Praxis Precision Medicines disclosed that its antisense oligonucleotide, elsunersen, achieved a 77% placebo‑adjusted seizure reduction in a Phase 1/2 EMBRAVE Part A trial of nine pediatric patients with SCN2A developmental and epileptic encephalopathy. 71% of the treated patients reached a >50% seizure reduction by period 6, and 57% experienced at least 28 days of seizure freedom. 100% of treated patients showed improvements in sleep, motor function, muscle tone, and attention, and no treatment‑related serious adverse events were reported.
The results represent the first statistically significant efficacy data for elsunersen and provide a strong foundation for the company’s ongoing EMBRAVE3 pivotal study. The data support a potential regulatory submission and position elsunersen as a first‑in‑class, disease‑modifying therapy for SCN2A‑DEE, a severe, drug‑resistant pediatric disorder with no approved targeted treatments.
Praxis’s financial position underpins the ability to pursue this program. The company reported a net loss of $303.3 million for 2025, but it holds a cash balance of $926 million as of December 31, 2025, and expects additional proceeds from a January 2026 offering to fund operations through 2028. The positive trial data strengthen the pipeline, enhance investor confidence, and could unlock a combined peak revenue potential of over $20 billion across its four late‑stage assets.
"We are thrilled to see the remarkable, consistent results from EMBRAVE Part A, showing 77% reduction in monthly seizures and disease‑modifying improvements in children with SCN2A early‑seizure onset DEE. We are well underway with our pivotal EMBRAVE3 study and look forward to sharing this placebo‑controlled data from the EMBRAVE Part A study with all key stakeholders," said Marcio Souza, president and chief executive officer.
Praxis’s shares traded higher following the announcement, driven by the strong efficacy and safety profile and the unmet need in SCN2A‑DEE. Investors viewed the 77% seizure reduction and the absence of serious adverse events as evidence that elsunersen could become the first disease‑modifying therapy for this rare disorder, thereby expanding the company’s commercial prospects.
SCN2A‑DEE is a severe genetic disorder characterized by early‑onset, drug‑resistant seizures and significant developmental delays. Elsunersen is an antisense oligonucleotide that reduces expression of the SCN2A gene, targeting the underlying gain‑of‑function mutations. The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and similar designations from the EMA, underscoring its potential to address a critical unmet medical need. The EMBRAVE3 study has been converted to a single‑arm, baseline‑controlled design in alignment with the FDA, reflecting the high unmet need and the company’s commitment to generating confirmatory data.
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