Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222 - Fixed Doses
Elsunersen · 2 trials · 4 indications
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following the 6th dose administration in the confirmatory phase.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 Arm 1: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 2: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 3: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Preliminary Safety | EXPERIMENTAL | Open-label PRAX-222 |
| Dose Escalation - PRAX-222 | EXPERIMENTAL | Initial dose escalation consisting of double-blind ascending doses of PRAX-222 |
| Dose Escalation - Placebo | PLACEBO_COMPARATOR | Double-blind placebo procedure |
| Optional Dose Escalation - PRAX-222 | EXPERIMENTAL | Optional dose escalation consisting of double-blind ascending doses of PRAX-222 |
| Optional Dose Escalation - Placebo | PLACEBO_COMPARATOR | Double-blind placebo procedure |
| Confirmatory Dosing - PRAX-222 | EXPERIMENTAL | Double-blind fixed-dose PRAX-222 |
| Confirmatory Dosing - Placebo | EXPERIMENTAL | Double-blind placebo procedure |
| Open-label PRAX-222 | EXPERIMENTAL | Open-label PRAX-222 |
| Name | Type | Description |
|---|---|---|
| 1mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| 0.5mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| PRAX-222 - Initial Dose | DRUG | PRAX-222 |
| PRAX-222 - Initial Ascending Doses | DRUG | Ascending doses of PRAX-222 |
| PRAX-222 - Optional Ascending Doses | DRUG | Escalation of PRAX-222 dose(s) |
| PRAX-222 - Fixed Doses | DRUG | Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222 |
| Placebo | PROCEDURE | Placebo procedure |
Inclusion Criteria: * Has a documented Gain of Function SCN2A variant confirmed through genetic testing. * Has onset of seizures prior to 3 months of age. * Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period. Exclusion Criteria: * Has any cli...
Elsunersen is an investigational RNA therapy being developed for SCN2A developmental and epileptic encephalopathy (SCN2A-DEE), a severe early-onset epilepsy syndrome. It is also being studied in epileptic encephalopathy associated with SCN2A variants. The drug is not yet approved for any indication and remains in clinical development.
Elsunersen targets SCN2A, the gene encoding the voltage-gated sodium channel Nav1.2. As an antisense oligonucleotide (ASO), it is designed to modulate SCN2A expression. This target class is denoted by the -sen suffix. The therapy aims to address the underlying genetic cause of SCN2A-DEE.
Elsunersen is being developed by Praxis Precision Medicines, Inc., which trades on the Nasdaq under the ticker PRAX. Praxis is a clinical-stage biopharmaceutical company focused on therapies for central nervous system disorders.
Elsunersen is in Phase 3 clinical development. The Phase 3 trial, NCT07019922, is currently recruiting pediatric participants with SCN2A-DEE. An earlier Phase 1 trial, NCT05737784, is active but no longer recruiting. Elsunersen has not been approved by the FDA.
Elsunersen is being evaluated in two registered clinical trials. The Phase 3 trial NCT07019922 is recruiting pediatric participants with SCN2A-DEE in the United States, Brazil, Germany, and Italy, with an enrollment target of 40. The Phase 1 trial NCT05737784, with 60 participants, is active but not recruiting in the United States and Brazil.
Yes, elsunersen is also known as PRAX-222. The names PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222 - Fixed Doses, and 1mg elsunersen refer to the same investigational compound. Elsunersen is the international nonproprietary name for PRAX-222.