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.
FDA Reviews Advance Without Planned Advisory Committees
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.
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.
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.
Commercial Preparation and Market Access Plans
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.
Pipeline Updates Include EMERALD Enrollment and Vormatrigine Redesign
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.
Second-Quarter Spending and Cash Position
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.
About Praxis Precision Medicines (NASDAQ:PRAX)
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.
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