French pharmaceutical company Servier said on Thursday that the first patient has been enrolled in the US in a Phase Ib/II first-in-human study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of its antisense oligonucleotide (ASO) molecule in children with KCNT1-related developmental and epileptic encephalopathy (KCNT1-DEE).

According to Servier, pathogenic variants in the KCNT1 gene lead to a devastating early-onset epilepsy syndrome associated with profound developmental impairment. The disease has a high degree of mortality among children, and there are currently no curative or disease-modifying treatments available. The asset being tested in this clinical trial is an investigational ASO, which is an innovative modality designed to target the genetic cause of KCNT1-DEE. It acts by degrading KCNT1 mRNA with the aim of modifying the disease in patients, and has been shown to be well-tolerated in pre-clinical studies.

The first-in-human clinical trial is enrolling children with KCNT1-DEE.