Bright Minds Biosciences Inc. (CSE:DRUG / NASDAQ:DRUG), a biotechnology company focused on developing novel drugs for the targeted treatment of neuropsychiatric disorders and refractory epilepsy, today announced the successful completion of its three-part Phase 1 study of BMB-101. The study, conducted in Adelaide, Australia, by CMAX Clinical Research, a clinical trial center specializing in a range of early-phase trials and first-in-human studies, evaluated the safety, tolerability, pharmacokinetic (PK), and food effect in healthy volunteers.

BMB-101 is a highly selective and potent 5-HT2C agonist being developed for the treatment of refractory epilepsies and other indications, such as psychosis, addiction, and impulse control disorders. BMB-101 demonstrated an excellent safety and tolerability profile. 5-HT2C target engagement was demonstrated by transient, dose-dependent increases in prolactin. BMB-101 exhibited predictable plasma pharmacokinetics with relatively small inter-individual variability. The current formulation allows for twice-a-day oral dosing, and with further formulation development, there may be potential for once-a-day dosing. Based on these observations, the Company believes that moderate doses of BMB-101 will fully engage 5-HT2C receptors, and therefore not be dose-limited by side effects, which will help to achieve maximal efficacy in future Phase 2 studies. Dose limited side effects exhibited by first generation 5-HT2C agonists have prevented exploiting the full potential of this pharmacological mechanism.

“We are highly encouraged by the Phase 1 study observations and results, which give us confidence in selecting doses of BMB-101 for testing in refractory epilepsies and other disorders where serotonin 2C agonists are indicated. Learnings from the study will inform our path forward as we seek to develop effective therapeutic options with convenient dosing regimens for patients.”

Mark A. Smith, M.D., Ph.D., Chief Medical Officer of Bright Minds.

“There is a great opportunity and an unmet need to develop improved treatments for these and potentially numerous other indications, including psychosis and addiction disorders. The successful and on-time completion of the study is an important achievement for us, as we continue to evolve from a drug discovery to a drug development stage company. BMB-101 is now a Phase 2 ready asset, and we look forward to sharing our further progress,” stated Ian McDonald, CEO of Bright Minds.

The Company is currently awaiting the qEEG (Quantitative Electroencephalogram) data and will provide a more detailed discussion of the Phase 1 results when available.

About the Phase 1 Study

Part 1 – Single Ascending Dose

  • 4 cohorts (6 drug and 2 placebo) – single dose (oral solution)
  • Reached the planned top dose of 180 mg/70 kg, which approached preclinical exposure limits
  • Well tolerated with predictable PK
  • Most common adverse event was oral paresthesias from liquid formulation

Part 2 – Food Effect

  • 12 subjects – crossover with and without breakfast, 120 mg/70kg
  • Effect of food on BMB-101 levels was relatively small, and therefore BMB-101 can be administered without the need for fasting

Part 3 – Multiple Ascending Dose

  • 4 cohorts (6 drug and 2 placebo) – twice a day dosing for seven days after meals
    • Reached a top dose of 150 mg/70 kg twice a day
  • Biomarkers for central target engagement: Prolactin release and qEEG

Good Manufacturing Practices (GMP) production completed for BMB-101 drug substance and drug product.

About BMB-101

BMB-101, a highly selective 5-HT2C, Gq-protein biased agonist, has demonstrated compelling activity in a host of in vitro and in vivo nonclinical tests. Compared to Lorcaserin, BMB-101 exhibits strong Gq biased signaling, coupled with minimal beta-arrestin recruitment. Bright Minds believes that G-protein biased signaling translates to better tolerance profile for this second-generation 5-HT2C agonist, making BMB-101 a best-in-class 5-HT2C agonist. Mechanistically, Serotonin (5-Hydroxytryptamine, 5-HT) is a monoamine neurotransmitter widely expressed in the central nervous system, and drugs modulating 5-HT have made a major impact in mental health disorders. Central 5-HT systems have long been associated with the control of ingestive behaviors and the modulation of the behavioral effects of psychostimulants, opioids, alcohol, and nicotine. Results of clinical trials and animal studies indicate that 5-HT2C receptor agonists may have therapeutic potential in the treatment of addiction by decreasing the intake of opioids as well as impulsive behavior that can escalate compulsive drug use. BMB-101 is a new chemical entity (NCE) and constitutes as a novel scaffold 5-HT2C agonist.

5-HT2C agonism is a well proven anticonvulsant mechanism. In translational animal models, BMB-101 demonstrated a significant reduction in both the number and intensity of epileptic seizures and is a promising candidate for the treatment of Dravet Syndrome and other epilepsies. The Phase 1 trial (NCT 05397041) has been completed and BMB-101 is now Phase 2 ready.

About Bright Minds

Bright Minds is focused on developing novel transformative treatments for neuropsychiatric disorders, epilepsy, and pain. Bright Minds has a portfolio of next-generation serotonin agonists designed to target neurocircuit abnormalities that are responsible for difficult to treat disorders such as resistant epilepsy, treatment resistant depression, PTSD, and pain. The Company leverages its world-class scientific and drug development expertise to bring forward the next generation of safe and efficacious drugs. Bright Minds’ drugs have been designed to potentially retain the powerful therapeutic aspects of psychedelic and other serotonergic compounds, while minimizing the side effects, thereby creating superior drugs to first-generation compounds, such as psilocybin.