Stoke Therapeutics Inc (NASDAQ:STOK) shares are trading lower after the company released new safety & efficacy data from STK-001 in the Phase 1/2a Studies (MONARCH & ADMIRAL) and the SWALLOWTAIL Open-Label Extension (OLE) study in children and adolescents with Dravet syndrome.
The studies involve patients aged 2 to 18 years with Dravet syndrome who received multiple doses of STK-001.
The data suggests promising results regarding decreased seizure frequency and improved cognition and behavior, pointing towards potential disease modification.
The Phase 1/2a MONARCH and ADMIRAL trials, conducted in the U.S. and U.K., respectively, are designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of STK-001.
In the combined analysis of these trials, the most significant reduction in seizure frequency was noted among patients who received two to three doses of 70mg STK-001.
Additionally, “through” and “at” analyses were performed to capture STK-001’s effect during and after dosing, respectively.
Patients who completed the Phase 1/2a MONARCH trial and met specific criteria were eligible for the SWALLOWTAIL OLE study.
Initial analysis indicated disease modification potential and durable reductions in seizure frequency with ongoing STK-001 treatment at 30mg, 45mg doses.
Significant improvements in cognition and behavior were also observed.
On the safety front, no significant issues were observed among the 74 patients treated with varying doses of STK-001. The most common treatment-emergent adverse events were elevated CSF protein, vomiting, and irritability. There were no drug-related discontinuations.
The company observed dose-dependent increases in drug exposure and STK-001 accumulation in CNS tissues, indicating sustained exposure in the brain.
Stoke Therapeutics anticipates additional data from the ongoing trials in Q1 2024.
Based on these results, the company will update Phase 3 planning in the first half of 2024.
Price Action: STOK shares are down 33.10% at $6.38 on the last check Tuesday.
