Takeda shares late-stage results for investigational soticlestat in rare epileptic disorders

18 Jun 2024
pmlive.com
Clinical ResultPhase 2Phase 3
Takeda shares late-stage results for investigational soticlestat in rare epileptic disorders
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Source: PMLiVE
Takeda has shared topline data from two late-stage trials of its experimental epilepsy drug soticlestat (TAK-935), with the CH24H inhibitor missing the studies’ primary endpoints but demonstrating efficacy in multiple key secondary endpoints.
Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) belong to a group of rare epilepsy syndromes that usually manifest during infancy or early childhood and are highly drug-resistant to many anti-seizure medications.
DS affects approximately one in every 15,000 to 21,000 people in the US, while LGS has an estimated prevalence of less than one in every 1,000.
The phase 3 SKYLINE trial evaluated soticlestat versus placebo, both given alongside standard of care, in patients with refractory DS and “narrowly missed” its primary endpoint of reduction from baseline in convulsive seizure frequency, Takeda said.
The company added that among the six key secondary endpoints, soticlestat showed “clinically meaningful and nominally significant” results in the responder rate, measures of caregiver and clinician global impression of improvement, and seizure intensity and duration scales over the 16-week treatment period.
The phase 3 SKYWAY study of soticlestat in LGS, which also compared the candidate against placebo on top of standard of care, missed its primary endpoint of reduction from baseline in major motor drop seizure frequency.
The candidate was generally well tolerated in both SKYLINE and SKYWAY and demonstrated a safety profile consistent with the findings of previous studies.
Sarah Sheikh, head, neuroscience therapeutic area unit and head, global development at Takeda, said: “Even with currently available therapies, we know that many patients with developmental and epileptic encephalopathies like DS and LGS still experience persistent unmet need across multiple dimensions, such as seizure burden and treatment tolerability.
“While we would have wished for more declarative results on the primary endpoints, we are encouraged by positive outcomes seen in the totality of the data…”
The announcement comes just under four years after Takeda reported that the phase 2 ELEKTRA study of soticlestat in children with DS or LGS met its primary endpoint of reduction in seizure frequency.
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