8:00 am
Virtual Coffee & Online Registration

8:45 am Chair’s Opening Remarks

  • Kirk Tanner Chief Scientific Officer , National Brain Tumor Society


9:00 am Examine Surgical Immunotherapy Trials Using Genes & Viruses for Brain Tumors


  • Examine the state of clinical trials for Glioblastoma
  • Consider rationale for gene and virus therapy to improve immune
  • Review scientific correlates for gene and virus therapy trials

9:30 am The Patient Perspective, Challenges Faced and Uniting our Purpose in the Fight Against GBM


  • Optimize the voice of the GBM community and the patient perspective
  • Assess COVID-19 and the added burden and difficulty for patients and
    their caregivers
  • Why we #LiveStacheStrong and how we can all unite to turn this disease
    from terminal to treatable?

10:00 am The Distinctive Design of ONCR-GBM: A MicroRNA Attenuated Oncolytic HSV for the Treatment of Glioma


  • Discuss why oncolytic viruses are a promising therapeutic approach for GBM
  • Examine ONCRGBM; an oncolytic herpes simplex virus, armed with a combination of immune transgenes selected to counteract the immune suppressive mechanisms in GBM tumors
  • Look at a progress report on ONCRGBM

10:30 am
Morning Break & Speed Networking

11:00 am PANEL DISCUSSION: Recognize the Financial Difficulty of Developing Glioblastoma Treatments and Moving Drugs to the Next Phase

  • James Garner Chief Executive Officer & Managing Director, Kazia Therapeutics Limited
  • Petra Hamerlik Associate Research Director/Bioscience, AstraZeneca
  • Ranjit S. Bindra Associate Professor of Therapeutic Radiology, Yale School of Medicine
  • Linda Liau Chair of Neurosurgery, UCLA


  • Explore the challenges of funding development
  • How can drug developers inspire and attract companies to invest in their novel preclinical glioblastoma development and pipeline?
  • How can the industry come together to overcome the paucity of funding?


11:45 am Kintara’s VAL-083: A First-in-Class Bifunctional Alkylating Agent with Promising Activity in MGMT Promoter- Unmethylated & Methylated Glioblastoma

  • James Perry Professor of Neurology, Neurologist , University of Toronto Temerty, Sunnybrook Research Institute
  • John de Groot Professor & Clinical Research Director of Neuro-Oncology, The University of Texas, MD Anderson Cancer Center


  • Examine the unmet need of an effective therapy for all GBM patients, and specifically unmethylated GBM patients
  • Evaluate updated clinical results from two Phase 2 trials of VAL-083 in newly diagnosed, adjuvant and recurrent GBM
  • Discuss clinical advantages of cytotoxic agents in treating GBM patients
  • Study Kintara’s VAL-083 participation in the Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) registrational study; newly diagnosed methylated, newly diagnosed unmethylated and recurrent GBM

12:15 pm Examine BDTX-189 – a Small Molecule Inhibitor Targeting Undrugged Oncogenic Driver Mutations of the ErbB Kinases EGFR & HER2

  • Elizabeth Buck Executive Vice President, Discovery & Translational Sciences, Black Diamond Therapeutics


  • Study how in preclinical models, BDTX-189 exhibits anti-tumor activity
    evidenced by potent tumor growth inhibition and tumor regression.
  • Examine BDTX-189 – cleared by the FDA and initiated to Phase 1/2
    clinical trials
  • Explore the preclinical data which illustrates how BDTX-189, a novel
    ATP-site small molecule, selectively inhibits the activity of a broad range
    of allosteric EGFR and HER2 mutants, producing growth regression of
    allosteric EGFR mutant patient-derived tumors in vivo

12:45 pm Targeting DRD2 and ClpP in High Grade Glioma with ONC201: Emerging Clinical Results and Future Trials

  • Josh Allen Chief Scientific Officer, Oncoceutics


  • Review DRD2 as therapeutic target in oncology
  • Update on the latest ONC201 clinical results in H3 K27M-mutant gliomas
  • Overview of ONC201 in other CNS tumors and emerging trials

1:15 pm
Networking Lunch

2:15 pm Study PI3K as a Key Target in Glioblastoma

  • James Garner Chief Executive Officer & Managing Director, Kazia Therapeutics Limited


  • Explore opportunities and challenges associated with modulating PI3K
    as a therapeutic strategy in glioblastoma
  • Examine The significance of biomarkers in patient selection
  • Review emerging clinical data from paxalisib, a brain penetrant PI3K /
    mTOR inhibitor

2:45 pm Target DDR Pathways in GBM, From Mechanistic Insights to Advances in the Clinic

  • Ranjit S. Bindra Associate Professor of Therapeutic Radiology, Yale School of Medicine


  •  Update on DDR pathways as they pertain to GBM
  • Overview on the latest DDR inhibitors, molecular biomarkers, and their
    use in clinical trials
  • Examine new trials targeting GBM with DDR inhibitors and DNA
    damaging agents

3:15 pm Determine the Functional Interplay Between Aberrant DNA Repair & Cellular Hierarchies in Glioblastoma


  • Examine the diverse causes of therapeutic resistance and how hierarchical organization of this disease is assumed a plausible cause of treatment failure, leading to tumor recurrence
  • Assess how DNA damage poses a serious threat to cell survival by compromising both genomic and epigenomic integrity
  • Study results providing mechanistic insights into how SPT6 regulates DNA repair and identifies SPT6 as a putative therapeutic target in glioblastoma


3:45 pm
Afternoon Refreshments

4:15 pm GBM Agile: Identify More Effective Therapies for the Right Patients Faster

  • Brian Alexander Associate Professor of Radiation Oncology, Harvard Medical School & Dana-Farber/ Brigham and Women’s Cancer Center, GBM Agile Global Coalition for Adaptive Research


  • Transform clinical trials by designing an adaptive platform trial: Adaptive Global Innovative Learning Environment for Glioblastoma (GBM AGILE)
  • How is GBM AGILE creating a rich biomarker repository and paving the way for Bayesian analysis of multiple GBM drugs?
  • Examine how GCAR aims to run similar adaptive Bayesian trials for other rare or deadly diseases: trailblazing adaptive platform trials

4:45 pm Use External Data to Design Efficient Clinical Trials in Glioblastoma with Externally Controlled Trial


  • Discuss sources of external data and challenges to data access and data-sharing
  • Design and evaluate the use of real-world evidence and clinical trial data to develop external control arms in GBM
  • Assess the utility of external data, including real world data and clinical trial data, to increase efficiency of clinical trials in GBM

5:15 pm Update on Response Assessment Endpoints in Neuro-oncology Clinical Trials

  • Susan Chang Director, Neuro & Oncology, University of Califorina, San Francisco


  • Provide an overview on the value of assessing response endpoints in clinical trials in neuro-oncology
  • Review current criteria for response assessment- strengths and challenges
  • Discuss novel imaging approaches to response assessment

5:45 pm PANEL DISCUSSION: Lessons Learned from Failed and Discontinued Clinical Trials for the Treatment of Glioblastoma

  • Susan Chang Director, Neuro & Oncology, University of Califorina, San Francisco
  • Jeffrey Skolnik Vice President, Inovio Pharmaceuticals
  • Robert Dillman Chief Medical Officer, AIVITA Biomedical
  • David Andrews Co-Founder, Chief Medical Officer, Imvax
  • David Reardon Clinical Director, Center for NeuroOncology, Dana-Farber Cancer Institute


  • Debate the common challenges with moving GBM clinical trials forward
  • What can we learn from the plethora of failed phase 2 trials?
  • With a high failure rate of phase 3 glioblastoma trials, panellists examine the need for greater reliability of earlier studies

6:30 pm
Chair’s Closing Remarks

  • Kirk Tanner Chief Scientific Officer , National Brain Tumor Society

6:45 pm
End of 2nd Glioblastoma Drug Development Summit