Huge pediatric epilepsy result
An early trial reported a new epilepsy drug reduced pediatric seizures by nearly 90% — a remarkably large effect size for severe childhood epilepsy, though the report notes the data are early. (x.com)
Zorevunersen (STK‑001) is an antisense oligonucleotide engineered to upregulate Nav1.1 expression from the nonmutant SCN1A allele. (clinicaltrials.gov) The results come from two open‑label Phase 1/2a multicenter trials—MONARCH in the U.S. and ADMIRAL in the U.K.—that enrolled 81 children aged 2–18, with 75 participants rolling into an open‑label extension. (news.northwestern.edu) Reported convulsive‑seizure reductions ranged from about 59% to as much as 91% across analyses, and participants who received two to three 70 mg doses showed a roughly 85% median drop at three months and 73% at six months. (epilepsy.org.uk) Investigators noted most adverse events were mild or moderate, but post‑lumbar‑puncture syndrome occurred in roughly 25% of patients and elevated cerebrospinal‑fluid protein was seen in about 45% during extensions. (emjreviews.com) Authors report sustained improvements in language, motor skills, behavior and quality‑of‑life measures during up to three years of follow‑up in the open‑label extension studies. (luriechildrens.org) A global randomized, double‑blind, sham‑controlled Phase 3 registrational trial called EMPEROR is underway targeting ~150 participants, with companies projecting a Phase 3 readout in mid‑2027 and a potential rolling NDA submission in the first half of 2027. (clinicaltrials.gov)
