Evaluation of the Safety and Tolerability of BHV-7000 in Adults with Refractory Focal Onset Epilepsy: A Phase 2 Multicenter Study

The primary objective of this Phase 2, global, multicenter study is to evaluate the **safety** and **tolerability** of BHV-7000 in subjects with **refractory focal onset epilepsy**. This is clinically relevant as it aims to determine the potential of BHV-7000 as a therapeutic option for patients who have not responded adequately to existing treatments. The study will assess the long-term safety profile of BHV-7000, which is administered in a prolonged-release tablet form, to ensure its suitability for chronic use in this patient population.

The clinical trial involves a total of **279 participants** diagnosed with **Refractory Focal Onset Epilepsy**. The study population includes both male and female subjects, with an age range that encompasses both adults and adolescents. Participants were selected based on their completion of the double-blind phase of prior parent studies, specifically BHV7000-302 or BHV7000-303. The trial includes females of childbearing potential, who are required to have a negative urine pregnancy test at the Baseline/Day 0 visit. The study population is characterized by a vulnerable group, indicating that special considerations are in place to ensure their safety and well-being throughout the trial. The trial does not specify any particular lifestyle considerations such as diet or physical activity for the participants.

The clinical trial is a Phase 4, global, multicenter study designed to evaluate the **safety** and tolerability of **BHV-7000** in subjects with **refractory focal onset epilepsy**. The trial employs a randomized, double-blind, controlled design to ensure the reliability and validity of the results. The estimated duration of the trial is from December 2024 to December 2026, with a maximum treatment period of 52 weeks for each participant. Participants will be administered **BHV-7000** in the form of a prolonged-release tablet, with a maximum daily dose of 75 mg and a total dose not exceeding 27,300 mg over the course of the study.

The sequence of study visits begins with an inclusion (screening) visit, where eligibility is confirmed based on criteria such as completion of the double-blind phase of prior parent studies BHV7000-302 or BHV7000-303, and a negative urine pregnancy test for females of childbearing potential. Follow-up visits are scheduled at regular intervals to monitor the safety and tolerability of the treatment, with assessments including the number of unique subjects with deaths, serious adverse events (SAEs), adverse events (AEs) leading to discontinuation, and laboratory abnormalities of grade 3 and 4. The end-of-study visit marks the conclusion of the participant's involvement, where final assessments are conducted to gather comprehensive safety data.

Participant involvement is expected to last up to 52 weeks, contingent upon adherence to the study protocol. Conditions that may lead to early termination from the study include the occurrence of severe adverse events or non-compliance with study procedures. The trial aims to provide valuable insights into the long-term safety profile of **BHV-7000**, contributing to the understanding of its use in managing **refractory focal onset epilepsy**.

The clinical trial involves the administration of the experimental medication **BHV-7000**, which is formulated as a **prolonged-release tablet**. The active substance, also named BHV-7000, is of chemical origin and is developed by Biohaven Therapeutics Ltd. The medication is administered orally, with a maximum daily dose of 75 mg and a total maximum dose of 27,300 mg over the course of the study. The treatment period extends up to 52 weeks. The study aims to evaluate the safety and tolerability of BHV-7000 in subjects with refractory focal onset epilepsy.

No non-experimental treatments, such as standard-of-care therapy, placebo, or comparator treatments, are specified in the trial data. The study does not include a pediatric formulation, and BHV-7000 is not classified as an orphan drug. Participant compliance with the dosing schedule will be monitored throughout the trial to ensure adherence to the prescribed regimen. The trial is designed to assess the long-term safety of BHV-7000, with a focus on its tolerability in the target patient population.

The efficacy of the clinical trial involving the investigational product **BHV-7000** will be assessed primarily through safety endpoints. The primary endpoint focuses on evaluating safety by monitoring the number of unique subjects experiencing deaths, serious adverse events (SAEs), adverse events (AEs) leading to discontinuation, moderate and severe AEs, and grade 3 and 4 laboratory abnormalities. These parameters will be systematically collected and analyzed to determine the safety profile of **BHV-7000** in subjects with refractory focal onset epilepsy.