Stoke Therapeutics has announced new data from two open-label Phase 1/2a studies and two open-label extension studies of its lead antisense therapy, STK-001, in children aged 2-18 years with Dravet syndrome. Dravet is a rare genetic epilepsy syndrome caused by haploinsufficiency.
The latest results showed substantial and durable reductions in convulsive seizure frequency (up to an 85% reduction 3 months after the last of 2 or 3 doses of 70mg) as well as improvements in multiple measures of cognition and behaviour. Two patients were seizure free at 3 and 6 months.
The improvements were seen in a highly refractory group of patients who were already taking the best available anti-seizure medicines. 85% of patients were routinely being treated with 3 anti-seizure medications.
STK-001 was generally well-tolerated. In the Phase 1/2a studies, 30% (24/81) of patients experienced a treatment-emergent adverse event that was related to the study drug. The most common were CSF protein elevations and procedural vomiting.
The Company plans to meet with regulatory agencies to discuss a registrational study that includes initial doses of 70mg followed by continued dosing at 45mg.
Stoke Therapeutics’ (STOK:NASDAQ) stock price quickly doubled on the news, finished the day 58% up as the Company announced a US$75m public offering.
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