Explore the Agenda
Workshop A
8:00 am Harnessing Omics Data & Molecular Subtyping to Inform Patient Stratification in Clinical & Translational Strategies in Glioblastoma Drug Development
- Join this workshop to deepen your understanding of how omics approaches and molecular subtyping are used to stratify glioblastoma patients and define target populations for new therapies
- Examining the strategic integration of multi-omics including genomics, transcriptomics, proteomics and epigenomics for a more comprehensive and clinically actionable GBM classification system
- Identifying and validating specific molecular features as robust markers for defining GBM subtypes with prognostic and predictive value
- Leveraging molecular subtyping further down the line in development to design biomarker-driven clinical trials to enrich patient experience and improve trial efficiency
- Identifying and validating novel biomarkers with a focus on their identification in discovery
- Looking towards personalized GBM treatment where therapy selection is guided by a patient’s molecular profile
Workshop D
8:00 am Advancing Imaging Techniques to Tackle Challenges Associated with Pseudo-progression & Pseudo-response in Glioblastoma Patients
- Join this workshop to discuss best practices and future innovations in brain imaging to distinguish between pseudo-responses and real changes more effectively in GBMs
- Exploring strategies to avoid serial brain biopsies GBM drug development to accurately assess dose selection and discover new biomarkers without invasive techniques
- Discussing advanced neuroimaging strategies to address the challenges of high glucose uptake in the brain such as novel PET ligands that target GBM metabolic pathways beyond glucose
- Evaluating the RANO assessment in modern GBM trials, focussing on how pseudo-progression and pseudo-responses can confound a drug’s true efficacy
- Exploring novel and complementary assessment methods, such as volumetric analysis, advanced imaging biomarkers, and the integration of iRANO criteria for GBM immunotherapies, that can provide a more accurate and reproducible picture of tumor response
- Addressing the practical challenges of standardizing new methodologies across different clinical sites and the regulatory considerations for their use as primary or secondary endpoints
10:30 am Morning Refreshments
Workshop B
11:00 am Exploring Strategies to Define Direct Vs Indirect Target Engagement with Glioblastomas
- Join this workshop to establish a clear framework for defining direct target engagement direct vs indirect target engagement
- Navigating the application of cutting-edge preclinical methodologies to confirm on-target binding with GBM cells such as mass spectrometry, thermal shift assays and radiolabels compounds for PET imaging to visualize drugtarget engagement.
- Analyzing changes in signalling pathways, gene expression and immune cell phenotypes within the GBM tumour microenvironment
- Advancing biomarker tracking within the blood-brain barrier to track drug effect with ctDNA and immune markers
- Deepening the understanding of direct vs indirect engagement to understand the design rationale of combination therapies
- Driving the analysis of PK/PD markers to monitor the emergence of drug resistance in recurring GBMs
Workshop E
11:00 am Assessing Current Clinical Biomarkers & Defining a Future Gold Standard for Surrogate Endpoints in Glioblastoma Clinical Development
- Join this workshop to establish a future direction towards achieving a gold standard surrogate endpoint for GBM clinical trials
- Discussing innovative, non-invasive strategies for dose selection and defining a biologically effective dose in GBM trials such as using advanced imaging biomarkers and liquid biopsies from CSF to overcome the infeasibility of serial brain biopsies
- Exploring the challenges in gaining regulatory approval and validate advanced imaging biomarkers to build a strong scientific case for their use as reliable indicators of therapeutics response
- Diving into the limitations of using ‘Progression Free Survival’ as a surrogate endpoint for overall survival in GBM due to pseudo-progression and designing trials that can use complementary endpoints to accurately differentiate between treatment-based inflammation and true disease progression
- Addressing the FDA’s hesitation to accept PFS as a standalone surrogate for accelerated approval in GBM
- Exploring alternative or composite endpoints that could meet regulatory standards
1:00 pm Lunch & Networking
Workshop C
2:00 pm Moving Towards Standardized Preclinical & Translational Study Methodology to Advance the Next Generation of Glioblastoma Models
- Join this workshop to collectively work towards standardized and effective preclinical GBM models to ensure convincing preclinical data is produced to reduce regulatory bottlenecks
- Acknowledging the current limitations with preclinical and translational models that mimic the human immune system for immunotherapy research in Glioblastoma
- Discussing strategies to overcome the challenges of in preserving cellular and mutational diversities in primary tumours to overcome the prolonged development time
- Advancing the use of K9 models as an improved model for paediatric Glioblastomas
- Exploring patient-derived glioblastoma organoid models to recapitulate the heterogeneity of glioblastomas to overcome the complexities of developing effective therapeutic strategies
- Evaluating the practical applications of glioblastoma organoids for rapidly testing patient-specific treatment strategies involving correlating mutation profiles with drug responses to advance personalized therapies
Workshop F
2:00 pm Reconsidering Approaches to Regulatory Interactions to Overcome Challenges in Dose Selection, Preclinical Data Package and Clinical Endpoint Selection
- Join this workshop to enjoy candid and actionable discussions around effectively communicating with regulatory agencies
- Exploring the necessary scientific and clinical data required to successfully advocate for regulatory waivers or alternative review pathways, particularly for a disease with a high failure rate and limited treatment options
- Innovative strategies for presenting a robust scientific rationale and a compelling data package that can persuade agencies to be more flexible, and for establishing a collaborative dialogue from the pre-IND stage through clinical development
- Discussing the validation of non-traditional endpoints and building strong scientific cases to convive regulators to accept them for accelerates approval, particularly given the
- challenges with endpoints like PFS and OS
- Discussing the benefits of a balanced perspective, where regulatory professionals from both the pharmaceutical industry and agencies can share their insights to better align on expectations and overcome shared challenges
- Discussing what a more flexible, adaptive regulatory framework for GBM drug development could look like including streamlining trial designs, facilitation biomarker driven
- patient selection and creating a clearer path to approval that incentivizes innovation whilst maintain patient safety