FDA places Bright Minds' BMB-101 DEE IND on partial clinical hold

FDA targets IND and related protocol

Bright Minds Biosciences said the FDA notified it by email that its IND for BMB-101 in developmental and epileptic encephalopathies and related Protocol BMB-101-105 had been placed on partial clinical hold after the agency's review. The proposed study is a randomized, placebo-controlled, double-blind Phase 2 trial. It is designed to evaluate efficacy and safety of ascending doses of BMB-101 up to a maximum tolerated dose for seizures associated with DEEs. DEEs are a group of severe epilepsy disorders.

The company said it believes the hold covers only the planned US Phase 2 trial and does not affect its other ongoing clinical trials. That scope matters because the FDA action is a partial hold on the IND and protocol, not a full stop on the company's other work.

Mouse toxicity and pediatric data gap

The FDA's primary reason was identified toxicity in mice and inadequate pharmacokinetic and juvenile toxicity data to support the proposed pediatric enrollment, the company said. Bright Minds disputes how the mouse data should be read. It said the toxicity seen in mice was not observed in any other species. The company believes the findings were incidental and not drug-related, based on its toxicity reports, peer reviewers and multiple independent toxicology expert opinions.

Those are the company's beliefs. Bright Minds said they should not be understood as FDA findings or as resolving the agency's stated concerns. The company was awaiting the FDA's full Partial Clinical Hold Letter when it disclosed the hold.

Prior BMB-101 safety data

Bright Minds has completed two clinical trials involving BMB-101. About 90 participants have received the investigational treatment across its clinical programmes, the company said. In Phase 1 studies involving 64 healthy volunteers, BMB-101 was reported to be well tolerated across the doses tested. No serious adverse events occurred, and reported adverse events were mild and consistent with the compound's serotonergic activity.

Those adult volunteer results do not address the FDA's pediatric objection. The agency cited inadequate juvenile toxicity data, a different dataset, and pharmacokinetic data it called inadequate.

Shares fall by half

Bright Minds shares closed 52.35% below the previous close on 2 November, the day of the disclosure. Investing.com reported the stock fell as much as 52% in morning trade that day. Volume was 12.8 times the stock's average daily volume in the session, according to StockTitan Argus data.

Levi & Korsinsky said it is investigating potential securities law violations. The firm said it is examining whether Bright Minds adequately disclosed known regulatory risks tied to preclinical toxicity findings and to insufficient pharmacokinetic and juvenile toxicity data. The investigation is pending. The firm is inviting shareholders who lost money to request a review of their losses.

What happens next

The FDA stated it would provide the full Partial Clinical Hold Letter within 30 days, including additional details, non-hold recommendations and study-design recommendations. Bright Minds said it intends to immediately request further clarification from the FDA. It plans to review the letter and refine the study in accordance with the agency's recommendations.

The company said additional data or studies may be required. It gave no assurance on when or whether the partial clinical hold will be lifted. It also gave no guarantee that future US trials will be permitted to proceed. Bright Minds said it aims to address the identified concerns and advance future US clinical trials.