Policy & Regulation
Stoke Therapeutics reports FDA approval for administration of higher single dose of STK-001 (70mg) in Phase 1/2a Dravet syndrome study
15 March 2023 -

Biotechnology company Stoke Therapeutics Inc (Nasdaq: STOK) revealed on Wednesday that the US Food and Drug Administration (FDA) will allow the administration of a higher single dose of STK-001 (70mg) in its ongoing Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome, a severe and progressive genetic epilepsy characterised by frequent, prolonged and refractory seizures beginning within the first year of life.

Stoke is developing STK-001, a proprietary antisense oligonucleotide (ASO), as the first potential new medicine to address the genetic cause of Dravet syndrome.

The company said that the new 70mg single dose cohort in MONARCH will complement the ongoing evaluation of multiple doses of 70mg in the ADMIRAL study.

Barry Ticho, MD, PhD, chief medical officer of Stoke Therapeutics, commented: "There is an urgent need for a disease modifying approach to treat Dravet syndrome, a devastating disease with effects that go beyond seizures and impact quality of life for patients and their families. We thank the FDA for its timely review of the clinical data from our ongoing studies. We expect data from this new dose cohort to provide information about the potential effects and durability of a single dose of 70mg. We look forward to dosing the first patient in the coming weeks and to be able to include preliminary findings in our anticipated data readout in the second half of 2023."