NEW YORK – Atalanta Therapeutics on Tuesday said it plans to submit an investigational new drug (IND) application to the US Food and Drug Administration this year seeking permission to begin testing an investigational RNAi drug, ATL-201, in patients with KCNT1-related epilepsy.
New preclinical data published in Epilepsia support Atalanta's plan to advance ATL-201, according to the Boston-based company. In a mouse model, a single dose of ATL-201 durably suppressed seizures and improved behavior through six months, as well as reduced KCNT1 transcript levels with favorable tolerability, Atalanta said.
"We are working diligently to prepare ATL-201 to enter the clinic in the hopes of bringing the first disease-modifying therapy for KCNT1-related epilepsy to the families who urgently need it," Atalanta CSO Aimee Jackson said in a statement. Jackson is a coauthor of the paper.
KCNT1-related epilepsy is a rare and severe seizure disorder that affects infants and children. It's caused by gain-of-function variants in the KCNT1 gene, which typically encodes a potassium channel in neurons. ATL-201, a divalent small interfering RNA (di-siRNA), aims to treat the condition through silencing of the KCNT1 gene transcript, thereby reducing KCNT1 protein levels.