Stoke Therapeutics Inc (NASDAQ:STOK) announced on October 7 that it plans to start administering STK-001 doses to children and teenage participants of the Phase 1/2a MONARCH Study.
The In Phase 1/2a MONARCH Study is a clinical study that is exploring the safety and efficacy of STK-001 as a potential treatment for dravet syndrome, an adverse type of epilepsy. The announcement about the plan to commence dose administration in subjects in the clinical study comes after talks with the Food and Drug Administration’s directive to hold off higher dose levels in the study.
Stoke Therapeutics has the green light to include extra higher dose levels in the single ascending dose (SAD) cohort. The company will evaluate three dose levels which will be 10 mg, 20 mg, and 30 mg as part of the study. Stoke Therapeutics also plans to include a multiple ascending dose (MAD) cohort in the MONARCH study based on toxicity data collected from a preclinical-repeat dose.
Stoke Therapeutics is working closely with the FDA to ensure the study is conducted correctly
The FDA reviewed the data and did not observe any adverse effects. Stoke also plans to send protocol amendments to the FDA. The amendments are courtesy of the changes that the company made to the MAD and SAD study cohorts.
“There is an urgent need for more effective medicines for people who are living with Dravet syndrome, so we are pleased to be moving ahead quickly with our plans to continue dosing children and adolescents in this important Phase 1/2a study of STK-001,” stated Stoke Therapeutics CEO, Edward M. Kaye.
The CEO also expressed gratitude to the FDA for its timely reviews and feedback. Mr. Kaye also noted that the researchers in the study have observed encouraging preclinical data and that they are excited to see how the three individual doses will perform. The researchers are also working on a revised protocol which they will submit to the FDA as soon as possible. Stoke Therapeutics has so far observed significant potential from STK-001 especially during preclinical trials involving mice models. Clinical human trials have also demonstrated strong potential.