Ionis starts Dravet syndrome drug trial

• Ionis Pharmaceuticals dosed the first participant in the Phase 1-2 ASCEND study of ION337 for Dravet syndrome.
• The trial will evaluate safety, tolerability and dosing of the investigational RNA-targeted medicine in children.
• Ionis said ION337 uses its NMA technology designed to support sustained activity and less frequent dosing.

Ionis Pharmaceuticals (NASDAQ:IONS) announced that the first participant has been dosed in the Phase 1-2 ASCEND study evaluating ION337, an investigational RNA-targeted medicine for children with Dravet syndrome, a rare neurological disorder.

The trial milestone follows Ionis’ continued development of neurological medicines and marks the company’s first wholly owned medicine developed using its next-generation N-Methylacetamide (NMA) technology platform.

The ASCEND study will evaluate ION337 in children aged 2 to 12 years with a clinical diagnosis of Dravet syndrome, including a six-month single ascending dose phase followed by a 24-month multiple ascending dose phase with planned dosing every six months.

Ionis said ION337 is designed to enhance the potency of splice-modulating antisense oligonucleotides and potentially support infrequent intrathecal dosing.

Dravet syndrome usually begins in infancy and is associated with prolonged seizures, developmental delays, cognitive impairments and increased risk of sudden death.

Ionis develops RNA-targeted medicines using antisense technology across neurological, cardiovascular and other disease areas, with ION337 representing a program within its expanding neurology pipeline.