8:00 am
Virtual Coffee & Online Registration

8:45 am Chair’s Opening Remarks


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

10:40 am
Morning Refreshments

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 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

12:15 pm Assess the Latest Data & EGFRlow Hypothesis & Trial of Onc201

  • Josh Allen Chief Scientific Officer, Oncoceutics


  • Session details TBC

12:45 pm
Networking Lunch

1:45 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

2:15 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

2:45 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

3:15 pm
Afternoon Refreshments


3:30 pm Realizing Potential: Taking GDC-0084 Forward in Glioblastoma

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


  • Identifying a target population: newly-diagnosed versus recurrent patients
  • Addressing the challenges and opportunities in designing a definitive clinical proof-of-concept for GBM
  • Defining a path-to-market for GDC-0084
  • Establishing the commercial prospects for a new drug in GBM

4:15 pm Clinical Case Study: Driving a New Therapeutic Strategy to Accelerate to the Clinic


  • Exportin 1 (XPO1) levels are elevated in most glioblastomas (GBM) versus glial cells and tumors that express higher XPO1 levels have poor prognosis with shorter survival; similar observations apply to other tumors
  •  Selinexor is first-in-class, oral, Selective Inhibitor of Nuclear Export (SINE) compound that forces the nuclear retention and activation of most tumor suppressor proteins (e.g., p53, p21, p27, pRB) and reduces levels of certain oncoproteins (e.g., c-myc, cyclin D1)
  • Selinexor was recently approved by the FDA to treat refractory multiple myeloma (XPOVIO™) and has good brain penetration with significant activity in xenograft and patient-derived orthotopic GBM models
  •  Selinexor has shown single agent activity in phase II (“KING” Study) in patients with relapsed or refractory GBM following ≥1 prior regimen with an ORR of ~10% and 6-cycle PFS of 30%, or 6-months PFS of 19%; dosing is once weekly oral with low grade gastrointestinal effects and mild thrombocytopenia

4:45 pm Clinical Case Study: Inovio’s Innovative Combination Trial


  • Optimizing T cell-generating therapies in combination with PD-1/PD-L1 inhibitors for GBM
  • Overview of the INO-5401 clinical trial and challenges to overcome in translation to phase 2 trials

5:15 pm PANEL DISCUSSION: Lessons Learned from Failed and Discontinued Clinical Trials for the Treatment of Glioblastoma


  • 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

6:00 pm
Chair’s Closing Remarks

6:15 pm
End of 2nd Glioblastoma Drug Development Summit