Bright Minds Biosciences has suffered a regulatory setback after the US Food and Drug Administration (FDA) placed a partial clinical hold on its investigational drug application for BMB-101, targeting Developmental and Epileptic Encephalopathies (DEEs).

 

The company said the FDA notified it by email that its Investigational New Drug (IND) application and related Protocol BMB-101-105 had been placed on partial clinical hold following the agency’s review.

 

The decision affects the planned US Phase 2 trial of BMB-101 in patients with DEEs, a group of severe epilepsy disorders. The proposed study is a randomised, double-blind, placebo-controlled trial designed to assess the drug’s safety and efficacy at escalating doses, up to the maximum tolerated dose.

 

The FDA’s primary concerns centre on toxicity identified in mice, along with what it considers inadequate pharmacokinetic and juvenile toxicity data to justify the proposed enrolment of paediatric patients.

 

Bright Minds, however, has challenged the significance of the mouse toxicity findings, arguing that the observations were not replicated in other animal species.

 

The company said that, based on its toxicity reports, peer reviews and multiple independent toxicology expert opinions, it believes the adverse findings in mice were incidental and not related to the drug.

 

However, these are the company’s interpretations and do not represent the FDA’s findings or resolve the regulator’s stated safety concerns.

 

Bright Minds believes the partial clinical hold is restricted to the planned US Phase 2 DEE trial and does not affect its other ongoing clinical studies.

 

The company is now awaiting the FDA’s formal Partial Clinical Hold Letter, which the regulator has said will be issued within 30 days. The letter is expected to provide further details of the concerns, recommendations that do not require a clinical hold and guidance on potential changes to the study design.

 

Bright Minds said it intends to seek immediate clarification from the FDA and review the regulator’s recommendations before refining the proposed study.

 

The company aims to address the identified concerns and advance future US clinical trials. However, additional data or studies may be required, and there is no guarantee that the partial hold will be lifted or that future US trials will be allowed to proceed.

 

The FDA’s decision marks a significant hurdle for Bright Minds’ planned paediatric epilepsy programme, with the next steps likely to depend on the regulator’s detailed assessment of the safety data and the additional evidence required to support the trial.