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Date
Thursday, Feb. 19, 2026 at 8:30 a.m. ET
Call participants
- Chief Executive Officer — Marcio Souza
- Chief Financial Officer — Tim Kelly
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Takeaways
- NDA submissions -- Praxis Precision Medicines (PRAX 1.65%) submitted two New Drug Applications (NDAs) this quarter for eulixacaltamide (for essential tremor) and rilucigene (for SCN2A-DEE), with rilucigene requesting priority review and eulixacaltamide requesting standard review.
- Eulixacaltamide dosing protocol -- The proposed NDA for eulixacaltamide includes both the standard titration regimen and an alternative protocol, which starts patients at 20 mg for longer than standard before dose escalation.
- Commercial launch focus -- Management stated, "we are laser focused at the highest possible quality launch" in the U.S., deferring ex-U.S. launches for eulixacaltamide despite global demand.
- Bermatrogene market penetration strategy -- Management described a conservative approach to first-line market penetration for bermatrogene in focal onset seizures, reflected in a peak revenue forecast of approximately $4 billion overall.
- Emerald study enrollment -- The EMERALD trial is enrolling a "very diverse group of patients," with no pre-specified subgroups or quotas for different etiologies in DEE.
- Patient retention data -- Preliminary retention data shows "incredibly high" persistence, with patients "really staying on the long run" for bermatrogene studies.
- Clinical data presentations -- Management expects new long-term follow-up and efficacy data for eulixacaltamide and other programs to be presented at the April Academy of Neurology conference.
- Bermatrogene open-label outcomes -- Preliminary results in open-label extension show reductions or removal of background therapies without loss of seizure control for patients on bermatrogene.
- Pricing guidance -- The company reaffirmed a ~$50,000 list price guidance for eulixacaltamide, noting consideration of Medicare Part D and Inflation Reduction Act (IRA) dynamics in the launch strategy.
- Synergy between launches -- Significant infrastructure and back-office synergies are expected in launching eulixacaltamide and rilucigene, though commercial field teams will operate independently due to market overlap timing.
- Regulatory agency feedback -- The FDA did not require additional preapproval clinical studies for the alternative eulixacaltamide titration protocol.
Summary
Praxis Precision Medicines advanced its late-stage pipeline by submitting NDAs for both eulixacaltamide and rilucigene, with regulatory review strategies shaped by business and payer considerations, such as timing launches to optimize value under Medicare and the IRA. Management detailed strong physician enthusiasm and high patient retention rates across ongoing studies, and confirmed focused commercial launches in the U.S. while deferring ex-U.S. expansion. No advisory committee signals have been received from the FDA to date for either NDA.
- Commercial trend data show substantial patient demand, with clinics and prescribers already mapped for rapid access upon U.S. launch.
- The EMERALD study's inclusive design targets phenotypically defined DEE, aiming for broad NDA label expansion by next year.
- The company highlighted the complementary positioning of pipeline assets, and expects data readouts from controlled and single-arm trials this year, possibly supporting further NDA submissions.
- Management described ongoing strategic investments in disease awareness campaigns and inventory builds ahead of launch, with heavier capital allocation directed to eulixacaltamide’s broader market.
Industry glossary
- DEE (Developmental and Epileptic Encephalopathy): A group of severe epilepsies with early-onset, drug-resistant seizures, and developmental impairment, often associated with genetic etiologies.
- SCN2A-DEE: A developmental and epileptic encephalopathy caused by mutations in the SCN2A gene, leading to severe, early-onset epilepsy.
- Essential tremor (ET): A neurological disorder characterized by involuntary, rhythmic shaking, most commonly affecting the hands.
- ASO (Antisense Oligonucleotide): Short, synthetic strands of nucleic acids designed to modulate gene expression for therapeutic purposes.
- IRA (Inflation Reduction Act): U.S. legislation affecting pharmaceutical pricing, Medicare negotiations, and prescription drug policy.
Full Conference Call Transcript
Operator: A log, so it can go into a fair bit of detail on the clinical program, which, of course, is very important for the 13,000 or so neurologists that are our audience. They are going to be there. Also, to expand the understanding on the overall community of this very strong clinical data sets. So we are going to be discussing other aspects like the response on the drug and what happens to these patients, and just how meaningful it is, the combination of all the endpoints, but particularly the primary measure here that is the MAGL 11. And you are going to see throughout all the presentations a very robust number of new data points.
We put a lot out there. So it is not to say that there is any scarcity of data on the Essential 3 program, but it is definitely more geared towards the future prescriber here, that is the neurologist. So incredibly excited, very grateful to the scientific community of AM to giving us the podium there to present the strong clinical there. Thank you. Our next question comes from the line of Ritu Baral with
Operator: TD Cowen. Your line is now open.
Athena Chen: Hi, guys. This is Athena Chen on for two. Thanks for taking the question. I have another one on Eulixa. You previously indicated that the label may include alternative titration schedules. What is the status on this? And are you currently running additional studies to support this? If the label does not include these schedules, how will you be educating and guiding prescribers upon launch? And then I have another follow-up.
Marcio Souza: Yeah, Achina. So maybe I will break it down, if I may, your questions into Right? So we have been discussing since you asked about education. And surprisingly, I would say there is very little education needed here. We have been presenting the data for advisor boards, for consultation meetings to a number of key opinion leaders in the country, very top key opinion leaders, and it is very clear that they see this incredibly robust and very, very easy to deal with the potential tolerability for a subset of patients here that we know might have that. Now going back to the matter of the label, as we previously discussed,
Athena Chen: we propose
Marcio Souza: not only submitted proposed label to the FDA, not only the standards titration that was done in the clinical study, right? So seven days 20 milligrams, seven days at 40, and then seven days at stay stable dose of 60. But also an alternative that we discussed with the FDA I think we move forward, as we continue to engage with the agents here, it is now that your view has to prevail in terms of what is the final labor is going to be, so it would be pretentious to us to say what is going to happen there. But it came in the hills off discussions with them and alignments.
The agents was incredibly clear with us that they did not expect us to conduct clinical studies preapproval on this regard. So as we always respect the opinion of the FDA and the guidance they give, of course, we are not doing I believe, and now that is my interpretation, that is because this is really not a safety issue, right? That is some polar bear that happens on a subset of patients. It is very quick. It resolves very quickly. And the efficacy, most importantly, is very strong. So when you put from a benefit risk perspective, that is both our interest, the prescriber’s interest, and the agent’s mandates that is all maintained quite nicely.
We are all going to see how this progress during the review and we are very enthusiastic about the ability to have serving not only the 70% or so of patients that stay and do really well, but hopefully 100% of the patients that try this drug. Got it.
Athena Chen: And as to the commercial prep for both Religigene and Yulixa, how much capital allocation should we be thinking about between the two programs?
Tim Kelly: I think in terms of allocation, you can imagine that with Alexa being a much broader market,
Tim Kelly: we will be probably putting more of the allocation there. I think we are looking at, as I said, the disease awareness campaign probably a bigger field for us as we get into that part of the work as well. The inventory build is going to be a bit bigger. So we want to be sure that I go back to the old line, you only get one chance to launch drug. And we want to be sure we are investing appropriately for a great launch. I think with the lutro gene, when we look at that market, particularly focused initially on the 2A8A population, it is a bit more focused.
So there is a lot of disease awareness for us to be doing there. It is a more focused effort though with those physicians but we want to be laying the groundwork for the indication expansion that we hope will come in 2027 with the EMERALD study. So it is a bit of a strategic move in how we will do the commercial prep for rigine also.
Athena Chen: Got it. Thank you. I will hop back in the queue.
Marcio Souza: Thank you. Hey,
Operator: Our next question comes from the line of Yatin Suneja with Guggenheim. Your line is now open.
Yatin Suneja: Hey, guys. Thank you for taking my questions. I have two questions. With regard to the POWERADE III study, can you just articulate to us how will that study help you move towards the frontline setting or to an FOS? So that is one. And then the second question is around the review timeline. So we, I think Marcia will good if you can address the review timeline for both the NDAs. We do get questions from investors in terms of priority review or not,
Marcio Souza: we will be good to sort of address it today. Thank you.
Marcio Souza: Yeah. Thanks. Thanks, Yatin. So starting with power three. Right? So just a reminder of what we are trying to accomplish here. So power one as the first study here for towards registration for bermatrigine. Reading out as we just narrowed here in Q2. They study roles a fair bit more than the original 230 patients that were planned. So we are very, very happy with how enthusiastic the investigators were on enrolling patients on this study. Particularly like when you consider competitively how well this study enrolls. Right? Power two, as we are enrolling quite nicely right now as well, would be the second registrational. But when you look into the markets, and it is something quite interesting.
What goes on in both contact seizures. You do have, I would say, a part of this market that is the more refractory patients or going to even argue hyper refractory being super treated, multiple ASMs, really struggling for years and years, possibly decades here. That is where most of drug developments and most of what the level four at lab two centers the key opinion leaders in epilepsy have been focusing But when you go to the rest of the market, and I probably a little facetious to call rest because it is the majority of the markets. Right?
The patients that are 70%, 80% of the patients with focal onset seizures they still cannot strive They still cannot carry on with their lives. There is all sorts of restrictions because they are having like break procedures from time to time and they really not doing well that market has stayed untapped. Until now. So speaking with those physicians and people who treat significant number of patients right, which it is a different subset of the ones we are looking. The needs there is for a drug that they can trust and understand how to remove the treatments so they can be confident and they are very enthusiastic about formatter gene. For that matter.
So we have been consultation with them for finalizing this design. We are getting this off the ground very soon. We thought today was the day to reinforce and celebrate submitting to NDAs. So we are going to be talking about that more in the near future. But what it does to the drug, while not required for registration, right, this is not a requirement as is the ever evolving requirement for registration in The United States as we are seeing this week. So we are very excited about what is to come.
But that but it is something we can see as moving to first line potentially on this drug in the future, which is from serving patients is what we all should be aiming for We are just in a very privileged position that bermatrogene might be the drug that allows patients and physicians to do that. On your second question about the review timelines, can imagine that the decisions we have to make are multiple faults. Right? So one obviously the timing of the submissions is now in the past.
The second is the entire understanding and relationship and workload and so forth with the agency, Good reminder here that while the N1 or division of neurology one and two are two different divisions. There is a lot of shared resource. We have two NDAs at the same time with them. Very obviously, ralutrogen is easier. I am going to say not because it is easier for a clinical or anything like that. It is just much, much less data by single study rare indication, and that by itself from a workload perspective is smaller, so we decided to request a priority review for that application. But we decided not to request for LexaCalc Mice for multiple reasons. Right?
Those that I just mentioned, but there is a broader business reason about the time of the launch and the maximization of the revenues over time for this drug. Particularly around PIC particularly around payer and overall dynamics of discounting and so on that happens year later in the launch. On a drug that can be and will be as big as elexacalt might, We cannot pick up the dollars and forget about the millions. On this one. So I think we need to be and we were very focused strategically on the overall value here.
Marcio Souza: Very good. Thank you so much.
Operator: Thank you. Our next question comes from the line of Joon So Lee with Truist Securities. Your line is now open.
Tim Kelly: Hey, guys. Congrats on all the progress, and thanks for taking our questions.
Marcio Souza: Just want to clarify the response from the prior question. Just answered. So you mentioned business reasons for not asking for prior review for liscalcamide in AT. Just to clarify, is that because asking for standard review as opposed to priority review re would result in almost a year delay
Joon So Lee: in being forced to negotiate under IRA. Is that is number question number one. Question number two, looking forward to your presentations at AEN. Can we expect any long term follow-up data? The reason I ask is long term efficacy came up as a key point for our payer KOLs. Regarding reauthorization of Olexa in ET. And the last question is, you know, in the past, you have telegraphed around $50,000 list price for lefcalcamide. Is that still the case? And can you help us understand your thought process behind the pricing strategy? Thank you.
Marcio Souza: Yeah.
Marcio Souza: June, I think we are you are in the right direction there. Right? In terms of what when you are looking at and we are forecasting this drug Of course, there are multiple dynamics, and you just name some of them. Right? Like, what payers and reauthorization and like, potential step edits and you name it. But the Inflation Reduction Act and the dynamics of the act right now is important consideration as well as a very important consideration This is a heavily Medicare Part D population. So we want to make sure we are both responsible on how we are launching also maximizing and giving the proper value for the drug.
And that includes looking to the life cycle of evolution of the current iteration of the act, when it impacts, when it kicks in, believe that is why you are going there. That is a pretty big difference in value. Depending on when that negotiation happens. It was a key consideration on our filing strategy. The second on the data to be presented I think multiple fold, yes, we are always going to be presenting more and more data to reinforce the short term and long term value of udexaclatamides in essential tremor, One point we believe there is a still room to explore here
Marcio Souza: is
Marcio Souza: really how strong the data is. I know you recently spoke to some payers and like, payer groups, and they I understand correctly, they agree with how strong this data is and how high the potential for this drug is. But it is the understanding is not clear yet in the market because we have not put this data out there. It is just how deep the effect is on a very large proportion of patients. And when you look into drugs, it is not one size fits all.
So we want to make sure that gets reinforced that gets holistically reviewed, Also very excited about having our principal investigators presenting this data to their peers They have been thrilled with the execution. With the results, and I think now is the time to let them take this stage and present this data as key opinion leaders in the field. So stay tuned. I know we are trying to cover a lot in this call. But stay tuned for April or I am sure you are going to be pleased.
Joon So Lee: Thank you so much, guys. Looking forward.
Operator: Thank you. Thank you. Our next question comes from the line of Andrew Say with Jefferies. Your line is now open.
Marcio Souza: Hey. Good morning. Congrats on all the
Marcio Souza: progress. Appreciate the updates. Maybe shifting to ralutagene, actually. You are pursuing this broader labeled EMERAL study for all DEEs. So how many different DEE patients in the phase three does the FDA want to give you a broad label, and how many different DEEs have you enrolled so far? And secondly, for that study, we think about what you wanna see, is it fair that you might be expecting efficacy to be similar or even stronger than what you saw in SCN two a, and maybe explain why. Thanks.
Marcio Souza: Yeah. Thanks, Andrew. So if you look into EMERALD right now, right, let me separate couple of things there. So one,
Tim Kelly: as
Marcio Souza: I mentioned in the remarks and then Tim mentioned as well in one of the answers, the goal
Joon So Lee: here
Marcio Souza: at this point in time is to get that sNDA by next year. And it should give you the confidence on what is happening on the study right now. Really incredible interest, incredible enthusiasm and engagement from the physician that are referring to the sites or participating in sites in this study. We took a very basic approach. Right? If you go and you look into the most recent definition of these Doctor. Shepherd’s recently published around this, really going back to the basics. What is a developmental epileptic encephalopathy? What are the drivers? Like, why should we be treated?
And relitrizine sits like on that junction of really helping the broadest population, least hypothetically right now since we are going to have to see the results
Athena Chen: in the
Marcio Souza: So we took the approach of phenotypically defined versus genotypically defined as patients for this study. What I can tell you right now without saying too much, is that it is a very diverse group of patients. That we have on the study. Both enrolled studies and patients in screening, diverse. Not completely unexpected or unexpected at all. And when we position and we discuss this study with the agency, I can never speak on their behalf, but I tell you our interpretation. Is that the idea here is to treat the disease is not to treat the cause, of the disease. Will be pretentious to any of us to try to do that.
And the understanding is that you cannot have a seizure without participation of Southern Channel in neurons. And therefore, this is like omnipresence type of mechanism that we can use. So that is the definition right now There is no subgroups or there are no quotas for difference, if I may, for different etiologies. So we are really looking into like an overall effect. When you go back to the last part of your question, and what to expect here, The we need to go back to translation. We need to go back to the preclinical data. Since we do not have the clinical data yet.
And when you go back there, we look into all different models, that we tested, all the fundamental electrophysiology and basic biology of the channel deposition the physical density of the channels in a critical juncture in neurons What we see is like quite overwhelming preclinical efficacy. So when we had that before, on SCN2A, on the different models with SCN8A on the autogenic model, think the way we are looking into that is like those are very good translational models. Now we have clinical data on those indications that translated well. So we expect to translate well as well.
I think would be a little bit too early to guide on how well the translation would be But you have got to remember from a overall GE perspective, there is basically nothing for these patients as well. So while I am incredibly excited about showing results, maybe the same, may be better than what we are seeing on two way and eight way It is absolutely not needed to deliver a very fundamental change on the way these patients are. Are treated right now. But as I said in the past, we are going to see this soon enough. So we are just going to keep our heads down executing on Emeralds.
And soon enough, we are going to be discussing hopefully, significant benefit for patients. On that population. Makes sense. Thank you. You bet. Our next question comes from the line of Douglas Tsao with H. C. Wainwright.
Operator: Your line is now open. Douglas, your line is open. Please check your mute button.
Marcio Souza: Sorry about that. I was on mute.
Marcio Souza: Marcio, maybe it is a little bit of a follow-up on that last question
Marcio Souza: in terms of
Marcio Souza: rilucigene, and then I have a follow-up on bromatrogene. But just sort of when you anticipate utilization, do you see sort of utility across the it sounds like you see utility across the entire D spectrum. And even in indications where there are sort of ASOs or sort of more targeted therapies being developed,
Joon So Lee: And, arguably, sort of vilucidium will sort of become a workhorse
Marcio Souza: used, you know, regardless of what other
Marcio Souza: may also be deployed for a particular patient population.
Marcio Souza: Thank you. Yeah. No. That is a very good question, Doug. The workhorse, probably, I would not use that term, but I am glad you did because I think that is one way to look in a drug like a in a toolbox, like, right rilutrogen, where physicians would have that available that they do not have right now. They have to ask too many questions too many tries, Like, unfortunately, a lot of those patients do not have that time to keep trying and optimizing And quite importantly, like, there is what? 20, 25 drugs that are normally tried in this population. Virtually none of them have been tried
Joon So Lee: And
Marcio Souza: officially like with randomized studies, properly developed in pediatric populations or in adolescents or early adults here, So we never talk about that other side of label use that we really do not know what you are doing. On those populations. So what we hear a lot from physicians here globally is that certainty of the drug that is being rationally developed for this indication. So there is a lot of enthusiasm there. Now, to think that this is a silver bullet, would be completely absurd. That is not such a thing as a super bullets in medicine. I think we all wish there was, but there is not.
So we do see this as a kind of an overall background therapy for some patients. Ideally, it would be monotherapy. For other patients, they are going to continue in other drugs. You mentioned ASOs particularly. I think ASOs are getting consolidated as kind of the second workhorse I think we are finally beyond some of the dreams we had about other modalities. On gene therapy and really understanding that is a very, very good mechanism. So combinations between ASOs, for example, and others in riletirgenia we expect to be the norm. We look forward for the moments where these discussions are about how the use is happening.
Versus all these hypotheticals because these patients do not have a lot of time and really excited about helping them. Okay.
Marcio Souza: Marcio. That is really helpful. And then just on for vatrogene, I am just curious.
Marcio Souza: In the RADIANT study,
Marcio Souza: as patients go sort of past that the initial point and we are in the open label extension. I am just curious, have you gotten data or seen data of patients who are withdrawing some of their background meds sort of a little bit of a preview or look, if you will, into the POWER three study and in particular, patients who are maybe able to pull off you know, sort of some of the more
Joon So Lee: problematic you know, sort of efficacious, but perhaps
Marcio Souza: sort of less tolerable drugs like cinobamate or carbamazepine, etcetera. Thank you.
Marcio Souza: Yeah. We are seeing across the boards reduction, elimination, removal, of background therapies as patients continue to have experience on the open label with formatterjee in that Physicians are very excited about that possibility. Would say patients are more excited about that. As you can imagine, of course, efficacy is king here. Right? But the queen is safety. For this patient then it is very hard for them to daily operate as humans when they are on all those medications. So it is always very important So not only that, right? I think one of the drugs you just mentioned, has just been like, put on a new warning for drug induced liver injury.
So you have got to think about the long term impacts of these drugs. And being chromatrogene, at least so far, like so clean, it is quite important for these patients as well. So we are seeing more and more enthusiasm Of course, every time you remove a drug, the first question you need to ask is, happens to the primary measure? What happens to seizures? And I am very happy to preliminarily report that we are seeing exactly what we were expecting to. Right? Maintenance of seizure control while really not being necessary to use What I believe you asked this from a clinical perspective, but I am going to jump into here into the commercial perspective.
As we believe the value proposition of by the very first time on a drug that you can sequentially reduce the use of other drugs is very different. Than a drug that is just from the top get used for a little bit, and maybe removed. So very excited. I think we are going to have more data and more discussions to talk about that. As when we have the power one results as well and giving an overall program updates.
Marcio Souza: Okay. Great. That is super helpful, and congrats on the progress.
Marcio Souza: Thank you. Our next question comes from the line of
Operator: Francois Brisebois with LifeSci Capital. Line is now open.
Tim Kelly: Hi. Thanks for taking the questions,
Marcio Souza: Congrats on all the progress. It is quite a 2025 for you guys. So maybe on eLsigner, there is a lot
Francois Brisebois: to touch on, but I do not think much has been touched on this one. Can you help us embrace data is coming soon? So just maybe set expectations there a little bit And I think there is so much going on with the company that maybe help us understand why it is so important that the, you know, the update on just having a single arm comparison for Embrace three and what that means. Yeah.
Marcio Souza: So thanks for that, Frank. The so ELsa, as we call, like, ELsa Nursery and slide, it definitely sometimes gets the backseat on this questions. So I appreciate We see in a sense, right, what linking your questions to dull questions is like an ecosystem All these drugs are all going to be used They are going to be different case on we use one more, the other, a lens or combinations, and you name it. In the future. So we have this cohort right now. Nine patients, three to one randomized to sham or drug. Our intention there was to continue to understand the safety, the efficacy, the PK of this drug But we are very excited.
Because every time you have control data, independently or even open label data, independently of the number of patients is yet another opportunity for us to understand the drug impacts, We know the study went really well with these patients, like did really well from a safety perspective. Since then we can monitor, like, generally, which is not a given, right, in any given disease. And we believe that it can be quite informative The FDA left the door open for us on discussions we had about the overall value of the datasets on the overall program. Right?
So we wanted to make sure we are we are very respectful and we are mindful of that, but it can have a very high value depending on the results. Here. In the next few months. Now when you look into the we are very pleased and slightly surprised that the agents was really pushing us to actually get in BRAVE-three to be controlled on baseline, I think they are definitely putting their money where their mouth is in terms of accelerating drug development for drugs with a high potential translatability plausible mechanism and this drug fits right in. All of that. We did switch the study globally to a single arm within patients randomized.
Well, I guess not randomizing now they are dosing on the study. And it has been like great experience with all the PIs and all the patients globally. So a little bit longer, on the timelines, not really long on biotech years, but long for us. Since there is a lot going on. But we do expect to be done with this study this year as well. Which will potentially get another, yet another NGA in the near future. Very different positioning, as you can imagine, commercially very complementary to the regulatory gene efforts. They were doing the same prescriber population overall same patient population.
So you can see how synergistic this can be on the overall life cycle of the company.
Francois Brisebois: No. That is very helpful. And then on eulxicalatamide, I do not think you mentioned anything about ex US efforts. I was just wondering, such a big market here. I assume ET is everywhere else. Any thoughts there that you can share? And then the launch, obviously, without prior review, this is maybe a little premature. But you have had so many trials and so many patients on this. I am just wondering in terms of line of sight, you know, in terms of the launch trajectory off the bat, do we know where these patients are Or any color there would be helpful.
Marcio Souza: Yeah. Yeah. Absolutely. So, maybe the easiest part of the question, you are absolutely no. Where these patients are I think we have daily motivation as patients keep calling our IR line, keep sending us message, and keep reminding us to keep pushing that they are waiting. It is quite motivating for me and for the rest of the team to keep moving there. And just like the millions of patients that we have mapped to prescribers, in The U.S. That in a sense, I think it answers the second question or the first question that I am answering second.
While that is huge unmet need outside of The U.S. and we appreciate that and we are very compassionate in relation to that, The focus of the company has to be in The U.S. right now.
Marcio Souza: The
Marcio Souza: we were laser focused on making sure the highest possible quality NDA was submitted. Now we are laser focused at the highest possible quality launch. Is done. For this. And we are looking to the magnitudes of those this launch, I think you would be you conceived and intended for us to get distracted. At this point in time with other geographies. So very good trajectory. Very good number of patients, as you can imagine, on open label extension right now, which would expect it would transition right away.
To commercial Other pools of patients that we believe are going to be right before and then just this spontaneous demands that we are seeing piling up on top of our database. Already for the launch. So we do not want to get over our skis here but this is definitely trending towards a successful launch.
Francois Brisebois: Excellent. Thank you. That is it for me.
Athena Chen: Thank you. Thank you. Our next question comes from the line of
Operator: Ami Fadia with Needham and Company. Your line is now open.
Athena Chen: Good morning. Thanks for taking my and congratulations on the submissions both the submissions this month. My question is on vometra gene and regarding how what you assumed for your peak revenue potential, particularly regarding utilization in first line? And if you could provide some color on how you see utilization in earlier lines of therapy impacting persistency of patients and duration of treatment And how will the POWER program you know, help you build the clinical data that supports or provide some color on how long patients stay on treatment as they get treated with vometra gene in earlier lines of therapy. Thank you.
Marcio Souza: No, thanks. Thanks, Ami. The So the retention we are seeing right now, right, as an early indicator, of what you
Marcio Souza: asking, it is incredibly high. And once we talk about
Marcio Souza: power run results, we are going to be able to talk about that as well. Patients not only participate in these studies, but really staying on the long run. So that gives us an early flavor of how retention in overall commercial is going to be. And of course, they are staying on drug differently from other drugs and different from other trials, because they are having benefits and because they are having safe experience with this drug. The way we currently forecast the movements between third line, second line, first line, is actually very responsible. I would say. So we are now looking for this at day one of launch.
We are not even looking to this at year one. Launch, we know it takes time for these things to happen. We know that the overall penetration is not like the highest
Francois Brisebois: but
Marcio Souza: when you look into our big revenue right around for billion dollars or so, in overall. Like, you can imagine that we just be hyper penetrating the first line because otherwise, this would be much, much higher. Than what we have right now. So that is a huge potential there. For upsides, as you can imagine. But at the same time, as we there are many firsts that happened for us. In the last few years. I think we are always very responsible to say what we knew, and what we did not.
And I think what we know right now is that there is incredible interest on utilizing this drug What we do not know is the dynamic of that. So we are really keeping I would say, very tempered our enthusiasm in terms of how the penetration is going to be there and that is reflected on our conservative to realistic one could call big revenue right now.
Francois Brisebois: Thank you.
Marcio Souza: Thank you.
Athena Chen: Our next question comes from the line of Brian Peter Skorney with
Operator: Baird. Your line is now open.
Tim Kelly: Hey, good morning, everyone. Thank you for taking my question. I guess we will get some guidance on NDA acceptance. I wanted to just get your preliminary thoughts on if the review division has given any indication on an advisory committee for either Relixitrain or Euloxa? I mean, it seems that adcoms are getting more rare under this current iteration of the FDA. And ralutrigine’s mechanism seems pretty straightforward with the data. It probably would not require one, but thoughts on EULXSA in particular?
Marcio Souza: Yeah. No. No indication whatsoever. At this point in time. One would not expect much of an indication
Tim Kelly: before
Marcio Souza: day 60s and day 74 interactions with the agency. But there is no indication right now, Brian.
Tim Kelly: Great. Thank you.
Operator: Thank you. Our next question comes from the line of Jay Olson with Oppenheimer. Your line is now open.
Marcio Souza: Hey, congrats on all the progress, and thank you for taking our As you plan your prelaunch activities for both Eulixa and ralutrigine,
Joon So Lee: can you talk about the potential synergies you can leverage between these two launches? And then
Francois Brisebois: eventually,
Joon So Lee: how those synergies could help set up your future launches of formatrogene and elsinersen? Thank you.
Marcio Souza: Certainly. So the a I will take from the borrowing side. Right? From an infrastructure perspective, back office perspective, lots of synergy. And how things are set up, and we have been doing a lot of that. On the backgrounds like how the systems are set up and so on. Until not that far in the in the past, we were actually considering a lot more synergy in the fields as well. On the approach to the markets. Because we believe before the involved positive results last year that we will have a little bit more time with proletrogene. So we would be able to leverage that.
And that change a lot once we are now really launching two drugs in about the same time, While that is a very significant overlap, in, I am going to call zip codes, right? Like we hospitals that have or clinics that have very high overlap of patients with HIT and GEs are focal We do not believe that it is prudent right now to take any distractions or taking a go to market strategy for both for individually for these and individually for a central tremor to max maximize each one of them.
Now, since your question extrapolated to the future, Jay, When you are looking to like, focal sense seizures, for example, generalized seizures, you name it, other things that might come in the future. That is very, very high overlap. Between prescribers for the majority of the epilepsy patients today and the majority of the essential tremors today. In the markets. So you can see how apraxia’s presence might be beneficial at that point in time. Now we are talking maybe two or three years from now and that we are going to be maximizing that now. Let me bring back to like, 60 days or so from now. You are going to be in Chicago with all these prescribers. Right?
If we were to count the universe of prescribers coming to the annual meeting of the Academy of Neurology we are talking about over 70% of the prescriptions for ET and focal in The U.S. are present at that meeting. So that is a natural overlap here We are just going to be maximizing that overlap more a few years from now than a few months from now.
Joon So Lee: Great. Thank you. Congrats again.
Marcio Souza: Thank you. Our next question comes
Operator: from the line of Kambiz Yazdi with BTIG. Your line is now open.
Joon So Lee: Morning, guys. Congrats on the NDA submissions. Three questions on my end. First, can you provide an update on the central tremor patient database? How is that already validated the size of the ET market? Second, the FDA’s default position is at one out and well controlled study combined with confirming evidence will serve as the basis of mark pending authorization. Of novel products do you think about that with regards to vermatrigine and FOS? And then my third and final question is, how should we think about the timing of ralutrigine EMERALD top line? Would an interim analysis be a possibility for EMERALD? Thank you so much. Thanks, Kevin. If I try to go through
Marcio Souza: through questions here if I understood them correctly. Right? So on and I think I missed the very first one, so I might ask you to repeat which one was on ET. Yeah. The first one was
Joon So Lee: can you provide an update on your essential tremor patient database? A little bit. How has that already validated, the size of the ET market?
Marcio Souza: Yeah. So we will continue to both validate and I think we intentionally did not talk about this today since now we are moving from the clinical focus one to the commercial focus effort, and we are going to give, like, a larger update at our commercial day in the near future there. So I am going to keep you holding your breath a little bit in relation to that. I am going to go to the Emerald and then go back to the second question. Of course, there is always an opportunity for Entrance. It is not a current plan. For Emeralds.
And the reason why it is not a current plan is really the pace of enrollments in Emeralds right now I think we have been realistic slash conservative about the timelines today, but there is really a very fast pace of enrollment that might not allow for that. And then on the last one on how we think about generation of evidence vis a vis the commissioner like, New England Journal of Medicine yesterday publication. We applauded I think it can be and will be.
I have built my trust on this on our country that is going to be used responsibly And we believe that in drugs where very clearly, like epilepsy, very clearly the second study was not that necessary in the past. Those are good case studies and best for the future. We could not possibly be trying to guide you today that is going to be the standard for our drugs. There is a lot of water that needs to go under that bridge. But we are enthusiastic about what that can have can do for drug development and for Praxis Precision Medicines, Inc. Particularly in the near future. So stay tuned.
Francois Brisebois: Thank you.
Operator: Our next question comes from the line of Justin Walsh with Jones Trading. Your line is now open.
Joon So Lee: Hi. Thanks for taking the question. With your clinical successes, have you been seeing increased attention paid to your cerebrum platform? And related to that, can you remind us how both cerebrum and solidus are differentiated in their ability to select quality candidates for your pipeline?
Marcio Souza: Yeah. Yeah. Absolutely. We do. As you can imagine, that is
Marcio Souza: thing
Marcio Souza: just in like a renaissance, maybe, I would say, on understanding that deep best mechanism to address a lot of this disease is through antisense oligonucleotides, So we are seeing a lot of interest across the board that On this, you are going hear more in the near future about how we are going to maximize Also believe that there is a different way We talked about standards today. I talked about plausible mechanism today. There are different things there to maximize We always follow two pillars. Right? The biology, when you are and what is the best way to address, and then the business on the other side. Without business, biology is irrelevant. Without biology, business is irrelevant.
I think we try to do both of them, and that is why we are here today. Discussing the successes and the future success. So I think that is stay tuned. But they are going to be a lot more on that platform as well.
Operator: Thank you. Our next question comes from the line of David Timothy Hoang with Deutsche Bank. Your line is now open.
Joon So Lee: Hi, there. Thanks for taking my question. So maybe first one on elixocalcimide in essential tremor. Could you just discuss a little bit about the distribution of the base? How well do you understand whether these prescribers will be based in, let us say, academic centers versus community This is a product that would be prescribed by, you know, general neurologists broadly. Then one on bromatrigine. As we think about the evolving landscape in focal epilepsy, there is several potassium channel focused therapies that are in late stage development in potentially coming to market soon. How do you think bromatrazine fits in amongst those products, and what and what would docs look at when selecting a therapy? Thanks a lot.
Marcio Souza: Thanks, Dave. The distribution, and I will say the distribution of patients and distribution of drug and the prescribing patterns very well understood. Think we have been talking about that for a bit. It was one of the very first functions and dollars that we built in the company I was arrogantly tell you that we have an exquisite understanding on this. And each one of the physicians that have case in The U.S. right now is the majority of them. Vast majority general urologists, and they are very eager and willing to engage with us.
Athena Chen: I think
Marcio Souza: on pharma and the competitive landscape, this was not and never going to be a zero sum game. We welcome We are cheering for the next readouts on the space coming up soon. And we believe that is really is in the best interest of patients that there are multiple positive results and drugs and we can use them And we just see the path to first line only happens with formattergine. So competitively, welcome Going to have some competition on the refractory patients on the third line, but there is no competition on the earlier lines. Yep. Thanks for your question.
Francois Brisebois: Thank you.
Operator: Our next question comes from the line of Ben Burnett with Wells Fargo.
Ben Burnett: Great. Thank you so much. I want to come back to an earlier question on you look elixocaltamide and just the potential to explore titrating patients. I think the you mentioned an alternative titration protocol. Guess curious if you could give us a little more color on this. And, I guess, would this alternative titration protocol start patients at a lower dose I and then secondly, you also talked about standard review for elixocalcamide, and I think you walked through a couple sort of business reasons for that. But it feels like it also would give you some time to maybe iron out a titration protocol. And is was that also a consideration?
Francois Brisebois: Yeah. Thanks, Ben.
Marcio Souza: The no. There was not a consideration, actually. I will say that was not an important consideration. Of course, it is always up to the FDA if they want to discuss more We had a very robust discussion about that topic. With the agents before It was very good to see that this is not a major concern but there is obviously an interest from us. And from then, when there is an efficacious drug that we try to actually expose and get the maximal amount of patients. That is the idea. It is not a lower dose. Right? So starting at 20 milligrams is just it is staying at 20 milligrams for longer.
Because what we see here, is what looks like it is really just a few days that they stay longer on that dose that tends to subside, the side effects, and then they have the opportunity to have the effects. So that is the that is the idea there.
Tim Kelly: I think
Marcio Souza: of course, that is thousands of things that can come up in conversations with the agency, but that was not one of them that we are planning as a main conversation for sure. Thank you.
Operator: I would now like to turn the call back over to Marcio Souza for closing remarks.
Joon So Lee: Yep.
Marcio Souza: Yes. Well, thank you, everyone. I think we run a little bit even over the allotted time.
So I appreciate you all hanging in with here, all the enthusiasm shared by all the analysts and our shareholders can say how much appreciate and all of us here at Praxis appreciates the patience that participate in all these studies that continue to engage with us as we are here very humbly submitting two NDAs, which I do not believe has ever been done by company our stage on the same quarter The real motivation for everyone that worked like, days and nights on the last several years, but particularly the last few months has been the fact that there is someone, as we say, outside of the door that we do not know that need these drugs.
So just want to dedicate these moments to all of them and thank them for participating in our studies. I will looking forward to interact with all of you soon, and thanks for tuning in.
Operator: This concludes today’s conference. Thank you for your participation. You may now disconnect.
