Bexicaserin Enters Phase III for Developmental and Epileptic Encephalopathy

Longboard Pharmaceuticals recently announced the launch of its Phase III DEEp OCEAN study to assess the potential of bexicaserin, a drug aimed at reducing seizures linked to developmental and epileptic encephalopathies (DEEs). This study, deemed the first of its kind by Chad Orevillo, Longboard’s executive vice president, marks a critical step in addressing a previously underserved need in epilepsy treatment.

DEEs are rare, severe disorders that typically begin in childhood and cause frequent, treatment-resistant seizures, which impair brain function and development. Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness.

Current treatment options remain limited, and many patients have not had opportunities to participate in trials specifically tailored to their condition.

DEEs present unique challenges for families, with symptoms that go beyond seizures to include movement, respiratory and sleep disorders, often making everyday care a considerable task.

Orevillo shared that DEEp OCEAN could “provide hope and access” for individuals with DEEs, as this pivotal trial is designed to study a broad range of DEEs and deliver a targeted therapeutic solution.

The DEEp OCEAN study, a global Phase III, double-blind, placebo-controlled trial, focuses on evaluating bexicaserin’s efficacy by tracking countable motor seizures in over 320 participants aged two to 65.

Bexicaserin, an investigational oral therapy, works by activating specific brain receptors, notably the 5-HT2C receptor, which helps regulate neurological functions. Unlike other treatments that may interact with multiple receptors and cause side effects, bexicaserin is designed to act selectively, which could improve its safety and tolerability.

Participants will undergo a five-week screening period, followed by dose titration and a 12-week maintenance phase on the highest tolerated dose. Those who complete the maintenance period can enter a DEEp open-label extension for further observation over a year. This extension aims to provide insights into long-term safety, addressing a common gap in epilepsy research.

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Bexicaserin has received the Food and Drug Administration’s (FDA) Breakthrough Therapy designation, signaling its potential to address an unmet need for DEE patients. This designation also offers a pathway to expedited regulatory review, potentially speeding up its journey to approval. Interim results from Longboard’s Phase II PACIFIC study, involving DEE participants aged 12 to 65, previously supported bexicaserin’s transition to this Phase III trial.

Last month, neuroscience-focused company Lundbeck announced a $2.6 billion acquisition of Longboard Pharmaceuticals, strengthening its presence in neuro-rare diseases. This acquisition supports Lundbeck’s Focused Innovator strategy, which aims to deliver therapies for rare neurological conditions with limited treatment options. With bexicaserin as the lead asset, Lundbeck gains a promising late-stage candidate for DEEs, including Dravet and Lennox-Gastaut syndromes.

Bexicaserin’s development has also gained regulatory support in Europe, where the European Medicines Agency’s (EMA) Paediatric Committee endorsed its potential use in children as young as two.

Bexicaserin is also currently being evaluated in the DEEp SEA study, a global Phase III trial focusing on reducing seizures in Dravet syndrome for patients aged two and older. This double-blind, placebo-controlled study will assess bexicaserin’s efficacy and safety over a 12-week maintenance period, with an option for eligible participants to join a year-long open-label extension.

In the announcement, Gabrielle Conecker, executive director of Decoding Developmental Epilepsies, emphasized the importance of reaching underserved patients with DEEs. She noted that DEEp OCEAN is a step towards inclusivity in clinical research. If successful, bexicaserin could offer new treatment avenues for patients who previously had limited or no options.