Phase 1/2a MONARCH clinical trial of STK-001 update

Stoke Therapeutics released interim safety, pharmacokinetic, and CSF exposure data from their Phase 1/2a MONARCH trial of STK-001 in individuals with Dravet syndrome.

• The data in this analysis comes from 21 patients in the Single Ascending Dose arm of the study (SAD: 1 dose with a 6-month follow-up) and an additional six patients from the Multiple Ascending Dose arm (MAD: 1 dose approximately every month for 3 months with a 6-month follow-up).
• So far, STK-001 has been well-tolerated without any study-drug related safety concerns in response to single administrations up to 30mg or multiple doses at 20mg.
• No patients were reported to have withdrawn from the study, and all patients that completed the Monarch study have elected to enroll in the Swallowtail Open-Label Extension study where they currently receive a 30mg dose every 4 months.
• The study is working to find the most effective dose range (mg) and timing of dosing by measuring the plasma and CSF concentrations of STK-001.
• The study reported very early trends of decreased seizure frequency in 8 out of 11 patients in the Single Ascending Dose (SAD) arm. This was seen across all dose levels (10, 20, and 30mg), and was more evident in the 2-12 year age group than the 13-18 year group. This is still very early in the study to assess these outcomes, and dosing has not yet been optimized.
• There are no updates on clinical presentation or Quality of Life yet.
• The Monarch trial is ongoing and patients are now being given 30mg doses in the MAD arm.
• The FDA will allow an additional dose increase for STK-001 to 45mg (previously 10, 20, and 30mg).
• The Admiral trial has begun in the UK, with two sites actively enrolling and the first patient has already received a dose. These studies are starting with dosing at 30mg and are approved to go up to 70mg.

Stoke Therapeutics is planning to present a poster at the American Epilepsy Society Annual meeting in the first week of December, 2021 that will hopefully include additional insights into these findings.

Find the press release from Stoke Therapeutics here.