Praxis Converts Elsunersen Study to Single-Arm Design After FDA Meeting

By Elias Schisgall

Praxis Precision Medicines is converting its EMBRAVE3 study of elsunersen for children suffering from a developmental disease into a single-arm study after meeting with the Food and Drug Administration.

The pharmaceutical company said the study would be converted from a double-blind, sham-controlled study into a single-arm, baseline-controlled study, allowing Praxis to move through the trial quicker.

The study will measure the medicine's effect on seizures among children with early-onset SCN2A developmental and epileptic encephalopathy, a developmental disease characterized by seizures among newborns.

Praxis said data from the study is expected in the first half of 2026.

"The Agency's recognition of both the urgency of the unmet need and the strong mechanistic rationale for elsunersen enables us to move with greater clarity and speed," Praxis Chief Executive Officer Marcio Souza said. "Converting 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."

Write to Elias Schisgall at elias.schisgall@wsj.com

(END) Dow Jones Newswires

December 09, 2025 17:00 ET (22:00 GMT)