BEDFORD -
Together, these data support the company's continued progress to develop STK-001 as the first disease-modifying medicine for the treatment of Dravet syndrome.
'The comprehensive set of data being presented at AES are giving us a very good understanding of how STK-001 works and its potential to address not only seizures, but many of the non-seizure effects of Dravet syndrome,' said
'Dravet syndrome goes far beyond seizures, and as children grow up, they experience a complex array of life-altering challenges, including developmental delays, movement and balance issues and delayed language and speech,' said
Highlights from the Company's presentations of data at the meeting, include: BUTTERFLY Natural History Study of Patients with Dravet Syndrome Ages 2 to 18: Despite treatment with the best available anti-seizure medicines, on average, patients continued to experience convulsive seizures over 24 months at similar frequency to baseline. No statistically significant change from baseline in the majority of Vineland-III measures (an established instrument for assessing developmental disabilities) was observed and the rate of improvement on multiple clinical measures, including key domains of the Vineland-III, was substantially below neurotypical peers. Gaps in neurodevelopment continued to widen throughout the study among patients with Dravet syndrome compared to their age-matched neurotypical peers.
MONARCH & ADMIRAL Interim Analyses: Single and multiple doses of STK-001 up to 70mg were generally well tolerated. The multiple dose 70mg cohort showed the greatest reductions in convulsive seizure frequency, outperforming all lower dose groups. Patients treated with 2 or 3 initial doses of 70mg experienced substantial and sustained reductions in convulsive seizure frequency.
SWALLOWTAIL & LONGWING Open Label Extension (OLE) studies: Approximately 90% of patients who completed participation in Phase 1/2a studies of STK-001 enrolled in one of these OLE studies. Multiple doses of STK-001 up to 45mg given every 4 months were generally well tolerated. In addition to durable reductions in convulsive seizure frequency throughout the course of treatment, data indicated substantial improvements in multiple assessments of cognition and behavior over 12 months. These data support the potential for disease-modification with STK-001.
PK Model for STK-001: A relationship between STK-001 brain exposures and convulsive seizure frequency was evaluated based on 72 patients treated in the Phase 1/2a studies (MONARCH and ADMIRAL) and the SWALLOWTAIL OLE study in children and adolescents with Dravet syndrome. The exposure-seizure analysis demonstrated that higher STK-001 brain exposure leads to greater reductions in convulsive seizure frequency (R=-0.23, P
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