Praxis Precision Medicines (NASDAQ:PRAX) used its fourth-quarter and full-year 2025 earnings call to highlight multiple regulatory and clinical milestones across its central nervous system portfolio, including two New Drug Application (NDA) submissions and several upcoming data catalysts. Management also outlined expanding pre-commercial preparations and reported a significantly strengthened cash position following recent equity financings.
Two NDA submissions and a shift toward commercialization
Chief Executive Officer Marcio Souza said 2025 featured “significant clinical achievements and regulatory events” across the company’s portfolio and noted Praxis has now submitted two NDAs: ulixacaltamide for essential tremor and relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). Souza said the company views 2026 as a continuation of portfolio expansion and part of a “transformation into a commercial company.”
Ulixacaltamide: Essential3 results, Breakthrough designation, and launch planning
Souza reviewed the company’s October disclosure of positive top-line results from the Essential3 program in essential tremor. He said Study 1 met its primary and all key secondary endpoints, including improvements in mADL11, disease progression rate, PGI, and CGI. Study 2 met its prespecified primary endpoint, showing superior maintenance of effect during the randomized withdrawal phase.
Based on the data package and the lack of disease-specific therapies with similar results, Praxis received FDA Breakthrough Therapy designation in December and held a pre-NDA meeting that month. Souza said the company has “recently completed the NDA submission to the FDA” and that commercial preparations are underway, including hiring and launch infrastructure. Praxis plans to begin a medical education campaign at the American Academy of Neurology (AAN) annual meeting in April, where it expects to present additional Essential3 data across roughly 15 presentations, including oral presentations with detailed clinical-program discussion geared toward neurologists.
On the call, management discussed titration and tolerability. Souza said the company proposed label language that includes both the trial titration schedule (20 mg for 7 days, 40 mg for 7 days, then 60 mg) and an alternative schedule discussed with FDA, emphasizing that final labeling decisions rest with the agency. He said FDA did not expect Praxis to conduct additional pre-approval studies on titration. In response to another question, Souza added the alternative approach would not start at a lower dose, but would keep patients at 20 mg longer, as the company observed tolerability issues tended to subside after a few extra days for some patients.
Souza estimated more than 7 million people in the U.S. live with essential tremor, with about 2 million considered “immediate needs for therapy” or an addressable population. He said Praxis believes ulixacaltamide has peak potential of over $10 billion annually. He also noted the company’s decision not to request priority review for ulixacaltamide was strategic, citing business considerations including the Medicare Part D-heavy nature of the population and the Inflation Reduction Act’s negotiation dynamics over the product lifecycle.
Relutrigine: NDA filed in SCN2A/SCN8A and broader DEE expansion plans
Souza said Praxis presented EMBOLD study data at the American Epilepsy Society (AES) meeting in December, describing “overwhelming efficacy” with clinically meaningful and statistically significant changes in seizure and developmental endpoints such as disruptive behavior, alertness, and communication. He characterized relutrigine’s effect as rapid and durable, with continued deepening over time, and said the company submitted its NDA earlier in 2026 in alignment with FDA.
Relutrigine has Rare Pediatric Disease designation, which management noted makes it eligible for a Pediatric Review Voucher upon approval. Souza estimated the initial addressable U.S. population for SCN2A and SCN8A DEE at roughly 10,000 patients, while noting there are more than 200,000 patients with DEE overall. The ongoing EMERALD study is evaluating relutrigine in a broader, phenotypically defined DEE population; Souza said there are no subgroup quotas for different etiologies in the trial design and described enrollment as progressing with strong physician engagement. If the initial NDA is approved and EMERALD is positive, he said Praxis expects to submit a supplemental NDA for broad DEE by 2027.
In discussing review strategy, Souza said the company requested priority review for relutrigine, in part because it is a smaller dataset in a rare indication compared with ulixacaltamide. Management also described pre-launch work for relutrigine, including key hires and inventory build, while noting the initial commercial focus will be more concentrated than ulixacaltamide’s broader market.
Vormatrigine and elsunersen: Upcoming readouts and registrational plans
For vormatrigine, a once-daily therapy in development for common epilepsies, management highlighted Phase 2 RADIANT data presented at AES. Souza said 58% of patients achieved at least a 50% seizure reduction at week 1 without titration, and that patients continuing to later timepoints showed a 100% median weekly seizure reduction at week 9 sustained through week 16. He also said the drug improved efficacy on top of other antiseizure medications.
Praxis expects top-line POWER1 results in the second quarter of 2026 and anticipates completing POWER2 enrollment by year-end. The company also plans to initiate POWER3, a monotherapy study, in the first half of 2026. Souza said POWER3 is intended to support movement toward earlier lines of therapy, though he noted it is not required for registration. In Q&A, Souza added that in open-label experience physicians are reducing or eliminating background therapies while maintaining seizure control, which he said supports the value proposition of a therapy that may allow sequential reduction of other medications.
For elsunersen, an antisense oligonucleotide for gain-of-function SCN2A DEE, Souza said FDA agreed in September to simplify the EMBRAVE3 registrational trial from a double-blind sham-controlled design to a single-arm, baseline-controlled study of about 30 patients. He said enrollment is moving quickly, with completion expected later in 2026 and a potential NDA in 2027. Separately, Praxis expects to report top-line results from the original nine patients in EMBRAVE Part A in the first half of 2026. Management noted elsunersen also has Rare Pediatric Disease designation and could qualify for a pediatric review voucher upon approval.
Financial results and cash runway
Chief Financial Officer Tim Kelly reported fourth-quarter operating expenses of $97 million, including $77.5 million in R&D and $19.5 million in G&A, compared with $71.4 million in operating expenses in Q4 2024. Full-year 2025 operating expenses were $326 million versus $209 million in 2024, with increases attributed to spending on the company’s Cerebrum and Solidus platforms to advance clinical programs.
Praxis ended the fourth quarter with $926 million in cash, equivalents, and marketable securities, up from $469 million at year-end 2024, driven primarily by net proceeds from an October 2025 follow-on offering and at-the-market stock sales, offset by operating cash use. Kelly said a January 2026 public offering added $621 million, bringing pro forma cash to approximately $1.5 billion, which he said is expected to fund operations into 2028. He also said the company expects a significant increase in spending in 2026 tied to commercial launch activities and continued pipeline investment.
Looking ahead, Souza said Praxis plans to hold an R&D day next quarter and a subsequent commercial day to outline launch strategy and readiness for ulixacaltamide and relutrigine.
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.
