Bright Minds launches Prader-Willi program

Executive Summary

• Bright Minds Biosciences has initiated a new Prader-Willi Syndrome (PWS) program, nominating BMB-105 as a new clinical candidate and launching the NOVA Phase 2a study for BMB-101 in PWS.
• The company provided an operational update on BMB-101 for absence seizures (AS) and developmental and epileptic encephalopathies (DDE), noting no safety signals and confirming top-line data release in January 2026.
• A Key Opinion Leader (KOL) event is scheduled for November 6, 2025, to discuss the PWS program, unmet medical needs, and the status of the ongoing epilepsy studies.

Key Details

• PWS Program Initiation:
  • Nominated BMB-105 as a new clinical candidate for the PWS program.
  • Commencing Phase 2a (NOVA) study for BMB-101 to assess efficacy, safety, and tolerability in PWS patients.
  • Commencing a randomized Phase 1 placebo-controlled study for BMB-105 in healthy volunteers to evaluate safety, tolerability, pharmacokinetics, and food effect.
  • Strategic approach: After the BMB-101 proof-of-pharmacology study, the company plans to select and advance BMB-105 as the dedicated compound for PWS, aiming to reduce time to market by approximately one year.
• BMB-101 Operational Update (Absence Seizures/DDE):
  • BMB-101 has been well tolerated with no drug-related serious adverse events or safety signals requiring protocol adjustments.
  • Exposure levels and tolerability are consistent with Phase 1 expectations, supporting future dose selection.
  • Open-label extension participation is proceeding well, with nearly all eligible patients electing to remain on therapy.
  • Top-line data for the current study will be released in early January 2026.
  • Planning for Phase 2/3 studies in DDE and Phase 2/3 in 2026.
• NOVA Clinical Study Design (BMB-101 for PWS):
  • Double-blind, randomized, Phase 2a study lasting up to 16 weeks.
  • Four-week screening period to establish hyperphagia and behavioral symptoms.
  • Randomization 1:1 to BMB-101 or placebo.
  • Four-week weekly ascending maximum tolerated dose (MTD) titration phase followed by an eight-week maintenance phase.
  • Five clinic visits and four telephone visits.
  • Optional unblinded, open-label phase for up to nine months (extendable) for participants who complete the maintenance phase.
  • Primary Endpoint: Change from baseline in hyperphagia questionnaire for clinical trials (HQ-CT) scores.
  • Secondary Endpoints: Change from baseline in hyperphagia severity (Caregiver and Clinician Global Impression of Severity), global impression of severity/improvement, and PWS-associated behavioral issues.
• KOL Event Details:
  • Date: November 6, 2025, at 10 a.m. ET.
  • Format: Virtual webcast with replay available.
  • Speakers include Dr. Jennifer Miller (University of Florida), Theresa V. Strong, PhD (Foundation for Prader Willi Research), and Elizabeth Roof, HSP, MA (Vanderbilt University).
  • Topics: Overview of PWS, unmet medical need, quality-of-life challenges, clinical assessment tools, and therapeutic rationale for 5-HT2C agonists.

Notable Quotes

• "The initiation of our PWS program and NOVA clinical study are exciting next steps as we continue our development efforts to serve patients with rare diseases... 5-HT2C agonism offers a novel mechanism for PWS by targeting the underlying aspects of the disease." — Ian McDonald, CEO and Co-Founder.
• "We believe it will pave the way forward for a pivotal study with BMB-105, our dedicated compound and expedite the development of the drug that aims at directly targeting the pathophysiology of PWS." — Ian McDonald.