Press Release: Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2026 Financial Results

FDA accepted the new drug application (NDA) for ulixacaltamide in Essential Tremor with a PDUFA target action date of January 29, 2027, and the NDA for relutrigine, with priority review, in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) with a PDUFA target action date of September 27, 2026

EMBRAVE Part A study results showed elsunersen treatment led to a 77% placebo-adjusted reduction in monthly seizures and demonstrated disease-modifying improvements in patients with early-seizure onset SCN2A-DEE

Topline results from the POWER1 study of vormatrigine in focal onset seizures expected in Q2 2026

Recruiting completed for relutrigine EMERALD study in broad DEEs, with topline results expected in Q4 2026

Cash and investments of approximately $1.4 billion as of March 31, 2026 maintains runway into 2028

Conference call today, May 7, 2026 at 8:30am

BOSTON, May 07, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system $(CNS)$ precision neuroscience biopharmaceutical company, today provided a corporate update and reported financial results for the first quarter of 2026.

"This quarter marks yet another inflection point for Praxis, with FDA acceptance of NDAs for both ulixacaltamide and relutrigine, positioning us for two U.S. launches within the next eight months as we accelerate our commercial roadmap to ensure readiness and market access upon approval. Our clinical pipeline continues to deliver, with EMBRAVE Part A data showing a 77% placebo-adjusted reduction in monthly seizures and disease-modifying improvements for elsunersen in early-onset SCN2A-DEE. Looking ahead, we expect topline results from the POWER1 study of vormatrigine in focal epilepsy this quarter, followed by the EMERALD readout in broad DEEs in the fourth quarter. Importantly, we remain well-capitalized to execute on this catalyst-rich period and deliver these therapies to patients," said Marcio Souza, president and chief executive officer.

Recent Highlights and Anticipated Milestones

Cerebrum$(TM)$ Small Molecule Platform

Ulixacaltamide for Essential Tremor $(ET)$: ET is one of the most common movement disorders, affecting approximately seven million patients in the U.S. Ulixacaltamide was the first investigational therapy to demonstrate positive results in a Phase 3 program in ET and was granted Breakthrough Therapy Designation by the FDA in December 2025.

   -- The FDA has accepted Praxis' NDA for ulixacaltamide for the treatment of 
      ET and has set a target action date under the Prescription Drug User Fee 
      Act (PDUFA) of January 29, 2027. 
 
   -- Commercial preparations and pre-launch activities continue to accelerate: 
 
          -- ESSENTIAL to me disease education campaign for healthcare 
             providers was launched in April 2026 to raise awareness of 
             Essential Tremor. 
 
          -- Commercial organization leadership hired, with build of 
             cross-functional commercial organization and infrastructure 
             on-track. 
 
          -- Distribution network is established, with commercial inventory 
             build in-progress ahead of launch. 
 
   -- At the recent American Academy of Neurology $(AAN)$ Annual Meeting, Praxis 
      shared several oral presentations and posters on ulixacaltamide. The oral 
      plenary session on Phase 3 results from Essential3 was recognized as an 
      Abstract of Distinction in Movement Disorders by the AAN. 

Relutrigine for DEEs: Relutrigine is a sodium channel modulator designed to precisely target the hyperexcitable state of sodium-channels, with therapeutic potential across developmental epilepsies. Relutrigine has been granted Breakthrough Therapy Designation and Orphan Drug Designation by the FDA.

   -- The FDA has accepted with priority review the relutrigine NDA for the 
      treatment of SCN2A and SCN8A DEEs, with a PDUFA target action date of 
      September 27, 2026. If approved, relutrigine will be the first therapy 
      for SCN2A/8A DEE and be eligible for a Pediatric Review Voucher. 
 
   -- Preparations for the commercial launch of relutrigine are progressing 
      well, including continued hiring within commercial and medical teams, 
      building sufficient inventory, establishing a comprehensive patient 
      support program and engaging with payers to ensure timely market access 
      upon potential approval. 
 
   -- Recruitment for the EMERALD study in broad DEEs is complete, with topline 
      results expected in the fourth quarter of 2026. Assuming successful 
      initial NDA approval of relutrigine, the EMERALD study, if positive, 
      would serve as the basis for a supplemental NDA submission in 2027. 

Vormatrigine for Focal Onset Seizures (FOS) and Generalized Epilepsy: An estimated 3.5 million people in the U.S. suffer from common epilepsies. Sodium channel therapy is the cornerstone of treatment for patients with epilepsy, yet currently approved drugs have significant safety and efficacy limitations. Vormatrigine is the most potent sodium-channel modulator ever developed for epilepsy and is designed to precisely target the hyperexcitable state of sodium-channels in adult common epilepsies.

   -- The POWER1 Phase 3 study for FOS is on track for topline results in the 
      second quarter of 2026. 
 
   -- POWER2, the second Phase 3 study for vormatrigine in FOS, continues to 
      progress towards completion in the second half of 2026 with topline 
      results anticipated in 2027. 
 
   -- The POWER3 study to evaluate vormatrigine as a monotherapy remains on 
      track to commence in the first half of 2026. 

Solidus(TM) Antisense Oligonucleotide $(ASO)$ Platform

   -- Elsunersen for early-seizure-onset SCN2A DEE: SCN2A early-onset DEE is a 
      rare, genetic epilepsy characterized by early-onset seizures and severe 
      impact on development. 
 
          -- Topline results from the EMBRAVE Part A Phase 1/2 study evaluating 
             SCN2A early onset seizure patients were announced in April 2026 
             and presented at AAN: 
 
                 -- Treatment with elsunersen led to a significant 77% 
                    placebo-adjusted seizure reduction from baseline (p=0.015). 
 
                 -- 71% of patients treated with elsunersen achieved >50% 
                    seizure reduction by period 6, with results sustained 
                    during the open label extension for up to one year. 
 
                 -- 57% of patients treated with elsunersen had at least a 
                    28-day period of seizure freedom. 
 
                 -- 100% of patients treated with elsunersen experienced 
                    improvements in sleep, motor function, muscle tone, 
                    attention or neuropsychomotor development compared to no 
                    observed improvements in placebo group. 
 
                 -- Elsunersen was well-tolerated, with no drug-related SAEs, 
                    no discontinuations and no neuroinflammation signals at 
                    doses up to 8 mg. 
 
                 -- Clinical updates from the elsunersen Emergency Use Program 
                    were also presented at AAN, highlighting durable seizure 
                    reduction and meaningful quality-of-life improvements 
                    across six patients treated globally, with more than 100 
                    doses administered to date. 
 
          -- Enrollment is progressing in the EMBRAVE3 registrational trial, 
             with topline results expected in 2027. 
 
   -- Praxis remains on track to nominate a development candidate for each of 
      its three early stage ASO therapeutic initiatives in the first half of 
      2026: 
 
          -- PRAX-080 is focused on targeting PCDH19 mosaic expression 
             disorder. 
 
          -- PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) 
             mutations, a leading cause of severe intellectual disability and 
             epilepsy in DEEs. 
 
          -- PRAX-100 targets SCN2A LoF mutations, the predominant genetic link 
             to de novo autism spectrum disorders. 

First Quarter 2026 Financial Results:

As of March 31, 2026, Praxis had $1.4 billion in cash, cash equivalents and marketable securities, compared to $926.1 million in cash, cash equivalents and marketable securities as of December 31, 2025. This increase of $473.9 million was primarily attributable to net proceeds from Praxis' January 2026 follow-on public offering and interest income on marketable securities, partially offset by cash used in operations. The Company's cash, cash equivalents and marketable securities as of March 31, 2026 are expected to fund operations into 2028.

Research and development expenses were $78.0 million for the first quarter of 2026, compared to $60.8 million for the first quarter of 2025. The increase in research and development expenses of $17.2 million was primarily attributable to $9.2 million in increased expenses related to the Company's Cerebrum(TM) platform, $3.8 million in increased personnel-related expenses and $3.0 million in increased expenses related to the Company's Solidus(TM) platform.

General and administrative expenses were $27.9 million for the first quarter of 2026, compared to $13.9 million for the first quarter of 2025. The increase in general and administrative expenses of $14.0 million was primarily attributable to $9.8 million in increased personnel-related expenses and $3.5 million in increased professional expenses.

Praxis incurred a net loss of $92.6 million for the first quarter of 2026, including $17.1 million of stock-based compensation expense, compared to $69.3 million for the first quarter of 2025, including $8.8 million of stock-based compensation expense.

As of March 31, 2026, Praxis had 27.9 million shares of common stock outstanding.

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May 07, 2026 08:00 ET (12:00 GMT)