Praxis Precision Medicines Inc. (NASDAQ: PRAX) announced results from its EMBRAVE Part A trial of elsunersen, an experimental treatment for pediatric patients with SCN2A developmental and epileptic encephalopathy.

The randomized, placebo-controlled Phase 1/2 trial enrolled nine patients aged 2-12 years, who received either elsunersen or a sham procedure every four weeks for 24 weeks. The company reported a 77% placebo-adjusted seizure reduction from baseline with statistical significance (p=0.015).

According to the trial data, 71% of elsunersen-treated patients achieved greater than 50% seizure reduction by period 6, with 57% experiencing at least a 28-day period of seizure freedom. All patients treated with elsunersen showed improvements in sleep, motor function, muscle tone, attention or neuropsychomotor development, compared to no improvements in the placebo group.

The company stated that elsunersen was well-tolerated with no drug-related serious adverse events, no discontinuations and no neuroinflammation signals at doses up to 8 mg. Most treatment-emergent adverse events were characterized as mild to moderate.

Elsunersen is an antisense oligonucleotide designed to decrease SCN2A gene expression in patients with gain-of-function SCN2A mutations. The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, along with Orphan Drug Designation and PRIME designation from the European Medicines Agency.

The company is conducting the pivotal EMBRAVE3 study and plans to present additional results at upcoming scientific meetings. Praxis develops therapies for central nervous system disorders through its small molecule platform Cerebrum and antisense oligonucleotide platform Solidus.