FDA Partially Places Phase 2 Study of BMB-101 on Clinical Hold Over Safety Data
Key Facts
The U.S. Food and Drug Administration placed Bright Minds Biosciences’ investigational new drug application and associated protocol for BMB-101-105 on partial clinical hold following the agency’s review, according to a company statement published on November 2. The application concerns a planned U.S. Phase 2 study to treat seizures associated with developmental and epileptic encephalopathies. The company said the decision is limited to this proposed study and does not affect its other ongoing clinical trials. The hold suspends administration of the investigational drug to participants in the study covered by it, according to the FDA’s definition. The announcement did not include a price or percentage for the stock’s trading after its release; EL7’s available price data ends on October 30, before the statement was published.
The study whose start is now on hold was designed as a randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of BMB-101 across escalating doses up to the maximum tolerated dose. Its proposed scope included treating seizures in patients with developmental and epileptic encephalopathies, a population in which enrolling children requires safety data appropriate for that age group. The FDA cited as its primary reason toxicity observed in rats, along with insufficient pharmacokinetic and toxicology data in juvenile animals to support the proposed participation of children. The hold therefore relates to nonclinical findings and the adequacy of the data package before the study begins, not to the suspension of an ongoing U.S. trial. The company said the toxicity findings in rats had not appeared in other species and that it considers them incidental and unrelated to the drug; this is the company’s assessment, not an agency conclusion.
The decision comes after Bright Minds said it had completed two clinical trials of BMB-101 and that about 90 participants had received the drug through its clinical programs as of November 2. The company said its Phase 1 studies included 64 healthy volunteers and that the drug was well tolerated at the doses tested, with no serious drug-related adverse events recorded. The open-label, multicenter BREAKTHROUGH study also included 24 adult patients: 15 in the absence-seizure group and 9 in the developmental and epileptic encephalopathies group. The company described the efficacy, safety and tolerability data associated with those results as preliminary and subject to change with further analyses and completion of audit and verification procedures. These results therefore provide prior clinical context for the drug, while the FDA’s current concerns relate to the toxicology and pharmacokinetic data needed to design the proposed study and enroll children.
The company is awaiting the complete clinical hold letter, which the FDA said it would provide within 30 days of the announcement, including further details and recommendations related to the hold and study design. Bright Minds said it would immediately seek further clarification, then review the letter and revise the study design in line with the agency’s recommendations. A response may require additional data or other studies, according to the company. There is no guarantee as to when the partial hold will be lifted or whether it will later allow U.S. trials to proceed, the company said. The next steps will therefore be determined after the company receives the FDA letter and reviews its requirements.