Ovid Therapeutics on Takeda's Phase 3 Study Results for Soticlestat

Ovid Therapeutics Inc., a biopharmaceutical firm focused on improving lives impacted by rare epilepsies and brain conditions, has reported findings from Takeda Pharmaceutical Company Limited's Phase 3 studies on soticlestat, a treatment for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). The studies, named SKYLINE and SKYWAY, have yielded mixed results.

The SKYLINE study, a multicenter, randomized, double-blind Phase 3 trial, aimed to evaluate soticlestat in patients with refractory DS. Despite narrowly missing its primary endpoint of reducing convulsive seizure frequency compared to placebo (with a p-value of 0.06), the study showed significant results in several secondary efficacy endpoints. These included improvements in seizure intensity, duration scales, responder rate, and measures of caregiver and clinician global impression of improvement, all achieving p-values of ≤ 0.008. Soticlestat's potential was also highlighted by previous Phase 2 ELEKTRA study findings, where it demonstrated a significant reduction in seizures from baseline compared to placebo in DS and LGS patients.

Conversely, the SKYWAY study, which evaluated soticlestat for refractory LGS, did not meet its primary endpoint of reducing major motor drop (MMD) seizure frequency. However, some pre-specified subgroups showed significant treatment effects on both the primary and secondary endpoints, necessitating further analysis.

Soticlestat was well tolerated in both studies, aligning with its known safety profile from earlier research. Takeda intends to discuss these findings with regulatory authorities to determine the next steps and plans to present the results at an upcoming scientific congress.

In a broader context, Ovid Therapeutics reflects on its independent R&D and financial strategies, emphasizing that their pipeline advancements are not reliant on soticlestat’s outcomes. The company has developed several promising candidates and platforms, including OV888, a ROCK2 inhibitor for cerebral cavernous malformations, and OV329, a GABA-aminotransferase inhibitor for refractory seizures. Ovid's KCC2 direct activator program, OV350, is also progressing, with first-in-human data expected by 2026.

The SKYLINE trial enrolled 144 subjects aged 2-21 years, all diagnosed with DS, and evaluated the efficacy, safety, and tolerability of soticlestat as an adjunctive therapy. Participants were randomized to receive either soticlestat or placebo in addition to their standard anti-seizure medications over a 16-week treatment period, consisting of a 4-week titration and a 12-week maintenance phase. Those who completed the study were offered the opportunity to continue in an open-label extension study, ENDYMION 2.

Similarly, the SKYWAY trial enrolled 270 subjects aged 2-55 years with LGS and followed the same structure in evaluating soticlestat. Both trials were adjudicated independently by the Epilepsy Study Consortium to ensure accurate diagnoses.

Ovid Therapeutics remains committed to advancing its pipeline with financial discipline. The company’s cash runway is projected to last through the first half of 2026, during which it anticipates achieving several clinical milestones, including safety and efficacy data from ongoing and upcoming studies of its various programs. These efforts underpin Ovid's mission to develop innovative treatments for complex neurological conditions, aiming to create significant value for patients and stakeholders alike.

Pending discussions with regulators about the soticlestat data, Takeda will determine the next regulatory steps, and Ovid will update its guidance on potential milestones and royalty payments linked to its agreement with Takeda. This strategic approach underscores Ovid's commitment to leveraging its scientific expertise and financial resources to bring novel therapies to patients in need.