ABI-009 safe in children with refractory epilepsy

In the treatment of paediatric patients with medically and surgically refractory epilepsy, the use of ABI-009 appears safe and tolerable, with early signs of efficacy, according to data from a phase I study.

The study included 12 children with medically intractable epilepsy (including tuberous sclerosis complex, focal cortical dysplasia, and hemimegalencephaly) and a history of resective surgery. These patients experienced more than eight seizures during the 30-day baseline. All of them received ABI-009 treatment, administered over 3 weeks with dosing cohorts of 5, 10, and 20 mg/m². Existing antiseizure medication regimens were maintained throughout the study.

After the 3-week treatment, the patients underwent observation during the 90-day follow-up period. Researchers collected data on seizure frequency, seizure type, adverse events, medical and laboratory assessments, the Quality of Life in Childhood Epilepsy (QOLCE), and the Nisonger Child Behavior Rating Form (NCBRF).

None of the patients missed their doses during the treatment period, and dose escalation progressed through all levels. The maximum tolerated dose was 20 mg/m².

Treatment-emergent adverse events (TEAEs) occurred in all 12 participants, with the majority being mild (80 percent). There were no reports of serious adverse events. None of the patients withdrew from the study voluntarily or by necessity due to TEAEs.

Weekly seizure rate decreased by a median of 37.6 percent. Treatment response, defined as ≥25-percent seizure reduction, was documented in 66.7 percent of patients, with seizure freedom achieved in two patients by follow-up.