $544.6 million in cash and expects that funding to last into the third quarter of 2028.Ultimately, this remains a pipeline story, and Exome's exit may reflect risk management, but the next meaningful driver of returns will likely be clinical and regulatory execution rather than institutional trading activity. Before you buy stock in Wave Life Sciences, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Wave Life Sciences wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $463,900!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,294,401!* Now, it’s worth noting Stock Advisor’s total average return is 978% — a market-crushing outperformance compared to 211% for the S&P 500. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built by individual investors for individual investors. See the 10 stocks » *Stock Advisor returns as of May 31, 2026. Jonathan Ponciano has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Guardant Health and Ionis Pharmaceuticals. The Motley Fool has a disclosure policy. This $5.5 Million Exit Raises Questions After Wave Life Sciences' Rough First Quarter was originally published by The Motley Fool

On May 15, 2026, Cormorant Asset Management disclosed a buy of 313,645 shares of Alumis (NASDAQ:ALMS), with the estimated transaction value at $7.84 million based on quarterly average pricing. According to a May 15, 2026 SEC filing, Cormorant Asset Management increased its position in Alumis by 313,645 shares during the first quarter. The firm’s estimated trade size was $7.84 million, calculated using the quarter’s average closing price. The stake’s value at quarter-end rose by $51.52 million, a figure that incorporates both buying activity and market price movements. Cormorant’s buy brings its Alumis stake to 4.37% of 13F assets as of March 31, 2026. Top holdings after the filing: NASDAQ:PRAX: $285.30 million (14.4% of AUM) NASDAQ:BBOT: $160.01 million (8.1% of AUM) NASDAQ:EYPT: $106.54 million (5.4% of AUM) NASDAQ:EWTX: $102.69 million (5.2% of AUM) NASDAQ:ERAS: $93.84 million (4.7% of AUM) As of May 14, 2026, Alumis shares were priced at $24.63, up about 400% over the past year and vastly outperforming the S&P 500’s roughly 25% gain in the same period. Alumis develops clinical-stage biopharmaceutical products targeting autoimmune and neuroinflammatory diseases, including ESK-001 and A-005, with a focus on allosteric TYK2 inhibitors. The company operates a research-driven model, advancing proprietary drug candidates through clinical trials. It targets healthcare providers and pharmaceutical partners addressing autoimmune and neurodegenerative conditions, with a primary focus on the biotechnology and healthcare sectors. Alumis is a clinical-stage biotechnology company specializing in the development of novel therapies for autoimmune and neuroinflammatory disorders. The company leverages its expertise in allosteric TYK2 inhibition to advance a pipeline of differentiated drug candidates. With a research-centric strategy and a focus on high unmet medical needs, Alumis aims to establish a competitive edge in the biopharmaceutical landscape. This is one of several buys Cormorant made last quarter into high-flying biotechs. With shares up roughly 400% over the past year, the fund added even more exposure, signaling confidence that the company’s late-stage autoimmune pipeline could continue driving upside from here.A lot of that optimism centers around envudeucitinib, Alumis’ oral TYK2 inhibitor for plaque psoriasis and lupus. Just last week, the company reported strong Phase 3 psoriasis data showing PASI 90 response rates above 60% and PASI 100 rates ofo about 40% by Week 24, results management said support the drug’s potential as a leading oral psoriasis therapy. Alumis also said it remains on track to submit an NDA in the fourth quarter of 2026, while potentially pivotal lupus data are expected in the third quarter.The balance sheet also gives the company flexibility. Alumis ended March with about $569.5 million in cash, cash equivalents, and marketable securities, which management says should fund operations into late 2027.As with its other buys (in Dianthus and Erasca), the opportunity is clear, but so is the risk. Expectations are now extremely high after the stock’s massive run, meaning future clinical data will matter far more than hype alone. Before you buy stock in Alumis, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Alumis wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $469,293!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,381,332!* Now, it’s worth noting Stock Advisor’s total average return is 993% — a market-crushing outperformance compared to 207% for the S&P 500. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built by individual investors for individual investors. See the 10 stocks » *Stock Advisor returns as of May 17, 2026. Jonathan Ponciano has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Alumis Stock Has Soared 400%. Cormorant Bought Another $8 Million Last Quarter was originally published by The Motley Fool

Image source: The Motley Fool. Thursday, May 7, 2026 at 8:30 a.m. ET Chief Executive Officer — Marcio Souza Chief Financial Officer — Tim Kelly Need a quote from a Motley Fool analyst? Email [email protected] Marcio Souza: Thank you, Dan, and good morning, everyone, and thanks for joining Praxis' First Quarter 2026 Conference Call. Building on a remarkable 2025, we have continued executing across our portfolio in our journey to become a commercial company with strong momentum building across 4 late-stage assets, representing more than $20 billion in peak sales potential. With the NDAs for ulixacaltamide and relutrigine accepted by the FDA and PDUFA date set, we're ramping up commercial efforts to support the 2 potential U.S. launches within the next 8 months while also making significant progress with our other clinical programs. It's also incredibly exciting to announce that we have completed recruitment for the EMBOLD study in the broad DEE population, with top-line results expected in the fourth quarter of this year, which we expect to support a potential supplemental NDA next year. We're also on track to report results from our POWER1 study for lamotrigine later this quarter. Also made exciting progress with our solids ASO platform with the positive results from the EMBRAVE Part A showing a disease-modifying effect of easinersen in SCN2A early onset DEE and substantial reduction in monthly seizures, amongst many other results. With key hires made in our commercial organization and a strong financial foundation, we're accelerating the delivery of life-altering treatments to patients with CNS disorders. Let me provide a bit more detail on each one of our programs. Let's start with Ulixa. FDA acceptance for Ulixa's NDA marks a meaningful step forward for the 7 million Americans living with essential tremor who currently have no ET-specifically developed treatments approved. We estimate that about 2 million of those people living with ET are in immediate need of a therapy that can clinically improve their daily lives, representing a potential for over $10 billion in peak sales. To unlock the benefit for patients and the value, we have been diligently preparing for our commercial launch based on the PDUFA date of January 29 next year. The commercial leadership team is in place with our field force plan to be hired and trained in advance of the launch, and we continue to expand and build the commercial infrastructure across multiple areas, like operations, marketing, access, and compliance. We have also successfully established a distribution network to ensure drug availability at launch at successful levels. Earlier this year, we conducted a very comprehensive observational study with physicians to understand their view of ET and ulixacaltamide. We surveyed more than 2,300 U.S. physicians who collectively manage tens of thousands of patients. The results were beyond encouraging. They validated the ulixacaltamide profile across efficacy, the breadth of benefits, and tolerability, reinforcing the more than $10 billion peak sales potential and the need for a drug like Ulixa in the market. Importantly, we also wanted to hear more details from patients and conduct a similar work with over 1,300 ET patients, which further validated the agreement between the needs of patients in terms of their functional benefits and the results of the Essential3 program. It's truly exciting to be in a place of such alignment amongst treating physicians, patients, and the results of our program. We're also very pleased with our robust presence at the American Academy of Neurology Annual Meeting last month. With 15 scientific presentations, including a plenary presentation highlighting the Essential3 program results, which received the AAN's abstract of Distinction and Movement Disorder Awards, which underscore the strong interest and engagement of the medical community. To further enhance our engagement with health care professionals, we have launched the Essential2 Me disease state campaign. Let's now move to our epilepsy programs. As we shared in March, in another pivotal moment for practice and patients, the FDA has accepted with priority review the NDA for relutrigine for seizures associated with SCN2A &8A-DEE. Those are severe patients affected early in life, and where the seizures are intractable from the very beginning. It's important to highlight that if approved, relutrigine would be eligible for a pediatric review voucher. With the PDUFA date of September 27, preparation for launch is moving full steam ahead with continued hiring of commercial roles, building sufficient inventory, establishing a comprehensive patient support program, and engaging with payers to ensure timely access upon potential approval. We remain confident in the clinical potential for relutrigine and the benefits to the broader DEE population. With recruitment in the EMBOLD study now completed in record time, it's clear that patients and investigators share our view. The potential launch in SCN2A &8A will build the foundation and the results of the EMBOLD later this year; if positive, it will significantly expand the commercial potential for relutrigine by several folds, considering the broad DEE population is comprised of over 200,000 patients in the United States. Let's now talk about vormatrigine, the most potent and selective sodium channel modulator ever developed for the 3.5 million people living with epilepsy in the United States. We have 3 key milestones in the near future for the program. The first is the readout of the 401 Phase III study later this quarter. Then the initiation of the POWER3 study, a milestone in the community, using all the exciting features of vormatrigine to deliver on what the majority of the market really needs. And then later in the year, the completion of the POWER2 Phase III study, which is evaluating doses of 20, 30, and 40 milligrams once daily. Enrollment is progressing well, and we're on track to finalize the study this year and report early next year. Lastly, let's talk about Elsunersen, the first ASO on our platform. also has a rare pediatric drug designation and is being developed for the treatment of early seizure onset patients with SCN2A mutations. We have recently reported the results of EMBRAVE Part A, which enrolled 9 children aged 2 to 12 who were randomized 3:1 to elsunersen or sham over 24 weeks. We are thrilled with the impressive 77% placebo-adjusted reduction in monthly seizures and the disease-modifying components seen across multiple domains in those patients, while maintaining the generally safe and well-tolerated profile. The overall data from both the EMBRAVE program, open-label extension, and emergency use program globally highlight durable seizure reduction and meaningful global gains, which further underscore the transformational potential of this drug. In conclusion, we're off to a great start with our momentum continuing to accelerate across our clinical portfolio, preparations for the commercial launch of ulixacaltamide and lamotrigine well underway, the completion of the EMERALD study enrollment, POWER1 top-line readout coming up, and many other achievements to come. Backed by a strong balance sheet and a long, multilayer IP portfolio across the programs, we're focused on rigorous execution and driving progress across our innovative first and best-in-class portfolio of CNS therapies. I'll now hand over the call to our CFO, Tim Kelly. Tim? Tim Kelly: Thank you, Marcio. Good morning, everybody, and thank you for joining today's call. I'll provide a quick summary of our first quarter financials. In Q1, our operating expenses were approximately $106 million, with $78 million of that for R&D and the remaining $28 million for SG&A, driven by ramping activities and hiring related to commercial launch preparations. During the first quarter, Praxis spent $86 million in operating cash compared to $53 million in the first quarter of 2025, reflecting greater clinical trial activity, headcount growth, and commercial launch preparations. As of March 31, 2026, Praxis had $1.4 billion in cash, cash equivalents, and marketable securities compared to $926 million as of December 31, 2025. This increase of approximately $474 million was primarily attributable to net proceeds from Praxis's January 2026 follow-on public offering and interest income on marketable securities, partially offset by the previously mentioned cash used in operations. The company's cash, cash equivalents, and marketable securities as of March 31, 2026, are expected to fund operations into 2028. With that, I will hand the call back to Marcio. Marcio Souza: Thank you, Tim. I appreciate the review. We're going to move now to Q&A. Howard, maybe you can provide the queue for us. Operator: [Operator Instructions] Our first question or comment comes from the line of Yasmeen Rahimi from Piper Sandler. Yasmeen Rahimi: Maybe also congrats on EMBOLD bringing to the finish line enrollment and data in 4Q as it's an important readout this year. Maybe remind us, what is the data that we have to support relutrigine working in a broader DEE population, both across preclinical and clinical data? And then also, what do you see on a blinded basis across safety and efficacy that continues to give you confidence in the high success in the EMBOLD study? And I'll jump back in the queue. Marcio Souza: Thanks, Yas. I'll take a step back here and maybe talk a little bit about the genesis of going to the broad DE population. When you look into seizure activities, particularly on those intractable conditions like DEEs, we know full well just how difficult those patients are to control. I know that when they can have at least some partial control for the most part, it is because that is a way to inhibit the block, better modulate their sodium channel activity, like you simply cannot have seizure activity without participation of those channels. So when we were conducting the EMBOLD program for SCN8A, the #1 question we're getting from physicians back then was, " When are you going to expand this to this [Indiscernible]? So we'll take that into mind in terms of the overall clinical proof of concept, payer idea, and the needs that we're seeing. But we had to slow down a little bit and do a lot of work. And you might have seen, and it's available on our website, a fair bit of the work in terms of different animal models, which are incredibly predictive in epilepsy, on understanding whether or not there was a good scientific rationale on top of the electrophysiology rationale, on top of the molecular rationale to go to this. Then, in the last part, we had to make sure that the FDA was in agreement that we could study in this population. Safety is paramount, making sure that we're actually understanding the populations we're in. So when we checked all those boxes, we're able to initiate the study. I think what is incredible, I would say, is just the level of interest. If you look into ourselves, if you look into other studies indeed before, if you look into competitive, and I'm going to put that very loosely, there were studies that are going right now, there was no interest in those others. It's pretty obvious by the pace of enrollment that is happening with us and with others out there. And the #1 reason why is that physicians are very confident in the Ada here, just like we are. So it gave us, of course, this extra ability to move things forward. But if I can turn to the business for a second, we are in the business of helping patients. But at the same time, the only way to continue to help them is to continue to generate proper positive returns to invest in the future. The expansion towards the DEE is like 20-fold what the initial indication for SCN2&8A is, which is incredibly important. But the second part makes not only scientific sense, but it's a tremendous upside in terms of the potential of this drug. Then lastly, I know we keep piling catalysts throughout the year, and you guys might be tired of us having so many readouts. But we thought it was very important to get that, shortly thereafter, to really, with a fresh, hopefully, approval at that point in time, give the FDA a lot of flexibility to actually look into just the additional data and potentially even qualify for certain accelerated mechanisms that have been available. So all in all, we see this as a tremendous and maybe the key updates today that we're giving in terms of value inflection for investors. Operator: Our next question or comment comes from the line of Ritu Baral from TD Cowen. Ritu Baral: A lot of client questions are just around the upcoming power readout and what our expectations should be. Knowing the baseline and knowing the relative baseline of other competitive therapies, how should investors, how should we be looking at placebo-adjusted seizure reduction, the safety profile? And then how might that data then frame the Power2 and 3 studies, given the different doses in those studies and the different dosing paradigms? Marcio Souza: Great set of questions there, Ritu. So, I think let's start with the baseline, that's what you mentioned. So if you look historically, at least in recent history, baseline for focal seizures in the refractory population, so 1 to 3 ASMs, and you know the drill there on the other criteria, have been hovering around like 9 to 11, 12 countable seizures on the previous 28 days. And I think we're probably a little higher than that, a tiny bit here for POWER1, which was what we're aiming for. We knew these patients were fairly refractory or very confident about the drug. We also wanted to make sure that once we establish there and have very clear efficacy as we expect, allow us to move incredibly quickly as well towards the ultimate goal of this drug that's being widely available for any patients with focal seizures and in the future, other types of seizures as well. That brings us to the second part of your question, which is the expectations. And I think that it's always dangerous to talk about expectations and setting bars, artificial bars, this late in the game, in terms of like literally like weeks, I would say, before readouts. But we've been fairly consistent on the expectation here throughout the years is, number one, as the severity increases, I think this is one of the few areas, and we're going to be very excited about science that the new drugs still deliver a lot. We just saw that recently with another program; we expect to see the same, like here, a drug delivered despite being piled up with a lot of other drugs. It is at a higher baseline, so more severity. And we've historically been giving that adjusted by placebo around 30% or so as quite meaningful because when we talk to physicians, when you look into the active prescription pattern, that seems to be a number that lands incredibly well. And then the last part is safety, as you mentioned. And we're very confident about the safety profile of this drug, both what we're seeing in the blinded, based on POWER1, or also what we are seeing on a blinded basis, POWER2, which was the very last topic you mentioned. So fairly comprehensively, I think we're excited about the upcoming readouts, another card to flip, another program to hopefully accelerate to bring to patients. POWER2 is going really well. So, as you saw as well in the press release, we reiterated finalizing the study by the end of the year and reading out early next year. So all in all, to think about a potential third or fourth drug or a potential third submission of an NDA or account for this NDA in 12 months is no joke, and we are very, very proud of that. Ritu Baral: Given that baseline, what about seizure-free, Marcio? Last question, I promise. Marcio Souza: No, no, like Nick one there. I think, again, we should unblind it. I do feel the #1 thing is really to make sure we have a very solid reduction. But of course, we want to see seizure freedom here as well. It's important. It's something we saw on the RADIANT data, that when you treat patients longer, by week 10, 12, your median seizure gets to 100 percent reduction. So of course, we want to see the more we treat, the longer we treat patients, a very deepening of effect, that's what started seeing. And I'll leave like that, but we're excited with the overall profile. Operator: Our next question or comment comes from the line of Tiago Fauth from Raymond James. Tiago Fauth: I had just one quick one on Ulixa. It's also related to the communication plan for the Street on the regulatory interactions still a huge area of focus with investors. So I'm curious what's the level of detail that the Street can expect around mid-cycle review, labeling discussions, CMC inspection scheduling, or anything related to that? Marcio Souza: Thanks, Tiago. So maybe, again, I'll take another step back here as well. So we are, of course, being communicated by the FDA when the expected mid-cycle meetings are going to happen for both programs, their communication pattern with us, when label negotiation is expected to start and be completed, and the entire cycle. So we have very good visibility from the agency's goals and from the conversations with us in relation to that. I can tell you that throughout this process, I think a very good shift, I would say, on the FDA is just the ability to really try to keep communicating with the companies throughout the process, and we are seeing that in both programs, which gives us -- I'm going to be cautiously optimistic here about a good level of comfort on how the process is moving on both drugs. Having said that, I think it would not be appropriate for us to give play-by-play. And at the mid-cycle, I think the expectation on our end is that they're going to be like no major concerns, keep reviewing, keep like finalizing, crossing the Ts and dotting the Is. And if that's the case, I think we should expect very little from ourselves. Of course, if something meaningful happens on those meetings, either on the view of like maybe they want to see something or the opposite. They are moving faster, and maybe they want to accelerate things. I think it would be appropriate for us to have a discussion. But we need to get to that point with both publications to be able to have a discussion. I know you asked Ulixa, but I want to make sure our equally lost child gets some attention here with relutrigine. Operator: Our next question or comment comes from the line of Francois Briesbo from LifeSci Capital. François Brisebois: Congrats on the EMBOLD recruitment there. I was just wondering, maybe if you can help us understand, obviously, the patient population size is quite different between IIa and 8A, and then going to broader. But I was just wondering on the broad side, how different are these patients? And my question is geared towards expectations. Is it that the IIa and 8A are so severe that if we look at that data, that kind of sets these artificial bars or whatnot on expectations for the broad DEEs? Or is that a dangerous game based on maybe the heterogeneity of the patients? Just a little more understanding of who you are going after with these broad DEEs? Marcio Souza: Yes. And thanks. I'll split that question into 2 parts. So, one, it is kind of insane to think this way, but it is the reality of the market. When you go to these patients and to the physicians, and we understand their clinical course and just how the disease is a downward slope, it only gets worse over time for those patients, unfortunately, leading to all sorts of complications and SEP and so on. It is very clear that improvement, any improvement, would be the bar, meaning stat sig is the bar for the ERL study. Of course, we want to deliver the best possible results for those patients, but I also think we have to be very careful about setting up the bar. And as I said, in relation to the previous study with POWER1 on Ritu's question. So here we are in a situation that is a very large market, but that is nothing. It's the end of the line. If you think about the patients, then over time, we expect to give them a lot. But I think for this study, we need to be happy if we see statistical significance as we expect to and some improvements. Now, to go to the other side of the question, how heterogeneous is this population? Our recruitment strategy was very clear as to what we want these patients to be diagnosed with. So that's very serious, has to be early, has to have a developmental impact together with seizure onset and a number of countable seizures at baseline. Having said that, we want the most diverse group of patients possible because that's what we're going after. That's what no other drug can do right now. And we accomplished that. By definition, one could argue that IIA and A are harder to treat, but this is more heterogeneous to treat. So if you can see even half, I would say, what we're seeing on EMBOLD would be just doing -- it's even hard to imagine how big a market that would be. But if we see just stabilization and improvement in these patients, that would be incredibly meaningful. So, on setting it up, what is meant to be a really major opportunity for all of us. François Brisebois: And maybe if I could sneak in on AAN, obviously, I think the plenary was passed, like about 8,000 people attending. And so can you just talk about your interactions with neurologists and movement disorder specialists, does that trigger any interest for ex U.S.? Can you share what you guys are thinking on the ex-U.S. stage for [indiscernible] Marcio Souza: Yes. Thanks for reminding us of something that I think we get so excited and so wanting to move forward as always, that sometimes we only stop, slow down, and smell the roses. Yes, being on that plenary at AAN in Chicago a couple of weeks back, and with about 8,000 physicians attending, being the first one to recognize the most important clinical study presented at the meeting. It was very emotional for some of us, for me, certainly. But the cort the most cort was actually the number of people who came afterwards to us and wanted us to go to their practice with our medical affairs team and present to the entire practice and orordentist start thinking and so on. So it just reinforced now there's a huge interest in ex-U.S. as you can imagine, we believe and to be very clear, this is, first and foremost, a U.S. opportunity right now. We're putting our heads down, and we're executing the U.S. There are multiple implications of current policies in the United States, particularly the pricing policies that I would say don't excite us too much to explore split strategies outside of the U.S. We wouldn't put at risk the U.S. business. So this is something that someone has to do globally. And of course, we're planning to eventually get there. But we're not really too excited about splitting the geographies with anyone else. Operator: Our next question or comment comes from the line of Yatin Suneja from Guggenheim. Yatin Suneja: Congrats. A very nice update. Maybe 2 questions for me. Marcio, you addressed the expectations for POWER1. So the follow-up question I have there is that at least in POWER1, we're going to get data on the 30-milligram QD dose. So could you maybe talk about the potential to capture additional efficacy with the 40-milligram in POWER2? Just love to hear from you, how should we think about the 30 and the 40 if there is any potential there? And then a broader question on the commercial side. I mean, obviously, you are undertaking 2 big launches in the next, let's say, 6 months or so. So could you talk about the manufacturing, supply chain, all of that stuff, where do you stand there? Marcio Souza: No, of course, yes, maybe even starting with that. So we imagine when we are planning these launches, we're like, okay, what is likely to happen? And that's how we set our base and how we set the financial expectations, and you see in our forward-looking statements, and overall, you are saying that. And then we go, and we talk to the market, and we start to be maybe a little conservative on some of those. And what if we're wrong on the upper side of that? And then what if we are wrong by several folds on the upper side of that? And that's how we have to plan supply in our view. And that's what we've been doing. We're glad for Ulixa, for example, to have dual like 2 completely independent drug substance manufacturers. They are being rock and rolled for us to have inventory. We're talking about metric tons of drugs here, of Ulixa, just to give you an idea of scale. This is not like a small scale in general, but a very large scale. And we're also quite happy with the fact that the process is relatively, I'm going to say, in the grand scheme of things, simple, and we've been able to align that with the FDA before the submission. So we're good there. Relutrigine, of course, the scale is smaller for 288, but it's not small for GE. So we're thinking about that as well, and we're preparing for that for the launch, too. Very different distribution strategies, as you can imagine, a much more full white glove-like one-on-one interactions all the way to the point of use with the relutrigine, and we are building like that. And then on the Ulixa, we want to do a one-to-one as well. But of course, there's a little bit less downstream here. So both the inventory and management of the patient taking the drug have been quite sorted out. If I go back to your POWER1, POWER2 interrelatedness question, 20, 30 milligrams of POWER1, we believe we're going to see very, very strong results there. But that begs the question: Is there even more to come? So if you look into recent history, very, very recent history, what we're seeing is companies dabbling with the issue of even a little bit more drug, and you trip the wire, and then the drug becomes completely useless from a clinical practice perspective. We saw that in our results a few weeks back, when there are 2 dose and the top dose cannot be used at all by patients despite delivering a little bit more efficacy on paper. But that is not the case here. We know that is not a limitation with Uoraserene. So when you think about the need of patients and the 3.5 million, not the 30,000 that maybe others are going after, that is the real market we're going after, and it requires understanding the heterogeneity of all those patients. That's why having the ability to deliver meaningful either 20, 30, or 40, you name it, is so important. So as not to create expectations that it could be even better. But of course, by definition, it could be even better there on POWER 2. So we'll get there soon, and we're going to be able to review it. I hope I answered your questions. Operator: Our next question or comment comes from the line of Kambiz Yazdi from BTIG. Kambiz Yazdi: Three for me. On Ulixa, with the Essential to Me disease education campaign launched in April, can you give us a sense for the early response from health care providers and how you think this initiative is going to translate into the top of the patient funnel heading into the PDUFA, maybe briefly on relutrigine. I know you touched base on Mario, but how were you able to enroll EMBOLD so rapidly compared to competitors in the DEE space? And lastly, on vormatrigine, you commented a little bit about blinded POWER1, POWER2 safety. How are you handling the investigator's option to reduce the dose of background medication in POWER1 relative to the RADIANT study? Marcio Souza: Sounds good. I'll try to tackle all of them so I can move on. So Essential to me was something we developed with patients and the way they see themselves. And I think once the reaction from the providers is fantastic. Like, people really love it. They see their patients. It reminds them to act, and so we're very happy with this. It will, and it's already doing a great job on building further our database prelaunch to understand really who would be the first in line here, both on the providers and on the patient. We're going to give more of an update next quarter as well. How did we enroll EMBOLD? I would say to go back in time, and people would ask, and maybe I'm going to be criticized by the comments, but I'm going to give it a try. We would ask Jordan how it could be Jordan. They would go back to the court, and would train and would understand the shots that worked and the ones that didn't. And it wouldn't take any wins as a win, but as an opportunity for the next win to work. I think we're never going to be as good as Jordan was, but I think we're pretty serious about every single day asking ourselves, how can we do better, because these patients need us. We're pretty good as well on actually getting sites that have a large number of patients and therefore, have the ability to enroll, and they understand us from the beginning, meaning they understand that our expectation is the highest quality, first and foremost. But also high speeds in terms of you don't sit on queries. We don't sit on the eligibility form. You don't sit on a meeting that's going to happen next week. You need us, we're there immediately. And I think our team is, if I were to say fantastic, they will be fantastic at doing all of this because everyone is here for one single reason is to help these patients. So that's as simple as that. And I think there was a last question that I actually forgot now. Kambiz Yazdi: Yes. Just briefly, on vormatrigine, you talked a little bit about blinded POWER1, POWER2 tolerability. How are you handling the investigator option to reduce dose and background in POWER1 relative to what was observed in RADIANT? Marcio Souza: Yes, absolutely. So what we learned from RADIANT, we've got both pragmatic and programmatic reduction systems in POWER1. One must be very confident in what the investigational drug does to allow for the background to be reduced. Others reduce the investigational drug, as you know, because they don't trust the drug so much, not in our case. So we allow that to happen. I will tell you, it happens partially, which is obviously very important. But I think it was very effective as well. It's the same algorithm for POWER2. I think physicians are very happy with how it's done. It's very safely done, although very logical, the way it's done, considering the fact that the background, sometimes on a placebo, creates circumstances as well, which is required. So it keeps the blinds, keeps the integrity, but at the same time, manages the study in general. So super happy with that as well. Operator: Our next question or comment comes from the line of Ami Fadia from Needham & Company. Unknown Analyst: This is [indiscernible] on for Ami. For focal onset epilepsy, are there any first-to-market dynamics you see if Xenon's product is first to market? Our KOL checks have been overwhelmingly positive for vomacigine, but I just wanted to understand if there are any other factors that may have an impact here? And also, have you had any early discussions with payers on what data you need to generate to support earlier line use? Marcio Souza: Yes. So I think when you look into the overall market, and we did a lot of work on this, it's very sad in a sense that when you go outside of the very small number of patients that are treated at Level 4 epilepsy centers, and you go to a much larger market outside of that, these patients are failing at very high proportions. They are like switching medications, like they're adding on top of that, and so on. So I said this before, I don't believe that it is a one-winner game here that if we had 10 other drugs for focal seizures, that would be needed. What we have right now is completely inadequate. Having said that, the biggest complication throughout the years is when you get a drug, and you really can't allow the physician to have the flexibility to tailor for their patients' needs. And I think what we're seeing with vormatrigine is that it gives a lot of flexibility in terms of what can be done, as we just discussed on the previous question, for example, and it's not the case for other potential competitors here. But also pretty confident about our overall pace. Being a few months behind, and reminding all of you, we were way more than several months behind the other DEE study not that long ago, and now we're reading out soon, and the other study is not even reading out until late next year. So I'm not sure if a few months is really a first-mover advantage. And I don't believe there was ever a mechanistic sodium channel modulator removed from the market, but there certainly was for the other mechanism that you mentioned, and all the safe signals are showing up there as well. So, I think the physicians we talk to are happy about having more options, but cautious about things that they've seen before not happening too well or working so well for patients on the safety side. So we'll play that. We'll play our press on the market, and I have no doubt we're going to win. Operator: Our next question or comment comes from the line of Andrew Tsai from Jefferies. Lin Tsai: Appreciate all the updates. I know we've talked a lot about the EMBOLD study. I did have one small pointed question about it. When you guys shared the IIaA data, 53% placebo-adjusted reduction overall, curious if you saw a consistent seizure reduction in both or each of the 2 DEE subgroups. Maybe that can give investors confidence that you'll see robust and consistent efficacy across the broader DEE subgroup. So, would it be possible to share the seizure reduction in both subgroups? And then for ET, I appreciate all the AAN analyses and so forth. And maybe based on your ongoing work on a friendlier titration schedule, ultimately, should this be approved, what kind of compliance rate do you expect to see in the real world? How long do you think Ulixa responders could be on the drug for? Marcio Souza: Thanks, Andrew. Maybe I'll start with ET, and then we can move back to IIA on EOL and in general. So when you ask physicians, and we had a number of advisory boards and this insanely large overall observational study, we asked them how they would manage and what the expectation is. I think the positive surprise for us was just like how comfortable they were in managing what we know to be a tolerability concern that happens on the first few days and first few weeks of this drug. We're going into this launch fully aware of that being the single most important driver of retention of patients in the long run, and they're incredibly comfortable, and we're actually telling them the things that we thought they could do in terms of like calling these patients and then advising them better, and so on and so forth. So very comfortable with that. Now, how does that translate to overall retention over time and financials? There are 2 approaches here that we can take. One is saying what the things we're thinking about, and that is your classical oral chronic therapy compliance and retention. So starts anywhere from the 60s to 80%. That's just overall numbers out there. And then what is the minimum to sustain the forecast we put in front of you all to be over $10 billion? That number is way smaller. So I'm not saying the number will be smaller. I'm saying I'm going to have to believe in something to go into a forecast, and we don't need to believe a lot to go to $10 billion plus. And that gives us insane comfort. We also have hundreds, hundreds of patients in the safety database, as you know, that have been on this drug for 6 months, 1 year, 2 years. So that gives us a very good indicator of how persistent all these patients are. The second question is actually very appropriate to be asked right now, the results on 2A and 8A, despite the fact that the manifestations, the electrophysiology, and the overall clinical manifestation of NaV1.2 deregulations and NaV 1.6 deregulations are very different. The results are quite similar and very good in terms of the overall reduction, developmental gains, and seizure freedom. So it gives us insane comfort that it is so similar across 2A and has all very good points. Thanks for reminding us to make the highlights. Operator: Our next question or comment comes from the line of David Hoang from Deutsche Bank. David Hoang: So I just had a couple here. Maybe back to vormatrigine in focal epilepsy across POWER1 and POWER2, I know you're looking at 3 doses, 20, 30, 40 mg. How many of those doses would you like to actually take to market? And what do you think would be the best number for commercial viability? And then in terms of the POWER 3 study, the monotherapy study that you also intend to conduct, could you talk a little bit about how that fits into your broader plans for vormatrigine? And why aren't other sponsors pursuing monotherapy studies like that? Marcio Souza: Thanks so much for the question, Dave. So 20, 30, 40, when you started S20 for POWER1, and the part is like normally, people start, if you go to the public documents for like other sponsors, for example, everyone is going to be like, " How close to the MESCC50 can I get when you translate that to humans? And I'm going to be pretty close to that because I can go much higher. We can go much higher here. So we're like, where we started the drug is going to be clearly effective. So that's where we started. And where do we end for now, when we believe that's kind of the maximum efficacy? And I think that's the bull that we are seeing. But a pretty big feedback from the thousands of neurologists that treat this patient is that flexibility is important for them as they're going through different patient types and different comorbid conditions. And so we intend to bring all of those to the market because we believe that gives the highest flexibility to physicians and our largest ability to obtain and keep market share. Now, POWER 3 is a different animal. POWER 3 requires the profile of vormatrigine. So the answer to your question is that others are not doing it because they cannot. Because you need to be able to use this drug as monotherapy. Now there are steps towards monotherapy. It's not an immediate reduction to monotherapy. It has to be done pretty safely, pretty thoughtfully. But patients fail because they can't be on one mechanism. The only mechanism that can, if tolerated, as we believe here, vormatrigine can truly stop any seizures is by acting on the IS and by modulating these channels. And we're the only ones that do that right now. Everything else is upstream of that mechanism. So that's why the confidence when you have a drug like vormatrigine, as I said in my prepared remarks, this is the most potent and most selective ever developed. So we have this data publicly available for anyone to scrutinize. So that is the confidence there. What it does is to move from what has been in a little bit of the definition of insanity on how these drugs are developed to get more and more severe patients, smaller pools of patients, and they no longer represent the broader market in those refractory studies. And you're never really addressing the true market, the true unmet needs that are those patients who are struggling to go back to work, struggling to stay driving, struggling to concentrate. A significant number of patients with epilepsy experience disabilities. They cost themselves, the health care system, and the social security system an insane amount of resources because the drugs they were putting are compounding the issue with the condition. And I think we have an obligation as a company with such a potent, interesting drug to go there and change and revolutionize, as we say, the treatment of epilepsy. That is the long answer to the question. Operator: Our next question or comment comes from the line of Olson Jay from OpCo. Jay Olson: Congrats on all the progress. This is Cheng on the line for Jay. Maybe a couple of us. First, on the Ulixa commercialization. Just curious about the feedback from A and the market research you conducted, what do you view as the most important driver of adoption? And then I think at the end, you also presented some data on the cerium channel modulator in pain. So I'm just also curious about your latest thinking around this opportunity and if there's anything we should expect in the near term? Marcio Souza: Yes, yes, of course. Thanks, Jay. So the most important thing for physicians, when we rank -- and as I mentioned before, and I appreciate you bringing up the question, was very extensive testing and really understanding the details from them. And there are a number of things they really like. They really like the fact that they have never had something targeted for essential tremor. So, having something that the most fundamental mechanism is C-type calcium modulation, and now they have something for that, or soon they're going to have something for that. The second was actually a little bit surprising to all of us. They absolutely lost the fact that in 2 weeks, for the majority of the patients, you have the ability to have a conversation with the patients and they really have an effect. They just don't have that right now in a sustainable manner. They like the fact that we tested the durability of the effect in Study 2. And I would say all those things together, and there are many others, really are going to be key drivers of adoption. And as I mentioned on one of the previous calls about like inventory and building, if anything, they're giving us indications they're going to prescribe to more patients than we originally thought they would. And then the second part of your question about our pain program. Well, I think we've been, as you can imagine, we're a CNS company. We need to look into areas of science that make sense for us based on our technology, on the one hand. On the other hand, we do have a fair bit of things that are moving forward as we discussed today, and many more catalysts to come. So we've been taking a measured approach in terms of exploring a number of other areas in science where our molecules and our platforms can play a significant role. And at AAN, we did present some of the work we've been doing in pain. And you will hear more from us in the future about how we are advancing quite significantly in that regard as well. Operator: Our next question or comment comes from the line of Douglas Tsao from H.C. Wainwright. Douglas Tsao: Marcio, just starting with EMBOLD, I'm just curious, congrats on completion of enrollment. Obviously, demand was very strong. I'm just curious if you have insight into the subsets of patients that you received? And were there any particular ones where you saw very strong demand to reflect sort of the magnitude of unmet need, because obviously, for so many of these, there's no approved therapy, and physicians are just basically trying to figure out as they go along, using sort of off-label ASMs. Marcio Souza: Yes. Doug, I'm going to be honest, when we went to the sites, and we talked to physicians who presented the protocol. Maybe what I haven't said in the call so far is that we disappointed a lot of physicians by telling them, no, no, no, you've got to stop. You can't enroll more patients in this study. We really got there pretty fast, and it seems like we have a deep understanding of this, exactly what you just said. There are so many patients out there that there is very little or nothing they can do, even patients who have other drugs technically approved, but they have failed already, or they try to have a suboptimal response. So it is very broad. Both the phenotype and genotype of patients we got here. And it is within what we expected. And unfortunately, maybe this is a coup in apology, we couldn't get all the patients that wanted to be on this study. But I think our promise is if the drug works, we're going to get this drug to them in the near future. So really, really happy with everything and all the dynamics here. Douglas Tsao: And if I can, on a follow-up in terms of vormatrigine. With POWER1, I'm just curious, do you have any insight in terms of the discontinuation rate so far? Because obviously, I think in RADIANT, there were some discontinuations, but we certainly heard from clinicians that they had patients who maybe dropped out, who they would have provided some additional counseling, just given the robustness ultimately of the drug, they would maybe encourage them to stay in. Additionally, given the fact that you titrate up in POWER1, if we're seeing any evidence that is having an effect? Marcio Souza: Yes, absolutely. So we're pretty happy with how we got reduced, I would say, the overall discontinuation here in terms of the historical with the program. I think some of the points you mentioned, people get more experience with the studies, and so on, they get more comfortable managing. So I'll say I think we're very comfortable with that. I think we're very comfortable with the new benchmark as well, when you consider that drugs people are excited about have like 22% to 25% discontinuation with a further 25% dose reduction on the top dose. That's definitely not what we are seeing. So we think we are very, very competitive here, considering recent competitive events. Operator: Our next question or comment comes from the line of Danielle Brill from Truist Securities. Unknown Analyst: Tyler here for Danielle. My question is regarding the total addressable market in essential tremor. So up to 7 million patients have essential tremor, but you recently said that there are approximately 1 million actively seeking treatment. So what's this gap between the predicted prevalent population and those seeking treatment? And with that, is education still needed in the field for more accurate diagnosis? And do higher volume academic centers typically have a more accurate diagnosis? Marcio Souza: Yes. So there's about like 2 million to 2.5 million patients right now, either who are being treated and seeing a physician at this moment or just done it and are sitting on the sidelines. So when you go from 7 to, let's say, 2 to 3, it is a drop that is not unheard of. I'll say there are many, many reasons here. It is not a misdiagnosed problem, and I want to clarify that. This is mostly something they know and they're either not speaking publicly or disclosing or discussing because they know there's a stigma, there's effect I think we just saw this week, Senator Collins speaking about her diagnose of essential tremor in the sense of people attacking that she might not be fit, which obviously is absurd, because this is a progressive disease only of movement, for example, but people are afraid of talking about things like that. It is a disease that, for the most part, runs in the family. So people are aware. It is a combination of 2 factors. One is the progress. It's how quickly and how far in the disease patients are. We call that the level of feasibility, and that's what gives us about $3 million. And the other is society, sometimes not being too ready for someone to raise their hand and say they have a condition. I think the last one is one where you have 2 or 3 family members, and a physician tells the first one, " There's nothing I can do right now because there are no good alternatives available. You did engage the other family members with ulixacalamide coming to the market; hopefully, soon those dynamics will change. So we bring this to the forefront. One of the reasons why our campaign is called Essential to Me because it's really what is essential for the patients' death. That's what we want to awaken to that. Operator: Our next question or comment comes from the line of Brian Skorney from Baird. Brian Skorney: You alluded to the opportunity to develop Ulixa and the oral T-type calcium channel antagonist in indications beyond ET. Obviously, you're pretty full with NDA reviews and running another few pivotal studies. So maybe it's a little bit in the background. But just wondering if you had any updated thoughts on the exploration of other indications where Ulixa could have an impact? And if there's any time frame you can provide, where we might hear an update on that. Marcio Souza: Yes. Brian, we selected 2 other indications we're going to be going through. We're just going through the last, I would say, checking everything here, taking the time, and making sure that we can discuss a little bit more publicly. We're discussing the format of that as well, whether it's an R&D Day or if it's a series of calls and experts with us. So sight just for a bit, and you're going to be able to talk about that. We are very excited, both scientifically, on the patient side, and market potential for either of those indications. Operator: That's all the time we have for Q&A at this time. I would like to turn the conference back over to Mr. Marcio De'Souza for any closing remarks. Marcio Souza: So thank you very much, everyone. I'm sorry for the questions we couldn't take on today's call. I think we're running a little bit ahead here. An incredibly exciting way to start the year. As you think back over the last 12 months, so much happened in really moving the needle for so many patients. Yet, another study they're finalizing, making sure that we flip the card, make sure that if there is a benefit, we're going to get that as quickly as possible. This year, with EMBRAVE Part A being positive, EMBRAVE 3 recruiting really well, EMBOLD coming up, POWER1 coming up, POWER2 coming up, new indications, as Brian just asked. So we're full steam ahead. The focus is very clear, is to deliver as much value for everyone, including our shareholders. And we promise you we'll have our heads down on the execution here and get everything done. Thanks for the interest, and we're going to be talking to you soon. Operator: Ladies and gentlemen, thank you for participating in today's conference. This concludes the program. You may now disconnect. Everyone, have a wonderful day. 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Interested in Praxis Precision Medicines, Inc.? Here are five stocks we like better. Praxis is preparing two potential U.S. launches after FDA accepted the NDAs for ulixacaltamide and Relutrigine, with PDUFA dates set for Jan. 29 and Sept. 27, respectively. Management said commercial buildout is already underway, including hiring, infrastructure, and distribution planning. Late-stage pipeline data are approaching, with top-line results from the EMERALD study expected in Q4 and POWER1 data for vormatrigine expected later this quarter. The company also highlighted positive EMBRAVE Part A results for elsunersen, including a 77% placebo-adjusted reduction in monthly seizures. Praxis ended the quarter with a strong balance sheet, reporting $1.4 billion in cash equivalents and marketable securities. The company said this should fund operations into 2028, even as spending rises to support clinical trials and launch preparation. Praxis Precision Medicines (NASDAQ:PRAX) said it is preparing for two potential U.S. product launches while advancing several late-stage clinical programs, as management outlined first-quarter 2026 results and pipeline updates on the company’s earnings call. President and Chief Executive Officer Marcio Souza said the company is building on “strong momentum” across four late-stage assets and is working toward becoming a commercial-stage company. He said Praxis is ramping up commercial efforts after the U.S. Food and Drug Administration accepted new drug applications for ulixacaltamide and Relutrigine, with PDUFA dates now set. → Insider Sales: Top AST SpaceMobile Insider Cuts Postion Over 30% Souza said Praxis sees more than $20 billion in peak sales potential across its late-stage portfolio. He also highlighted the completion of recruitment in the EMERALD study in a broad developmental and epileptic encephalopathy, or DEE, population, with top-line results expected in the fourth quarter. The company also expects results from the POWER1 study of vormatrigine later this quarter. Praxis is preparing for a potential U.S. launch of ulixacaltamide in essential tremor following FDA acceptance of the drug’s NDA. Souza said the PDUFA date is Jan. 29 of next year. → Light Speed Returns: Corning Cashes In on NVIDIA Growth Souza said the company estimates that 7 million Americans live with essential tremor and that about 2 million are in immediate need of a therapy that can clinically improve daily life. He said that represents more than $10 billion in potential peak sales. Praxis has put its commercial leadership team in place and plans to hire and train its field force ahead of launch, Souza said. The company is also building commercial infrastructure in operations, marketing, access and compliance, and has established a distribution network to support availability at launch. → Years in the Making, AMD’s Upside Movement Has Just Begun Souza said Praxis conducted an observational study involving more than 2,300 U.S. physicians and a separate effort involving more than 1,300 essential tremor patients. He said the physician research validated ulixacaltamide’s profile across efficacy, breadth of benefit and tolerability, while patient work supported alignment between patients’ functional needs and the Essential3 program results. At the American Academy of Neurology annual meeting, Praxis presented 15 scientific presentations, including a plenary presentation on the Essential3 program results. Souza said the presentation received the AAN’s Abstract of Distinction in Movement Disorder Award. The company has also launched the ESSENTIAL to me disease-state campaign. Praxis also received FDA acceptance with priority review for the NDA for Relutrigine for seizures associated with SCN2A and SCN8A-DEE. Souza said the PDUFA date is Sept. 27 and that, if approved, Relutrigine would be eligible for a pediatric review voucher. Commercial preparations are “moving full steam ahead,” Souza said, including hiring for commercial roles, building inventory, establishing a patient support program and engaging with payers. Souza said the completed enrollment of the EMERALD study in the broader DEE population could substantially expand Relutrigine’s commercial potential if results are positive. He said the broad DEE population includes more than 200,000 patients in the U.S. During the question-and-answer session, Souza said the rationale for studying Relutrigine beyond SCN2A and SCN8A was based on the role of sodium channels in seizure activity, feedback from physicians during the EMBOLD program, animal model work and FDA agreement that Praxis could study the broader population. He said the company viewed broad DEE as “20-fold” the initial SCN2A/SCN8A opportunity. Asked about expectations for EMERALD, Souza said statistical significance would be an important threshold, while noting the severity of the patient population. He said Praxis intentionally sought a diverse group of patients with diagnosed DEEs, early onset, developmental impact and a baseline number of countable seizures. Praxis expects top-line results later this quarter from POWER1, a Phase 3 study of vormatrigine in focal onset seizures. Souza described vormatrigine as the “most potent and selective sodium channel modulator” developed for epilepsy and said the company has three upcoming milestones for the program: POWER1 results, initiation of POWER3 and completion of POWER2 later this year. POWER2 is evaluating 20 mg, 30 mg and 40 mg once-daily doses, with enrollment progressing and results expected early next year, according to Souza. Asked by analysts about POWER1 expectations, Souza said historical baseline seizure counts in focal seizure studies in refractory populations have typically been around nine to 12 countable seizures over the prior 28 days. He said POWER1 may be “a little higher than that.” Souza said Praxis has previously characterized a roughly 30% placebo-adjusted reduction as meaningful based on physician feedback and prescribing patterns. Souza also said the company is confident in vormatrigine’s safety profile based on blinded POWER1 and POWER2 observations. On dosing, he said Praxis intends to bring 20 mg, 30 mg and 40 mg doses to market if supported, because neurologists have emphasized the importance of flexibility across patient types and comorbid conditions. Souza highlighted positive results from EMBRAVE Part A for elsunersen, the first antisense oligonucleotide in Praxis’ Solidus platform. The drug is being developed for early seizure onset patients with SCN2A mutations and has rare pediatric drug designation. EMBRAVE Part A enrolled nine children ages 2 to 12 and randomized them three-to-one to elsunersen or sham over 24 weeks. Souza said the study showed a 77% placebo-adjusted reduction in monthly seizures and disease-modifying effects across multiple domains, while maintaining what he described as a generally safe and well-tolerated profile. Souza said data from EMBRAVE, its open-label extension and emergency use programs globally showed durable seizure reduction and meaningful global gains. Chief Financial Officer Tim Kelly said Praxis reported first-quarter operating expenses of approximately $106 million, including $78 million in research and development expenses and $28 million in selling, general and administrative expenses. He said SG&A was driven by ramping activities and hiring tied to commercial launch preparations. Praxis used $86 million in operating cash during the quarter, compared with $53 million in the first quarter of 2025. Kelly attributed the increase to greater clinical trial activity, headcount growth and commercial launch preparations. As of March 31, 2026, Praxis had $1.4 billion in cash equivalents and marketable securities, compared with $926 million at Dec. 31, 2025. Kelly said the increase was primarily due to net proceeds from the company’s January 2026 follow-on public offering and interest income on marketable securities, partially offset by operating cash use. The company expects its cash equivalents and marketable securities to fund operations into 2028. In closing remarks, Souza said Praxis remains focused on execution across multiple expected catalysts, including EMERALD, POWER1, POWER2 and potential new indications. He said the company is working to move quickly if clinical programs show benefit for patients. Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on discovering and developing precision therapies for disorders driven by neuronal excitability. The company applies translational neuroscience and genetic insights to design small molecule drugs that target specific ion channels and receptor subtypes implicated in neurological and psychiatric conditions. Its research aims to address unmet needs in rare epilepsies, essential tremor, treatment-resistant depression and other central nervous system (CNS) disorders. The company's pipeline includes several lead candidates at various stages of development. The article "Praxis Precision Medicines Q1 Earnings Call Highlights" was originally published by MarketBeat. View MarketBeat's top stocks for May 2026.