Bright Minds Biosciences (DRUG) Study Result summary

Study design and patient population

• Phase II open-label, multicenter study of BMB-101 in adults with drug-resistant absence seizures and DEE, conducted at five epilepsy centers in Australia, enrolling 24 patients (15 Absence, 9 DEE) with a mean age of 30 years.

• Enrolled highly refractory patients with extensive treatment histories, including those with Lennox-Gastaut, Dravet, and Rett syndromes; some had neuromodulation devices.

• Patients were on multiple anti-seizure medications, with up to 16 prior failed treatments in absence and 9 in DEE.

• Baseline median seizure count was 22 for both cohorts; study included baseline, titration, and maintenance phases, with seizure tracking via diaries and 24-hour ambulatory EEGs.

• No limit on number of concomitant therapies, reflecting real-world clinical practice.

Efficacy results

• BMB-101 achieved a 73.1% median reduction in absence seizures and a 74.4% reduction in seizure burden, both statistically significant.

• In DEE, there was a 63.3% median reduction in major motor seizures, with 60.3% in Lennox-Gastaut and 76.1% in other DEE subtypes.

• Homogeneous seizure reduction observed across absence cohort, with no super-responders or non-responders; robust reduction regardless of seizure duration.

• Objective EEG endpoints confirmed robust efficacy, with blinded independent reads.

• Some patients experienced seizure freedom, including a Rett syndrome subject with 100% reduction for 43 days.

Safety and tolerability

• BMB-101 was generally well tolerated, with no drug-related serious adverse events; most adverse events were mild or moderate.

• Most common adverse events included respiratory infections, fatigue, constipation, headache, and drowsiness; fatigue rates matched placebo arms in similar studies.

• Three severe adverse events occurred, but only one (dry mouth) was related and transient, resolving without dose change.

• No significant effects on vital signs, labs, or ECGs.

• Tolerability profile supports chronic use in polytherapy populations.