Objectives & Work Plan

Advance BEA-17 through chemistry, manufacturing and controls, plus GLP preclinical safety studies, aiming to produce a pharmaceutical and non-clinical package that can support a future Clinical Trial Application.^{[Beactica (2025)]}

Develop candidate predictive and pharmacodynamic biomarker readouts intended to support future patient selection, translational decisions, and assessment of BEA-17 activity.

Aim to build the regulatory documentation and biomarker strategy needed to support a future European CTA, subject to successful completion of the planned studies and regulatory review.^{[CORDIS GLIOBREAK]}

GLIOBREAK will optimise the BEA-17 manufacturing route for scalability and reproducibility, establish analytical characterisation of the drug substance, and qualify stability-indicating methods aligned with regulatory expectations.

The project will conduct GLP-compliant toxicology studies with ADME profiling, CNS penetration assessment, bioanalytical method qualification, and safety pharmacology covering cardiovascular, CNS, and respiratory systems.

The intended output is a documented drug candidate supported by chemistry and non-clinical safety data designed to support a future CTA or IND. Submission will depend on successful study completion and regulatory feedback.^{[CORDIS GLIOBREAK]}

Single-cell RNA sequencing and multi-omics profiling will define transcriptional and epigenetic signatures linked to BEA-17 sensitivity, especially tumours with high LSD1/CoREST activity and immune suppression.

Matched biopsy profiling will track changes in LSD1/CoREST levels, chromatin state, immune gene expression, and ERV de-repression, with liquid biopsy approaches added where feasible.

The biomarker programme aims to generate a candidate assay concept and patient signature. Any decision thresholds or companion-diagnostic use would require further analytical and prospective clinical validation under the applicable regulatory framework.^{[EU IVDR 2017/746]}

The regulatory workstream will consolidate evidence from manufacturing, formulation, pharmacokinetics, toxicology, in vivo efficacy, and bioanalysis, identifying and closing the gaps that must be addressed before a Clinical Trial Application.

GLIOBREAK aims to assemble quality, non-clinical, and supporting clinical documentation intended to support a future European CTA and first-in-human study planning, subject to study outcomes and regulatory feedback.^{[CORDIS GLIOBREAK]}

Candidate patient-selection and pharmacodynamic biomarkers could inform a future clinical-development strategy. Assay development and any use in a first-in-human study would require further validation and consideration of the applicable EU regulatory framework.^{[EU IVDR 2017/746]}