Day Two

Thursday, January 20, 2022

8:00 am Chair’s Opening Remarks

A Deep Dive into DNA Damage Repair for GBM

8:10 am Small molecule epichaperome inhibitors to treat cancers and neurodegenerative disease


  • Icapamespib (PU-AD) a small molecule epichaperome inhibitor is being
    developed for brain cancers and neurodegenerative diseases
  • Icapamespib is BBB permeable, inhibits epichaperome activity,
    initiates degradation of disease associated proteins, normalizes cellular
    pathways and causes cancer cell death or recovery of neuronal functions
  • In preclinical studies icapamespib decreased tumor growth in human GBM
    explant xenografts, initiated degradation of oncoproteins and increased survival

8:30 am Targeting the DNA Damage Response in Glioma: Emerging New Concepts and Novel Directions

  • Ranjit Bindra Professor, Therapeutic Radiology, Yale University School of Medicine


  • Overview of the DNA damage response (DDR) and how it can be targeted therapeutically for GBM
  • Review on promising new DDR inhibitors and DNA damaging agents, which are either in development or in the clinic for GBM
  • Discuss emerging DDR targets, key hurdles, and future directions in the GBM space

9:00 am Tumor treating fields (TTFields) in glioblastoma

  • Chirag Patel Clinical Assistant Professor, Stanford University


  • Overview of tumor treating fields (TTFields), a form of alternating electric fields therapy in cancer
  • Review the effects of TTFields on DNA damage repair (DDR) in glioblastoma
  • Discuss the membrane permeabilizing effects of TTFields in glioblastoma and combination strategies with other therapies’

9:30 am Morning Break & Networking

Financing BioPharma for Rapid GBM Drug Development

10:40 am Panel: It’s All in the Money – Funding Challenges & Opportunities in the GBM Space


  • 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?

Optimizing Immunotherapies for GBM Drug Development

11:40 am Inovio’s DNA Medicine for the Treatment of Glioblastoma


  • Using novel DNA-encoded medicines to treat human cancers such as GBM
  • Generating robust, specific anti-tumor T cells against GBM antigens
  • Combining DNA medicine with immune checkpoint inhibitors to build
    clinical responses

12:10 pm Targeting EGFR in in glioblastoma with a novel brain-penetrant small molecule EGFR-TKI


  • A novel EGFR-TKi with extraordinary brain-selective distribution ( >20-fold brain to plasma ratio)
  • It has a favorable oral PK and safety profile with a low systemic toxicity
  • Superior activity against GBM in preclinical models (patient-derived and GEM models)

12:40 pm Mechanisms of Immunosuppression

  • Michael Lim Professor & Chair Department of Neuro Surgery, Stanford University School of Medicine


  • Understand current landscape of immunotherapy for brain tumors
  • Understand the current obstacles for developing effective immunotherapies for brain tumors

1:10 pm Lunch & Networking

2:10 pm Presentation Slot Reserved for Istari Oncology


2:40 pm Leveraging the Lysosome to Treat Glioblastoma


  • BXQ-350 – a unique, first in class, lysosomal protein (in lipid nanovesicle) involved in cellular homeostasis
  • Phase 1 clinical trial findings of extraordinary safety and tolerability with monotherapy effect
  • Planned clinical trials in newly diagnosed GBM – combination therapy
    with radiation and temozolomide

3:10 pm Optimization of an Oncolytic HSV-1 for the Treatment of Glioblastoma


  • Optimization of oHSV backbone 7 Transgenes
  • Development of clinical candidate oncolytic virus

3:40 pm Afternoon Break & Refreshments

4:10 pm Targeting Clonal Heterogeneity In Treatment-Refractory GBM With Empiric Polytherapy


  • Review the biological profile of treatment-refractory glioblastoma
  • Explore the application of an integrative multi-omics target discovery platform for human GBM which incorporates DNA cellular barcoding, glycocapture profiling of the cell surface proteome, RNA sequencing, single-cell sequencing and genome-wide CRISPR screening of primary and recurrent GBM
  • Review the rationale for combinatorial polytherapy for targeting intratumoral heterogeneity and therapy-driven clonal evolution found in GBM recurrence

4:40 pm Novel Hydrogel-Based Localized Treatment For Glioblastoma


  • Discovery of a novel target for Glioblastoma using a machine learning algorithm
  • Development of a tailored hydrogel-based drug delivery system to be used as adjunct to surgery
  • Presentation of preclinical in vitro and in vivo data supporting clinical

5:10 pm End of Day Two