Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2025 Financial Results

On track for six major study readouts across four programs over the next 12 months

Ready to initiate pivotal studies in two developmental and epileptic encephalopathy (DEE) programs in mid-year 2025: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A GoF with elsunersen

Vormatrigine continues to generate a best-in-class safety profile with new data demonstrating no food effect and higher dosing tolerability

Praxis to host a virtual investor event on Friday, May 2, 2025 to discuss its DEE portfolio [registration link]

Cash and investments of $472 million as of March 31, 2025, maintains runway into 2028

BOSTON, May 02, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system $(CNS)$ disorders characterized by neuronal excitation-inhibition imbalance, today provided a corporate update and reported financial results for the first quarter 2025.

"We have made substantial progress in advancing our promising late-stage epilepsy portfolio and are firing on all cylinders through this unpredictable time in the industry," said Marcio Souza, president and chief executive officer of Praxis. "With multiple upcoming catalysts and data readouts, we expect a transformative year at Praxis. In the near-term, we anticipate topline results for vormatrigine from the RADIANT study in epilepsy by mid-2025, and from the POWER1 study in the second half of 2025. Vormatrigine continues to demonstrate an ideal profile and strong competitive differentiation"

Mr. Souza continued, "The registrational cohort of the EMBOLD study is recruiting strongly and is on-track for topline results no later than the first half of 2026, and we will soon initiate the registrational EMERALD study to investigate relutrigine more broadly in DEEs. The EMBRAVE study with elsunersen is progressing very well, with topline results expected in the first half of next year to support the registrational package, and we will initiate the Phase 3 EMBRAVE3 trial for elsunersen by mid-2025. While the interim analysis of Essential3 study 1 for ulixacaltamide was not what we expected, we are on track to complete both study 1 and study 2 for topline readout in the third quarter of this year. Our solid financial position ensures we have sufficient funding for all studies supporting our late-stage programs, with runway into 2028."

Recent Highlights and Anticipated Milestones

Cerebrum$(TM)$ Small Molecule Platform

   -- Vormatrigine for Focal Onset Seizures $(FOS.AU)$ 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 the currently approved drugs have significant safety and 
      efficacy limitations. Vormatrigine is the most potent sodium-channel 
      modulator ever designed to precisely target the hyperexcitable state of 
      sodium-channels in adult common epilepsies. 
 
          -- At the American Association of Neurology $(AAN)$ conference earlier 
             this quarter, Praxis presented further data from recently 
             completed Phase 1 studies [link], where vormatrigine continued to 
             demonstrate an ideal profile with strong competitive 
             differentiation. 
 
                 -- Results from an additional 45 mg multiple ascending dose 
                    $(MAD.AU)$ cohort demonstrated a 20x exposure on the MES EC50 
                    scale achieved in subjects, the most of any anti-seizure 
                    medicine, with a tolerable safety profile. 
 
                 -- The Phase 1 food effect study demonstrated that food intake 
                    does not significantly impact vormatrigine absorption and 
                    therefore vormatrigine can be administered with or without 
                    food, which increases flexibility in dosing and ease of use 
                    for patients. 
 
          -- Enrollment and progress across the ENERGY program continues to 
             advance 
 
                 -- Praxis provided an update on the EMPOWER registrational 
                    study [link]at AAN, which continues to attract epilepsy 
                    patients and be a source for identifying candidate patients 
                    for the RADIANT and POWER1 studies, with over 3,000 
                    patients registered to date 
 
                 -- The RADIANT Phase 2 study in FOS and generalized epilepsy 
                    is progressing, with topline results expected mid-year 2025 
 
                 -- The POWER1 Phase 2/3 registrational study for treatment 
                    resistant FOS is enrolling, with topline results expected 
                    in the second half of 2025 
 
                 -- The POWER2 Phase 2/3 registrational study for treatment 
                    resistant FOS is on track to initiate in the second half of 
                    2025 
 
   -- Relutrigine for DEEs: Relutrigine is a sodium channel modulator with 
      therapeutic potential across developmental epilepsies. Relutrigine is 
      currently being evaluated in the EMBOLD study in SCN2A and SCN8A DEEs, 
      and we will be initiating the EMERALD study in a broader, pan-DEE patient 
      population. Relutrigine has Rare Pediatric Disease Designation in three 
      indications: SCN1A epilepsy (Dravet syndrome), SCN2A and SCN8A DEEs 
 
          -- The EMBOLD registrational cohort 2 continues to enroll strongly, 
             with topline results expected no later than the first half of 
             2026, followed by potential NDA filing. 
 
          -- Praxis has completed discussions with the FDA on a trial for broad 
             DEEs and will initiate the EMERALD registrational study in 
             mid-2025. The study design will be shared at today's DEE virtual 
             analyst event. 
 
   -- Ulixacaltamide for Essential Tremor $(ET)$: ET is an inadequately managed, 
      undertreated and high burden disease with a prevalence of seven million 
      patients in the U.S. The Essential3 program is the first Phase 3 study in 
      the disease and includes two registrational studies: Study 1 is a 
      parallel design, placebo-controlled study (N=400) and Study 2 is a 
      randomized withdrawal study (N=200). Since beginning recruitment in 
      November 2023, over 200,000 patients have demonstrated interest in 
      participating in the study. 
 
          -- On February 28, Praxis shared the results of a pre-planned interim 
             analysis of Study 1. The Independent Data Monitoring Committee 
             recommended that the study be stopped for futility, due to the 
             results being unlikely to meet the primary efficacy endpoint under 
             the parameters set by the statistical model. 
 
          -- Praxis has decided to continue the studies to completion and will 
             share topline results in the third quarter of 2025. After 
             reviewing the results of both studies, Praxis will make a decision 
             if there is sufficient evidence to support an NDA filing. 

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

   -- Elsunersen for early-seizure-onset SCN2A DEE: SCN2A GoF-DEE is a rare, 
      genetic epilepsy characterized by early-onset seizures and severe impact 
      on development. Praxis has two studies supporting its registrational 
      filing. 
 
          -- The EMBRAVE Part A Phase 1/2 study is continuing to enroll. 
             Patients are randomized 3:1 drug to sham for a six-month period on 
             a once-monthly dose, with the potential to escalate from 1 mg to 8 
             mg. Topline results are expected in the first half of 2026. 
 
          -- The EMBRAVE3 registrational study for SCN2A GoF-DEE is on track to 
             initiate in mid-2025. Cohort 1 of the study will enroll up to 40 
             patients ages 2 to 18 years, 1:1 between drug and sham for a 
             six-month period with once monthly intrathecal dosing of 1 mg per 
             visit, with a primary endpoint measuring reduction in seizure 
             frequency. Cohorts 2 and 3 will evaluate patients from ages 1 to 2 
             and 0 to 1 years, respectively. 
 
          -- In April 2025, the experience of an emergency-use patient dosed on 
             elsunersen was published in Nature Medicine [linkl providing 
             preliminary insights on the safety and efficacy of elsunersen in a 
             preterm infant with early-onset SCN2A developmental and epileptic 
             encephalopathy. 
 
   -- Praxis remains on track to nominate a development candidate for each of 
      its early stage ASO therapeutic initiatives in 2025 
 
          -- PRAX-080: Focused on targeting PCDH19 mosaic expression 
 
          -- PRAX-090: Designed to address SYNGAP1 loss-of-function (LoF) 
             mutations, a leading cause of severe intellectual disability and 
             epilepsy in DEEs. 
 
          -- PRAX-100: Targeting SCN2A LoF mutations, the predominant genetic 
             link to de novo autism spectrum disorders $(ASD.NZ)$. 

First Quarter 2025 Financial Results:

As of March 31, 2025, Praxis had $472.0 million in cash, cash equivalents and marketable securities, compared to $469.5 million in cash, cash equivalents and marketable securities as of December 31, 2024. The increase of $2.5 million is primarily attributable to net proceeds from at-the-market offerings of common stock offset by cash used in operating activities. The Company's cash, cash equivalents and marketable securities as of March 31, 2025 are expected to fund operations into 2028.

(MORE TO FOLLOW) Dow Jones Newswires

May 02, 2025 08:30 ET (12:30 GMT)