Praxis Precision Medicines, Inc. Common Stock 2026 Q2 Earnings Call

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 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 Steven 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 become a commercial company. This quarter is the one where that stopped being a plan and it started materializing into the organization. We have two NDAs in late-stage review with the FDA, with both approvals expected in about 6 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 1st launch hired and trained, and a distribution network established and inventory being built. I want to spend most of my time today on some key regulatory developments and what we have been building. Let me start with ulixacaltamide.

Essential tremor affects over 7 million Americans, and there is still no FDA-approved therapy developed specifically to treat the condition. With the potential approval coming up by January next year, ulixacaltamide is poised to change that. Speaking about the NDA review, the FDA completed its mid-cycle communications with us and 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 are 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 lifecycle 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 ulixa further. That work is about expanding the value for patients and Praxis way beyond the initial launch year. Turning to relutrigine. SCN2A and SCN8A are amongst the most severe epilepsies we know of. Seizure onset in infancy, profound developmental delays, and no approved treatment. The addressable population is roughly 10,000 patients in the United States.

As we disclosed last quarter, we submitted additional sensitive 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 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 ulixa, 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 etiologies, amongst many others not genetically defined. That 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 a supplemental NDA approval in 2027. It is 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, the centralized study for ET, as well as the interim analysis, we're extremely pleased with the outcome of the inspections. One note on how we communicate from now on. Given the stage of discussions on both applications, we do not intend to provide further regulatory updates until the expected action dates. I would ask you to read our silence between now and January as discipline rather than a signal of any kind. Let me turn to 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 endpoints of reduction in monthly focal seizures frequency from baseline to week 12.

It did meet a key secondary endpoint with a significantly greater proportion of patients on 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 finding says 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're 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 DE caused by gain-of-function variant based on the results of the EMBRAVE Part A study. That's our third breakthrough designation since July last year. Three designations across three different assets on two different platforms. 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? Thank you, Marcio, and good morning, everybody.