Bright Minds Biosciences (DRUG) investor relations material

Key program updates and clinical data

• Lead program BMB-101 targets serotonin 5-HT2C receptors for refractory absence seizures and developmental and epileptic encephalopathies (DEE), with robust efficacy shown in a small, highly refractory patient population.

• Phase I studies showed no serious adverse events and good tolerability, with only transient side effects at high doses.

• In clinical trials, BMB-101 achieved a 73% median reduction in absence seizures and 63% in DEE, with improvements seen across seizure durations and sleep architecture.

• No new seizure types or significant safety concerns emerged; adverse events were mostly mild and manageable.

• Global phase III studies in DEE and absence seizures are being initiated, with regulatory discussions ongoing in the US, Europe, and Australia.

Regulatory and development strategy

• Pre-IND meeting completed for DEE; pre-IND for absence planned, with EEG endpoints to be finalized with agencies.

• Enrollment for absence studies expected to be strong in the US and Australia; DEE studies to leverage established investigator networks across US, Europe, and Australia.

• DEE pivotal trials will include all comers, not limited to specific syndromes, focusing on the final common seizure pathway.

• Recent financing will fund multiple readouts for epilepsy and Prader-Willi programs, and advance BMB-105 through phase I.

Market opportunity and competitive landscape

• Both DEE and absence indications are seen as multi-billion dollar opportunities due to high unmet need and limited effective therapies.

• Existing drugs like ethosuximide and valproate have significant limitations; BMB-101 offers broad efficacy across seizure types.

• Recruitment for absence studies faces little competition, while DEE studies will compete with other ongoing trials.