The US Food and Drug Administration (FDA) has placed a partial clinical hold on Bright Minds Biosciences’ Phase II seizure trial, with the company’s stock sinking more than 50% on the news.
The FDA notified the company via email that its investigational new drug application (IND) for developmental and epileptic encephalopathies (DEEs) and related protocol for BMB-101-105 has been placed on a partial clinical hold.
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The FDA hold relates to identified toxicity in mice, and inadequate pharmacokinetic (PK) and juvenile toxicity data to support a proposed paediatric enrolment.
The company was planning to launch a Phase II randomised, placebo-controlled, double-blind study designed to evaluate the efficacy and safety of ascending doses of BMB-101, up to a maximum tolerated dose, for the treatment of seizures associated with DEEs.
DEEs are a group of brain disorders that are characterised by frequent seizures that begin in early childhood. Because of the complex physiology behind these seizures, traditional antiseizure medications often do not work.
The company believes that the partial clinical hold covers only the planned DEE study and will not affect other ongoing studies.
The company states that the mouse toxicity was not seen in any other species and that according to toxicity reports, peer reviewers, and multiple independent toxicology expert opinions, the adverse mouse findings were “incidental and not drug-related”.
While the company is awaiting the FDA’s full partial holder letter, it will also request further clarification from the FDA. Based on the FDA’s views, the study will be refined accordingly.
After the clinical hold was announced, the company’s stock dropped by 52.34% to $26.20 on 2 November close from a 30 October close of $54.98.
The company has completed two clinical trials with BMB-101, enrolling a total of 88 participants.
This included the Phase I, placebo-controlled single-ascending-dose, multiple-ascending-dose and food-effect studies conducted in 64 healthy volunteers, in which BMB-101 was well tolerated at all doses tested.
The company also evaluated the drug in the Phase II BREAKTHROUGH trial, for which topline data were released on 6 January. The study enrolled 24 adults with drug-resistant absence seizures or DEE, most of whom were taking three to five other anti-seizure medications.
In the study, 79.6% of treatment-emergent adverse events were mild, and 17.2% were moderate. No drug-related serious adverse events occurred. Six DEE patients completed the main study treatment with a median reduction in motor seizures of 63.3%. All five DEE patients who entered the open-label extension remain on therapy with BMB-101.
GlobalData reports that the adult epilepsy market across the seven major markets (7MM: the US, Germany, France, Italy, Spain, the UK, and Japan) is projected to grow from $11.4bn in 2025 to $17.8bn by 2035.
