ARCADE and ENDYMION Studies

October 12, 2020

ARCADE Study

 
ARCADE is a Phase 2 open-label, signal-finding pilot study designed to inform the potential for future development of soticlestat in CDD and Dup15q. The study enrolled 20 patients, ages 2 to 55 years, with refractory epileptic seizures associated with CDD (n=12) or Dup15q (n=8) and consisted of a four- to six-week screening period to establish baseline seizure frequency, followed by a 20-week treatment period, including an eight-week titration/dose optimization period and a 12-week maintenance period. Patients in the study were allowed to be on one to six concomitant anti-epileptic drugs (AEDs), with the majority of patients concomitantly treated with at least four AEDs, representing a highly refractory patient population. The primary objective of the ARCADE study was to determine percent change from baseline in motor seizure frequency during the 12-week maintenance period. Afterward, patients were offered the chance to continue soticlestat treatment in the ENDYMION open-label extension study, and all patients who completed ARCADE elected to roll over into ENDYMION.

Data from Dup15q Syndrome
Dup15q patients also exhibited a variety of seizures types, including motor (tonic and atonic), myoclonic and absence seizures. In Dup15q patients (n=8), there was an increase in median motor seizure frequency of 13% during the 20-week study period (eight-week dose titration and 12-week maintenance period) and an increase of 12% during the 12-week maintenance period (primary objective of the study). However, longer-term data from the four Dup15q patients who reached nine months of continuous treatment with soticlestat in EN