Takeda Pharmaceutical and Ovid Therapeutics have reported positive top-line data from the Phase II ELEKTRA clinical trial of soticlestat in children with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS).
Soticlestat is a highly selective oral inhibitor of the cholesterol 24-hydroxylase enzyme. Takeda and Ovid are developing the drug to treat rare developmental and epileptic encephalopathies (DEEs), including DS and LGS.
A total of 141 patients aged two to 17 were enrolled in ELEKTRA and 126 completed the trial.
The trial met its primary endpoint with a 27.8% median decrease in convulsive seizure (DS) and drop seizure (LGS) frequency from baseline during the 12-week maintenance period.
This represents a statistical significance compared to a 3.1% median increase with placebo.
Soticlestat also led to a 29.8% median reduction in convulsive (DS) and drop seizure (LGS) frequency versus a 0% change in median seizure frequency in the placebo arm during the 20-week treatment period.
In the cohort of 51 participants, treatment with soticlestat showed a 33.8% median decrease in convulsive seizure frequency compared to a 7% median increase with placebo during the 20-week treatment period.
Based on these findings, the companies intend to discuss with regulatory authorities regarding launch of a Phase III registrational programme for the drug in DS patients.
In the LGS cohort of 88 participants, the drug demonstrated a 20.6% median reduction in drop seizure frequency compared to a 6% median reduction in patients on placebo during the treatment period.
Additional analyses are underway to inform the next steps for the development of the drug in this patient population.
Safety data showed that the drug was generally well-tolerated, with a profile consistent with those of prior studies and without any new safety signals.
Takeda Pharmaceutical Neuroscience Therapeutic Area Unit head Sarah Sheikh said: “It is exciting to see these positive results and to advance soticlestat into late stage clinical development, initially for the potential treatment of children with Dravet syndrome who need more options to manage treatment-resistant seizures.
“Soticlestat and its novel mechanism of action were discovered at Takeda and we are enthusiastic about continuing to advance the science and clinical programmes as one aligned team, in strong partnership with Ovid Therapeutics.”
Earlier this month, Takeda partnered with AbbVie and Amgen on I-SPY COVID trial.