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PRAX-562

Phase 2

SCN2A Encephalopathy | Small molecule | Other |Praxis Precision Medicines, Inc.|Last Updated: Jan 29, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment77
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05818553A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)PHASE2 ACTIVE NOT_RECRUITING 77Aug 2, 2023Mar 1, 2027Jan 29, 20268 United States, Israel +2
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Study Endpoints
Primary Endpoints
PART A (Cohorts 1 and 2) RDB: To evaluate the safety and tolerability of PRAX 562 in pediatric participants with SCN2A- and SCN8A- DEEs
16 weeks

Changes from baseline in monthly (28-day) motor seizure frequency

PART B (Cohorts 1 and 2) OLE: To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs
48 weeks

Incidence and severity of TEAEs

Secondary Endpoints
PART A (Cohorts 1 and 2) RDB: To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs
16 weeks
Plasma concentrations of PRAX-562
16 weeks
Seizure Frequency (OLE Extension)
48 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/PlaceboEXPERIMENTALEligible participants from each cohort will be randomized in a 1:1 ratio to either 0.5 milligrams/kilograms/day (mg/kg/day) PRAX-562 for 16 weeks (PRAX-562 arm) or 0.5 mg/kg/day PRAX-562 for 12 weeks and matching placebo for 4 weeks (PRAX-562/placebo arm) administered orally or via gastrostomy tube (G-tube).
Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562EXPERIMENTALEligible participants will receive 0.5mg/kg/day administered orally or via G-tube for up to 144 weeks.
Part A: Randomized, Double-Blind 1.0 mg/kg/day PRAX-562 or PRAX-562/PlaceboEXPERIMENTALEligible participants from each cohort will be randomized in a 1:1 ratio to either 1.0 milligrams/kilograms/day (mg/kg/day) PRAX-562 for 16 weeks (PRAX-562 arm) or 1.0 mg/kg/day PRAX-562 for 12 weeks and matching placebo for 4 weeks (PRAX-562/placebo arm) administered orally or via gastrostomy tube (G-tube).
Open-Label Extension Treatment 1.0 mg/kg/day PRAX-562EXPERIMENTALEligible participants will receive 1.0 mg/kg/day administered orally or via G-tube for up to 144 weeks.
Interventions
NameTypeDescription
PRAX-562DRUGOnce daily oral or G-tube treatment.
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Eligibility Criteria
Age Range1 Year — 18 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Has a documented variant in SCN2A with onset of seizures occurring in the first 3 months of life or has a diagnosis of SCN8A-DEE supported by both clinical and genetic findings. * Has a seizure frequency as follows: * At least 8 countable motor seizures in the 4 weeks immed...

Countries:United StatesIsraelSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05818553primaryCompletionDate: changed
LOWMay 24, 2026NCT05818553studyFirstPostDate: changed