March 1, 2023

Phase 1 Study of BMB-101 in Healthy Volunteers Moves to Last Part 

Article published by Dravet Syndrome News


A Phase 1 clinical trial that enrolled healthy volunteers to test BMB-101, Bright Minds Biosciences’ investigational treatment for Dravet syndrome, has moved to its final part, the company announced in a corporate update. 


This first-in-human trial of the oral therapy, which is testing BMB-101 at different doses, now is in its multiple ascending dose (MAD) phase. 

The trial’s first two parts — evaluating a single ascending dose (SAD) and the effect of food on the medication — are now complete. 


According to Bright Minds, the therapy has shown a favorable safety profile, and was tolerated well both with and without food. The study is expected to be completed, with available data, between April and June. 


“We are pleased with the progress of our Phase 1 first-in-human trial of BMB-101 and believe there are large potential market opportunities for the treatment of refractory [hard-to-treat] epilepsies,” said Ian McDonald, CEO and co-founder of Bright Minds Biosciences.

BMB-101 is an orally available molecule that works by activating a specific serotonin receptor, called 5-HT2C, while avoiding others that could cause side effects. Serotonin is a major brain chemical messenger whose abnormally low activity and levels have been reported to be associated with seizure onset and Dravet syndrome. 


As such, activating certain serotonin receptors may have protective effects against seizures. 


In preclinical studies, BMB-101 showed a protective effect against seizures in a zebrafish model of Dravet syndrome and in two rodent models of seizures. Also, repeated dosing of BMB-101, for nearly a month, was deemed safe and well-tolerated in mice and dogs. 


The Phase 1 trial (NCT05397041), which began dosing in September 2022, is underway at CMAX Clinical Research, in Adelaide, Australia. The study aims to evaluate the Dravet syndrome treatment candidate’s safety, tolerability, and pharmacokinetics against a placebo in an estimated 76 healthy adults. Pharmacokinetics are a medication’s movement into, through, and out of the body. 

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