Stoke Therapeutics Highlights Strategic Priorities and Anticipated Milestones for 2024

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced strategic priorities and anticipated milestones for 2024.

“Over the last three years we have generated a comprehensive set of data from 81 patients that support STK-001 as potentially the first disease-modifying medicine for Dravet syndrome. 2024 is all about advancing this potential new medicine toward a registrational study,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The recent 2-year data from our BUTTERFLY natural history study make it clear that even the best available anti-seizure medicines are inadequate. These data show continued high seizure burden and stagnation of neurodevelopment, which are in stark contrast to the improvements we see across the studies of STK-001. Results from clinical studies anticipated in the first quarter are expected to give us the data necessary to request meetings with regulators to discuss our Phase 3 plans.”

Updates and Anticipated Milestones

STK-001: Dravet Syndrome

• In December, at the American Epilepsy Society annual meeting, the Company presented data supporting the continued advancement of STK-001 as a disease-modifying potential new medicine for the treatment of Dravet syndrome. Two-year natural history study data showed a lack of improvement among patients who are taking the best available anti-seizure medicines. In contrast, substantial and sustained reductions in seizure frequency and improvements in cognition and behavior were observed in clinical studies of STK-001. Modeling data suggest that higher STK-001 drug exposure in brain leads to greater seizure reductions. Single and multiple doses of STK-001 up to 70mg have been generally well tolerated to date.
• In the first quarter of 2024, the Company plans to report additional clinical and modeling data from 81 patients treated in the Phase 1/2a studies of STK-001 (MONARCH and ADMIRAL) and the two ongoing open-label extension studies (OLE) (SWALLOWTAIL and LONGWING), including:
  • Safety, pharmacokinetic (PK) modeling, and cerebrospinal fluid (CSF) results;
  • Seizure frequency data from ~20 patients who received 1, 2, or 3 initial doses of 70mg of STK-001 and were followed for six months;
  • The effects of repeat doses of STK-001 (30mg, 45mg) on seizure frequency and cognition and behavior from patients treated in the SWALLOWTAIL and LONGWING OLE studies.
• Pending the results of the Q1 data readout, the Company plans to proceed with Phase 3 preparation activities, including discussions with global regulatory agencies, availability of chronic toxicology data, preparation of the investigator brochure, submission of a final protocol to regulatory agencies and institutional review boards (IRB).

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