管理層發言
Good day, and thank you for standing by. Welcome to the Praxis Precision Medicine Second Quarter 2026 Financial Results Conference Call. 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.
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 timelines and financial projections. While these forward-looking statements represent Praxis' views as of today, they should not be relied upon as representing the company's views in the future. Praxis may update these statements in the future, but is not taking on an obligation to do so. Please refer to Praxis' most recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business.
Joining us on today's call are Marcio 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?
Thank you, Dan. Good morning, everyone. Thank you for joining Praxis' Second Quarter 2026 Conference Call. Three months ago, I told you this would be the year Praxis becomes a commercial company. This quarter is the one where that stopped being a plan and started materializing into the organization. We have two NDAs in late-stage review with the FDA with both approvals expected in about six months. It's extremely exciting to bring both ulixacaltamide to essential tremor patients and relutrigine to SCN2A and SCN8A patients. We have commercial leadership in place, a field force for the first launch hires is trained, and a distribution network established and inventory being built. I want to spend most of my time today on some key regulatory developments and what we have been building. Let me start with ulixacaltamide. Essential tremor affects over 7 million Americans, and there is still no FDA-approved therapy developed specifically to treat the condition.
With the potential approval coming up by January next year, ulixacaltamide is poised to change that. Speaking about the NDA review, the FDA completed its mid-cycle communications with us and we are very pleased with the progress and discussions with the agency. In that meeting, the agency identified no efficacy-related significant issues and stated that it does not plan to request an advisory committee meeting. I would characterize the discussion as expected and very forward-looking. On the commercial build itself, leadership is in place and all core capabilities are where we expect them to be at this stage. We will be ready ahead of PDUFA to launch ulixacaltamide for essential tremor 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 essential tremor 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 practices 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, the agency 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 developmental and epileptic encephalopathies, exceeded its target with approximately 200 patients enrolled spanning more than 50 distinct genetically defined etiologies, among many others not genetically defined. There is a trial population that did not exist as a cohort even five years ago. Assuming the study is positive and the initial review for relutrigine in SCN2A/SCN8A is 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-of-its-kind decentralized study for essential tremor 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 topline 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 seizure frequency 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 says 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 Breakthrough Therapy Designation for elsunersen for seizures associated with SCN2A-DEE caused by gain-of-function variants 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 gives us and discussing a comprehensive plan with the agency in the near future. Parallel to that, EMBRAVE3 continues to enroll well with topline results expected next year. We're incredibly pleased with all the progress made on all fronts this quarter, and we look forward to a successful rest of the year. Let me now turn the call to our CFO, Tim Kelly. Tim?
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.
Thank you, Tim. Really appreciate the update. Now we're going to move into Q&A. Operator?
分析師問答
Our first question comes from Yasmeen Rahimi from Piper Sandler.
Congrats on an incredible update that I think was very, very timely and important to us, especially as there has been some noise around an advisory committee. 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, 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?
Thanks, Yasmeen. I absolutely share the sentiment that you just expressed. These are incredibly complex programs, as we discussed in our remarks, and 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 very similar to what we've discussed before publicly. So, we checked that box quite nicely as well. And of course, the cherry on top, it's always good to have the FDA in the house checking everything and making sure they agree. We always knew we were doing everything correctly, but it's good that they agree with our assessment on data integrity, documentation, communications, procedures, and safety of subjects in the studies. So we turn a page to talk about what we really like talking about: the millions of Americans that are not served currently in the United States. We've been very diligent in hiring a world-class sales, marketing, market access, medical, and operations team. I can say this is probably an opportunity of a lifetime if you are in one of those positions to launch these drugs and transform patients' lives. I'll hand over to Megan to discuss a little bit about the phenotype and what we are seeing at this stage.
Thanks, Marcio. So yes, as Marcio was sharing, 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. From the phenotype standpoint, we are hiring individuals with multiple launch experience in the rare neurology space — hunters who will go out and do a phenomenal job for us. In the case of relutrigine, the team is hired and trained. We have the next few months to really be active in account profiling, which will set us up quite well from a launch readiness perspective. The ulixacaltamide field force build-out is well underway and also on track for launch timing.
Our next question comes from Ritu Baral of TD Cowen.
Marcio, I wanted to dig down into your comment about the mid-cycle review meeting. You mentioned the word "forward-looking." First, could you comment on if there were any surprises during the meeting? Any new topics that were unexpected? And second, 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 a quick follow-up to that last point: what aspects of commercial launch prep are you preparing to optimize that commercial experience to optimize compliance?
Yes, absolutely. I appreciate the question on what "forward-looking" means here. It was not meant to be vague; in the context of this application, forward-looking refers to approval considerations, labeling, promotion, and making sure these patients have access. That's what I meant by that. The conversation itself in the room had a rare type of feeling—at least in my view—where I actually felt peaceful. That's the way I would describe it: they were transparent and collaborative. All the elements necessary to make a decision have been on the table. On surprise, not really. Maybe my surprise was how much of the discussion turned into proper use of the drug—labeling and proper use discussions that normally occur later in the process. So when you are marching toward proper use in these conversations, I consider that exceptionally positive. The level of collaboration, the depth and breadth of the discussion, the number of people in the meeting, and the presence of leadership and support from leadership — all of that indicated this is an application that matters to the agency as much as it matters to us.
Regarding titration, the topic did come up, which is completely expected. I was positively surprised by how much alignment we have on that. While I cannot and should not predict what will end up in the label, I can tell you unequivocally that there is a very good understanding that when patients start ulixacaltamide, about 20% may have some tolerability issues that do not translate into safety concerns. If they stay on the drug, those issues tend to subside, and they experience substantial efficacy that we view as meaningful compared to other compounds. Any reasonable person would look to maximize tolerability pathways so patients can get to the benefit, and I think we're really close to figuring that out. How this translates to commercial: you can imagine that, even within a conservative addressable population at launch, there are many patients who could benefit; we want every patient to have the best experience and remain on therapy when appropriate. Megan can discuss what we are doing to support that commercial experience.
Sure. To recap, the focus out of the gates for the ulixacaltamide launch will absolutely be on ensuring a high-quality first experience so that we build physician confidence and durable patient persistence. From provider feedback via advisory boards and clinical engagement, it starts with clear expectations in the initial conversation: communicating that there can be a rapid onset of effect with meaningful change and that there might be some tolerability issues, which neurologists are comfortable managing. From a patient program and services perspective, Tim mentioned we're well underway in establishing the infrastructure we need to support this. We're building a modern hub that is fully integrated from the front end to receive the prescription through fulfillment, so we have line of sight on the first prescription fill and can provide services at the pharmacy and through our integrated network to help patients get started quickly and titrate through the early weeks. This is an absolute priority and we're feeling very good about where we are in the build. Physician enthusiasm is strong to get patients started on this therapy.
Our next question comes from François Brisebois from LifeSci Capital.
On relutrigine, I was wondering: you mentioned there are about 50 separate genetic etiologies involved in EMERALD. Would you say the study population is relatively enriched for indications which have a history of sensitivity to sodium channel blockers, or not?
Thanks, François. I'll hand that one to Steve to discuss.
Yes. Looking at the size of the trial, that spread of etiologies is precisely what you would expect given the distribution of prevalence in that group of patients. The mix of patients we anticipate who would be pharmacosensitive to a sodium channel mechanism is represented in that cohort.
Okay, great. Then can you comment on the powering of EMERALD? Based on the study numbers, is there like a placebo-level median percent change you're looking for or testing against?
Yes. With the caveat that this is a truly heterogeneous DEE study both genetically and non-genetically defined, there are many historical data we can reference. When you go through the analysis, there are a few points. First, overall response at the 50% benchmark is a clear and meaningful endpoint, and placebo response in such severely affected patients is typically very small. These patients are so severe: the median baseline countable seizures in EMERALD is over 50 per 28 days, so the likelihood of natural regression is very low. As you move up to 75% or 90% response, placebo rates become vanishingly small. So modeling was straightforward from our perspective, and we are very pleased with both the powering and, importantly, the mix of patients who are pharmacosensitive to the mechanism. Stay tuned for results, but we are bullish on what we expect to see.
Our next question comes from Kevin Strang of Goldman Sachs.
On vormatrigine: you alluded to the design being more important than the drug itself. Can you walk us through specific learnings from POWER1 on dose and design that give you confidence to restart the program?
Yes, absolutely. We'll reserve some details until we finalize the amended designs and present them, not as hedging but as a disciplined approach. A few high-level points: dose clearly played a role, and so did duration at a given dose. It's not only whether the dose was 20 or 30 mg, but the amount of time patients were on that dose mattered — six weeks at dose vs. longer made a difference. Other elements include the number of prior medication failures in the entry criteria, which was high and could be tightened. All of these parameters are relatively straightforward to adjust. Once we make these changes, and without overstretching expectations, we believe POWER2 and eventually POWER3 will show effects at levels consistent with the drug's potential in these populations. We're finalizing details with advisors and will provide a full update soon; we intend to start these studies.
Our next question comes from Tiago Fauth of Raymond James.
For EMERALD and Dravet comparisons: conventional sodium channel blockers can be contraindicated in Dravet because they can worsen seizures, yet you had strong preclinical data in Dravet models. What does that tell you about the mechanism of relutrigine relative to conventional sodium channel blockers? And what does that imply about potential activity across other DEEs? Also, how should we think about enrichment criteria like seizure burden to offset unknowns or risk from non-ion channel DEEs in the mix?
Great question. I'll hand to Steve to address the mechanism in detail, because he's the expert on the neurobiology.
Thanks, Marcio. Sodium channels play a central role in determining neuronal excitability and in epilepsy; they're the gatekeepers of excitability. When sodium channels themselves are altered by mutations, they are a clear target. Beyond that, sodium channels are a point of physiological convergence downstream of many different etiologies that lead to DEEs, whether genetic or acquired. Because of that downstream role, they remain viable targets even when the primary mutation is not in a sodium channel. In loss-of-function mutations, loss of sodium channel function can lead to compensatory upregulation of other elements that still produce excitability issues. Modulation of sodium channels can therefore control excitability broadly. Additionally, the way a sodium channel modulator interacts with channels is important: relutrigine's mechanism and profile distinguish it from other agents. That was our initial therapeutic hypothesis and we've continued to follow it through the trials.
Our next question comes from Douglas Tsao of H.C. Wainwright.
Congrats on the progress. On vormatrigine, you plan to restart POWER2 and POWER3 with different designs. Do you think those two studies will be enough to support a potential filing, given they will be different studies in design and what they aim to demonstrate?
Thanks, Doug. Yes, we do think those two studies can be sufficient to support a filing. There are steps ahead, including conversations with the agency to discuss potential labeling, but from a historical and policy perspective, we're optimistic. The regulatory landscape in epilepsy has evolved and we feel bullish about the path, though of course we will validate that through formal interactions.
Quick follow-up on relutrigine: given ongoing enrollment in EMERALD and ulixacaltamide programs, have you heard any clinician feedback about whether they are preferentially referring patients to one study versus the other based on mechanism, i.e., conscious or unconscious enrichment?
I get the point. While anecdotal conversations with clinicians can suggest that they might choose a study based on where they think a patient might best respond, that's not unexpected. For example, where serotonergic mechanisms are established, clinicians may try those first; that's reasonable for trial execution but would be a poor market strategy long-term. For EMERALD, we randomized about 200 patients relatively quickly, and the final mix suggests that many of the patients are those historically more likely to respond to our mechanism. Whether there was conscious or unconscious segmentation across sites, what matters is that both mechanisms can work and the market should welcome multiple mechanisms. We are supportive of multiple successful therapies for these conditions.
Our next question comes from Lin Tsai of Jefferies.
Thanks for the updates. On essential tremor, is there a chance approval could come earlier than expected, especially given the completed mid-cycle review and inspection? Will you be entering final labeling discussions soon? And how prepared would you be to launch in Q4 if there was an early approval?
I appreciate the question. The next formal steps include late-cycle discussion and label negotiations, and those are underway. As we mentioned earlier, we're not going to provide regulatory updates between now and the action date, but we have set the goal to be ready for launch well ahead of PDUFA for multiple reasons: it's the right thing to do, we have the capital to do it, and physicians and patients are already asking when the drug will be available. So we will be ready and are prepared to maximize the opportunity in the event of an earlier-than-expected approval.
Our next question comes from Yatin Suneja of Guggenheim.
Excellent updates. Staying with essential tremor: could you talk about the payer work you've done? Marcio, in the past you've discussed pricing — can you share payer feedback and how to think about step edits relative to propranolol and the fact that many patients are on generics?
Yes. From a payer perspective, we've been very active. We've done analytical benchmarking and engaged plan administrators proactively, and recently they've been reaching out to talk to us. I was positively surprised by their understanding of the need here and their interest in being ready on day one. Our planning assumptions include step edits through propranolol as a prudent scenario, although that's not necessarily what will happen broadly. Importantly, many essential tremor patients cannot medically take propranolol — about half of the market may be unable to use a beta blocker due to cardiac or other contraindications — so that represents a substantial portion of the addressable market. Also, the Essential3 study showed ulixacaltamide has additive efficacy on top of propranolol, so that supports access discussions. Regarding pricing, the more we talk to payers, the more confident we are that our initial pricing assumptions are grounded. While we are not disclosing a final price, our initial planning range was roughly $50,000 to $100,000 per year, and we are comfortable that range is appropriate to operate in general.
Our next question comes from Kambiz Yazdi of U.S. Bancorp BTIG.
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/SCN8A population?
Start with what's necessary and then what's possible. The necessary bar is statistical significance in the study — these patients have extremely high seizure burden and have tried many interventions, so demonstrating a consistent reduction in seizures is meaningful. Biologically, and as Steve discussed earlier, there are reasons to believe efficacy in broader DEEs could be similar, and potentially in some cases comparable to EMBOLD, though we will stay true to the data. We are hopeful and will provide more details as we progress.
Our next question comes from Jay Olson of Oppenheimer.
Following up on relutrigine: you've commented previously that pooled masked data from EMERALD show dynamics different from historical DEE placebo cohorts. Can you talk about the most important factor behind that observation? And how do you compare information across thresholds like 50%, 75% or 100% seizure reduction?
All response thresholds — 50%, 75%, 90%, 100% — are important and we track the full distribution. One point we haven't discussed as much is what happens when patients transition to open label. Many patients have been on open label for several months, and that data, combined with the double-blind information, departs from historical expectations. That departure is notable given the severity and seizure burden of these patients. While we must remain cautious and vigilant until study completion, these observations are encouraging.
Our next question comes from Ami Fadia of Needham & Company.
Two questions: for ulixacaltamide, what mix of patients do you expect across commercial, Medicare and Medicaid at launch? Where will initial patients come from — older longstanding ET patients or younger patients starting earlier? And on EMERALD, across the 50 etiologies, could response rates vary mechanistically across etiologies?
This launch will likely unfold in stages. Our conservative addressable population at launch is around two million patients. A majority — perhaps around three-quarters — will be Medicare or Medicare Advantage, so initially we'll see more older patients. Family members often drive interest and demand, and there will be uptake in younger cohorts over time. The prevalence of essential tremor increases with age, so the patient population will grow organically. We expect long-term double-digit market growth due to demographics. On EMERALD, response rates may not be identical across all etiologies — heterogeneity exists — but we expect consistent positive signals across the overall cohort.
The efficacy across etiologies is something we'll continue to analyze and report, but our expectation is consistent positive signals across the broad mix.
Thanks, Tim. We're hopeful and realistic: we expect consistent benefit across many etiologies and will let the data speak.
Our next question comes from Brian Skorney of Baird.
Can you characterize areas of focus for the agency in the mid-cycle review for ulixacaltamide? Which FDA groups took the most time — clinical, statistical, safety? Were senior leaders or specific reviewers present? Also, any insight into whether the FDA is thinking about DEA scheduling?
Those meetings are comprehensive and include the groups you mentioned — clinical, statistical, safety — and leadership representation given the importance of the application and its breakthrough designation. The meeting was collaborative and focused largely on how to get this drug to patients, with broad representation from relevant FDA divisions. There were no major surprises or findings. As for DEA scheduling, that was not a focal public topic in the meeting; much of the agenda was on public health and access. Overall, we view the meeting's tenor as very positive.
Our next question comes from Alex on for Danielle Brill of Truist.
Given the two mid-cycle reviews were close in time, were there noticeable differences in tenor, pushback or body language between the FDA reviews for ulixacaltamide versus relutrigine?
I would say no difference in body language; both meetings were collegial. Ulixacaltamide's application is larger with more people involved given the scope, but overall both reviews were collaborative and positive. The inspections closed with no findings and the mid-cycle discussions were productive for both applications.
Our next question comes from David Hoang of Deutsche Bank.
On ulixacaltamide's potential commercial launch: could you talk about the prescriber base? Will this be primarily neurologists, or would primary care physicians feel comfortable prescribing? What size sales force would you need to support a successful launch? And can you remind us of your latest assumptions on peak sales for ulixacaltamide?
I'll start with peak sales: we've been conservative in our planning and model peak sales around $10 billion globally. We believe that to be a conservative estimate given the unmet need and market dynamics. Megan can speak to the prescriber base and sales force sizing.
David, neurologists are our primary focus at launch due to their influence on essential tremor care and patient volume. Our call target sizing is roughly 13,000 to 15,000 accounts, which supports a field force of around 300. We are well underway with hiring and expect to be prepared for launch upon PDUFA.
Our next question comes from Josh on for Leonid Timichev of RBC Capital Markets.
For relutrigine, with initial patient targeting, will you go after the most severe patients or go more broadly earlier? How might that align with how clinicians typically use a novel antiseizure agent?
It's difficult to compare severity across conditions, but our feedback from the SCN2A Family Foundation meeting and clinicians indicates these patients are among the most severely affected. We'll emphasize proper use: thorough assessment and optimization of background medications before initiating relutrigine. Launch strategy will focus on appropriate patient selection to maximize benefit, retention and long-term access. We're getting strong feedback from physicians and patient groups.
Our next question comes from Rudy Li of Wolfe Research.
For ulixacaltamide, what gives you confidence that titration can improve discontinuation in practice? What data or evidence supports your titration proposal, and how should we think about discontinuation rates in the real world?
That's a great question and one we have spent a lot of time on, including discussions with the agency. We have data showing that if patients stay on the drug just a bit longer through early tolerability issues, the probability of both tolerating the drug and achieving benefit increases substantially. In clinical studies, patients are less likely to be biased toward staying on treatment compared to a real-world clinical office conversation where a physician and patient can work through early tolerability with more personalized support. We have mathematical and clinical evidence indicating that short extensions through early titration materially improve outcomes. Our proposed label language and our commercial patient-support hub are intended to give clinicians tools to manage those early weeks and help patients stay on therapy to realize benefit. We care about every patient who can benefit — beyond peak revenue, it's about delivering a therapy that works for people who currently have no targeted options.
Our next question comes from Orfia on for Ben Burnett of Wells Fargo.
Congrats on over-enrolling EMERALD. For relutrigine, can you share what proportion of EMERALD patients are on XCOPRI or another sodium blocker at baseline? What are your expectations for incremental efficacy in patients already on such agents? And on cash runway: given relutrigine and elsunersen are eligible for pediatric review vouchers, do you plan to monetize those on approval and is that contemplated in your runway?
We have a representative distribution of background medications among EMERALD participants. Many patients have tried multiple medications, and discontinuation and tolerability have been low in the study. We're confident in both the efficacy signal and safety profile relative to background therapies. Specific proportions on individual concomitant medications we'll report in future data releases.
On runway: our cash position gives us flexibility to invest in launches and build two field teams and supporting infrastructure. We do anticipate receiving a pediatric review voucher if relutrigine is approved for SCN2A and SCN8A given orphan designation, and that provides optionality. We view potential PRV proceeds as additional flexibility rather than central to our runway assumptions. If realized, they would enable continued investment but are not necessary for our runway plan into 2028.
This concludes the question-and-answer session. I would now like to turn it back to Marcio for closing remarks.
Yes. Thank you so much. I hope we were comprehensive today given the updates. It's absolutely amazing the palpable energy we have every single day here in the office with the sales team and in our interactions with physicians. As we move toward commercialization, we'll discuss the clinical and regulatory less and focus more on patients and access. I want to thank everyone — including critics and supporters — for feedback that helped us get here. I couldn't be prouder on behalf of patients; we receive stories every day from patients transitioning on to open-label programs, compassionate use, or emergency access, and particularly those wanting to be on ulixacaltamide. Those stories keep us going. Thanks enormously for your support and we look forward to continued conversations in the near future.
Thank you for your participation in today's conference. This does conclude the program, and you may now disconnect.