Conference Day Two

Thursday 16th March 2023

8:30 am Registration & Welcome Coffee

8:55 am Chair’s Opening Remarks

Facing the Funding Dilemma: Investors, Non-Profits & Biotechs Weigh In On the Variety of Funding Avenues for GBM Research

9:00 am Changes in the financing landscape for glioma drug development & ONC201

  • Michael Andriole Chief Financial Officer & Chief Business Officer, Chimerix Inc


  • Historical challenges of glioma drug development and funding constraints
  • Improving landscape of investing in glioma drug development
  • Applying lessons learned to ONC201 (dordaviprone) development

9:15 am Financing the Unfinanceable : An Optimistic Perspective From Europe


  • Let’s learn from history: the temozolomide precedent.
  • Let’s learn from others: the rare diseases examples.
  • Being wise, not too big, not too early : a living dog is better than a dead lion.”

9:30 am Panel Discussion: Navigating the Catch-22 With GBM Funding: How Can Investors & Companies Work Together to Solve This Dilemma?
Moderated by: Scott Rakestraw


  • Addressing how to attract investment for the indication that the proposition has not yet been clinically proven
  • Discussing gaining support for novel strategies, including combination trials especially when monotherapy has not been proven in isolation
  • Evaluating preclinical models in their reliability to reflect clinical outcomes and which models appeal to investors the most
  • Comparing the two main sources of funding- public vs private funding
  • Transforming development and business models in GBM research

10:30 am Morning Networking Break


Meet and connect with your peers in this dedicated networking session.

Exciting Opportunities on the Horizon: Exploring Next Wave Therapeutic Modalities

11:00 am How Do Human Glioblastoma-Derived Tumorspheres Translate to an Orthotopic Intracranial Model System?


  • Identifying potentially efficacious agents with 3D spheroids developed from primary patient tissue
  • Testing single agent and combinatorial effect predictions with radiation in an in vitro setting
  • Comparing in vitro x-ray radiation to in vivo intracranial radiation + TMZ 

11:30 am Targeted Oncolytic Viruses Armed With Immune-Modulating Transgene Delivery for Enhanced Activity


  • Understanding the critical components of efficient oncolytic virus entry and spread in GBM cells
  • Unpacking the value of arming purposeful transgenes/payloads to attack the immune suppressive GBM microenvironment

12:00 pm PI3K Inhibition With Hypoinsulinaema – A Novel Approach to Maximizing the Impact of Targeted Therapy

  • James Garner Chief Executive Officer, Kazia Therapeutics Limited


  • Reviewing the role of the PI3K pathway in glioblastoma
  • Evaluating the rationale for combining PI3K pathway inhibition with hypoinsulinaemia
  • Overview of an ongoing clinical trial to explore the combination

12:30 pm Delivering On the Promise of Glioblastoma Immunotherapy

  • Natalie Artzi Assistant Professor of Medicine, Harvard Medical School


  • Local delivery in the brain eliminates the need to cross the BBB, opening new
    GBM treatment opportunities
  • Combination therapy that can educate the immune system and generate
    immune memory is required to obtain long-lasting anti-tumor responses
  • Sustained delivery of the therapy is required to bridge the innate and adaptive
    immune responses to obtain immune memory

1:00 pm Lunch Break


Take this chance to meet the expert speakers, connect with your peers and explore our exhibition booths.

2:00 pm Viral Immunotherapy for Solid Tumors: The New Frontier

  • Paul Peter Tak Chief Executive Officer & President, Candel Therapeutics


  • CAN-2409 treatment is a systemic immunotherapy delivered intra-tumorally, which induces a CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases. It is being tested in the clinic across multiple solid tumors
  • CAN-3110 is a replication-competent HSV gene construct, which is able to replicate specifically in tumor cells while sparing healthy tissue. It is being evaluated in patients with recurrent high-grade glioma
  • The enLIGHTEN™ Discovery Platform makes it possible to design new viral immunotherapies that could synergize with CAR-T cells and other cell therapies

2:30 pm Targeting OLIG2 in GBM and Medulloblastoma Using CT-179, an Orally Delivered Small Molecule Entering Clinical Development


  • Scientific rationale for targeting OLIG2 in GBM, medulloblastoma, and other
  • Preclinical data supporting the use of CT-179 alone and in combination with
    standard of care therapies
  • Overview of the early-stage clinical development plan for CT-179 in adults with recurrent and newly diagnosed GBM

3:00 pm Afternoon Break

Spotlighting Nucleic Acid Approaches to Treating GBM- From RNA Vaccines to DNA Drugs

3:30 pm Making Glioblastoma a Target for Immunotherapy: A Road From Biomarker Discovery to Harnessing the Tumor Microenvironment

  • Adilia Hormigo Leader of the Neuro-Oncology Program, Professor in the Departments of Oncology, Neurosurgery and Microbiology and Immunology, Albert Einstein College of Medicine


  • Discussing the use of vaccines for the treatment of glioblastomas
  • Identifying circulating biomarkers, the past and the present
  • Addressing hurdles to overcome to implement the next steps

4:00 pm Uncovering the Use of DNA Medicines for the Treatment of Glioblastoma

  • Jeffrey Skolnik Vice President - Clinical Development, Inovio Pharmaceuticals


  • Explore how DNA as a therapeutic can be employed in the treatment of GBM
  • Discover how combination agents work together to build an immune response against tumor antigens
  • What additional advances can be utilized to continue to optimize clinical outcomes?

The Next Step in GBM Research: Combination Approaches

4:30 pm DNA Modifiers: A Novel Therapeutic Strategy for Targeting Known DDR-Deficient GBMs


  • Design and discovery of novel covalent DNA modifying agents (DNA Modifiers) that exploit existing glioma DDR defects such as MGMT and overcome common clinical resistance mechanisms.
  • KL-50: the first DNA Modifier to generate a dynamic bifunctional DNA modification that exploits differences in repair kinetics to generate time- and MGMT-dependent DNA interstrand crosslinks
  • Proof of principle of KL-50 efficacy and tolerability across numerous GBM cell lines, invivo, and in PDX models with expansion of platform to other DDR biomarkers

5:00 pm Heavy Atom Nanoparticles for Radiation Enhancement of Glioblastoma

  • Henry Smilowitz Associate Professor, University of Connecticut Health Center


  • Heavy atom nanoparticles (e.g. iodine nanoparticles or INPs) can be used for radiation enhancement
  • The XOFT irradiator can be used to implement INP-RT of resected tumors
  • Combination therapies with INP-RT: Potential for synergy with radiation sensitizers, chemotherapy and immunotherapy

5:30 pm Chair’s Closing Remarks End of the 4th Annual Glioblastoma Drug Development Summit