Biohaven shared new data this week on their experimental epilepsy medicine opakalim, including an update involving a child with KCNQ2-DEE. In the six-month follow-up, the company reported that the child remained clinically stable with ongoing seizure control, and an overnight EEG showed about a 50% reduction in seizure counts compared to before starting treatment. The medicine was also reported to be well-tolerated during the treatment period.

The child in this update had previously experienced daily tonic seizures despite three concurrent ASMs, including a first-generation Kv7 activator. Prior attempts to taper off this Kv7 activator resulted in status epilepticus, ICU admission, and developmental regression, so transitioning to opakalim without these complications is significant progress

While promising, it is important to remember that these findings come from a single person receiving treatment through compassionate use, meaning more research is needed to understand how opakalim may work across the broader KCNQ2 community. Still, these early results are encouraging, as they support the idea that more precisely targeted therapies can achieve seizure control with fewer side effects. Because opakalim activates Kv7.2/7.3 channels more selectively than first-generation activators, researchers hope this approach reduces off-target effects such as sleepiness, dizziness, fatigue, and memory issues.

If you feel your loved one’s seizures are not currently well managed and that they might benefit from access to an investigational potassium channel activator, start by discussing the opportunity with your physician. You can also find more information about the compassionate use program and access helpful links in our blog post.

Biohaven expects additional clinical trial results later in 2026. More information is available in the press release on the Biohaven website.