Bright Minds Biosciences (DRUG) investor relations material

Pipeline overview and scientific rationale

• Focus on serotonin 5-HT2 family, with lead clinical asset in phase II for developmental and epileptic encephalopathies (DEE) and Absence Epilepsy.

• New program initiated for Prader-Willi syndrome using a novel 5-HT2C molecule, with additional preclinical assets targeting neuropsychiatric and pain disorders.

• Lead asset demonstrates G protein-biased agonism at 5-HT2C, aiming to avoid tolerance seen with other agents.

• Differentiation from competitors includes improved pharmacokinetics, better tolerability, and no need for refrigeration.

• Biomarker and QEEG data support receptor engagement and potential cognitive benefits.

Clinical development and trial design

• Phase II studies underway for Absence Epilepsy and DEE, with Absence using EEG-based endpoints and enrolling about 10 patients.

• DEE trial is multi-center in Australia, with plans for global phase 2/3 including US and Europe.

• Efficacy benchmarks: 50% reduction in seizures in 50% of patients for Absence, and 40-60% reduction expected in DEE.

• Data readout expected in early January, with pivotal studies planned for both indications in the first half of next year.

• Two pivotal studies required for each indication, with regulatory alignment ongoing, especially for Absence EEG endpoints.

Market opportunity and competitive landscape

• DEE market estimated at $1.5–2 billion in peak sales, with large unmet need and few approved therapies.

• Absence Epilepsy represents a potential 275,000–500,000 patient population, with no new branded agents in decades.

• Current Absence treatments are outdated or have significant side effects, highlighting opportunity for new entrants.

• Lundbeck is a key competitor in DEE, expected to reach market first, but Absence indication would be first to market.