Bright Minds Biosciences (DRUG) investor relations material

Program overview and pipeline milestones

• Lead asset BMB-101, a highly selective 5-HT2C agonist, showed strong phase II results in treatment-resistant absence seizures and developmental epileptic encephalopathies (DEE).

• Prader-Willi syndrome (PWS) program is in phase II, with data expected in the first half of 2027.

• Additional 5-HT2C agonist entering phase I soon, with data anticipated in the coming months.

• Several 5-HT2A assets are in preclinical development and could enter the clinic within months of a go decision.

Clinical efficacy and safety data

• BMB-101 demonstrated a 73% reduction in absence seizures and a 63% reduction in major motor seizures in DEE, with robust effects across refractory populations.

• Notable individual responses included 100% seizure reduction in a Rett syndrome subject and 50% reduction in a Dravet patient previously unresponsive to fenfluramine.

• Adverse events were mostly mild to moderate and transient, with a favorable safety profile compared to older drugs.

• No cardiac valvulopathy or weight gain cap issues seen with older 5-HT2B-binding drugs.

Regulatory and development plans

• Pre-IND meetings with FDA for both DEE and absence indications were positive, with agreement on endpoints, titration, and dosing.

• IND filings and initiation of phase III trials for both indications planned for later this year; DEE study will be global, absence study US-only.

• Phase III trials will be double-blind, placebo-controlled, targeting 100 completers per study.

• Data from these studies could be available in 2024, with enrollment expected to take about a year.