Summary of Bright Minds Biosciences FY Conference Call Company Overview - **Company**: Bright Minds Biosciences (NasdaqCM:DRUG) - **Focus**: Development of therapies targeting serotonin receptors, specifically the 5-HT2 family - **Current Clinical Assets**: - BMB-101 in Phase 2 for two types of epilepsy: developmental and epileptic encephalopathies (DEEs) and absence epilepsy - New program targeting Prader-Willi syndrome (PWS) with a 5-HT2C molecule - Several preclinical assets focusing on the 5-HT2A receptor for potential use in pain and neuropsychiatric disorders [2][60] Key Points and Arguments Clinical Development - **BMB-101**: - A G protein-based agonist at the 5-HT2C receptor, differentiating it from other compounds like fenfluramine and BMB-101 by avoiding beta-arrestin pathway activation, which can lead to tolerance [3][4] - Better pharmacokinetics (PK) with a proposed once-daily formulation compared to BMB-101, which requires refrigeration and is taken three times a day [4][7] - Phase 1 data indicates better tolerability and a linear dosing response, reducing side effects compared to BMB-101 [8][9] Epilepsy Indications - **Absence Epilepsy**: - The FDA is interested in absence seizures due to the lack of effective treatments; EEG is a reliable measurement tool for these seizures [16][17] - The benchmark for approval is a 50% reduction in seizures in 50% of patients, adjusted for placebo effects [17][18] - Enrollment target for the study is about 10 patients, with doses ranging from 0.67 to 2 mg/kg [28][31] - **Developmental and Epileptic Encephalopathies (DEEs)**: - Majority of patients will likely be from the Lennox-Gastaut syndrome (LGS) population, with a similar benchmark for efficacy as absence epilepsy [36][38] - Multi-center trial being conducted in Australia, with plans for a global Phase 2/3 trial [37][44] Market Opportunity - **DEEs**: Potential peak sales for treatments in this area are estimated at $1.5 billion to $2 billion [49] - **Absence Epilepsy**: Bright Minds aims to be the first branded agent in this indication, with a patient population estimated at around 275,000 to 500,000 annually [49][51] Prader-Willi Syndrome (PWS) - **Mechanistic Rationale**: Genetic link to 5-HT2C receptors in PWS patients; targeting these receptors may alleviate symptoms including hyperphagia and neuropsychiatric issues [60] - **Clinical Evidence**: Previous studies with fenfluramine showed improvements in weight and neuropsychiatric symptoms, supporting the rationale for 5-HT2C agonism in PWS [60] Financials and Capitalization - The company is operating with a focus on capital efficiency, with current funding covering ongoing and upcoming studies [69] Additional Important Information - Future studies planned for both DEE and absence epilepsy, with a need for regulatory alignment on seizure counting methods [46][48] - The company is also advancing a second asset, BMB-105, to potentially save time in development [62][63] - The upcoming data release is expected in early January, strategically timed to capture investor attention [42]