Praxis Precision Medicines announced that the U.S. Food and Drug Administration approved a conversion of its EMBRAVE3 study of the antisense oligonucleotide elsunersen into a single‑arm, baseline‑controlled design. The change follows a Type C meeting held on December 09 2025 in which the company presented data and received the agency’s agreement to proceed with the new design.
Under the revised protocol, 30 patients with SCN2A‑developmental and epileptic encephalopathy (DEE) will receive 24 weeks of active treatment followed by an open‑label extension. The study will enroll all participants in a single arm, eliminating a placebo group while preserving a rigorous, approvable pathway. The primary endpoint will be the change from baseline in countable motor seizures, and the study will also collect safety and pharmacodynamic data.
The FDA’s willingness to accept a single‑arm design is a significant de‑risking event for Praxis. By reducing the number of patients from 40 to 30 and eliminating a control arm, the company can accelerate data collection and shorten the time to regulatory submission. The change also positions elsunersen for a potential breakthrough therapy designation and orphan drug status, which could provide accelerated review and market exclusivity benefits.
Analysts responded positively to the regulatory alignment. Following the announcement, several firms raised their price targets for Praxis, with Guggenheim increasing its target to $760.00. The market reaction reflects confidence that the accelerated pathway will reduce uncertainty and speed the path to approval for a disease‑modifying therapy in a rare, high‑need population.
CEO Marcio Souza emphasized the patient‑centric impact of the decision: “The FDA’s agreement to convert EMBRAVE3 to a single‑arm, baseline‑controlled study ensures that every child entering the trial will receive active treatment from day one, while preserving a rigorous and approvable pathway.” He added that the move aligns with Praxis’s broader pipeline strategy, which includes ulixacaltamide for essential tremor and relutrigine for SCN2A/SCN8A DEEs.
Overall, the FDA’s approval of the accelerated trial design marks a pivotal milestone for Praxis. It not only de‑risks the clinical program for elsunersen but also signals the agency’s support for innovative trial designs in rare neurological disorders, potentially benefiting the company’s broader precision‑neurology portfolio.
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