Stoke Touts Early Signs Of Efficacy For Antisense Drug In Dravet Syndrome

Stoke Therapeutics Inc STOK announced highlights from five presentations related to the ongoing clinical development of STK-001 being made at the American Epilepsy Society 2021 Annual Meeting.

• STK-001 is under development to target the underlying cause of Dravet syndrome, a severe and progressive genetic epilepsy.
• STK-001 was well-tolerated in single and multiple doses in 22 Dravet syndrome patients between 2 and 18 years old.
• 12 of the 17 evaluable patients at the time (70.6%) saw reductions from baseline in convulsive seizure frequency.
• Median reductions in seizure frequency of 17% to 37% were seen across all cohorts.
• The drug appeared well-tolerated after 12 weeks, with the most common side effects being headache, vomiting, seizure, irritability, and back pain. 
• Though five of 22 patients (22.7%) experienced a serious adverse event, none of them were related to the drug.
• Patients in MONARCH are eligible to continue treatment in the SWALLOWTAIL extension study, which is currently enrolling. 
• Stoke plans to enroll 90 patients in the U.S. in the MONARCH study.
• Related Link: Stoke Therapeutics Posts Encouraging Interim Safety Data From Early-Stage Dravet Syndrome Trial.
• BofA initiated coverage of Stoke Therapeutics with a Buy rating and a $43 price target. 
• Analyst Greg Harrison adds that Stoke's lead program STK-001 in development for Dravet syndrome could be the first to address refractory epilepsy, with potential for peak sales of over $2 billion.
• Price Action: STOK shares are down 10.50% at $21.35 during the market session on the last check Friday.