Servier has enrolled the first patient in the United States in a Phase Ib/II first-in-human clinical study of an antisense oligonucleotide (ASO) therapy for children with KCNT1-related developmental and epileptic encephalopathy (KCNT1-DEE). The trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the investigational ASO in a rare, severe pediatric epilepsy syndrome with no curative or disease‑modifying treatments.
KCNT1-DEE is caused by pathogenic variants in the KCNT1 gene that lead to an early‑onset epilepsy syndrome associated with profound developmental impairment and high mortality in children. In many affected infants, seizures begin within the first days or months of life, and some never leave the hospital. Servier’s ASO candidate is intended to target the genetic cause of the disorder by degrading KCNT1 mRNA, with the aim of modifying disease course. The company said the compound has been shown to be well tolerated in preclinical studies.
Servier framed the trial as part of a broader neurology strategy focused on accelerating drug development for rare neurological disorders and highlighted that the ASO was discovered and developed by its internal research teams. Patient advocates from the KCNT1 Epilepsy Foundation described the first U.S. enrollment as a meaningful step for families who have been waiting for targeted treatments and emphasized the urgency of advancing effective therapies, given the rapid and devastating progression of the disease.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!