Phase 1 study of BMB-101 in healthy volunteers moves to last part

Bright Minds' Dravet syndrome treatment shows promise in trial

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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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.

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Preclinical Results Pave Way for Human Trial of BMB-101 Oral Therapy

Testing possible doses of Dravet syndrome treatment

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.

In its SAD part, involving 32 participants, the investigational therapy was administered as an oral solution in four evaluated doses. According to Bright Minds, BMB-101 was well tolerated by individuals at all four doses. The most common adverse effect was a prickling sensation in the mouth.

The therapy also showed a favorable pharmacokinetics profile, with the highest dose approaching preclinical exposure limits.

In part two, 12 new participants were randomly assigned to receive a single dose of BMB-101, taken with or without a standard high-fat breakfast. Participants then crossed over to the other group.

Data showed that the experimental therapy was well tolerated with and without food, and that eating had a relatively small effect on its pharmacokinetics. This suggests that BMB-101 can be administered without the need for fasting, according to Bright Minds.

In the ongoing MAD part, up to 32 participants will be randomly assigned to receive either BMB-101 or a placebo, twice a day for seven days, after meals. A total of four doses will be tested. The first dose group is now complete.

BMB-101 also is being evaluated as a potential treatment for opioid use disorder, binge eating disorder, and Alzheimer’s disease.

Bright Minds also has completed procedures to produce BMB-101 according to good manufacturing practices requirements, which ensure products are manufactured according to quality standards.