Takeda announced disappointing topline data from two phase 3 trials of soticlestat in rare forms of epilepsy, but still isn't ready to give up on the Ovid Therapeutics-partnered prospect.
Soticlestat "narrowly missed" the primary endpoint in the SKYLINE study, which evaluated soticlestat plus standard of care versus placebo plus standard of care in patients with refractory Dravet syndrome. Despite not reducing convulsive seizure frequency, soticlestat did show clinically meaningful results in some secondary endpoints, including the responder rate, measures of caregiver and clinician global impression of improvement, and seizure intensity and duration scales over the 16-week treatment period.
In the SKYWAY study, which evaluated soticlestat plus standard of care versus placebo plus standard of care in patients with refractory Lennox-Gastaut syndrome, soticlestat missed the novel primary endpoint of reduction from baseline in Major Motor Drop seizure frequency as compared to placebo.
Takeda noted that in both trials, "some pre-specified subgroups of patients also showed nominally significant treatment effects on the primary and secondary efficacy endpoints of caregiver and clinician global impression of improvement, and seizure intensity and duration scales over the 16-week treatment period." The drugmaker says further analyses are being conducted.
Soticlestat is a first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase that was developed to treat children and adults with two rare forms of epilepsy — Dravet Syndrome and Lennox-Gastaut Syndrome. Ovid Therapeutics co-developed soticlestat through phase 2 and then sold its rights to Takeda in 2021.
Takeda put down $196 million upfront to pick up the prospect, with another $660 million in potential milestone payments down the line.
Now, Takeda says it plans to engage with regulatory authorities to discuss the totality of the data generated by the two studies to determine next steps.
