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.

• Patients were highly refractory, with extensive treatment histories, including those with Lennox-Gastaut, Dravet, Rett syndromes, and some with neuromodulation devices.

• Most DEE patients had failed nearly 10 prior antiseizure treatments, and absence patients had up to 16 prior failed therapies.

• 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.

• Robust efficacy observed regardless of seizure duration, with consistent reductions across subgroups and no super-responders or non-responders.

• Objective EEG endpoints confirmed robust efficacy, with blinded independent reads; BMB-101 was the first drug to demonstrate efficacy in absence seizures using 24h EEG.

• Some patients experienced seizure freedom, including a Rett syndrome subject with 100% reduction for 43 days and significant improvement in previously unresponsive patients.

Safety and tolerability

• BMB-101 was safe and well-tolerated, with no drug-related serious adverse events; about 80% of TEAEs 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 drug-related and transient, resolving without dose change.

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

• Tolerability profile supports chronic use in polytherapy populations; only one patient discontinued due to a drug-related event.